表紙:遺伝子治療市場:第4版
市場調査レポート
商品コード
986216

遺伝子治療市場:第4版

Gene Therapy Market (4th Edition) by Therapeutic Approach (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas

出版日: | 発行: Roots Analysis | ページ情報: 英文 550 Pages | 納期: 即日から翌営業日

価格
価格表記: USDを日本円(税抜)に換算
本日の銀行送金レート: 1USD=110.86円
遺伝子治療市場:第4版
出版日: 2021年01月31日
発行: Roots Analysis
ページ情報: 英文 550 Pages
納期: 即日から翌営業日
  • 全表示
  • 概要
  • 図表
  • 目次
概要

現在までに、10の承認された遺伝子治療があり、世界中で1,000を超える製品候補が臨床試験で評価されています。この分野における現在の研究ペースと製品開発活動を考慮すると、専門家は、遺伝子治療を含む臨床研究イニシアチブの数が毎年17%ずつ増加する可能性があると考えています。

当レポートでは、世界の遺伝子治療市場について調査分析し、現在の市場情勢と将来の可能性について、体系的な情報を提供しています。

目次

第1章 序文

第2章 エグゼクティブサマリー

第3章 イントロダクション

第4章 遺伝子導入ベクター

  • 章の概要
  • 遺伝子導入のウイルス/非ウイルス法
  • 遺伝子組み換え療法のウイルスベクター
  • ウイルスベクターの種類
  • 非ウイルスベクターの種類

第5章 規制情勢と償還シナリオ

  • 章の概要
  • 北米の規制ガイドライン
  • 欧州の規制ガイドライン
  • アジア太平洋地域の規制ガイドライン
  • 償還シナリオ
  • 遺伝子治療で一般に提供される決済モデル

第6章 市場概要

  • 章の概要
  • 遺伝子治療市場:臨床・商業パイプライン
  • 遺伝子治療市場:初期段階のパイプライン
  • 遺伝子治療の候補:特殊指定
  • グリッド表現:分析 (開発段階別、治療領域別、治療アプローチ別)

第7章 競合情勢

  • 章の概要
  • 遺伝子治療市場:開発業者のリスト
  • 主要企業:分析 (製品候補の数別)

第8章 上市済みの遺伝子治療

  • 章の概要
  • Gendicine (Shenzhen Sibiono GeneTech)
  • Oncorine (Shanghai Sunway Biotech)
  • Rexin-G (Epeius Biotechnologies)
  • Neovasculgen (Human Stem Cells Institute)
  • Imlygic (Amgen)
  • Strimvelis (Orchard Therapeutics)
  • Luxturna (Spark Therapeutics)
  • Zolgensma (Novartis Gene Therapies)
  • Collategene (AnGes)
  • Zyntelgo (bluebird bio)

第9章 主要な商品化戦略

  • 章の概要
  • 成功する医薬品発売戦略:フレームワーク
  • 成功する医薬品発売戦略:製品の差別化
  • 製品開発の段階に基づく一般的に採用されている商品化戦略
  • 現在承認されている遺伝子治療のリスト
  • 遺伝子治療に焦点を当てた企業が採用した主要な商品化戦略
  • 結論

第10章 後期段階(フェーズII/III 以降)の遺伝子治療

  • 章の概要
  • GS010
  • PTC-AADC
  • BMN 270
  • rAd-IFN/Syn3
  • TAVO
  • OTL-200
  • EB-101
  • ProstAtak
  • BIIB111
  • LentiGlobin BB305?
  • Lenti-D
  • FCX-007
  • CG0070
  • Vigil
  • VM202
  • VGX-3100
  • Invossa
  • KB103
  • PF-06939926
  • PF-06838435
  • PF-07055480
  • SPK-8011
  • AMT-061
  • VB-111
  • OTL-101
  • BIIB112
  • FLT180a
  • LYS-SAF302
  • NFS-01
  • OTL-103

第11章 新興技術

第12章 主要な治療分野

  • 章の概要
  • 分析:治療分野別、特殊指定別
  • 腫瘍疾患
  • 神経障害
  • 代謝障害
  • 眼科疾患
  • 遺伝性疾患

第13章 特許分析

  • 章の概要
  • 遺伝子治療市場:特許リスト
  • 遺伝子編集市場:特許リスト
  • 知的財産ポートフォリオ全体:分析 (組織のタイプ別)

第14章 M&A

第15章 資金調達・投資分析

第16章 臨床試験分析

第17章 コスト価格分析

第18章 大手製薬企業:遺伝子治療に関連するイニシアチブの分析

第19章 需要分析

第20章 市場予測・機会分析

  • 章の概要
  • 範囲と制限
  • 主な前提条件と予測手法
  • 世界の遺伝子治療市場
  • 遺伝子治療市場:価値創造分析
  • 遺伝子治療市場:製品別の売上予測
    • Gendicine
    • Oncorine
    • Rexin-G
    • Neovasculgen
    • Strimvelis
    • Imlygic
    • Luxturna
    • Zolgensma
    • Collategene
    • Zyntelgo
    • GS010
    • PTC-AADC
    • BMN 270
    • rAd-IFN / Syn3
    • TAVO
    • OTL-200
    • EB-101
    • ProstAtak
    • BIIB111
    • LentiGlobinBB305
    • Lenti-D
    • FCX-007
    • CG0070
    • Vigil?
    • VM202
    • VGX-3100
    • Invossa
    • KB103
    • PF-06939926
    • PF-06838435
    • PF-07055480
    • SPK-8011
    • AMT-061
    • VB-111
    • OTL-101
    • BIIB112
    • FLT180a
    • LYS-SAF302
    • NFS-01
    • OTL-103

第21章 ベクター製造

第22章 ケーススタディ:遺伝子治療のサプライチェーン

第23章 結論

第24章 インタビュー記録

第25章 付録1:表形式データ

第26章 付録2:企業・組織のリスト

図表

List of Figures

  • Figure 3.1 Evolution of Gene Therapies
  • Figure 3.2 Ex Vivo Gene Therapies
  • Figure 3.3 In Vivo Gene Therapies
  • Figure 3.4 Mechanism of Action of Gene Therapies
  • Figure 3.5 Genome Editing: Historical Milestones
  • Figure 3.6 Genome Editing Techniques
  • Figure 3.7 Advantages and Disadvantages of Gene Therapies
  • Figure 4.1 Gene Transfer: Viral and Non-Viral Methods
  • Figure 5.1 Centralized Regulatory Pathway for Market Approval in the US
  • Figure 5.2 Centralized Regulatory Pathway for Market Approval in the Europe
  • Figure 5.3 Manufacturing Requirements for Gene Therapy Products in China
  • Figure 5.4 Key Elements of Quality Control in China
  • Figure 5.5 Centralized Regulatory Pathway for Market Approval in Japan
  • Figure 6.1 Gene Therapies: Distribution by Phase of Development
  • Figure 6.2 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development
  • Figure 6.3 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Therapeutic Area
  • Figure 6.4 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
  • Figure 6.5 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used
  • Figure 6.6 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene
  • Figure 6.7 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Modification
  • Figure 6.8 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used and Modification
  • Figure 6.9 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene Therapy
  • Figure 6.10 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Route of
  • Figure 6.11 Early Stage Pipeline of Gene Therapies: Distribution by Phase of Development
  • Figure 6.12 Early Stage Pipeline of Gene Therapies: Distribution by Therapeutic Area
  • Figure 6.13 Early Stage Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
  • Figure 6.14 Early Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used
  • Figure 6.15 Early Stage Pipeline of Gene Therapies: Distribution by Type of Gene
  • Figure 6.16 Early Stage Pipeline of Gene Therapies: Distribution by Therapeutic Approach
  • Figure 6.17 Early Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used and Modification
  • Figure 6.18 Early Stage Pipeline of Gene Therapies: Distribution by Type of Gene Therapy
  • Figure 6.19 Early Stage Pipeline of Gene Therapies: Distribution by Route of Administration
  • Figure 6.20 Gene Therapies: Distribution by Special Designation(s) Awarded
  • Figure 6.21 Grid Representation: Distribution by Phase of Development, Therapeutic Area and Therapeutic Approach
  • Figure 7.1 Gene Therapy Developers: Distribution by Year of Establishment
  • Figure 7.2 Gene Therapy Developers: Distribution by Company Size
  • Figure 7.3 Gene Therapy Developers: Distribution by Location of Headquarters
  • Figure 7.4 Gene Therapy Developers: Distribution by Year of Establishment, Company Size and Location of Headquarters
  • Figure 7.5 Gene Therapy Developers: Most Prominent Hubs
  • Figure 7.6 Gene Therapy Developers: Distribution of Players based in North America
  • Figure 7.7 Gene Therapy Developers: Distribution of Players based in Europe
  • Figure 7.8 Gene Therapy Developers: Distribution of Players based in Asia-Pacific
  • Figure 7.9 Gene Therapy Developers: Key Players
  • Figure 7.10 Gene Therapy Developers: Distribution by Key Players and Phase of Development
  • Figure 8.1 Gendicine®: Development Timeline
  • Figure 8.2 Oncorine®: Development Timeline
  • Figure 8.3 Oncorine®: Mechanism of Action
  • Figure 8.4 Rexin-G®: Development Timeline
  • Figure 8.5 Neovasculgen®: Development Timeline
  • Figure 8.6 Imlygic®: Development Timeline
  • Figure 8.7 Imlygic®: Mechanism of Action
  • Figure 8.8 Strimvelis®: Development Timeline
  • Figure 8.9 Luxturna™: Development Timeline
  • Figure 8.10 Zolgensma™: Development Timeline
  • Figure 8.11 Collategene®: Development Timeline
  • Figure 8.12 Zyntelgo™: Development Timeline
  • Figure 9.1 Successful Drug Launch Strategy: ROOTS Framework
  • Figure 9.2 Successful Drug Launch Strategy: Product Differentiation
  • Figure 9.3 Commonly Adopted Commercialization Strategies based on Phase of Development of the Product
  • Figure 9.4 Harvey Ball Analysis: Commercialization Strategies Adopted by Companies Focused on Gene Therapies
  • Figure 9.5 Approved Gene Therapies: Historical Timeline of Geographical Expansion
  • Figure 9.6 Approved Gene Therapies: Snapshot of Promotional Activities on Product Websites
  • Figure 9.7 Key Commercialization Strategies: Harvey Ball Analysis by Ease of Implementation, Value Addition and Current Adoption
  • Figure 11.1 Gene Editing: Key Application Areas
  • Figure 11.2 Gene Editing: Emerging Technology Platforms
  • Figure 12.1 Gene Therapies: Distribution by Therapeutic Area and Special Designation(s) Awarded
  • Figure 12.2 Gene Therapies for Oncological Disorders: Distribution by Target Indication and Phase of Development
  • Figure 12.3 Gene Therapies for Oncological Disorders: Distribution by Type of Vector Used
  • Figure 12.4 Gene Therapies for Nervous System Disorders: Distribution by Target Indication and Phase of Development
  • Figure 12.5 Gene Therapies for Nervous System Disorders: Distribution by Type of Vector Used
  • Figure 12.6 Gene Therapies for Metabolic Disorders: Distribution by Target Indication and Phase of Development
  • Figure 12.7 Gene Therapies for Metabolic Disorders: Distribution by Type of Vector Used
  • Figure 12.8 Gene Therapies for Ophthalmic Diseases: Distribution by Target Indication and Phase of Development
  • Figure 12.9 Gene Therapies for Ophthalmic Diseases: Distribution by Type of Vector Used
  • Figure 12.10 Gene Therapies for Genetic Disorders: Distribution by Target Indication and Phase of Development
  • Figure 12.11 Gene Therapies for Genetic Disorders: Distribution by Type of Vector Used
  • Figure 13.1 Gene Therapy Patent Portfolio: Distribution by Type of Patent
  • Figure 13.2 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2016-2020
  • Figure 13.3 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
  • Figure 13.4 Gene Therapy Patent Portfolio: Distribution by Geography
  • Figure 13.5 Gene Therapy Patent Portfolio: Distribution by Region (North America)
  • Figure 13.6 Gene Therapy Patent Portfolio: Distribution by Region (Europe)
  • Figure 13.7 Gene Therapy Patent Portfolio: Distribution by Region (Asia-Pacific)
  • Figure 13.8 Gene Therapy Patent Portfolio: Distribution by CPC Symbols
  • Figure 13.9 Gene Therapy Patent Portfolio: Emerging Focus Areas
  • Figure 13.10 Gene Therapy Patent Portfolio: Leading Industry Players
  • Figure 13.11 Gene Therapy Patent Portfolio (Sangamo Therapeutics and Genzyme): Benchmarking by Patent Characteristics
  • Figure 13.12 Gene Therapy Patent Portfolio (Other Leading Industry Players): Benchmarking by Patent Characteristics
  • Figure 13.13 Gene Therapy Patent Portfolio: Distribution by Patent Age (2016-2020)
  • Figure 13.14 Gene Therapy Patent Portfolio: Valuation Analysis
  • Figure 13.15 Gene Editing Patent Portfolio: Distribution by Type of Patent
  • Figure 13.16 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2016-2020
  • Figure 13.17 Gene Editing Patent Portfolio: Distribution by Type of Patent and Publication Year
  • Figure 13.18 Gene Editing Patent Portfolio: Distribution by Geography
  • Figure 13.19 Gene Editing Patent Portfolio: Distribution by Region (North America)
  • Figure 13.20 Gene Editing Patent Portfolio: Distribution by Region (Europe)
  • Figure 13.21 Gene Editing Patent Portfolio: Distribution by Region (Asia-Pacific)
  • Figure 13.22 Gene Editing Patent Portfolio: Distribution by CPC Symbols
  • Figure 13.23 Gene Editing Patent Portfolio: Emerging Focus Areas
  • Figure 13.24 Gene Editing Patent Portfolio: Leading Industry Players
  • Figure 13.25 Gene Editing Patent Portfolio (Sangamo Therapeutics and CRISPR Therapeutics): Benchmarking by Patent Characteristics
  • Figure 13.26 Gene Editing Patent Portfolio (Other Leading Industry Players): Benchmarking by Patent Characteristics
  • Figure 13.27 Gene Editing Patent Portfolio: Distribution by Patent Age (2016-2020)
  • Figure 13.28 Gene Editing Patent Portfolio: Valuation Analysis
  • Figure 13.29 Gene Therapy and Gene Editing Patent Portfolio: Cumulative Distribution by Type of Organization
  • Figure 14.1 Mergers and Acquisitions: Cumulative Year-Wise Trend (2015-2020)
  • Figure 14.2 Mergers and Acquisitions: Distribution by Type of Agreement
  • Figure 14.3 Mergers and Acquisitions: Distribution by Year and Type of Agreement
  • Figure 14.4 Mergers and Acquisitions: Continent-wise Distribution
  • Figure 14.5 Mergers and Acquisitions: Distribution by Intercontinental and Intracontinental Acquisitions
  • Figure 14.6 Mergers and Acquisitions: Country-wise Distribution
  • Figure 14.7 Acquisitions: Distribution by Key Value Drivers
  • Figure 14.8 Acquisitions: Distribution by Year of Acquisition and Key Value Drivers
  • Figure 14.9 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company's Product
  • Figure 14.10 Mergers and Acquisitions: Distribution by Year of Acquisition and Phase of Development of the Acquired Company's Product and
  • Figure 14.11 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company's Product and Deal Amount
  • Figure 14.12 Mergers and Acquisitions: Distribution by Therapeutic Area
  • Figure 15.1 Funding and Investment Analysis: Cumulative Year-wise Distribution by Number of Instances, 2015-2020
  • Figure 15.2 Funding and Investment Analysis: Cumulative Amount Invested, 2015-2020 (USD Million)
  • Figure 15.3 Funding and Investment Analysis: Distribution of Instances by Type of Funding, 2015-2020
  • Figure 15.4 Funding and Investment Analysis: Distribution of the Total Amount Invested by Type of Funding, 2015-2020 (USD Million)
  • Figure 15.5 Funding and Investment Analysis: Distribution by Year and Type of Funding, 2015-2020 (USD Million)
  • Figure 15.6 Funding and Investment Analysis: Distribution by Year, Type of Funding and Amount Invested, 2015-2020 (USD Million)
  • Figure 15.7 Funding and Investment Analysis: Summary of Investments, 2015-2020 (USD Million)
  • Figure 15.8 Funding and Investments: Distribution by Focus Area
  • Figure 15.9 Funding and Investment Analysis: Distribution by Geography
  • Figure 15.10 Funding and Investment Analysis: Regional Distribution of Funding Instances
  • Figure 15.11 Funding and Investment Analysis: Most Active Players, based on the Number of Funding Instances, 2015-2020 (USD Million)
  • Figure 15.12 Funding and Investment Analysis: Most Active Players, based on the Amount Invested, 2015-2020 (USD Million)
  • Figure 15.13 Funding and Investment Analysis: Distribution by Type of Investors
  • Figure 15.14 Funding and Investment Analysis: Distribution by Leading Investors
  • Figure 15.15 Funding and Investment Analysis: Distribution of Investments by Phase of Development of the Company's Product
  • Figure 15.16 Funding and Investment Analysis: Distribution by Therapeutic Area
  • Figure 15.17 Funding and Investment Summary, 2015-2020 (USD Million)
  • Figure 16.1 Clinical Trial Analysis: Distribution by Trial Registration Year, Pre 2010-2020
  • Figure 16.2 Clinical Trial Analysis: Distribution by Trial Status
  • Figure 16.3 Clinical Trial Analysis: Distribution by Trial Phase
  • Figure 16.4 Clinical Trial Analysis: Distribution by Therapeutic Area
  • Figure 16.5 Clinical Trial Analysis: Distribution by Geography
  • Figure 16.6 Clinical Trial Analysis: Geographical Distribution by Trial Registration Year
  • Figure 16.7 Clinical Trial Analysis: Geographical Distribution by Trial Status
  • Figure 16.8 Clinical Trial Analysis: Geographical Distribution by Trial Status and Trial Phase
  • Figure 16.9 Clinical Trial Analysis: Geographical Distribution by Therapeutic Area
  • Figure 16.10 Clinical Trial Analysis: Distribution by Type of Sponsor
  • Figure 16.11 Clinical Trial Analysis: Distribution by Prominent Treatment Sites
  • Figure 16.12 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Registration Year
  • Figure 16.13 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status
  • Figure 16.14 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
  • Figure 16.15 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area
  • Figure 16.16 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Location of Trial Site
  • Figure 16.17 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status and Location of Trial Site
  • Figure 16.18 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status, Phase and Location of Trial Site
  • Figure 16.19 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area and Location of Trial Site
  • Figure 17.1 Gene Therapy: Pricing Model Based on Patient Segment
  • Figure 18.1 Big Pharma Players: Heat Map Analysis of Top Pharmaceutical Companies
  • Figure 18.2 Big Pharma Players: Analysis by Therapeutic Area
  • Figure 18.3 Big Pharma Players: Analysis by Type of Vector Used
  • Figure 18.4 Big Pharma Players: Analysis by Therapeutic Approach
  • Figure 18.5 Big Pharma Players: Analysis by Type of Gene Therapy
  • Figure 18.6 Big Pharma Players: Heat Map Analysis of Other Players
  • Figure 19.1 Demand Analysis: Clinical Demand for Gene Therapies
  • Figure 19.2 Demand Analysis: Commercial Demand for Gene Therapies
  • Figure 20.1 Global Gene Therapy Market, 2020-2030: Base Case Scenario (USD Billion)
  • Figure 20.2 Gene Therapy Market: Distribution by Therapeutic Approach, 2020, 2025 and 2030
  • Figure 20.3 Gene Therapy Market: Distribution by Type of Gene Therapy, 2020, 2025 and 2030
  • Figure 20.4 Gene Therapy Market: Distribution by Type of Vector Used, 2020, 2025 and 2030
  • Figure 20.5 Gene Therapy Market: Distribution by Therapeutic Area, 2020, 2025 and 2030
  • Figure 20.6 Gene Therapy Market: Distribution by Route of Administration, 2020, 2025 and 2030
  • Figure 20.7 Gene Therapy Market: Distribution by Geography, 2020, 2025 and 2030
  • Figure 20.8 Gene Therapy Market: Country-wise Distribution of Asia-Pacific Region, 2020, 2025 and 2030
  • Figure 20. 9 Gendicine® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.10 Oncorine® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.11 Rexin-G® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.12 Neovasculgen® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.13 Strimvelis® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.14 Imlygic® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.15 Luxturna™ Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.16 Zolgensma™ Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.17 Collategene® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.18 Zyntelgo™ Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.19 GS010 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.20 PTC-AADC Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.21 BMN 270 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.22 rAd-IFN/Syn3 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.23 TAVO™ Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.24 OTL-200 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.25 EB-101 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.26 ProstAtak® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.27 BIIB111 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.28 LentiGlobin BB305® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.29 Lenti-D™ Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.30 FCX-007 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.31 CG0070 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.32 Vigil® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.33 VM202 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.34 VGX-3100 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.35 Invossa™ Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.36 KB103 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.37 PF-06939926 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.38 PF-06838435 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.39 PF-07055480 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.40 SPK-8011 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.41 AMT-061 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.42 VB-111 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.43 OTL-101 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.44 BIIB112 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.45 FLT180a Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.46 LYS-SAF302 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.47 NFS-01 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 20.48 OTL-103 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 21.1 Viral Vectors: Manufacturing Steps
  • Figure 22.1 Steps Involved in the Development of Gene Therapies
  • Figure 22.2 Overall Development Process of Cell-based Gene Therapies
  • Figure 22.3 Key Responsibilities of Stakeholders Involved in Advanced Therapies Supply Chain
  • Figure 22.4 Key Steps Involved in the Implementation of a Supply Chain Strategy
  • Figure 22.5 Keys Steps Involved in the Logistics Process of Autologous Therapies
  • Figure 22.6 Comparison of Logistics Processes for Autologous and Allogeneic Therapies
  • Figure 22.7 Global Regulatory Authorities for Gene Therapy Supply Chain
  • Figure 22.8 Overview of Supply Chain Orchestration Platform
  • Figure 22.9 Functions of Key Technological Solutions for Cell and Advanced Therapies Supply Chain Management
  • Figure 23.1 Concluding Remarks: Summary of the Current Market Landscape
  • Figure 23.2 Concluding Remarks: Summary of the Current Developer Landscape
  • Figure 23.3 Concluding Remarks: Recent Mergers and Acquisitions
  • Figure 23.4 Concluding Remarks: Recent Funding and Investments
  • Figure 23.5 Concluding Remarks: Market Sizing and Opportunity Analysis

List of Tables

  • Table 3.1 Comparison of Ex Vivo and In Vivo Techniques
  • Table 3.2 Vectors Used for Targeted Gene Delivery to Tissues / Organs
  • Table 3.3 Routes of Administration of Gene Therapies: Advantages and Disadvantages
  • Table 4.1 Viral Vectors: Key Features
  • Table 5.1 Approved Advanced Therapies Medicinal Products (ATMPs) and their Reimbursement Status in Europe
  • Table 5.2 Payment Options Available for Gene Therapies
  • Table 6.1 Gene Therapy Market: Clinical and Commercial Pipeline
  • Table 6.2 Gene Therapy Market: Early Stage Pipeline
  • Table 6.3 Gene Therapy Market: Special Designation(s) Awarded
  • Table 7.1 Gene Therapy Market: List of Developers
  • Table 8.1 Gene Therapies: Marketed and Approved Products
  • Table 8.2 Shenzhen Sibiono GeneTech: Company Overview
  • Table 8.3 Gendicine®: Patent Portfolio
  • Table 8.4 Gendicine®: Status of Development
  • Table 8.5 Shanghai Sunway Biotech: Company Overview
  • Table 8.6 Oncorine®: Status of Development
  • Table 8.7 Epeius Biotechnologies: Company Overview
  • Table 8.8 Rexin-G®: Status of Development
  • Table 8.9 Human Stem Cell Institute: Company Overview
  • Table 8.10 Neovasculgen®: Status of Development
  • Table 8.11 Amgen: Company Overview
  • Table 8.12 Imlygic®: Status of Development
  • Table 8.13 Imlygic®: Recommended Dose and Schedule
  • Table 8.14 Imlygic®: Determination of Injection Volume based upon Lesion Size
  • Table 8.15 Orchard Therapeutics: Company Overview
  • Table 8.16 Strimvelis®: Status of Development
  • Table 8.17 Spark Therapeutics: Company Overview
  • Table 8.18 Luxturna™: Status of Development
  • Table 8.19 Novartis Gene Therapies: Company Overview
  • Table 8.20 Zolgensma™: Status of Development
  • Table 8.21 AnGes: Company Overview
  • Table 8.22 Collategene®: Status of Development
  • Table 8.23 bluebird bio: Company Overview
  • Table 8.24 Zyntelgo™: Status of Development
  • Table 9.1 Luxturna™: Presentations in Conferences Before Product Approval
  • Table 9.2 Zolgensma™: Presentations in Conferences Before Product Approval
  • Table 9.3 Zyntelgo™: Presentations in Conferences Before Product Approval
  • Table 9.4 Luxturna™: Presentations in Conferences After Product Approval
  • Table 9.5 Strimvelis™: Presentations in Conferences After Product Approval
  • Table 9.6 Zyntelgo™: Presentations in Conferences After Product Approval
  • Table 9.7 Approved Gene Therapies: Patient Support Services Available on Product Websites (Comparative Analysis)
  • Table 10.1 Gene Therapies: Late Stage (Phase II/III and Above) Drug Candidates
  • Table 10.2 GS010: Drug Profile
  • Table 10.3 PTC-AADC: Drug Profile
  • Table 10.4 BMN 270: Drug Profile
  • Table 10.5 rAd-IFN/Syn3: Drug Profile
  • Table 10.6 TAVO™: Drug Profile
  • Table 10.7 OTL-200: Drug Profile
  • Table 10.8 EB-101: Drug Profile
  • Table 10.9 ProstAtak®: Drug Profile
  • Table 10.10 BIIB111: Drug Profile
  • Table 10.11 LentiGlobin BB305®: Drug Profile
  • Table 10.12 Lenti-D™: Drug Profile
  • Table 10.13 FCX-007: Drug Profile
  • Table 10.14 CG0070: Drug Profile
  • Table 10.15 Vigil®: Drug Profile
  • Table 10.16 VM202: Drug Profile
  • Table 10.17 VGX-3100: Drug Profile
  • Table 10.18 Invossa™: Drug Profile
  • Table 10.19 KB103: Drug Profile
  • Table 10.20 PF-06939926: Drug Profile
  • Table 10.21 PF-06838435: Drug Profile
  • Table 10.22 PF-07055480: Drug Profile
  • Table 10.23 SPK-8011: Drug Profile
  • Table 10.24 AMT-061: Drug Profile
  • Table 10.25 VB-111: Drug Profile
  • Table 10.26 OTL-101: Drug Profile
  • Table 10.27 BIIB112: Drug Profile
  • Table 10.28 FLT180a: Drug Profile
  • Table 10.29 LYS-SAF302: Drug Profile
  • Table 10.30 NFS-01: Drug Profile
  • Table 10.31 OTL-103: Drug Profile
  • Table 11.1 Gene Editing Platforms
  • Table 11.2 CRISPR Therapeutics: Funding Instances
  • Table 11.3 CRISPR Therapeutics: Collaborations
  • Table 11.4 Editas Medicine: Funding Instances
  • Table 11.5 Editas Medicine: Collaborations
  • Table 11.6 Intellia Therapeutics: Funding Instances
  • Table 11.7 Intellia Therapeutics: Collaborations
  • Table 11.8 Cellectis: Funding Instances
  • Table 11.9 Cellectis: Collaborations
  • Table 11.10 bluebird bio: Funding Instances
  • Table 11.11 bluebird bio: Collaborations
  • Table 11.12 Sangamo Therapeutics: Funding Instances
  • Table 11.13 Sangamo Therapeutics: Collaborations
  • Table 11.14 Gene Switch Technology Platforms
  • Table 11.15 Intrexon: Funding Instances
  • Table 11.16 Intrexon: Collaborations
  • Table 11.17 MeiraGTx: Funding Instances
  • Table 11.18 MeiraGTx: Collaborations
  • Table 11.19 Gene Therapy: Technology Platforms
  • Table 12.1 Gene Therapies for Oncological Disorders
  • Table 12.2 Gene Therapies for Nervous System Disorders
  • Table 12.3 Gene Therapies for Metabolic Disorders
  • Table 12.4 Gene Therapies for Ophthalmic Diseases
  • Table 12.5 Gene Therapies for Genetic Disorders
  • Table 13.1 Gene Therapy Patent Portfolio: List of Top CPC Symbols
  • Table 13.2 Gene Therapy Patent Portfolio: Summary of Benchmarking Analysis
  • Table 13.3 Gene Therapy Patent Portfolio: Categorizations based on Weighted Valuation Scores
  • Table 13.4 Gene Therapy Patent Portfolio: List of Leading Patents (in terms of Highest Relative Valuation)
  • Table 13.5 Gene Editing Patent Portfolio: List of Top CPC Symbols
  • Table 13.6 Gene Editing Patent Portfolio: Summary of Benchmarking Analysis
  • Table 13.7 Gene Editing Patent Portfolio: Categorizations based on Weighted Valuation Scores
  • Table 13.8 Gene Editing Patent Portfolio: List of Leading Patents (in terms of Highest Relative Valuation)
  • Table 14.1 Gene Therapy: List of Mergers and Acquisitions, 2015-2020
  • Table 14.2 Acquisitions: Key Value Drivers
  • Table 14.3 Mergers and Acquisitions: Information on Phase of Development and Therapeutic Area of the Acquired Company's Gene Therapy Product
  • Table 15.1 Gene Therapy Market: Funding and Investments, 2020-2030
  • Table 15.2 Funding and Investment Analysis: Summary of Investments
  • Table 15.3 Funding and Investment Analysis: Summary of Venture Capital Funding
  • Table 16.1 Clinical Trial Analysis: Emerging Molecules to Watch
  • Table 16.2 Clinical Trial Analysis: Most Important Trials to Watch
  • Table 17.1 Pricing Model: Price of Marketed Gene / Cell Therapies
  • Table 17.2 Pricing Model: Price of Marketed Targeted Drugs
  • Table 17.3 Pricing Model: Opinions of Experts / Other Analysts
  • Table 20.1 Gene Therapies: Expected Launch Years of Advanced Stage Drug Candidates
  • Table 20.2 Gendicine®: Target Patient Population
  • Table 20.3 Gendicine®: Net Present Value (USD Million)
  • Table 20.4 Gendicine®: Value Creation Analysis (USD Million)
  • Table 20.5 Oncorine®: Target Patient Population
  • Table 20.6 Oncorine®: Net Present Value (USD Million)
  • Table 20.7 Oncorine®: Value Creation Analysis (USD Million)
  • Table 20.8 Rexin-G®: Target Patient Population
  • Table 20.9 Rexin-G®: Net Present Value (USD Million)
  • Table 20.10 Rexin-G®: Value Creation Analysis (USD Million)
  • Table 20.11 Neovasculgen®: Target Patient Population
  • Table 20.12 Neovasculgen®: Net Present Value (USD Million)
  • Table 20.13 Neovasculgen®: Value Creation Analysis (USD Million)
  • Table 20.14 Strimvelis®: Target Patient Population
  • Table 20.15 Strimvelis®: Net Present Value (USD Million)
  • Table 20.16 Strimvelis®: Value Creation Analysis (USD Million)
  • Table 20.17 Imlygic®: Target Patient Population
  • Table 20.18 Imlygic®: Net Present Value (USD Million)
  • Table 20.19 Imlygic®: Value Creation Analysis (USD Million)
  • Table 20.20 Luxturna™: Target Patient Population
  • Table 20.21 Luxturna™: Net Present Value (USD Million)
  • Table 20.22 Luxturna™: Value Creation Analysis (USD Million)
  • Table 20.23 Zolgensma™: Target Patient Population
  • Table 20.24 Zolgensma™: Net Present Value (USD Million)
  • Table 20.25 Zolgensma™: Value Creation Analysis (USD Million)
  • Table 20.26 Collategene®: Target Patient Population
  • Table 20.27 Collategene®: Net Present Value (USD Million)
  • Table 20.28 Collategene®: Value Creation Analysis (USD Million)
  • Table 20.29 Zynteglo™: Target Patient Population
  • Table 20.30 Zynteglo™: Net Present Value (USD Million)
  • Table 20.31 Zynteglo™: Value Creation Analysis (USD Million)
  • Table 20.32 GS010: Target Patient Population
  • Table 20.33 GS010: Net Present Value (USD Million)
  • Table 20.34 GS010: Value Creation Analysis (USD Million)
  • Table 20.35 PTC-AADC: Target Patient Population
  • Table 20.36 PTC-AADC: Net Present Value (USD Million)
  • Table 20.37 PTC-AADC: Value Creation Analysis (USD Million)
  • Table 20.38 BMN 270: Target Patient Population
  • Table 20.39 BMN 270: Net Present Value (USD Million)
  • Table 20.40 BMN 270: Value Creation Analysis (USD Million)
  • Table 20.41 rAd-IFN/Syn3: Target Patient Population
  • Table 20.42 rAd-IFN/Syn3: Net Present Value (USD Million)
  • Table 20.43 rAd-IFN/Syn3: Value Creation Analysis (USD Million)
  • Table 20.44 TAVO™: Target Patient Population
  • Table 20.45 TAVO™: Net Present Value (USD Million)
  • Table 20.46 TAVO™: Value Creation Analysis (USD Million)
  • Table 20.47 OTL-200: Target Patient Population
  • Table 20.48 OTL-200: Net Present Value (USD Million)
  • Table 20.49 OTL-200: Value Creation Analysis (USD Million)
  • Table 20.50 EB-101: Target Patient Population
  • Table 20.51 EB-101: Net Present Value (USD Million)
  • Table 20.52 EB-101: Value Creation Analysis (USD Million)
  • Table 20.53 ProstAtak®: Target Patient Population
  • Table 20.54 ProstAtak®: Net Present Value (USD Million)
  • Table 20.55 ProstAtak®: Value Creation Analysis (USD Million)
  • Table 20.56 BIIB111: Target Patient Population
  • Table 20.57 BIIB111: Net Present Value (USD Million)
  • Table 20.58 BIIB111: Value Creation Analysis (USD Million)
  • Table 20.59 LentiGlobin BB305®: Target Patient Population
  • Table 20.60 LentiGlobin BB305®: Net Present Value (USD Million)
  • Table 20.61 LentiGlobin BB305®: Value Creation Analysis (USD Million)
  • Table 20.62 Lenti-D™: Target Patient Population
  • Table 20.63 Lenti-D™: Net Present Value (USD Million)
  • Table 20.64 Lenti-D™: Value Creation Analysis (USD Million)
  • Table 20.65 FCX-007: Target Patient Population
  • Table 20.66 FCX-007: Net Present Value (USD Million)
  • Table 20.67 FCX-007: Value Creation Analysis (USD Million)
  • Table 20.68 CG0070: Target Patient Population
  • Table 20.69 CG0070: Net Present Value (USD Million)
  • Table 20.70 CG0070: Value Creation Analysis (USD Million)
  • Table 20.71 Vigil®: Target Patient Population
  • Table 20.72 Vigil®: Net Present Value (USD Million)
  • Table 20.73 Vigil®: Value Creation Analysis (USD Million)
  • Table 20.74 VM202: Target Patient Population
  • Table 20.75 VM202: Net Present Value (USD Million)
  • Table 20.76 VM202: Value Creation Analysis (USD Million)
  • Table 20.77 VGX-3100: Target Patient Population
  • Table 20.78 VGX-3100: Net Present Value (USD Million)
  • Table 20.79 VGX-3100: Value Creation Analysis (USD Million)
  • Table 20.80 Invossa™: Target Patient Population
  • Table 20.81 Invossa™: Net Present Value (USD Million)
  • Table 20.82 Invossa™: Value Creation Analysis (USD Million)
  • Table 20.83 KB103: Target Patient Population
  • Table 20.84 KB103: Net Present Value (USD Million)
  • Table 20.85 KB103: Value Creation Analysis (USD Million)
  • Table 20.86 PF-06939926: Target Patient Population
  • Table 20.87 PF-06939926: Net Present Value (USD Million)
  • Table 20.88 PF-06939926: Value Creation Analysis (USD Million)
  • Table 20.89 PF-06838435: Target Patient Population
  • Table 20.90 PF-06838435: Net Present Value (USD Million)
  • Table 20.91 PF-06838435: Value Creation Analysis (USD Million)
  • Table 20.92 PF-07055480: Target Patient Population
  • Table 20.93 PF-07055480: Net Present Value (USD Million)
  • Table 20.94 PF-07055480: Value Creation Analysis (USD Million)
  • Table 20.95 SPK-8011: Target Patient Population
  • Table 20.96 SPK-8011: Net Present Value (USD Million)
  • Table 20.97 SPK-8011: Value Creation Analysis (USD Million)
  • Table 20.98 AMT-061: Target Patient Population
  • Table 20.99 AMT-061: Net Present Value (USD Million)
  • Table 20.100 AMT-061: Value Creation Analysis (USD Million)
  • Table 20.101 VB-111: Target Patient Population
  • Table 20.102 VB-111: Net Present Value (USD Million)
  • Table 20.103 VB-111: Value Creation Analysis (USD Million)
  • Table 20.104 OTL-101: Target Patient Population
  • Table 20.105 OTL-101: Net Present Value (USD Million)
  • Table 20.106 OTL-101: Value Creation Analysis (USD Million)
  • Table 20.107 BIIB112: Target Patient Population
  • Table 20.108 BIIB112: Net Present Value (USD Million)
  • Table 20.109 BIIB112: Value Creation Analysis (USD Million)
  • Table 20.110 FLT180a: Target Patient Population
  • Table 20.111 FLT180a: Net Present Value (USD Million)
  • Table 20.112 FLT180a: Value Creation Analysis (USD Million)
  • Table 20.113 LYS-SAF302: Target Patient Population
  • Table 20.114 LYS-SAF302: Net Present Value (USD Million)
  • Table 20.115 LYS-SAF302: Value Creation Analysis (USD Million)
  • Table 20.116 NFS-01: Target Patient Population
  • Table 20.117 NFS-01: Net Present Value (USD Million)
  • Table 20.118 NFS-01: Value Creation Analysis (USD Million)
  • Table 20.119 OTL-103: Target Patient Population
  • Table 20.120 OTL-103: Net Present Value (USD Million)
  • Table 20.121 OTL-103: Value Creation Analysis (USD Million)
  • Table 21.1 Small Scale Cell Culture Systems
  • Table 21.2 Contract Manufacturing Service Providers for Viral Vectors and Plasmid DNA
  • Table 25.1 Gene Therapies: Distribution by Phase of Development
  • Table 25.2 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development
  • Table 25.3 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Therapeutic Area
  • Table 25.4 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
  • Table 25.5 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used
  • Table 25.6 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene
  • Table 25.7 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Modification
  • Table 25.8 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Vector Used and Modification
  • Table 25.9 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Type of Gene Therapy
  • Table 25.10 Clinical and Commercial Pipeline of Gene Therapies: Distribution by Route of Administration
  • Table 25.11 Early Stage Pipeline of Gene Therapies: Distribution by Phase of Development
  • Table 25.12 Early Stage Pipeline of Gene Therapies: Distribution by Therapeutic Area
  • Table 25.13 Early Stage Pipeline of Gene Therapies: Distribution by Phase of Development and Therapeutic Area
  • Table 25.14 Early Stage Pipeline of Gene Therapies: Distribution by Type of Vector Used
  • Table 25.15 Early Stage Pipeline of Gene Therapies: Distribution by Type of Gene
  • Table 25.16 Early Stage Pipeline of Gene Therapies: Distribution by Therapeutic Approach
  • Table 25.17 Early Stage Pipeline of Gene Therapies: Distribution by Vector Used and Modification
  • Table 25.18 Early Stage Pipeline of Gene Therapies: Distribution by Type of Gene Therapy
  • Table 25.19 Early Stage Pipeline of Gene Therapies: Distribution by Route of Administration
  • Table 25.20 Gene Therapies: Distribution by Special Designation Awarded
  • Table 25.21 Gene Therapy Developers: Distribution by Year of Establishment
  • Table 25.22 Gene Therapy Developers: Distribution by Company Size
  • Table 25.23 Gene Therapy Developers: Distribution by Location of Headquarters
  • Table 25.24 Gene Therapy Developers: Distribution by Year of Establishment, Company Size and Location of Headquarters
  • Table 25.25 Gene Therapy Developers: Key Players
  • Table 25.26 Gene Therapy Developers: Distribution by Key Players and Phase of Development
  • Table 25.27 Gene Therapies: Distribution by Therapeutic Area and Special Designation(s) Awarded
  • Table 25.28 Gene Therapies for Oncological Disorders: Distribution by Target Indication and Phase of Development
  • Table 25.29 Gene Therapies for Oncological Disorders: Distribution by Type of Vector Used
  • Table 25.30 Gene Therapies for Nervous System Disorders: Distribution by Target Indication and Phase of Development
  • Table 25.31 Gene Therapies for Nervous System Disorders: Distribution by Type of Vector Used
  • Table 25.32 Gene Therapies for Metabolic Disorders: Distribution by Target Indication and Phase of Development
  • Table 25.33 Gene Therapies for Metabolic Disorders: Distribution by Type of Vector Used
  • Table 25.34 Gene Therapies for Ophthalmic Diseases: Distribution by Target Indication and Phase of Development
  • Table 25.35 Gene Therapies for Ophthalmic Diseases: Distribution by Type of Vector Used
  • Table 25.36 Gene Therapies for Genetic Disorders: Distribution by Target Indication and Phase of Development
  • Table 25.37 Gene Therapies for Genetic Disorders: Distribution by Type of Vector Used
  • Table 25.38 Gene Therapy Patent Portfolio: Distribution by Type of Patent
  • Table 25.39 Gene Therapy Patent Portfolio: Cumulative Distribution by Publication Year, 2016-2020
  • Table 25.40 Gene Therapy Patent Portfolio: Distribution by Type of Patent and Publication Year
  • Table 25.41 Gene Therapy Patent Portfolio: Distribution by Geography
  • Table 25.42 Gene Therapy Patent Portfolio: Distribution by Region (North America)
  • Table 25.43 Gene Therapy Patent Portfolio: Distribution by Region (Europe)
  • Table 25.44 Gene Therapy Patent Portfolio: Distribution by Region (Asia-Pacific)
  • Table 25.45 Gene Therapy Patent Portfolio: Leading Industry Players
  • Table 25.46 Gene Therapy Patent Portfolio: Distribution by Patent Age (2016-2020)
  • Table 25.47 Gene Editing Patent Portfolio: Distribution by Type of Patent
  • Table 25.48 Gene Editing Patent Portfolio: Cumulative Distribution by Publication Year, 2016-2020
  • Table 25.49 Gene Editing Patent Portfolio: Distribution by Type of Patent and Publication Year
  • Table 25.50 Gene Editing Patent Portfolio: Distribution by Geography
  • Table 25.51 Gene Editing Patent Portfolio: Distribution by Region (North America)
  • Table 25.52 Gene Editing Patent Portfolio: Distribution by Region (Europe)
  • Table 25.53 Gene Editing Patent Portfolio: Distribution by Region (Asia-Pacific)
  • Table 25.54 Gene Editing Patent Portfolio: Leading Industry Players
  • Table 25.55 Gene Editing Patent Portfolio: Distribution by Patent Age (2016-2020)
  • Table 25.56 Gene Therapy and Gene Editing Patent Portfolio: Cumulative Distribution by Type of Organization
  • Table 25.57 Mergers and Acquisitions: Cumulative Year-Wise Trend (2015-2020)
  • Table 25.58 Mergers and Acquisitions: Distribution by Type of Agreement
  • Table 25.59 Mergers and Acquisitions: Distribution by Year and Type of Agreement
  • Table 25.60 Mergers and Acquisitions: Continent-wise Distribution
  • Table 25.61 Mergers and Acquisitions: Country-wise Distribution
  • Table 25.62 Acquisitions: Distribution by Key Value Drivers
  • Table 25.63 Acquisitions: Distribution by Year of Acquisition and Key Value Drivers
  • Table 25.64 Mergers and Acquisitions: Distribution by Phase of Development of the Acquired Company's Product
  • Table 25.65 Mergers and Acquisitions: Distribution by Year of Acquisition and Phase of Development of the Acquired Company's Product
  • Table 25.66 Mergers and Acquisitions: Distribution by Therapeutic Area
  • Table 25.67 Funding and Investment Analysis: Cumulative Year-wise Distribution by Number of Instances, 2015-2020
  • Table 25.68 Funding and Investment Analysis: Cumulative Amount Invested, 2015-2020 (USD Million)
  • Table 25.69 Funding and Investment Analysis: Distribution of Instances by Type of Funding, 2015-2020
  • Table 25.70 Funding and Investment Analysis: Distribution of the Total Amount Invested by Type of Funding, 2015-2020 (USD Million)
  • Table 25.71 Funding and Investment Analysis: Distribution of Year and Type of Funding, 2015-2020 (USD Million)
  • Table 25.72 Funding and Investments: Distribution by Focus Area
  • Table 25.73 Funding and Investment Analysis: Distribution by Geography
  • Table 25.74 Funding and Investment Analysis: Regional Distribution of Funding Instances
  • Table 25.75 Funding and Investment Analysis: Most Active Players, based on the Number of Funding Instances, 2015-2020 (USD Million)
  • Table 25.76 Funding and Investment Analysis: Most Active Players, based on the Amount Invested, 2015-2020 (USD Million)
  • Table 25.77 Funding and Investment Analysis: Distribution by Type of Investors
  • Table 25.78 Funding and Investment Analysis: Distribution by Leading Investors
  • Table 25.79 Funding and Investment Analysis: Distribution of Investments by Phase of Development of the Company's Product
  • Table 25.80 Funding and Investment Analysis: Distribution by Therapeutic Area
  • Table 25.81 Clinical Trial Analysis: Distribution by Trial Registration Year, Pre 2010-2020
  • Table 25.82 Clinical Trial Analysis: Distribution by Trial Status
  • Table 25.83 Clinical Trial Analysis: Distribution by Trial Phase
  • Table 25.84 Clinical Trial Analysis: Distribution by Therapeutic Area
  • Table 25.85 Clinical Trial Analysis: Geographical Distribution by Trial Registration Year
  • Table 25.86 Clinical Trial Analysis: Distribution by Type of Sponsor
  • Table 25.87 Clinical Trial Analysis: Distribution by Prominent Treatment Sites
  • Table 25.88 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Registration Year
  • Table 25.89 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Status
  • Table 25.90 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
  • Table 25.91 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Therapeutic Area
  • Table 25.92 Demand Analysis: Clinical Demand for Gene Therapies
  • Table 25.93 Demand Analysis: Commercial Demand for Gene Therapies
  • Table 25.94 Global Gene Therapy Market, 2020-2030: Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.95 Gene Therapy Market: Distribution by Therapeutic Approach, 2020, 2025 and 2030
  • Table 25.96 Gene Therapy Market: Distribution by Type of Gene Therapy, 2020, 2025 and 2030
  • Table 25.97 Gene Therapy Market: Distribution by Type of Vector Used, 2020, 2025 and 2030
  • Table 25.98 Gene Therapy Market: Distribution by Therapeutic Area, 2020, 2025 and 2030
  • Table 25.99 Gene Therapy Market: Distribution by Route of Administration, 2020, 2025 and 2030
  • Table 25.100 Gene Therapy Market: Distribution by Geography, 2020, 2025 and 2030
  • Table 25.101 Gene Therapy Market: Country-wise Distribution of Asia-Pacific Region, 2020, 2025 and 2030
  • Table 25.102 Gendicine® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.103 Oncorine® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.104 Rexin-G® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.105 Neovasculgen® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.106 Strimvelis® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.107 Imlygic® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.108 Luxturna™ Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.109 Zolgensma™ Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.110 Collategene® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.111 Zyntelgo™ Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.112 GS010 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.113 PTC-AADC Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.114 BMN 270 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.115 rAd-IFN/Syn3 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.116 TAVO™ Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.117 OTL-200 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.118 EB-101 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.119 ProstAtak® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.120 BIIB111 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.121 LentiGlobin BB305® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.122 Lenti-D™ Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.123 FCX-007 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.124 CG0070 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.125 Vigil® Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.126 VM202 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.127 VGX-3100 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.128 Invossa™ Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.129 KB103 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.130 PF-06939926 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.131 PF-06838435 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.132 PF-07055480 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.133 SPK-8011 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.134 AMT-061 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.135 VB-111 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.136 OTL-101 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.137 BIIB112 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.138 FLT180a Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.139 LYS-SAF302 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.140 NFS-01 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)
  • Table 25.141 OTL-103 Sales Forecast (Till 2030): Conservative, Base and Optimistic Scenarios (USD Million)

List of Companies

The following companies / institutes / government bodies and organizations have been mentioned in this report.

  • 1. 4BIO Capital
  • 2. 4D Molecular Therapeutics
  • 3. 5AM Ventures
  • 4. AavantiBio
  • 5. aaVective
  • 6. AAVogen
  • 7. AbbVie
  • 8. Abby Grace Foundation
  • 9. Abeona Therapeutics
  • 10. Abintus Bio
  • 11. Adage Capital Management
  • 12. Adrenas Therapeutics
  • 13. Advantagene (also known as Candel Therapeutics)
  • 14. Advaxis
  • 15. Adverum Biotechnologies
  • 16. Aevitas Therapeutics
  • 17. Affinia Therapeutics
  • 18. Agent Capital
  • 19. Agilis Biotherapeutics
  • 20. AGTC
  • 21. Aisling Capital
  • 22. AJU IB Investment
  • 23. Akouos
  • 24. Alexandria Venture Investments
  • 25. Allergan
  • 26. Ally Bridge Group
  • 27. Alpha Holdings
  • 28. AlphaVax
  • 29. Altheia Science
  • 30. Altitude Life Science Ventures
  • 31. Amarna Therapeutics
  • 32. Ambulero
  • 33. American Gene Technologies™
  • 34. Amgen
  • 35. Amicus Therapeutics
  • 36. Amplo
  • 37. Anaeropharma Science
  • 38. AnGes
  • 39. Angionetics
  • 40. Annapurna Therapeutics (acquired by Avalanche Biotechnologies)
  • 41. ApolloBio
  • 42. Apple Tree Partners
  • 43. ARCH Venture Partners
  • 44. Arix Bioscience
  • 45. Arkin Bio Ventures
  • 46. ArrowMark Partners
  • 47. Arthrogen
  • 48. ARTIS Ventures
  • 49. Aruvant Sciences
  • 50. ASC Therapeutics
  • 51. Asia Alpha
  • 52. Asklepios BioPharmaceutical (AskBio)
  • 53. Aspa Therapeutics
  • 54. Astellas Pharma
  • 55. AstraZeneca
  • 56. Atlas Venture
  • 57. Atsena Therapeutics
  • 58. Audentes Therapeutics
  • 59. Audition Therapeutics
  • 60. Auriga Partners
  • 61. Avalanche Biotechnologies
  • 62. AVROBIO
  • 63. Axovant Gene Therapies
  • 64. Baillie Gifford
  • 65. Bain Capital Life Sciences
  • 66. Baldota Group
  • 67. Bamboo Therapeutics
  • 68. Bayer
  • 69. Beam Therapeutics
  • 70. Berkeley Catalyst Fund
  • 71. Biogen
  • 72. BioInvent International
  • 73. BioMarin Pharmaceutical
  • 74. Biomatics Capital
  • 75. Bionic Sight
  • 76. BioVec Pharma
  • 77. Bioverativ (a Sanofi company)
  • 78. Blue Bear Ventures
  • 79. bluebird bio
  • 80. BlueWater Angels
  • 81. Boehringer Ingelheim
  • 82. Boxer Capital
  • 83. Bpifrance
  • 84. Brace Pharma Capital
  • 85. Brain Neurotherapy Bio
  • 86. BrainVectis
  • 87. Breakout Ventures
  • 88. BridgeBio
  • 89. Bristol-Myers Squibb
  • 90. Brookside Capital
  • 91. BVF Partners
  • 92. California Institute for Regenerative Medicine
  • 93. Calimmune
  • 94. Cambridge Enterprise
  • 95. Cambridge Gene Therapy
  • 96. Cambridge Innovation Capital
  • 97. Cancer Research Institute
  • 98. CapDecisif Management
  • 99. Cardiogen Sciences
  • 100. Carmine Therapeutics
  • 101. Casdin Capital
  • 102. Case Western Reserve University
  • 103. Castle Creek Biosciences
  • 104. Catalyst Biosciences
  • 105. CavoGene LifeSciences
  • 106. Celenex (Nationwide Children's Hospital Spin Out)
  • 107. CellGenTech
  • 108. Celsion
  • 109. CG Oncology
  • 110. Chiba University
  • 111. Children's Medical Research Foundation
  • 112. Children's Oncology Group
  • 113. Coalition for Epidemic Preparedness Innovations (CEPI)
  • 114. CODA Biotherapeutics
  • 115. Codexis
  • 116. Columbia University
  • 117. Columbus Venture Partners
  • 118. CombiGene
  • 119. Consort Medical
  • 120. Copernicus Therapeutics
  • 121. Cormorant Asset Management
  • 122. Cowen Group
  • 123. Cowen Private Investments
  • 124. CRISPR Therapeutics
  • 125. CSL Behring
  • 126. CureDuchenne Ventures
  • 127. Cynvec
  • 128. Cyprium Therapeutics
  • 129. Cystic Fibrosis Foundation Therapeutics
  • 130. DARPA
  • 131. Deerfield Management
  • 132. DEFTA Partners
  • 133. Denovo Biopharma
  • 134. Dexcel Pharma
  • 135. Dimension Therapeutics
  • 136. DiNAQOR
  • 137. Discovery Genomics
  • 138. DNAlite Therapeutics
  • 139. DNAtrix
  • 140. Driehaus Capital Management
  • 141. Duke University
  • 142. Dyno Therapeutics
  • 143. EB Medical Research Foundation
  • 144. EcoR1 Capital
  • 145. Editas Medicine
  • 146. Edmond de Rothschild Investment Partners
  • 147. Eight Roads Ventures
  • 148. Emendo Biotherapeutics
  • 149. Emory University
  • 150. Emrys Bio
  • 151. Encoded Therapeutics
  • 152. Endsulin
  • 153. Enochian BioSciences
  • 154. Entos Pharmaceuticals
  • 155. Epeius Biotechnologies
  • 156. EPS Holdings
  • 157. Errant Gene Therapeutics
  • 158. Esco Ventures
  • 159. ESTEVE
  • 160. European Innovation Council Accelerator
  • 161. European Investment Bank
  • 162. Eventide Asset Management
  • 163. Evotec
  • 164. Excision BioTherapeutics
  • 165. Exegenesis Bio
  • 166. Exonics Therapeutics
  • 167. Expression Therapeutics
  • 168. ExSight Capital
  • 169. Eyevensys
  • 170. FerGene (Ferring Pharmaceuticals subsidiary)
  • 171. FF Science
  • 172. Fibrocell Science
  • 173. Fidelity Management & Research Company
  • 174. Flagship Pioneering
  • 175. Flash Therapeutics
  • 176. Flerie Invest AB
  • 177. Flexion Therapeutics
  • 178. Fondation Sanfilippo Suisse
  • 179. Foresite Capital
  • 180. Forge Biologics
  • 181. Forte Biosciences
  • 182. Fosun Capital
  • 183. FoxKiser
  • 184. F-Prime Capital
  • 185. Franklin Templeton Investments
  • 186. Frazier Healthcare Partners
  • 187. Freeline Therapeutics
  • 188. Friedreich's Ataxia Research Alliance
  • 189. Fund+
  • 190. Fundación Sanfilippo B
  • 191. Galia Gestion
  • 192. Gemini Therapeutics
  • 193. Genable Technologies
  • 194. Gene Therapy Research Institution
  • 195. GeneCure Biotechnologies
  • 196. Genelux
  • 197. GeneMedicine
  • 198. GeneOne Life Science
  • 199. GeneQuine Biotherapeutics
  • 200. Generation Bio
  • 201. Genespire
  • 202. Genethon
  • 203. GeneTx
  • 204. Genexine
  • 205. Genprex
  • 206. GenSight Biologics
  • 207. GenVec
  • 208. Ghost Tree Capital Group
  • 209. GO CAPITAL
  • 210. GOFAR
  • 211. GOG Foundation
  • 212. Google Ventures
  • 213. Gradalis
  • 214. Green Cross Holdings
  • 215. GSK
  • 216. Gyroscope Therapeutics
  • 217. Handl Therapeutics
  • 218. Harvard University
  • 219. Hatteras Venture Partners
  • 220. Hayfin Capital Management
  • 221. HBM Healthcare Investments
  • 222. HealthCap
  • 223. Helixmith
  • 224. Hemera Biosciences
  • 225. Herantis Pharma
  • 226. Hiroshima Venture Capital
  • 227. Histogenics
  • 228. Holostem Terapie Avanzate
  • 229. Homology Medicines
  • 230. HORAMA
  • 231. Horizon Technology Finance
  • 232. Hovione Scientia
  • 233. Huapont Life Sciences
  • 234. Human Stem Cells Institute PJSC (HSCI)
  • 235. Idinvest Partners
  • 236. Ikarovec
  • 237. Illumina Ventures
  • 238. Ilya Pharma
  • 239. Immusoft
  • 240. Imperial Innovations
  • 241. Innovate UK
  • 242. Innovation Network Corporation of Japan
  • 243. Inotek Pharmaceuticals
  • 244. Inovio Pharmaceuticals
  • 245. Inserm Transfert
  • 246. Institute of Myology
  • 247. Intellia Therapeutics
  • 248. Intrexon
  • 249. Invus
  • 250. Israel Innovation Authority
  • 251. IVERIC bio
  • 252. Janssen Pharmaceuticals
  • 253. Janus Capital Management
  • 254. Jennison Associates
  • 255. Johnson & Johnson Development
  • 256. Juda Capital
  • 257. K2 Venture Capital
  • 258. Kiwoom Securities
  • 259. Kleiner Perkins
  • 260. Kodikaz Therapeutic Solutions
  • 261. Kolon TissueGene
  • 262. Korea Investment Partners
  • 263. Kriya Therapeutics
  • 264. Krystal Biotech
  • 265. Kubota Vision
  • 266. Kurma Partners
  • 267. Kyorin Pharmaceutical
  • 268. Lacerta Therapeutics
  • 269. Leerink Partners
  • 270. Legend Capital
  • 271. Leiden University Medical Center
  • 272. Lentistem® Biotech
  • 273. LEXEO Therapeutics
  • 274. LifeArc
  • 275. Lilly Asia Ventures
  • 276. Lime Asset Management
  • 277. Limelight Bio
  • 278. Lipigon Pharmaceuticals
  • 279. LogicBio Therapeutics
  • 280. Lokon Pharma
  • 281. Longitude Capital
  • 282. Ludwig Cancer Research
  • 283. Lundbeckfonden Ventures
  • 284. Lysogene
  • 285. Mangrove Partners
  • 286. Maryland Technology Development
  • 287. Matrix Capital Management
  • 288. Maverick Ventures
  • 289. Medison Ventures
  • 290. MeiraGTx
  • 291. Memorial Sloan Kettering Cancer Center
  • 292. Mercia Technologies
  • 293. Merck
  • 294. MERITZ Securities
  • 295. Mesa Verde Venture Partners
  • 296. Michigan Economic Development
  • 297. MidCap Financial
  • 298. Milo Biotechnology
  • 299. Mirae Asset Financial Group
  • 300. Mita Securities
  • 301. Mitsubishi Tanabe Pharma
  • 302. Mitsubishi UFJ Asset Management
  • 303. Momotaro-Gene
  • 304. MultiVir
  • 305. Muscular Dystrophy Association
  • 306. Mustang Bio
  • 307. Myonexus Therapeutics
  • 308. Myosana Therapeutics
  • 309. NanoCor Therapeutics
  • 310. Nanogenic Solutions
  • 311. Narya Capital
  • 312. National Cancer Institute
  • 313. National Institute of Allergy and Infectious Diseases
  • 314. National Institute of Health
  • 315. National MPS Society
  • 316. Nationwide Children's Hospital
  • 317. Navega Therapeutics
  • 318. NeoMed
  • 319. Nerveda
  • 320. Netherlands Enterprise Agency
  • 321. NeuExcell Therapeutics
  • 322. Neuracle Genetics
  • 323. Neurimmune
  • 324. Neurocrine Biosciences
  • 325. Neurogene
  • 326. Neurophth Therapeutics
  • 327. New Energy and Industrial Technology Development Organization
  • 328. New Enterprise Associates
  • 329. New Leaf Venture Partners
  • 330. Nightstar Therapeutics
  • 331. Ninevah Therapeutics
  • 332. Noga Therapeutics
  • 333. Northpond Ventures
  • 334. Novartis
  • 335. Novo Holdings
  • 336. Novo Nordisk
  • 337. Oberland Capital
  • 338. Ocugen
  • 339. Odylia Therapeutics
  • 340. Okayama University
  • 341. Omega Funds
  • 342. Omnes Capital
  • 343. Oncolys BioPharma
  • 344. Onconetics Pharmaceuticals
  • 345. Oncos Therapeutics
  • 346. OncoSec Medical
  • 347. OncoSenX
  • 348. OrbiMed
  • 349. Orbit Biomedical
  • 350. ORCA Therapeutics
  • 351. Orchard Therapeutics
  • 352. ORI Capital
  • 353. Osage University Partners
  • 354. Otonomy
  • 355. Oxford BioMedica
  • 356. Oxford Finance
  • 357. Oxular
  • 358. Panmure Gordon
  • 359. Paragon Biosciences
  • 360. Parker Institute for Cancer Immunotherapy
  • 361. Partners Innovation Fund
  • 362. Passage Bio
  • 363. Pattern Bioscience
  • 364. Pavilion Capital
  • 365. PBM Capital
  • 366. Pentwater Capital Management
  • 367. Perceptive Advisors
  • 368. PeriphaGen
  • 369. Perseverance Capital Management
  • 370. Pfizer
  • 371. Pharmakon Advisors
  • 372. Phoenix Nest
  • 373. PhorMed
  • 374. Pivotal bioVenture Partners
  • 375. PlasmaTech Biopharmaceuticals
  • 376. Pontifax Venture Capital
  • 377. POSCO Capital
  • 378. Poseida Therapeutics
  • 379. Precigen
  • 380. Precision BioSciences
  • 381. Prevail Therapeutics
  • 382. Progenics Pharmaceuticals
  • 383. PsiOxus Therapeutics
  • 384. PTC Therapeutics
  • 385. RA Capital Management
  • 386. RBV Capital
  • 387. Red Sanfilippo Foundation
  • 388. Redbiotec
  • 389. Redmile Group
  • 390. Redpin Therapeutics
  • 391. Regeneron Pharmaceuticals
  • 392. REGENXBIO
  • 393. Renova Therapeutics
  • 394. Rentschler Biopharma
  • 395. Rev1 Ventures
  • 396. Reyon Pharmaceutical
  • 397. Ridgeback Capital Investments
  • 398. Ring Therapeutics
  • 399. Roche
  • 400. Rock Springs Capital
  • 401. Rocket Pharmaceuticals
  • 402. Roswell Park Comprehensive Cancer Center
  • 403. RoverMed BioSciences
  • 404. RTW Investments
  • 405. Ryboquin
  • 406. Samsara BioCapital
  • 407. Sanfilippo Children's Foundation
  • 408. Sangamo Therapeutics
  • 409. Sanofi
  • 410. Sanofi Genzyme
  • 411. Santen Pharmaceutical
  • 412. Sarepta Therapeutics
  • 413. Scancell
  • 414. SDL Ventures
  • 415. Seattle Children's Research Institute
  • 416. Selecta Biosciences
  • 417. Sequoia Capital China
  • 418. Seraph Research Institute
  • 419. Seventure Partners
  • 420. Sham Innovation Sante
  • 421. Shanghai Sunway Biotech
  • 422. Shenzhen Qianhai Taxus
  • 423. Shinhan Capital
  • 424. Shinsei Corporate Investment
  • 425. Shire
  • 426. Sibiono GeneTech
  • 427. SillaJen
  • 428. Sofinnova Ventures
  • 429. Solid Biosciences
  • 430. Sorrento Therapeutics
  • 431. SOSV
  • 432. SOTIO
  • 433. Spark Therapeutics
  • 434. Sphera Global Healthcare Fund
  • 435. Spirovant Sciences
  • 436. SR-Tiget
  • 437. St. Jude Children's Research Hospital
  • 438. Stanford University
  • 439. StrideBio
  • 440. Surveyor Capital
  • 441. SwanBio Therapeutics
  • 442. Syncona
  • 443. Synpromics
  • 444. T. Rowe Price
  • 445. Takeda Pharmaceutical
  • 446. Tamid Bio
  • 447. Target ALS Foundation
  • 448. Targovax
  • 449. Taysha Gene Therapies
  • 450. Temasek
  • 451. Tenaya Therapeutics
  • 452. The Army Small Business Innovation Research
  • 453. The Column Group
  • 454. TheraBiologics
  • 455. Theravectys
  • 456. TNK Therapeutics
  • 457. Tocagen
  • 458. Tolerion
  • 459. TPG Capital
  • 460. Transgene
  • 461. Transhuman Capital
  • 462. Trucode Gene Repair
  • 463. UCL Technology Fund
  • 464. UK Cystic Fibrosis Gene Therapy Consortium
  • 465. Ultragenyx Pharmaceutical
  • 466. uniQure Biopharma
  • 467. Universitat Autònoma de Barcelona (UAB)
  • 468. University College London
  • 469. University Hospitals Cleveland Medical Center
  • 470. University of California San Diego School of Medicine
  • 471. University of Florida
  • 472. University of Massachusetts
  • 473. University of North Carolina at Chapel Hill
  • 474. University of Pennsylvania
  • 475. University of Pittsburgh
  • 476. University of Washington
  • 477. Urovant Sciences
  • 478. Valor Equity Partners
  • 479. V-Bio Ventures
  • 480. VBL Therapeutics
  • 481. VCN Biosciences
  • 482. Vectalys
  • 483. venBio
  • 484. Venrock
  • 485. Versant Ventures
  • 486. Vertex Pharmaceuticals
  • 487. Verve Therapeutics
  • 488. Vida Ventures
  • 489. ViGeneron (spin-off of the Ludwig-Maximilians-University)
  • 490. Viking Global Investors
  • 491. Virttu Biologics
  • 492. Vivet Therapeutics
  • 493. Vivo Capital
  • 494. Voyager Therapeutics
  • 495. VREX Therapeutics
  • 496. Washington Research Foundation
  • 497. Washington State University's Office of Commercialization
  • 498. Waverly Capital
  • 499. Weill Cornell Medicine
  • 500. Wellcome Trust
  • 501. Whitesun Healthcare Ventures
  • 502. WI Harper Group
  • 503. Wize Pharma
  • 504. Woodford Investment Management
  • 505. WuXi AppTec
  • 506. Wyvern Pharmaceuticals
  • 507. XyloCor Therapeutics
  • 508. Ysios Capital
  • 509. Ziopharm Oncology
目次

Title:
Gene Therapy Market (4th Edition)
by Therapeutic Approach (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas (Autoimmune Disorders, Cardiovascular Diseases, Genetic Disorders, Hematological Disorders, Metabolic Disorders, Muscle-related Diseases, Oncological Disorders, Ophthalmic Diseases and Others), Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2020-2030.

Overview:

Over time, several gene therapies have been developed for the treatment of both simple (single defect) and complex (multiple defects) genetic disorders. In fact, there are 10 approved gene therapies (recent examples include Zolgensma®, Zynteglo™ and Collategene®), till date, and more than a thousand product candidates being evaluated in clinical trials, worldwide. Considering the current pace of research and product development activity in this field, experts believe that the number of clinical research initiatives involving gene therapies are likely to grow by 17% annually. In this context, the USFDA released a notification, mentioning that it now expects to receive twice as many gene therapy applications each year, starting 2020. Despite the ongoing pandemic, it is worth highlighting that gene therapy companies raised approximately USD 5.5 billion in capital investments, in 2020 alone. This is indicative of the promising therapeutic potential of this emerging class of pharmacological interventions, which has led investors to bet heavily on the success of different gene therapy candidates in the long term.

Several technology platforms are currently available for discovery and development of various types of gene therapies. In fact, advances in bioanalytical methods (such as genome sequencing), and genome editing and manipulation technologies (such as molecular switches), have enabled the development of novel therapy development tools / platforms. In fact, technology licensing is a lucrative source of income for stakeholders in this industry, particularly for those with proprietary gene editing platforms. Given the growing demand for interventions that focus on the amelioration of the underlying (genetic) causes of diseases, it is expected that the gene therapy pipeline will continue to steadily expand. Moreover, promising results from ongoing clinical research initiatives are likely to bring in more investments to support therapy product development initiatives in this domain. Therefore, we are led to believe that the global gene therapy market is poised to witness significant growth in the foreseen future.

Scope of the Report:

The "Gene Therapy Market (4th Edition) by Therapeutic Approach (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Therapy (Ex vivo and In vivo), Type of Vectors used (Adeno Associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Plasmid DNA, Retrovirus and Others), Target Therapeutic Areas (Autoimmune Disorders, Cardiovascular Diseases, Dermatological Disorders, Genetic Disorders, Hematological Disorders, Metabolic Disorders, Muscle-related Diseases, Oncological Disorders, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebellar, Intradermal, Intramuscular, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal and Others), and Key Geographical Regions (US, EU5 and rest of the world): Industry Trends and Global Forecasts, 2020-2030" report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies. The study also features an elaborate discussion on the future potential of this evolving market. Amongst other elements, the report features:

  • A detailed review of the overall market landscape of gene therapies and gene editing therapies, including information on phase of development (marketed, clinical, preclinical and discovery) of pipeline candidates, key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), type of vector used, type of gene, therapeutic approach (gene augmentation, oncolytic viral therapy and others), type of gene therapy (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any).
  • A detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain.
  • An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors.
  • A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies.
  • Detailed profiles of marketed and late stage (phase II/III and above) gene therapies, including development timeline of the therapy, information on the current development status, mechanism of action, affiliated technology, strength of patent portfolio, dosage and manufacturing details, as well as details related to the developer company.
  • An elaborate discussion on the various commercialization strategies that can be adopted by drug developers across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing.
  • A review of the various emerging technologies and therapy development platforms that are being used to design and manufacture gene therapies, featuring detailed profiles of technologies that were / are being used for the development of four or more products / product candidates.
  • An in-depth analysis of various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2016. The analysis assesses several relevant parameters associated with the patents, including type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed / granted), and patent valuation.
  • A detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company' product and target therapeutic area.
  • An analysis of the investments made at various stages of development in companies that are focused in this area, between 2015-2020, including seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings.
  • A detailed geographical clinical trial analysis of completed, ongoing and planned studies of numerous gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.
  • An analysis of the various factors that are likely to influence the pricing of gene therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies.
  • An analysis of the big biopharma players engaged in this domain, featuring a heat map based on parameters, such as number of gene therapies under development, funding information, partnership activity and strength of patent portfolio.
  • An informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.
  • A case study on the prevalent and emerging trends related to vector manufacturing, along with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors.
  • A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, for the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2020-2030. The report also features the likely distribution of the current and forecasted opportunity across [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vectors used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry's growth.

The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals:

  • Adam Rogers (CEO, Hemera Biosciences)
  • Al Hawkins (CEO, Milo Biotechnology)
  • Buel Dan Rodgers (Founder & CEO, AAVogen)
  • Christopher Reinhard (CEO and Chairman, Gene Therapeutics (previously known as Cardium Therapeutics))
  • Michael Triplett (CEO, Myonexus Therapeutics)
  • Robert Jan Lamers (CEO, Arthrogen)
  • Ryo Kubota (CEO, Chairman & President, Acucela)
  • Tom Wilton (CBO, LogicBio Therapeutics)
  • Jeffrey Hung (CCO, Vigene Biosciences)
  • Cedric Szpirer (Executive & Scientific Director, Delphi Genetics)
  • Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory)
  • Molly Cameron (Corporate Communications Manager, Orchard Therapeutics)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

Key Questions Answered:

  • Who are the leading industry players engaged in the development of gene therapies?
  • How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products?
  • Which types of vectors are most commonly used for effective delivery of gene therapies?
  • What are the key regulatory requirements for gene therapy approval, across various geographies?
  • Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development?
  • What are the different pricing models and reimbursement strategies currently being adopted for gene therapies?
  • What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies?
  • Who are the key CMOs / CDMOs engaged in supplying viral / plasmid vectors for gene therapy development?
  • What are the key value drivers of the merger and acquisition activity in the gene therapy industry?
  • Who are the key stakeholders that have actively made investments in the gene therapy domain?
  • Which are the most active trial sites (in terms of number of clinical studies being conducted) related to this domain?
  • How is the current and future market opportunity likely to be distributed across key market segments?

Chapter Outlines:

Chapter 2 provides an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.

Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It further highlights the different types of gene therapies (namely somatic and germline therapies, and in vivo and ex vivo therapies), potential application areas of such products and route of administration of these therapeutic interventions. In addition, it provides information on the concept of gene editing, highlighting key historical milestones, applications and various techniques used for gene editing. The also chapter includes a discussion on the advantages and disadvantages associated with gene therapies. Further, it features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.

Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages and limitations of currently available vectors.

Chapter 5 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, Australia, China, Hong Kong, Japan and South Korea. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.

Chapter 6 includes information on over 800 gene therapies and gene editing therapies that are currently approved or are in different stages of development. It features a detailed analysis of pipeline molecules, based on several relevant parameters, such as key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), phase of development (marketed, clinical, preclinical and discovery), type of vector used, type of gene, type of gene therapy (ex vivo and in vivo), therapeutic approach (gene augmentation, oncolytic viral therapy and others), route of administration and special drug designation (if any). Further, we have presented a grid analysis of gene therapies based on phase of development, therapeutic area and therapeutic approach.

Chapter 7 provides a detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. Further, we have presented a logo landscape of product developers in North America, Europe and the Asia-Pacific region on the basis of company size.

Chapter 8 provides detailed profiles of marketed gene therapies. Each profile includes information about the innovator company, its product pipeline (focused on gene therapy only), development timeline of the therapy, its mechanism of action, target indication, current status of development, details related to manufacturing, dosage and sales, the company's patent portfolio and collaborations focused on its gene therapy product / technology.

Chapter 9 features an elaborate discussion on the various strategies that can be adopted by therapy developers across key commercialization stages, including prior to drug launch, during drug launch and post-launch. In addition, it presents an in-depth analysis of the key commercialization strategies that have been adopted by developers of gene therapies approved during the period 2015-2020.

Chapter 10 provides detailed profiles of drugs that are in advanced stages of clinical development (phase II/III and above). Each drug profile provides information on the current developmental status of the drug, its route of administration, developers, primary target indication, special drug designation received, target gene, dosage, mechanism of action, technology, patent portfolio, clinical trials and collaborations (if any).

Chapter 11 provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. In addition, it features brief profiles of some of the key technologies. Each profile features details on the various pipeline molecules that have been / are being developed using the technology, its advantages and the partnerships that have been established related to the technology platform. Further, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.

Chapter 12 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include genetic disorders, metabolic disorders, nervous system disorders, oncological disorders and ophthalmic diseases.

Chapter 13 provides an overview of the various patents that have been filed / granted in relation to gene therapy and gene editing technologies. It also features a detailed analysis, highlighting the prevalent trends related to type of patent, publication year, regional applicability, CPC symbols, emerging areas and leading industry players (in terms of number of patents filed). In addition, it features a competitive benchmarking analysis of the patent portfolios of leading industry players and patent valuation. For the purpose of this analysis, we have taken into consideration patents that have been filed / granted since 2016.

Chapter 14 features a detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company' product and target therapeutic area.

Chapter 15 presents details on various funding instances, investments and grants reported within the gene therapy domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and subsequent offerings) received by the companies between 2015 and 2020, highlighting the growing interest of the venture capital community and other strategic investors in this market.

Chapter 16 presents a detailed, geographical clinical trial analysis of completed, ongoing and planned studies focused on gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.

Chapter 17 highlights our views on the various factors that may be taken into consideration while deciding the price of a gene therapy. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt in order to decide the price of its proprietary products.

Chapter 18 highlights top big biopharma players engaged in the field of gene therapy, featuring a heat map analysis based on several parameters, including therapeutic area, type of vector used, therapeutic approach and type of gene therapy.

Chapter 19 features an informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.

Chapter 20 presents an elaborate market forecast analysis, highlighting the future potential of the market till the year 2030. It also includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II/III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vector used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic area (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world).

Chapter 21 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. In addition, it features the challenges that exist in this domain. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.

Chapter 22 provides a glimpse of the gene therapy supply chain. It discusses the steps for implementing a robust model and provides information related to the global regulations for supply chain. Moreover, the chapter discusses the challenges associated with supply chain of gene therapies. In addition, it features the technological solutions that can be adopted for the management of gene therapy supply chain.

Chapter 23 summarizes the overall report, wherein we have mentioned all the key facts and figures described in the previous chapters. The chapter also highlights important evolutionary trends that were identified during the course of the study and are expected to influence the future of the gene therapy market.

Chapter 24 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interviews held with Adam Rogers (CEO, Hemera Biosciences), Al Hawkins (CEO, Milo Biotechnology), Buel Dan Rodgers (Founder & CEO, AAVogen), Christopher Reinhard (CEO & Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)), Michael Triplett (CEO, Myonexus Therapeutics), Robert Jan Lamers (CEO, Arthrogen), Ryo Kubota (CEO, Chairman & President, Acucela), Tom Wilton (CBO, LogicBio Therapeutics), Jeffrey Hung (CCO, Vigene Biosciences), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) and Molly Cameron (Corporate Communications Manager, Orchard Therapeutics). In addition, a brief profile of each company has been provided.

Chapter 25 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 26 is an appendix, which contains a list of companies and organizations mentioned in this report.

TABLE OF CONTENTS

1. PREFACE

  • 1.1. Scope of the Report
  • 1.2. Research Methodology
  • 1.3. Key Questions Answered
  • 1.4. Chapter Outlines

2. EXECUTIVE SUMMARY

3. INTRODUCTION

  • 3.1. Context and Background
  • 3.2. Evolution of Gene Therapies
  • 3.3. Classification of Gene Therapies
    • 3.3.1. Somatic and Germline Therapies
    • 3.3.2. Ex Vivo and In Vivo Therapies
  • 3.4. Routes of Administration
  • 3.5. Mechanism of Action of Gene Therapies
  • 3.6. Overview of the Basic Concepts of Gene Editing
    • 3.6.1. Evolution of Genome Editing
    • 3.6.2. Applications of Genome Editing
    • 3.6.3. Genome Editing Techniques
  • 3.7. Advantages and Disadvantages of Gene Therapies
  • 3.8. Ethical and Social Concerns Related to Gene Therapies
  • 3.9. Constraints and Challenges Related to Gene Therapies
    • 3.9.1. Therapy Development Concerns
    • 3.9.2. Manufacturing Concerns
    • 3.9.3. Commercial Viability Concerns

4. GENE DELIVERY VECTORS

  • 4.1. Chapter Overview
  • 4.2. Viral and Non-Viral Methods of Gene Transfer
  • 4.3. Viral Vectors for Genetically Modified Therapies
  • 4.4. Types of Viral Vectors
    • 4.4.1. Adeno-associated Viral Vectors
      • 4.4.1.1. Overview
      • 4.4.1.2. Design and Manufacturing
      • 4.4.1.3. Advantages
      • 4.4.1.4. Limitations
    • 4.4.2. Adenoviral Vectors
      • 4.4.2.1. Overview
      • 4.4.2.2. Design and Manufacturing
      • 4.4.2.3. Advantages
      • 4.4.2.4. Limitations
    • 4.4.3. Lentiviral Vectors
      • 4.4.3.1. Overview
      • 4.4.3.2. Design and Manufacturing
      • 4.4.3.3. Advantages
      • 4.4.3.4. Limitations
    • 4.4.4. Retroviral Vectors
      • 4.4.4.1. Overview
      • 4.4.4.2. Design and Manufacturing
      • 4.4.4.3. Advantages
      • 4.4.4.4. Limitations
    • 4.4.5. Other Viral Vectors
      • 4.4.5.1. Alphavirus
      • 4.4.5.2. Foamy Virus
      • 4.4.5.3. Herpes Simplex Virus
      • 4.4.5.4. Sendai Virus
      • 4.4.5.5. Simian Virus
      • 4.4.5.6. Vaccinia Virus
  • 4.5. Types of Non-Viral Vectors
    • 4.5.1. Plasmid DNA
    • 4.5.2. Liposomes, Lipoplexes and Polyplexes
    • 4.5.3. Oligonucleotides
    • 4.5.4. Other Non-Viral Vectors
    • 4.5.5. Gene Delivery Using Non-Viral Vectors
      • 4.5.5.1. Biolistic Methods
      • 4.5.5.2. Electroporation
      • 4.5.5.3. Receptor Mediated Gene Delivery
      • 4.5.5.4. Gene Activated Matrix (GAM)

5. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO

  • 5.1. Chapter Overview
  • 5.2. Regulatory Guidelines in North America
    • 5.2.1. The US Scenario
    • 5.2.2. The Canadian Scenario
  • 5.3. Regulatory Guidelines in Europe
  • 5.4. Regulatory Guidelines in Asia-Pacific
    • 5.4.1. Chinese Scenario
    • 5.4.2. Japanese Scenario
    • 5.4.3. South Korean Scenario
    • 5.4.4. Australian Scenario
    • 5.4.5. Hong Kong Scenario
  • 5.5. Reimbursement Scenario
    • 5.5.1. Challenges Related to Reimbursement
  • 5.6. Commonly Offered Payment Models for Gene Therapies

6. MARKET OVERVIEW

  • 6.1. Chapter Overview
    • 6.1.1. Analysis by Phase of Development of Gene Therapy
  • 6.2. Gene Therapy Market: Clinical and Commercial Pipeline
    • 6.2.1. Analysis by Phase of Development
    • 6.2.2. Analysis by Therapeutic Area
    • 6.2.3. Analysis by Type of Vector Used
    • 6.2.4. Analysis by Type of Gene
    • 6.2.5. Analysis by Therapeutic Approach
    • 6.2.6. Analysis by Type of Gene Therapy
    • 6.2.7. Analysis by Route of Administration
  • 6.3. Gene Therapy Market: Early Stage Pipeline
    • 6.3.1. Analysis by Phase of Development
    • 6.3.2. Analysis by Therapeutic Area
    • 6.3.3. Analysis by Type of Vector Used
    • 6.3.4. Analysis by Type of Gene
    • 6.3.5. Analysis by Therapeutic Approach
    • 6.3.6. Analysis by Type of Gene Therapy
  • 6.4. Gene Therapy Candidates: Special Designations
    • 6.4.1. Analysis by Special Designation(s) Awarded
  • 6.5. Grid Representation: Analysis by Phase of Development, Therapeutic Area and Therapeutic Approach

7. COMPETITIVE LANDSCAPE

  • 7.1. Chapter Overview
  • 7.2. Gene Therapy Market: List of Developers
    • 7.2.1. Analysis by Year of Establishment
    • 7.2.2. Analysis by Company Size
    • 7.2.3. Analysis by Geography
  • 7.3. Key Players: Analysis by Number of Product Candidates

8. MARKETED GENE THERAPIES

  • 8.1. Chapter Overview
  • 8.2. Gendicine® (Shenzhen Sibiono GeneTech)
    • 8.2.1. Company Overview
    • 8.2.2. Development Timeline
    • 8.2.3. Mechanism of Action and Vectors Used
    • 8.2.4. Target Indication(s)
    • 8.2.5. Current Status of Development
    • 8.2.6. Manufacturing, Dosage and Sales
  • 8.3. Oncorine® (Shanghai Sunway Biotech)
    • 8.3.1. Company Overview
    • 8.3.2. Development Timeline
    • 8.3.3. Mechanism of Action and Vectors Used
    • 8.3.4. Target Indication(s)
    • 8.3.5. Current Status of Development
    • 8.3.6. Manufacturing, Dosage and Sales
  • 8.4. Rexin-G® (Epeius Biotechnologies)
    • 8.4.1. Company Overview
    • 8.4.2. Development Timeline
    • 8.4.3. Mechanism of Action and Vector Used
    • 8.4.4. Target Indication(s)
    • 8.4.5. Current Status of Development
    • 8.4.6. Manufacturing, Dosage and Sales
  • 8.5. Neovasculgen® (Human Stem Cells Institute)
    • 8.5.1. Company Overview
    • 8.5.2. Development Timeline
    • 8.5.3. Mechanism of Action and Vectors Used
    • 8.5.4. Target Indication(s)
    • 8.5.5. Current Status of Development
    • 8.5.6. Manufacturing, Dosage and Sales
  • 8.6. Imlygic® (Amgen)
    • 8.6.1. Company Overview
    • 8.6.2. Development Timeline
    • 8.6.3. Mechanism of Action and Vectors Used
    • 8.6.4. Target Indication(s)
    • 8.6.5. Current Status of Development
    • 8.6.6. Manufacturing, Dosage and Sales
  • 8.7. Strimvelis® (Orchard Therapeutics)
    • 8.7.1. Company Overview
    • 8.7.2. Development Timeline
    • 8.7.3. Mechanism of Action and Vectors Used
    • 8.7.4. Target Indication(s)
    • 8.7.5. Current Status of Development
    • 8.7.6. Manufacturing, Dosage and Sales
  • 8.8. Luxturna™ (Spark Therapeutics)
    • 8.8.1. Company Overview
    • 8.8.2. Development Timeline
    • 8.8.3. Mechanism of Action and Vector Used
    • 8.8.4. Target Indication(s)
    • 8.8.5. Current Status of Development
    • 8.8.6. Manufacturing, Dosage and Sales
  • 8.9. Zolgensma™ (Novartis Gene Therapies)
    • 8.9.1. Company Overview
    • 8.9.2. Development Timeline
    • 8.9.3. Mechanism of Action and Vector Used
    • 8.9.4. Target Indication(s)
    • 8.9.5. Current Status of Development
    • 8.9.6. Manufacturing, Dosage and Sales
  • 8.10. Collategene® (AnGes)
    • 8.10.1. Company Overview
    • 8.10.2. Development Timeline
    • 8.10.3. Mechanism of Action and Vector Used
    • 8.10.4. Target Indication(s)
    • 8.10.5. Current Status of Development
    • 8.10.6. Manufacturing, Dosage and Sales
  • 8.11. Zyntelgo™ (bluebird bio)
    • 8.11.1. Company Overview
    • 8.11.2. Development Timeline
    • 8.11.3. Mechanism of Action and Vector Used
    • 8.11.4. Target Indication(s)
    • 8.11.5. Current Status of Development
    • 8.11.6. Manufacturing, Dosage and Sales

9. KEY COMMERCIALIZATION STRATEGIES

  • 9.1. Chapter Overview
  • 9.2. Successful Drug Launch Strategy: ROOTS Framework
  • 9.3. Successful Drug Launch Strategy: Product Differentiation
  • 9.4. Commonly Adopted Commercialization Strategies based on Phase of Development of Product
  • 9.5. List of Currently Approved Gene Therapies
  • 9.6. Key Commercialization Strategies Adopted by Companies Focused on Gene Therapy
    • 9.6.1. Strategies Adopted Before Therapy Approval
    • 9.6.2. Strategies Adopted During / Post Therapy Approval
  • 9.7. Concluding Remarks

10. LATE STAGE (PHASE II/III AND ABOVE) GENE THERAPIES

  • 10.1. Chapter Overview
  • 10.2. GS010
  • 10.3. PTC-AADC
  • 10.4. BMN 270
  • 10.5. rAd-IFN/Syn3
  • 10.6. TAVO™
  • 10.7. OTL-200
  • 10.8. EB-101
  • 10.9. ProstAtak®
  • 10.10. BIIB111
  • 10.11. LentiGlobin BB305®
  • 10.12. Lenti-D™
  • 10.13. FCX-007
  • 10.14. CG0070
  • 10.15. Vigil®
  • 10.16. VM202
  • 10.17. VGX-3100
  • 10.18. Invossa™
  • 10.19. KB103
  • 10.20. PF-06939926
  • 10.21. PF-06838435
  • 10.22. PF-07055480
  • 10.23. SPK-8011
  • 10.24. AMT-061
  • 10.25. VB-111
  • 10.26. OTL-101
  • 10.27. BIIB112
  • 10.28. FLT180a
  • 10.29. LYS-SAF302
  • 10.30. NFS-01
  • 10.31. OTL-103

11. EMERGING TECHNOLOGIES

  • 11.1. Chapter Overview
  • 11.2. Gene Editing Technologies
    • 11.2.1. Overview
    • 11.2.2. Applications
  • 11.3. Emerging Gene Editing Platforms
    • 11.3.1. CRISPR / Cas9 System
    • 11.3.2. TALENs
    • 11.3.3. megaTAL
    • 11.3.4. Zinc Finger Nuclease
  • 11.4. Gene Expression Regulation Technologies
  • 11.5. Technology Platforms for Developing / Delivering Gene Therapies

12. KEY THERAPEUTICS AREAS

  • 12.1. Chapter Overview
  • 12.2. Analysis by Therapeutic Area and Special Designation(s) Awarded
  • 12.3. Oncological Disorders
    • 12.3.1. Analysis by Target Indication
    • 12.3.2. Analysis by Type of Vector Used
  • 12.4. Nervous System Disorders
    • 12.4.1. Analysis by Target Indication
    • 12.4.2. Analysis by Type of Vector Used
  • 12.5. Metabolic Disorders
    • 12.5.1. Analysis by Target Indication
    • 12.5.2. Analysis by Type of Vector Used
  • 12.6. Ophthalmic Diseases
    • 12.6.1. Analysis by Target Indication
    • 12.6.2. Analysis by Type of Vector Used
  • 12.7. Genetic Disorders
    • 12.7.1. Analysis by Target Indication
    • 12.7.2. Analysis by Type of Vector Used

13. PATENT ANALYSIS

  • 13.1. Chapter Overview
  • 13.2. Gene Therapy Market: List of Patents
    • 13.2.1. Scope and Methodology
      • 13.2.1.1. Analysis by Publication Year
      • 13.2.1.2. Analysis by Publication Year and Type of Patent
      • 13.2.1.3. Analysis by Geography
      • 13.2.1.4. Analysis by CPC Symbols
      • 13.2.1.5. Analysis by Emerging Focus Areas
      • 13.2.1.6. Leading Players: Analysis by Number of Patents
      • 13.2.1.7. Patent Benchmark Analysis
      • 13.2.1.8. Patent Valuation Analysis
  • 13.3. Gene Editing Market: List of Patents
    • 13.3.1. Scope and Methodology
      • 13.3.1.1. Analysis by Publication Year
      • 13.3.1.2. Analysis by Publication Year and Type of Patent
      • 13.3.1.3. Analysis by Geography
      • 13.3.1.4. Analysis by CPC Symbols
      • 13.3.1.5. Analysis by Emerging Focus Areas
      • 13.3.1.6. Leading Players: Analysis by Number of Patents
      • 13.3.1.7. Patent Benchmark Analysis
      • 13.3.1.8. Patent Valuation Analysis
  • 13.4. Overall Intellectual Property Portfolio: Analysis by Type of Organization

14. MERGERS AND ACQUISITIONS

  • 14.1. Chapter Overview
  • 14.2. Merger and Acquisition Models
  • 14.3. Gene Therapy Market: Mergers and Acquisitions
    • 14.3.1. Analysis by Year of Agreement
    • 14.3.2. Analysis by Type of Agreement
    • 14.3.3. Analysis by Geography
      • 14.3.3.1. Continent-wise Distribution
      • 14.3.3.2. Intercontinental and Intracontinental Deals
      • 14.3.3.3. Country-wise Distribution
    • 14.3.4. Analysis by Key Value Drivers
      • 14.3.4.1. Analysis by Key Value Drivers and Year of Acquisition
    • 14.3.5. Analysis by Phase of Development of the Acquired Company's Product
    • 14.3.6. Analysis by Therapeutic Area

15. FUNDING AND INVESTMENT ANALYSIS

  • 15.1. Chapter Overview
  • 15.2. Types of Funding
  • 15.3. Gene Therapy Market: Funding and Investment Analysis
    • 15.3.1. Analysis by Number of Funding Instances
    • 15.3.2. Analysis by Amount Invested
    • 15.3.3. Analysis by Type of Funding
    • 15.3.4. Analysis by Type of Funding and Year
    • 15.3.5. Analysis of Amount Invested by Type of Gene Therapy
    • 15.3.6. Analysis of Amount Invested by Geography
    • 15.3.7. Most Active Players: Analysis by Number of Funding Instances and Amount Invested
    • 15.3.8. Key Investors: Analysis by Number of Funding Instances
    • 15.3.9. Analysis by Phase of Development
    • 15.3.10. Analysis by Therapeutic Area
  • 15.4. Concluding Remarks

16. CLINICAL TRIAL ANALYSIS

  • 16.1. Chapter Overview
  • 16.2. Scope and Methodology
  • 16.3. Gene Therapy Market: Clinical Trial Analysis
    • 16.3.1. Analysis by Trial Registration Year
    • 16.3.2. Analysis by Trial Status
    • 16.3.3. Analysis by Trial Phase
    • 16.3.4. Analysis by Therapeutic Area
    • 16.3.5. Analysis by Geography
    • 16.3.6. Geographical Analysis by Trial Registration Year
    • 16.3.7. Geographical Analysis by Trial Status
    • 16.3.8. Geographical Analysis by Trial Status and Trial Phase
    • 16.3.9. Geographical Analysis by Therapeutic Area
  • 16.4. Analysis by Type of Sponsor
  • 16.5. Analysis by Prominent Treatment Sites
  • 16.6. Gene therapy Market: Analysis of Enrolled Patient Population
    • 16.6.1. Analysis by Trial Registration Year
    • 16.6.2. Analysis by Trial Status
    • 16.6.3. Analysis by Trial Phase
    • 16.6.4. Analysis by Therapeutic Area
    • 16.6.5. Analysis by Location of Trial Site
    • 16.6.6. Analysis by Trial Status and Location of Trial Site
    • 16.6.7. Analysis by Trial Status, Trial Phase and Location of Trial Site
    • 16.6.8. Analysis by Therapeutic Area and Location of Trial Site
  • 16.7. Concluding Remarks
    • 16.7.1. Emerging Molecules to Watch
    • 16.7.2. Most Important Trials to Watch

17. COST PRICE ANALYSIS

  • 17.1. Chapter Overview
  • 17.2. Gene Therapy Market: Factors Contributing to the Price of Gene Therapies
  • 17.3. Gene Therapy Market: Pricing Models
    • 17.3.1. Based on Associated Product / Component Costs
    • 17.3.2. Based on Competition
    • 17.3.3. Based on Patient Segment
    • 17.3.4. Based on Opinions of Industry Experts

18. BIG PHARMA PLAYERS: ANALYSIS OF GENE THERAPY RELATED INITIATIVES

  • 18.1. Chapter Overview
  • 18.2. Gene Therapy Market: List of Most Prominent Big Pharmaceutical Players
    • 18.2.1. Analysis by Therapeutic Area
    • 18.2.2. Analysis by Type of Vector Used
    • 18.2.3. Analysis by Therapeutic Approach
    • 18.2.4. Analysis by Type of Gene Therapy
  • 18.3. Other Big Pharma Players

19. DEMAND ANALYSIS

  • 19.1. Chapter Overview
  • 19.2. Assumptions and Methodology
  • 19.3. Clinical Demand for Gene Therapies
  • 19.4. Commercial Demand for Gene Therapies

20. MARKET FORECAST AND OPPORTUNITY ANALYSIS

  • 20.1. Chapter Overview
  • 20.2. Scope and Limitations
  • 20.3. Key Assumptions and Forecast Methodology
  • 20.4. Global Gene Therapy Market, 2020-2030
    • 20.4.1. Gene Therapy Market: Analysis by Type of Gene Modification
    • 20.4.2. Gene Therapy Market: Analysis by Type of Therapy
    • 20.4.3. Gene Therapy Market: Analysis by Type of Vector Used
    • 20.4.4. Gene Therapy Market: Analysis by Therapeutic Area
    • 20.4.5. Gene Therapy Market: Analysis by Route of Administration
    • 20.4.6. Gene Therapy Market: Analysis by Geography
  • 20.5. Gene Therapy Market: Value Creation Analysis
  • 20.6. Gene Therapy Market: Product-wise Sales Forecasts
    • 20.6.1. Gendicine®
      • 20.6.1.1. Target Patient Population
      • 20.6.1.2. Sales Forecast
      • 20.6.1.3. Net Present Value
      • 20.6.1.4. Value Creation Analysis
    • 20.6.2. Oncorine®
      • 20.6.2.1. Target Patient Population
      • 20.6.2.2. Sales Forecast
      • 20.6.2.3. Net Present Value
      • 20.6.2.4. Value Creation Analysis
    • 20.6.3. Rexin-G®
      • 20.6.3.1. Target Patient Population
      • 20.6.3.2. Sales Forecast
      • 20.6.3.3. Net Present Value
      • 20.6.3.4. Value Creation Analysis
    • 20.6.4. Neovasculgen®
      • 20.6.4.1. Target Patient Population
      • 20.6.4.2. Sales Forecast
      • 20.6.4.3. Net Present Value
      • 20.6.4.4. Value Creation Analysis
    • 20.6.5. Strimvelis®
      • 20.6.5.1. Target Patient Population
      • 20.6.5.2. Sales Forecast
      • 20.6.5.3. Net Present Value
      • 20.6.5.4. Value Creation Analysis
    • 20.6.6. Imlygic®
      • 20.6.6.1. Target Patient Population
      • 20.6.6.2. Sales Forecast
      • 20.6.6.3. Net Present Value
      • 20.6.6.4. Value Creation Analysis
    • 20.6.7. Luxturna™
      • 20.6.7.1. Target Patient Population
      • 20.6.7.2. Sales Forecast
      • 20.6.7.3. Net Present Value
      • 20.6.7.4. Value Creation Analysis
    • 20.6.8. Zolgensma™
      • 20.6.8.1. Target Patient Population
      • 20.6.8.2. Sales Forecast
      • 20.6.8.3. Net Present Value
      • 20.6.8.4. Value Creation Analysis
    • 20.6.9. Collategene®
      • 20.6.9.1. Target Patient Population
      • 20.6.9.2. Sales Forecast
      • 20.6.9.3. Net Present Value
      • 20.6.9.4. Value Creation Analysis
    • 20.6.10. Zyntelgo™
      • 20.6.10.1. Target Patient Population
      • 20.6.10.2. Sales Forecast
      • 20.6.10.3. Net Present Value
      • 20.6.10.4. Value Creation Analysis
    • 20.6.11. GS010
      • 20.6.11.1. Target Patient Population
      • 20.6.11.2. Sales Forecast
      • 20.6.11.3. Net Present Value
      • 20.6.11.4. Value Creation Analysis
    • 20.6.12. PTC-AADC
      • 20.6.12.1. Target Patient Population
      • 20.6.12.2. Sales Forecast
      • 20.6.12.3. Net Present Value
      • 20.6.12.4. Value Creation Analysis
    • 20.6.13. BMN 270
      • 20.6.13.1. Target Patient Population
      • 20.6.13.2. Sales Forecast
      • 20.6.13.3. Net Present Value
      • 20.6.13.4. Value Creation Analysis
    • 20.6.14. rAd-IFN/Syn3
      • 20.6.14.1. Target Patient Population
      • 20.6.14.2. Sales Forecast
      • 20.6.14.3. Net Present Value
      • 20.6.14.4. Value Creation Analysis
    • 20.6.15. TAVO™
      • 20.6.15.1. Target Patient Population
      • 20.6.15.2. Sales Forecast
      • 20.6.15.3. Net Present Value
      • 20.6.15.4. Value Creation Analysis
    • 20.6.16. OTL-200
      • 20.6.16.1. Target Patient Population
      • 20.6.16.2. Sales Forecast
      • 20.6.16.3. Net Present Value
      • 20.6.16.4. Value Creation Analysis
    • 20.6.17. EB-101
      • 20.6.17.1. Target Patient Population
      • 20.6.17.2. Sales Forecast
      • 20.6.17.3. Net Present Value
      • 20.6.17.4. Value Creation Analysis
    • 20.6.18. ProstAtak®
      • 20.6.18.1. Target Patient Population
      • 20.6.18.2. Sales Forecast
      • 20.6.18.3. Net Present Value
      • 20.6.18.4. Value Creation Analysis
    • 20.6.19. BIIB111
      • 20.6.19.1. Target Patient Population
      • 20.6.19.2. Sales Forecast
      • 20.6.19.3. Net Present Value
      • 20.6.19.4. Value Creation Analysis
    • 20.6.20. LentiGlobin BB305®
      • 20.6.20.1. Target Patient Population
      • 20.6.20.2. Sales Forecast
      • 20.6.20.3. Net Present Value
      • 20.6.20.4. Value Creation Analysis
    • 20.6.21. Lenti-D™
      • 20.6.21.1. Target Patient Population
      • 20.6.21.2. Sales Forecast
      • 20.6.21.3. Net Present Value
      • 20.6.21.4. Value Creation Analysis
    • 20.6.22. FCX-007
      • 20.6.22.1. Target Patient Population
      • 20.6.22.2. Sales Forecast
      • 20.6.22.3. Net Present Value
      • 20.6.22.4. Value Creation Analysis
    • 20.6.23. CG0070
      • 20.6.23.1. Target Patient Population
      • 20.6.23.2. Sales Forecast
      • 20.6.23.3. Net Present Value
      • 20.6.23.4. Value Creation Analysis
    • 20.6.24. Vigil®
      • 20.6.24.1. Target Patient Population
      • 20.6.24.2. Sales Forecast
      • 20.6.24.3. Net Present Value
      • 20.6.24.4. Value Creation Analysis
    • 20.6.25. VM202
      • 20.6.25.1. Target Patient Population
      • 20.6.25.2. Sales Forecast
      • 20.6.25.3. Net Present Value
      • 20.6.25.4. Value Creation Analysis
    • 20.6.26. VGX-3100
      • 20.6.26.1. Target Patient Population
      • 20.6.26.2. Sales Forecast
      • 20.6.26.3. Net Present Value
      • 20.6.26.4. Value Creation Analysis
    • 20.6.27. Invossa™
      • 20.6.27.1. Target Patient Population
      • 20.6.27.2. Sales Forecast
      • 20.6.27.3. Net Present Value
      • 20.6.27.4. Value Creation Analysis
    • 20.6.28. KB103
      • 20.6.28.1. Target Patient Population
      • 20.6.28.2. Sales Forecast
      • 20.6.28.3. Net Present Value
      • 20.6.28.4. Value Creation Analysis
    • 20.6.29. PF-06939926
      • 20.6.29.1. Target Patient Population
      • 20.6.29.2. Sales Forecast
      • 20.6.29.3. Net Present Value
      • 20.6.29.4. Value Creation Analysis
    • 20.6.30. PF-06838435
      • 20.6.30.1. Target Patient Population
      • 20.6.30.2. Sales Forecast
      • 20.6.30.3. Net Present Value
      • 20.6.30.4. Value Creation Analysis
    • 20.6.31. PF-07055480
      • 20.6.31.1. Target Patient Population
      • 20.6.31.2. Sales Forecast
      • 20.6.31.3. Net Present Value
      • 20.6.31.4. Value Creation Analysis
    • 20.6.32. SPK-8011
      • 20.6.32.1. Target Patient Population
      • 20.6.32.2. Sales Forecast
      • 20.6.32.3. Net Present Value
      • 20.6.32.4. Value Creation Analysis
    • 20.6.33. AMT-061
      • 20.6.33.1. Target Patient Population
      • 20.6.33.2. Sales Forecast
      • 20.6.33.3. Net Present Value
      • 20.6.33.4. Value Creation Analysis
    • 20.6.34. VB-111
      • 20.6.34.1. Target Patient Population
      • 20.6.34.2. Sales Forecast
      • 20.6.34.3. Net Present Value
      • 20.6.34.4. Value Creation Analysis
    • 20.6.35. OTL-101
      • 20.6.35.1. Target Patient Population
      • 20.6.35.2. Sales Forecast
      • 20.6.35.3. Net Present Value
      • 20.6.35.4. Value Creation Analysis
    • 20.6.36. BIIB112
      • 20.6.36.1. Target Patient Population
      • 20.6.36.2. Sales Forecast
      • 20.6.36.3. Net Present Value
      • 20.6.36.4. Value Creation Analysis
    • 20.6.37. FLT180a
      • 20.6.37.1. Target Patient Population
      • 20.6.37.2. Sales Forecast
      • 20.6.37.3. Net Present Value
      • 20.6.37.4. Value Creation Analysis
    • 20.6.38. LYS-SAF302
      • 20.6.38.1. Target Patient Population
      • 20.6.38.2. Sales Forecast
      • 20.6.38.3. Net Present Value
      • 20.6.38.4. Value Creation Analysis
    • 20.6.39. NFS-01
      • 20.6.39.1. Target Patient Population
      • 20.6.39.2. Sales Forecast
      • 20.6.39.3. Net Present Value
      • 20.6.39.4. Value Creation Analysis
    • 20.6.40. OTL-103
      • 20.6.40.1. Target Patient Population
      • 20.6.40.2. Sales Forecast
      • 20.6.40.3. Net Present Value
      • 20.6.40.4. Value Creation Analysis

21. VECTOR MANUFACTURING

  • 21.1. Chapter Overview
  • 21.2. Overview of Viral Vector Manufacturing
  • 21.3. Viral Vector Manufacturing Processes
    • 21.3.1. Mode of Vector Production
    • 21.3.2. Adherent and Suspension Cultures
    • 21.3.3. Unit and Parallel Processes
    • 21.3.4. Cell Culture Systems for Production of Viral Vectors
    • 21.3.5. Culture Media Specifications
  • 21.4. Bioprocessing of Viral Vectors
    • 21.4.1. Adeno-associated Viral Vector Production
    • 21.4.2. Adenoviral Vector Production
    • 21.4.3. Lentiviral Vector Production
    • 21.4.4. ? -Retroviral Vector Production
  • 21.5. Challenges Associated with Vector Manufacturing
  • 21.6. Companies Offering Contract Services for Viral and Plasmid Vectors

22. CASE STUDY: GENE THERAPY SUPPLY CHAIN

  • 22.1. Chapter Overview
  • 22.2. Overview of the Gene Therapy Supply Chain
  • 22.3. Implementation of Supply Chain Models
  • 22.4. Logistics Associated with Gene Therapies
    • 22.4.1. Autologous and Allogeneic Therapies
  • 22.5. Regulatory Supply Chain Across the Globe
  • 22.6. Challenges Associated with Gene Therapy Supply Chain
  • 22.7. Optimizing Cell and Advanced Therapies Supply Chain Management
    • 22.7.1. Enterprise Manufacturing Systems
    • 22.7.2. Laboratory Information Management Systems
    • 22.7.3. Inventory Management Systems
    • 22.7.4. Quality Management Systems
    • 22.7.5. Logistics Management Systems
    • 22.7.6. Patient Management Systems
    • 22.7.7. Electronic Clinical Outcome Assessments Systems
    • 22.7.8. Supply Chain Orchestration Platforms
  • 22.8. Recent Developments and Upcoming Trends

23. CONCLUSION

24. INTERVIEW TRANSCRIPTS

  • 24.1. Chapter Overview
  • 24.2. Adam Rogers, CEO, Hemera Biosciences
  • 24.3. Al Hawkins, CEO, Milo Biotechnology
  • 24.4. Buel Dan Rodgers, Founder and CEO, AAVogen
  • 24.5. Christopher Reinhard, CEO and Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)
  • 24.6. Michael Tripletti, CEO, Myonexus Therapeutics
  • 24.7. Robert Jan Lamers, CEO, Arthrogen
  • 24.8. Ryo Kubota, CEO, Chairman and President, Acucela
  • 24.9. Tom Wilton, CBO, LogicBio Therapeutics
  • 24.10. Jeffrey Hung, CCO, Vigene Biosciences
  • 24.11. Cedric Szpirer, Executive & Scientific Director, Delphi Genetics
  • 24.12. Marco Schmeer, Project Manager and Tatjana Buchholz, Marketing Manager, PlasmidFactory
  • 24.13. Molly Cameron, Corporate Communications Manager, Orchard Therapeutics

25. APPENDIX 1: TABULATED DATA

26. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS