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遺伝子治療の世界市場の分析・予測:〜2022年

Global Gene Therapy Market Analysis & Forecast to 2022

発行 Kelly Scientific Publications 商品コード 528231
出版日 ページ情報 英文 310 Pages
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遺伝子治療の世界市場の分析・予測:〜2022年 Global Gene Therapy Market Analysis & Forecast to 2022
出版日: 2018年03月01日 ページ情報: 英文 310 Pages
概要

当レポートでは、遺伝子治療の市場を調査し、現在の世界市場規模と予測、治療領域・地域・技術別による市場規模と予測、遺伝子治療技術・治療遺伝子の開発に関連した課題・遺伝子治療のデメリットに関する考察、製品情報、地域別による遺伝子治療薬の市販化状況、ベクターとして使用される各種ウィルスの詳細分析、および主要企業のプロファイルなどをまとめています。

第1章 イントロダクション

第2章 遺伝子治療:基本

  • 遺伝子治療技術
  • 遺伝子治療のルート
  • CRISPR:遺伝子治療における最新技術
  • 遺伝子治療に関連した課題
  • 遺伝子治療のデメリット
  • 製品候補・製品のコンセプト

第3章 遺伝子治療の歴史的な概要

  • 遺伝子治療の形成期
  • 遺伝子治療の実りの多い年

第4章 遺伝子治療における製品候補:フェーズIII

  • CardioNovo (Generx)
  • Collategene
  • LentiGlobin
  • Lenti-D
  • VM-202
  • Invossa
  • GS010
  • ADA-Lentiviral Gene Therapy

第5章 遺伝子治療における製品候補:フェーズII

第6章 遺伝子治療薬の市販化・コスト・認可

  • 法外な価格の理由
  • 遺伝子治療薬を手頃な価格にするための戦略
  • 遺伝子治療薬の史上初の認可の年

第7章 遺伝子治療産業の強み

  • 適切な遺伝子デリバリー媒体の開発
  • CRISPRベースの治療薬
  • 早期の臨床遺伝子治療薬プロトコル
  • 世界における遺伝子治療薬の臨床試験の状況
  • 大手製薬企業は遺伝子治療薬へ進出

第8章 遺伝子デリバリーのベクター

  • 非ウィルスベクター
  • 遺伝子治療向けウィルスベクター

第9章 遺伝子治療薬の臨床アプリケーション

  • レーバー先天性黒内障 (LCA)
  • シュタルガルト病 (SMD)
  • 先天性脈絡膜欠如
  • レーバー遺伝性視神経萎縮症 (LHON)
  • パーキンソン病
  • 脊髄性筋萎縮症
  • アルツハイマー病
  • 嚢胞性線維症 (CF)
  • X 連鎖性副腎白質ジストロフィー、ほか

第10章 市場分析

  • 世界の遺伝子治療市場:市場セグメント別
  • 世界の遺伝子治療市場:地域別
  • 中国における遺伝子治療薬の市販化
  • フィリピンにおける遺伝子治療薬の市販化
  • ロシアにおける遺伝子治療薬の市販化
  • 欧州における遺伝子治療薬の市販化
  • 米国における遺伝子治療
  • 韓国における遺伝子治療薬の市販化
  • 既存・見込み企業にとっての課題
  • 遺伝子治療の将来展望
  • 遺伝子治療製品候補薬の潜在的市場

第11章 ウィルスベクターの製造・物流

  • ウィルスベクターの主な製造企業
  • 治験におけるAVVベクターが標的と知る主な疾患
  • レンチウイルスベクターを開発する主要企業
  • 製造工程
  • 請負製造
  • 治療遺伝子の標的化遺伝子導入
  • 遺伝子治療の物流戦略
  • 遺伝子治療を含めたバイオ医薬品の臨床試験のコスト

第12章 企業プロファイル

目次
Product Code: 2018GT

Growing at a CAGR of over x% the global gene therapy market is forecast to hit $363 million by 2022 from $x million in 2017. Strengthened by recent approvals of Kymriah, Yescarta and Luxturna in the US, and a committed European, Japanese and Chinese environment, gene therapy is set to become a significant player in the bio-pharmaceutical industry. The space covers many therapeutic areas specifically, oncology, rare diseases, Parkinson's, HIV, severe combined immuno-deficiencies (SCID) and hemophilia. Gene therapy is driven by over 2,200 clinical trials globally, with over 55% of this occurring in the US, followed by Europe, Canada and China. Recently, in November 2017, the FDA indicated that gene therapies will now qualify for a fast approval process, which will bring more therapies to market faster. However, the space also has significant challenges, such as manufacturing logistics, reimbursement and its high cost. This 310 page market analysis cutting-edge report tackles this growing but challenging industry, it highlights its strengths, weaknesses and opportunities and provides a comprehensive account of major companies, clinical trials and technological advancement.

Since the FDA approved Kymriah (tisagenlecleucel), Yescarta (Axicabtagene ciloleucel) and Luxturna (voretigene neparvovec-rzyl) in 2017, the US gene therapy space has expanded significantly, underlined by the fact that over 55% of completed and ongoing trials are located in this geographic. Growth in the gene therapy industry has resulted in new commercial initiatives and the emergence of new startups and spin-off biotechs. Furthermore, gene therapy specifically has raised well over $600 million of venture capital in the last five years. Early stage companies have raised seed, Series A and Series B investment steadily since the market took off, including Spark Therapeutics, Avalanche Biotech, uniQure, Voyager Therapeutics, Editas Medicine and GenSight.

In 2017, the gene therapy market for technologies, services and products was estimated to be worth $x million, with a potential to reach $363 million by 2022. The main market space is cancer which currently holds x% market share. This indication generated $x million in 2017 and will generate $x million in 2022. This is followed by rare diseases, cardiovascular, neurological and ocular indications. Looking at the market by technology, at present, gene product therapeutics generate the majority of revenue with over $x million in 2017, growing to $x million by 2022. Viral vectors are set to generate $x million in 2017, and will rise to $x million in 2022, and by then gene therapy services such as vector development and transfection will hit $x million. At present, the Americas have penetrated the market significantly with 65% geographic share, followed by Europe (x%) and the RoW (x%).

Gene therapy products approved between the years 2003 and 2017 include Gendicine, Oncorine, Rexin-G, Neovasculgen, Glybera, Imlygic, Strimvelis, Zalmoxis, Kymriah, Yescarta and Luxturna. Gendicine was approved for head and neck squamous cell carcinoma and has been in the Chinese market since 2003. Rexin-G was approved in the Philippines back in 2007 for the treatment of primary and metastatic cancer. Oncorine was approved in China in 2005 for nasopharyngeal carcinoma. The Russian market has Neovasculgen from 2011 for the treatment of peripheral arterial disease (PAD) and critical limb ischemia. The first gene therapy approved in E.U. was Glybera in 2012 for the treatment of familial lipoprotein lipase deficiency (LPL), however in October 2017 it was pulled from the market due to lack of patient demand. In 2015, Imlygic was approved in E.U. and also in the U.S. to treat melanoma, and Phase II results released in 2017 indicated its efficacy in combination with the checkpoint-inhibitor, Yervoy. In the E.U., Strimvelis was approved in 2016 for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID). In 2016, Zalmoxis was approved in E.U. for the treatment of leukemia. 2017 was a bumper year for gene therapy with Kymriah, Yescarta and Luxturna all gaining FDA approval.

Renewed interest has encouraged start-up companies to affiliate with academic centers for tech know-how. As clinical trials advance towards licensure, more meticulous product characterization using improved analytical methods and progressively higher regulatory compliance will be required. Some of the ongoing clinical trials are closing on to produce promising results, including one for hemophilia B caused by the deficiency of Factor IX using a recombinant adeno-associated virus (AAV) as a vector. The product candidate if succeeds will be a relatively cheaper alternative to the expensive and lifelong factor replacement therapy.

A second example of a successful outcome in gene therapy are studies conducted by independent laboratories focusing on sub-retinal delivery of recombinant AAV expressing retinal pigment epithelial RPE65 for Leber Congenital Amaurosis Type 2. A third example is the clinical trial involving nine children with X-linked severe combined immunodeficiency (SCID-X1) treated with autologous bone marrow CD34+ cells transduced with a self-inactivating (SIN) γ-retroviral vector expressing the IL-2 receptor γ-chain. The most significant achievement in gene therapy is the spectacular clinical results obtained by many independent teams using CAR-T-cell technology. This novel strategy involves ex vivo gene transfer using recombinant retroviral or lentiviral vectors of chimeric antigen receptors consisting of antibody-binding domains fused to T-cell-signaling domains into patient T lymphocytes.

As gene therapies are generally meant for one time or short duration treatments, they are customized to individuals confined to small patient populations. Therefore, manufacturing firms are expected to seek premium prices for these therapies. Because of this, these therapies will have to face valuation and reimbursement challenges. Stakeholders will show reservations about the hefty price tags and they will require significant data to be convinced. With the removal of Gylbera from the EU market in 2017, due to the fact that only one patient was treated with the drug, all eyes are focused on the number of end patients that will be treated, and their ability to pay. To that end, launching of new drugs may have to be delayed in order to collect more data for payers. Furthermore, annuity based reimbursement agreements and pay-for-performance scenarios will have to be tackled.

This report provides the reader with:

  • Current Global Market Worth and Forecast with CAGR Through 2022
  • Sub-Market Worth by Therapeutic Area (Cancer, Rare Diseases, Cardiovascular, Neurological, Ocular) and Forecast with CAGR Through 2022
  • Sub-Market Worth by Geography (Americas, Europe, RoW) and Forecast with CAGR Through 2022
  • Sub-Market Worth by Technology (Gene Product, Service, Viral Vectors) and Forecast with CAGR Through 2022
  • Insight into gene therapy technologies, challenges associated with developing therapeutic genes and disadvantages of gene therapy.
  • Full outline of the gene therapy industry from the formative years through to products discovered during 1990 and 2017.
  • Detailed descriptions of commercialized products approved between 2003 and 2017 that include: Gendicine, Rexin-G, Oncorine, Neovasculgen, Glybera, Imlygic, Strimvelis, Zalmoxis, Kymriah, Yescarta and Luxturna.
  • Description of seven of the Phase III product candidates that include: Generx, Collategene, LentiGlobin, Lenti-D, VM-202, Invosa and GS-010.
  • Description of 21 Phase II product candidates that are set to have significant market share.
  • Commercialization status of gene therapies in by geographic region
  • Evaluation of gene therapy pricing
  • Description of the firstever warranty offer by GSK for Strimvelis.
  • A detailed analysis of various types of viruses used as vectors.
  • Description of clinical applications of gene therapy and the various genetic and infectious diseases addressed by gene therapy.
  • Description of 77 companies that are directly and indirectly associated with gene therapy industry.

Key Questions Answered in this Report:

  • What is the size of gene therapy market?
  • What is the CAGR and market size over the next five years?
  • What are the different sub-markets and their worth/CAGR over the next five years?
  • What is gene augmentation therapy?
  • What is suicide gene therapy?
  • How is ex vivo gene delivery different from in vivo gene delivery?
  • What are the types of gene therapies classified on the basis of targeted cell types?
  • What is the role of CRISPR technology in gene therapy?
  • What are the approved gene therapy products?
  • How many gene therapy product candidates have reached the Phase III stage?
  • How many Phase II gene therapy product candidates are there?
  • What is the commercialization status of gene therapies in E.U. member countries?
  • What are the prices of gene therapy products?
  • What are the reasons for this extortionate pricing of gene therapies?
  • Which company is offering warranty for its gene- therapy product?
  • What is the current strength of gene therapy industry?
  • Is it true that the real strength of gene therapy industry is based on the number of clinical trials?
  • What is the total number of ongoing clinical trials as of 2017?
  • What is the distribution of clinical trials by geography?
  • Which countries are associated with gene therapy clinical trials?
  • What are the major indications addressed by the clinical trials?
  • Which genes are transferred in these clinical trials?
  • How many Big Pharma are associated with the gene therapy industry?
  • What are non-viral and viral vectors?
  • What are the various features of viral vectors?
  • Which viral vectors are predominantly used in gene therapy clinical trials?
  • What are the major diseases addressed by therapeutic genes?
  • Where is the gene therapy market heading, and what opportunities and challenges will it face?

Table of Contents

1.0 Introduction

  • 1.1 Executive Summary
  • 1.2 About this Report
  • 1.3 Key Questions Answered in this Report

2.0 Gene Therapy: The Basics

  • 2.1 Gene Therapy Techniques
    • 2.1.1 Gene Augmentation Therapy
    • 2.1.2 Gene Inhibition Therapy
    • 2.1.3 Suicide Gene Therapy
  • 2.2 Gene Therapy Routes
    • 2.2.1 Ex Vivo Gene Delivery Route
    • 2.2.2 In vivo Gene Delivery Route
  • 2.3 CRISPR: Latest Technology in Gene Therapy
  • 2.4 Challenges Associated with Developing Gene Therapies
  • 2.5 Disadvantages of Gene Therapy
    • 2.5.1 Short-Lived Nature of Gene Therapy
    • 2.5.2 Immune Response
    • 2.5.3 Problems with Viral Vectors
    • 2.5.4 Multigenic Disorders
    • 2.5.5 Insertional Mutagenesis
  • 2.6 Concept to Product Candidates and Products

3.0 Historic Overview of Gene Therapy

  • 3.1 The Formative Years of Gene Therapy
  • 3.2 The Productive Years of Gene Therapy
    • 3.2.1 Gendicine
    • 3.2.2 Oncorine
    • 3.2.3 Rexin-G
    • 3.2.4 Neovasculgen
    • 3.2.5 Glybera
    • 3.2.6 Imlygic
    • 3.2.7 Strimvelis
    • 3.2.8 Zalmoxis

4.0 Gene Therapy Phase III Product Candidates

  • 4.1 CardioNovo (Generx)
  • 4.2 Collategene
  • 4.3 LentiGlobin
  • 4.4 Lenti-D
  • 4.5 VM-202
  • 4.6 Invossa
  • 4.7 GS010
  • 4.8 ADA-Lentiviral Gene Therapy

5.0 Gene Therapy Phase II Product Candidates

  • 5.1 hF-IX gene/Hemophilia B
  • 5.2 NaGlu gene/San Fillipo B Syndrome
  • 5.3 StarGen/Stargardt disease
  • 5.4 UshStat/Usher Syndrome Type1B
  • 5.5 Retinostat/Wet AMD
  • 5.6 Lenti-D/Childhood Cerebral ALD
  • 5.7 LentiGlobin/Beta Thalassemia
  • 5.8 SB-728/HIV/AIDS
  • 5.9 CERE-110/Alzheimer's disease
  • 5.10 AATD/Alpha-1 Antitrypsin deficiency
  • 5.11 RS1 gene/X-linked juvenile Retinoschisis (XLRS)
  • 5.12 SPK-CHM/Choroideremia
  • 5.13 SPK-FIX/Hemophilia B
  • 5.14 AMG0001/Primary Lymphedema
  • 5.15 AMG0001/Ischemic heart disease
  • 5.16 WASp /Wiskott-Aldrich syndrome
  • 5.17 X-linked CGD
  • 5.18 GSK2696273/ADA gene
  • 5.19 VM106/Chronic Granulomatous Disease
  • 5.20 VY-AADC01/Parkinson's disease

6.0 Commercialization, Cost and Warranty of Gene Therapies

  • 6.1 Reasons for the Extortionate Price
    • 6.1.1 High Cost of Manufacturing
    • 6.1.2 High Cost of Delivery
    • 6.1.3 Lack of Comparative Studies as Evidence for Reimbursement Scheme
    • 6.1.4 Lack of Competition
    • 6.1.5 Potential for Cure
  • 6.2 Strategies to Make Gene Therapy Affordable
  • 6.3 The Age of First-Ever Warranty for Gene Therapy

7.0 The Strength of Gene Therapy Industry

  • 7.1 Development of Appropriate Gene Delivery Vehicles
  • 7.2 CRISPR-Based Therapy
  • 7.3 Early Clinical Gene Therapy Protocols
  • 7.4 Worldwide Scenario of Gene Therapy Clinical Trials
    • 7.4.1 Geographical Distribution of Gene Therapy Clinical Trials
    • 7.4.2 Gene Therapy Clinical Trials by Country
    • 7.4.3 Major Indications Addressed by Gene Therapy Clinical Trials
    • 7.4.4 Gene Types Transferred
    • 7.4.5 Status of Gene Therapy Clinical Trials
  • 7.5 Big Pharma's Bold Venturing into Gene Therapy
    • 7.5.1 Novartis' Deal with GenVec
    • 7.5.2 Strategic Alliance between GlaxoSmithKline and Fondazione Telethon
    • 7.5.3 Amgen's Acquisition of BioVex
    • 7.5.4 Chiesi's Rights to uniQure's Glybera
    • 7.5.5 Bayer's Collaboration with Dimension Therapeutics
    • 7.5.6 Genzyme's (Sanofi) Collaboration with University of Florida
    • 7.5.7 Pfizer's Collaboration with Spark Therapeutics
    • 7.5.8 Biogen Idec's Collaboration with AGCT
    • 7.5.9 Sanofi's Tie-Up with Voyager Therapeutics
    • 7.5.10 Bristol Myers Squibb's Investment in uniQure
    • 7.5.11 Joint Venture between Bayer and CRISPR Therapeutics AG
    • 7.5.12 Flow of Funds to Gene Therapy Companies
  • 7.6 Venture Investments by Body Systems

8.0 Vectors for Gene Delivery

  • 8.1 Non-Viral Vectors
    • 8.1.1 Inorganic Particles
      • 8.1.1.1 Poly(lactic-co-glycolic acid) (PLGA) and poly lactic acid (PLA)
      • 8.1.1.2 Chitosan
      • 8.1.1.3 Poly(ethylene imine) (PEI)
      • 8.1.1.4 Dendrimers
      • 8.1.1.5 Polymethacrylates
    • 8.1.2 Synthetic/Natural Biodegradable Particles
      • 8.1.2.1 Cationic Liposomes
      • 8.1.2.2 Lipid Nanoemulsions
      • 8.1.2.3 Solid Lipid Nanoparticles (SLN)
      • 8.1.2.4 Poly-L-Lysine
    • 8.1.3 Physical Methods
      • 8.1.3.1 Needle Injection
      • 8.1.3.2 Gene Gun
      • 8.1.3.3 Electroporation
      • 8.1.3.4 Sonoporation
      • 8.1.3.5 Photoporation
      • 8.1.3.6 Magnetofection
      • 8.1.3.7 Hydroporation
  • 8.2 Viral Vectors for Gene Therapy
    • 8.2.1 Key Properties of Viral Vectors
    • 8.2.2 Best-Suited Viral Vectors for Gene Therapy
    • 8.2.3 Potential Barriers for the Use of Viral Vectors
    • 8.2.4 Types of Viral Vectors
      • 8.2.4.1 Adenovirus Vectors
      • 8.2.4.2 Retrovirus Vectors
      • 8.2.4.3 Adeno-Associated Virus (AAV)
      • 8.2.4.4 Lentivirus Vectors
      • 8.2.4.5 Vaccinia Virus
    • 8.2.5 Clinical Translation of Viral Vectors
    • 8.2.6 Viral Vectors used in Clinical Trials
    • 8.2.7 Culture Systems for Viral Vector Production

9.0 Clinical Applications of Gene Therapy

  • 9.1 Leber Congenital Amaurosis (LCA)
    • 9.1.1 Frequency
    • 9.1.2 Genetic Basis of Leber Congenital Amaurosis
    • 9.1.3 Pattern of Inheritance
    • 9.1.4 Gene Therapy for LCA
  • 9.2 Stargardt Macular Degeneration (SMD)
    • 9.2.1 Frequency of SMD
    • 9.2.2 Genetic Basis of SMD
    • 9.2.3 Pattern of Inheritance
    • 9.2.4 Gene Therapy
  • 9.3 Choroideremia
    • 9.3.1 Frequency
    • 9.3.2 Genetic Basis of Choroideremia
    • 9.3.3 Pattern of Inheritance
    • 9.3.4 Gene Therapy
  • 9.4 Leber Hereditary Optic Neuropathy (LHON)
    • 9.4.1 Frequency of LHON
    • 9.4.2 Genetic Basis of LHON
    • 9.4.3 Pattern of Inheritance
    • 9.4.4 Gene Therapy
  • 9.5 Parkinson Disease
    • 9.5.1 Frequency of Parkinson Disease
    • 9.5.2 Genetic Basis of Parkinson Disease
    • 9.5.3 Inheritance of Parkinson Disease
    • 9.5.4 Current Treatment for Parkinson Disease
    • 9.5.5 Gene Therapy for Parkinson Disease
  • 9.6 Spinal Muscle Atrophy
    • 9.6.1 Genetic Basis
    • 9.6.2 Pattern of Inheritance
    • 9.6.3 Gene Therapy
  • 9.7 Alzheimer Disease
    • 9.7.1 Frequency
    • 9.7.2 Genetic Basis
    • 9.7.3 Pattern of Inheritance
    • 9.7.4 Gene Therapy
  • 9.8 Cystic Fibrosis
    • 9.8.1 Genetic Basis
    • 9.8.2 Inheritance of CF
    • 9.8.3 Frequency of CF
    • 9.8.4 Currently Available Treatments for CF
    • 9.8.5 Gene Therapy for Cystic Fibrosis
  • 9.9.1 Frequency
    • 9.9.2 Genetic Basis
    • 9.9.3 Pattern of Inheritance
    • 9.9.4 Currently Available Treatments
    • 9.9.5 Gene Therapy
  • 9.10 X-Linked Adrenoleukodystrophy
    • 9.10.1 Frequency
    • 9.10.2 Genetic Basis
    • 9.10.3 Pattern of Inheritance
    • 9.10.4 Gene Therapy
  • 9.11 Pompe Disease
    • 9.11.1 Frequency
    • 9.11.2 Genetic Basis
    • 9.11.3 Pattern of Inheritance
    • 9.11.4 Gene Therapy
  • 9.12 Batten Disease (CLN3 Disease)
    • 9.12.1 Frequency of Batten Disease
    • 9.12.2 Genetic Basis of Batten Disease
    • 9.12.3 Pattern of Inheritance
    • 9.12.4 Gene Therapy for Batten Disease
  • 9.13 Metachromatic Leukodystrophy
    • 9.13.1 Frequency
    • 9.13.2 Genetic Basis
    • 9.13.3 Pattern of Inheritance
    • 9.13.4 Gene Therapy
  • 9.14 Sanfilippo Syndrome
    • 9.14.1 Frequency
    • 9.14.2 Genetic Basis
    • 9.14.3 Patern of Inheritance
    • 9.14.4 Gene Therapy
  • 9.15 Hunter Syndrome
    • 9.15.1 Genetic Basis
    • 9.15.2 Pattern of Inheritance
    • 9.15.3 Gene Therapy
  • 9.16 Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
    • 9.16.1 Frequency
    • 9.16.2 Genetic Basis
    • 9.16.3 Pattern of Inheritance
    • 9.16.4 Gene Therapy
  • 9.17 X-Linked SCID
    • 9.17.1 Frequency
    • 9.17.2 Genetic Basis
    • 9.17.3 Pattern of Inheritance
    • 9.17.4 Gene Therapy
  • 9.18 Chronic Granulomatous Disease (CGD)
    • 9.18.1 Prevalence of CGD
    • 9.18.3 Pattern of Inheritance
    • 9.18.4 Current Treatments for CGD
    • 9.18.5 Gene Therapy for CGD
  • 9.19 Wischott Aldrich syndrome (WAS)
    • 9.19.1 Frequency
    • 9.19.2 Genetic Basis
    • 9.19.3 Pattern of Inheritance
  • 9.20 Hemophilia
    • 9.20.1 Frequency of Hemophilia
    • 9.20.2 Genetic Basis
    • 9.20.3 Inheritance of Hemophilia
    • 9.20.4 Currently Available Treatments for Hemophilia
    • 9.20.5 Gene Therapy for Hemophilia
  • 9.21 Sickle Cell Anemia
    • 9.21.1 Frequency of Sickle Cell Anemia
    • 9.21.2 Genetic Basis of Sickle Cell Anemia
    • 9.21.3 Inheritance of Sickle Cell Anemia
    • 9.21.4 Current Treatment for Sickle Cell Anemia
    • 9.21.5 Gene Therapy for Sickle Cell Anemia
  • 9.22 Beta Thalassemia
    • 9.22.1 Frequency
    • 9.22.2 Genetic Basis
    • 9.22.3 Pattern of Inheritance
    • 9.22.4 Gene Therapy
  • 9.23.1 Frequency
    • 9.23.2 Genetic Basis
    • 9.23.4 Gene Therapy
  • 9.25 Limb Girdle Muscle Dystrophy2C/2D
    • 9.25.1 Frequency
    • 9.25.2 Genetic Basis
    • 9.25.3 Pattern of Inheritance
    • 9.25.4 Gene Therapy
  • 9.26 Duchenne and Becker Muscular Dystrophy
    • 9.26.1 Frequency
    • 9.26.2 Genetic Basis
    • 9.26.3 Pattern of Inheritance
  • 9.27 Human Immunodeficiency Virus (HIV)
    • 9.27.1 Currently Available Treatments
  • 9.28 Epidermolysis Bullosa
    • 9.28.1 Frequency
    • 9.28.2 Genetic Basis
    • 9.28.3 Pattern of Inheritance
    • 9.28.4 Gene Therapy
  • 9.29 Leukemia
    • 9.29.1 Currently Available Treatments
    • 9.29.2 Genetic Basis of Leukemia
    • 9.29.3 Gene Therapy Strategies for Leukemia
  • 9.30 Ovarian Cancer
    • 9.30.1 Frequency of Ovarian Cancer
    • 9.30.2 Genetic Basis of Ovarian Cancer
    • 9.30.3 Currently Available Treatments for Ovarian Cancer
    • 9.30.4 Gene Therapy for Ovarian Cancer
  • 9.31 Pancreatic Cancer
    • 9.31.1 Frequency of Pancreatic Cancer
    • 9.31.2 Currently Available Treatments for Pancreatic Cancer
    • 9.31.3 Genetic Basis
    • 9.31.4 Gene Therapy for Pancreatic Cancer
  • 9.32 Head and Neck Cancers
    • 9.32.1 Frequency of Head and Neck Cancers
    • 9.32.2 Currently Available Treatments for Head and Neck Cancers
    • 9.32.3 Gene Therapy for Head and Neck Cancers
  • 9.33 Melanoma
    • 9.33.1 Frequency of Melanoma
    • 9.33.2 Genetic Basis of Melanoma
    • 9.33.3 Pattern of Inheritance
    • 9.33.4 Available Treatments for Melanoma
    • 9.33.5 Gene Therapy for Melanoma
  • 9.34 Prostate Cancer
    • 9.34.1 Frequency of Prostate Cancer
    • 9.34.2 Genetic Basis of Prostate Cancer
    • 9.34.3 Pattern of Inheritance
    • 9.34.4 Currently Available Treatments for Prostate Cancer
    • 9.34.5 Gene Therapy for Prostate Cancer
  • 9.35 Breast Cancer
    • 9.35.1 Frequency of Breast Cancer
    • 9.35.2 Genetic Basis of Breast Cancer
    • 9.35.3 Pattern of Inheritance
    • 9.35.4 Currently Available Treatments for Breast Cancer
    • 9.35.5 Gene Therapy
  • 9.36 Fabry Disease
    • 9.36.1 Frequency of Fabry Disease
    • 9.36.2 Genetic Basis of Fabry Disease
    • 9.36.3 Pattern of Inheritance
    • 9.36.4 Currently Available Treatments for Fabry Disease
    • 9.36.5 Gene Therapy for Fabry Disease
  • 9.37 Hypercholesterolemia
    • 9.37.1 Frequency of Hypercholesterolemia
    • 9.37.2 Genetic Basis of Hypercholesterolemia
    • 9.37.3 Current Medications for Hypercholesterolemia
    • 9.37.4 Gene Therapy for Familial Hypercholesterolemia
  • 9.38 Huntington Disease
    • 9.38.1 Frequency of Huntington Disease
    • 9.38.2 Genetic Basis of Huntington Disease
    • 9.38.3 Inheritance of Huntington Disease
    • 9.38.4 Current Treatment for Huntington Disease
    • 9.38.5 Gene Therapy for Huntington Disease
  • 9.39 Tay-Sachs Disease
    • 9.39.1 Frequency of Tay-Sachs Disease
    • 9.39.2 Genetic Basis of Tay-Sachs Disease
    • 9.39.3 Pattern of Inheritance
    • 9.39.4 Currently Available Treatments
    • 9.39.5 Gene Therapy for Tay-Sachs Disease

10.0 Market Analysis

  • 10.1 Global Market for Gene Therapy by Market Segment
  • 10.2 Global Gene Therapy Market by Geography
  • 10.3 Commercialization of Gene Therapy in China
  • 10.4 Commercialization of Gene Therapy in the Philippines
  • 10.5 Commercialization of Gene Therapy in Russia
  • 10.6 Commercialization of Gene Therapy in Europe
    • 10.6.1 Gene Therapy in France
    • 10.6.2 Gene Therapy Landscape in U.K.
    • 10.6.3 Gene Therapy Efforts in Germany
    • 10.6.4 Australia's Participation in Gene Therapy
    • 10.6.5 New Regulatory Framework in Japan
  • 10.7 Gene Therapy in the U.S.
    • 10.7.1 Likely FDA Approval for Leukemia Treatment
  • 10.8 Commercialization of Gene Therapy in South Korea
    • 10.9 Additional Addresseable Markets for Gene Therapy
  • 10.10 Challenges for Existing and Prospective Players
    • 10.10.1 Challenges in Valuation
    • 10.10.2 Challenges in Reimbursement
    • 10.10.3 Challenges in Commercialization
  • 10.11 Future Outlook for Gene Therapy
  • 10.12 Potential Market for Gene Therapy Product Candidates
    • 10.12.1 CardioNovo (Generx)
    • 10.12.2 Collategene
    • 10.12.3 Lentiglobin BB305
    • 10.12.4 VM-202
    • 10.12.5 Invossa
    • 10.12.5 GS010
    • 10.12.7 ADA Lentivirus
    • 10.12.8 Lenti-D

11.0 Manufacturing of Viral Vectors and Logistics

  • 11.1 Major Manufacturing Companies of Viral Vectors
  • 11.2 Major Diseases Targeted by AAV Vectors in Clinical Trials
  • 11.3 Major Companies Developing Lentiviral Vectors
  • 11.4 Manufacturing Process
  • 11.5 Contract Manufacturing
  • 11.6 Targeted Delivery of Therapeutic Genes
  • 11.7 Logistics Strategies for Gene Therapies
    • 11.7.1 Threat to Gene Therapeutics during Transit
    • 11.7.2 Impact of Varying Environmental Events on Cell and Gene Therapy Products
    • 11.7.3 Pharmaceutical Cold Chain Logistics
    • 11.6.4 Clinical Logistics
  • 11.7 Cost of Clinical Trials for Biopharmaceuticals including Gene Therapy
    • 11.7.1 Clinical Trial Expenditure by Disease

12.0 Company Profiles

  • 12.14d Molecular Therapeutics LLC
    • 12.1.1 AAV Vectors
    • 12.1.24D's Partnership with Pfizer
    • 12.1.34D's Partnership with uniQure
    • 12.1.44D's Partnership with Roche
    • 12.1.54D's Partnership with AGTC
    • 12.1.64D's Partnership with Benitec
    • 12.1.74D's Product Pipeline
  • 12.2 Abeona Therapeutics LLC
    • 12.2.1 Abeona's Clinical Trial Programs
      • 12.2.1.1 ABO-102 Phase I/II Clinical Trial
      • 12.2.1.2 ABO-101 Phase I/II Clinical Trial
  • 12.3 Advanced Cell & Gene Therapy LLC
    • 12.3.1 Consulting Services
  • 12.4 Advantagene Inc.
    • 12.4.1 Gene Mediated Cytotoxic Immunotherapy (GMCI)
  • 12.5 Adverum Biotechnologies Inc.
  • 12.6 Addgene Inc.
    • 12.6.1 Viral Service
  • 12.7 Agilis Biotherapeutics LLC
    • 12.7.1 Agilis Engineered DNA Therapeutics
    • 12.7.2 DNA Therapeutics for AADC Deficiency
    • 12.7.3 DNA Therapeutics for Friedreich's ataxia (FA)
    • 12.7.4 DNA Therapeutics for Angelman's Syndrome
  • 12.8 Angionetics Inc.
    • 12.8.1 Technology
    • 12.8.2 Generx Therapeutic Positioning
    • 12.8.3 Product Pipeline
    • 12.8.4 Addresseable Market
  • 12.9 Applied Genetic Technologies Corporation (AGTC)
    • 12.9.1 AGTC's Technology
  • 12.10 AnGes MG Inc.
    • 12.10.1 HGF Plasmid
    • 12.10.2 NF-kB Dekoy Oligonucleotide
    • 12.10.3 DNA Therapeutic Vaccines
    • 12.10.4 AnGes' Alliance Partners
  • 12.11 Asklepios BioPharmaceutical Inc.
    • 12.11.1 Asklepios' Collaborators
  • 12.12 Audentes Therapeutics Inc.
    • 12.12.1 Audentes' Technology
  • 12.13 AveXis Inc.
    • 12.13.1 AVXS-101
  • 12.14 AvroBio Inc.
    • 12.14.1 Lentiviral Vectors for Rare Diseases
    • 12.14.2 Cytokine IL-12 for Cancer Immunotherapy
    • 12.14.3 Gene Vector
    • 12.14.4 AvroBio's Programs
  • 12.15 Benitec Biopharma
    • 12.15.1 ddRNAi Technology
    • 12.15.2 Benitec's In-House Programs
    • 12.15.3 Benitecs Licensed Programs
  • 12.16 BioCancell Therapeutic Inc.
    • 12.16.1 The H19 Gene
    • 12.16.2 BC-819
    • 12.16.3 BC-821
  • 12.17 BioMarin Pharmaceutical Inc.
    • 12.17.1 BMN
  • 12.18 Bluebird bio Inc.
    • 12.18.1 Bluebird's Program Pipeline
    • 12.18.2 Lenti-D
    • 12.18.3 LentiGlobin
    • 12.18.4 Bluebird's Partnership with Celgene
    • 12.18.5 Bluebird's Partnership with FivePrime
    • 12.18.6 Bluebird's Partnership with Kite Pharma
    • 12.18.7 Bluebird's Partnership with ViroMed
  • 12.19 Brammer Bio LLC
    • 12.19.1 Cell Therapy Services
    • 12.19.2 Ex Vivo Gene Therapy
    • 12.19.3 Viral Vector Manufacturing
  • 12.20 Cellectis S.A.
    • 12.20.1 UCART19
    • 12.20.2 UCART123
    • 12.20.3 UCART38 & UCARTCS1
    • 12.20.4 UCART22
  • 12.21 Clontech Laboratories Inc.
  • 12.22 Cobra Biologics Ltd.
    • 12.22.1 DNA Services
    • 12.22.2 Virus Services
    • 12.22.3 Protein Services
    • 12.22.4 Microbiota Services
    • 12.22.5 Fill and Finish Services
  • 12.23 Copernicus Therapeutics Inc.
    • 12.23.1 Technology
    • 12.23.2 Cystic Fibrosis (CF) Program
    • 12.23.3 Retinitis Pigmentosa (RP) Program
    • 12.23.4 Parkinson's Disease (PD) Program
  • 12.24 Dimension Therapeutics Inc.
    • 12.24.1 Dimension's Gene Therapy Programs
  • 12.25.1 SB Therapeutics
  • 12.26 Editas Medicine Inc.
    • 12.26.1 CRISPR/Cas9 & TALENs
  • 12.27 Fibrocell Sciences Inc.
    • 12.27.1 Fibroblast's Pipeline
    • 12.27.2 FCX-007
    • 12.27.3 FCX-013
  • 12.28 Florida Biologix
    • 12.28.1 Services
  • 12.29 Freeline Therapeutics Ltd.
    • 12.29.1 Freeline's Platform
  • 12.30 Genable Technologies Ltd.
    • 12.30.1 RhoNova
  • 12.31 Genethon
    • 12.31.1 Product Pipeline
  • 12.32 Genlantis
    • 12.32.1 DNA Transfection
    • 12.32.2 siRNA Transfection
    • 12.32.3 Neuronal Transfection
    • 12.32.4 Customer Services
    • 12.32.5 Protein Delivery/Transfection
    • 12.32.6 siRNA Generation Kits
    • 12.32.7 siRNA Transfection
    • 12.32.8 Dicer Enzyme Kits
  • 12.33 GenSight Biologics S.A.
    • 12.33.1 GS010
    • 12.33.2 GS030
  • 12.34.1 AdenoVerse Technology
    • 12.34.2 Antigen Discovery
    • 12.34.3 Cell Lines
    • 12.34.4 GenVec's Product Pipeline
      • 12.34.4.1 GGF166 for Hearing Loss
      • 12.34.4.2 GV2311 - RSV Vaccine
      • 12.34.4.3 GV2207 - HSV-2 Immunotherapeutic
  • 12.35 Ichor Medical Systems Inc.
    • 12.35.1 Technology
  • 12.36 Immune Design Corp.
    • 12.36.2 GLASS
  • 12.37 Immusoft Corp.
    • 12.37.1 Gene Delivery Technology
    • 12.37.2 Key Indications being Addressed
  • 12.38 Inovio Pharmaceuticals Inc.
    • 12.38.1 Inovio's Technology
    • 12.39 Intellia Therapeutics Inc.
  • 12.39.1 CRISPR/CAS9
    • 12.39.2 Intellia's Programs
  • 12.40 Juventa Therapeutics Inc.
    • 12.40.1 Non-Viral Gene Therapy
    • 12.40.2 JVS-100
  • 12.41 Kite Pharma Inc.
    • 12.41.1 Chimeric Antigen Receptor (CAR)
    • 12.41.2 T cell Receptor (TCR)
    • 12.41.3 Cancer Programs
    • 12.41.4 Kite Pharma's Partnership with National Cancer Institute (NCI)
    • 12.41.5 Kite Pharma's Partnership with The Netherlands Cancer Institute (NKI)
    • 12.41.6 Kite Pharma's Partnership with Adimab
    • 12.41.7 Kite Pharma's Collaboration with Alpine Immune Sciences (AIS)
    • 12.41.8 Kite Pharma's Collaboration with Amgen
    • 12.41.9 Kite Pharma's Partnership with Bluebird Bio
    • 12.41.10 Kite Pharma's Partnership with Cell Design Labs
    • 12.41.11 Kite Pharma's Collaboration with Genentech
    • 11.41.12 Kite Pharma's Partnership with GE Global Research
    • 12.41.13 Kite Pharma's Partnership with Leiden University Medical Center
    • 12.41.14 Kite Pharma's Partnership with Leukemia & Lymphoma Society (LLS)
    • 12.41.15 Kite Pharma's Partnership with The Tel-Aviv Sourasky Medical Center
    • 12.41.16 Kite Pharma's Partnership with The UCLA David Geffen School of Medicine
  • 12.42 Kolon Life Sciences Inc.
    • 12.42.1 Invossa
    • 12.42.2 KLS-1010
    • 12.42.3 KLS-2020
    • 12.42.4 KLS3020
  • 12.43 Lentigen Technology Inc.
    • 12.43.1 Lentiviral Vectors for Translational Research
    • 12.43.2 Lenti-Viral Vector Technology
    • 12.43.3 Clinical Trial Support
  • 12.44 Lysogene S.A.S.
    • 12.44.1 Lysogene's rAAV Vectors
    • 12.44.2 CNS Administration
    • 12.44.3 MPS IIIA
    • 12.44.4 GMI Gangliosidosis
    • 12.44.5 Partnership with Alcyone Lifesciences Inc.
    • 12.44.6 Partnership with University of Massachusetts
    • 12.44.7 Partnership with Auburn University
    • 12.44.8 Partnership with University of Manchester
  • 12.45 Medgenics Inc.
    • 12.45.1 Transduced Autologous Restorative Gene Therapy (TARGT)
  • 12.46 Mirus Bio LLC
    • 12.46.1 TransIT - Lenti Transfection Reagent
    • 12.46.2 Ingenio Electroporation Kits
  • 12.47 Mologen AG
    • 12.47.1 Technologies
    • 12.47.1.1 dSLIM
    • 12.47.1.2 EnanDIM
    • 12.47.1.3 MIDGE
    • 12.47.2 Allogeneic Tumor Cell Bank
  • 12.48 NanoCor Therapeutics Inc.
    • 12.48.1 Biological Nanoparticle (BNP) Technology
    • 12.48.2 Carfostin
  • 12.49 Nature Technology Corp. (NTC)
    • 12.49.1 Vector Cell Lines
    • 12.49.2 DNA Manufacturing
    • 12.49.3 Cloning & QC
    • 12.49.4 Protein Products
    • 12.49.5 Technology & Consulting
  • 12.50 NightstaRx Ltd.
    • 12.50.1 Project AAV2 REP1
  • 12.51 Novasep Process SAS
    • 12.51.1 Manufacturing Services for Customers
    • 12.51.2 Contract Manufacturing Services
    • 12.51.3 Purification Technologies
  • 12.52 Omnia Biologics Inc.
    • 12.52.1 Services
      • 12.52.1.1 Preclinical and GMP Manufacturing
      • 12.52.1.2 Process Development
      • 12.52.1.3 Cell and Viral Banking
      • 12.52.1.4 Aseptic Filling
  • 12.53 ORCA Therapeutics B.V.
    • 12.53.1 Technology
    • 12.53.2 ORCA-010
    • 12.53.3 Oncolytic Viruses Expressing p35
    • 12.53.4 RNA Interference
  • 12.54 OrphageniX
    • 12.54.1 Technology
  • 12.55 Oxford BioMedica plc
    • 12.55.1 LentiVector Gene Delivery Technology
    • 12.55.2 OXB-102
    • 12.55.3 OXB-201 (RetinoStat)
    • 12.55.4 CAR-T Cell Therapy
    • 12.55.5 OXB-301 (TroVax)
    • 12.55.6 SAR422459
    • 12.55.7 SAR421869
    • 12.55.8 Partnership with Sanofi
    • 12.55.9 Partnership with GlaxoSmithKline
    • 12.55.10 Partnership with Novartis
    • 12.55.11 Partnership with Immune Design Corp.
  • 12.56 Oxford Genetics Ltd.
    • 12.56.1 DNA Services
      • 12.56.1.1 High Throughput Cloning Services
      • 12.56.1.2 DNA Design and Protein Optimization Services
    • 12.56.2 Cell Line Services
      • 12.56.2.1 Cell Line Development
      • 12.56.2.2 Custom Cell Engineering
    • 12.56.3 Protein and Virus Services
      • 12.56.3.1 Protein Expression and Antibody Engineering
      • 12.56.3.2 Virus Construction and Production
    • 12.56.4 Standard DNA Services
    • 12.56.4.1 DNA Synthesis
    • 12.56.4.2 Custom Cloning
    • 12.56.4.3 Plasmid Preparation
  • 12.57 REGENXBIO Inc.
    • 12.57.1 NAV Technology
    • 12.57.2 RGX-501
    • 12.57.3 RGX-314
    • 12.57.4 RGX-111
    • 12.57.5 RGX-121
    • 12.57.6 RGX-321
  • 12.58 Renova Therapeutics Inc.
    • 12.58.1 Renova's Pipeline
  • 12.59 RetroSense Therapeutics LLC
    • 12.59.1 RST-001
  • 12.60 Sangamo Biosciences Inc.
    • 12.60.1 Sangamo's Technology
    • 12.60.2 Sangamo's Product Pipeline
    • 12.60.3 Sangamo's Research Collaborations
  • 12.61 Sarepta Therapeutics Inc.
    • 12.61.1 RNA Medicine
    • 12.61.2 RNA Modulation by PMO
    • 12.61.3 Sarepta's Programs for Duchenne Muscular Dystrophy (DMD)
    • 12.61.4 Sarepta's Programs for Infectious Diseases
  • 12.62 Shanghai Sunway Biotech Co. Ltd.
    • 12.62.1 Oncorine
  • 12.63 SiBiono GeneTech Co. Ltd.
    • 12.63.1 Gendicine
  • 12.64 Sirion Biotech GmbH
    • 12.64.1 Adenovirus
    • 12.64.2 Lentivirus
    • 12.64.4 Adeno-Associated Virus
    • 12.64.5 AdenoBOOST & LentiBOOST
    • 12.64.6 AdenONE
  • 12.65 Spark Therapeutics Inc.
    • 12.65.1 Spark Therapeutics' Pipeline
  • 12.66 Takara Bio Inc.
    • 12.66.1 Takara's Gene Therapy
      • 12.66.1.1 Oncolytic Virus HF10
      • 12.66.1.2 Engineered T-Cell Therapy (siTCR Gene Therapy)
      • 12.66.1.3 CAR Gene Therapy
      • 12.66.1.4 MazF Gene Therapy
  • 12.67 Taxus Cardium Pharmaceutical Group Inc.
    • 12.67.1 Generx
  • 12.68 Tocagen Inc.
    • 12.68.1 Technology
    • 12.68.2 Toca511 & Toca FC
  • 12.69 ToolGen Inc.
    • 12.69.1 Gene Editing
    • 12.69.2 RNA-Guided Endonucleases (RGEN)
  • 12.70 Transgene SA
    • 12.70.1 TG4010
    • 12.70.2 Pexa-Vec
    • 12.70.3 TG6002
    • 12.70.4 TG1050
    • 12.70.5 TG4001
    • 12.70.6 Immunotherapy against Tuberculosis
  • 12.71 uniQure N.V.
    • 12.71.1 Glybera
  • 12.72 Vascular Biologics Ltd.
    • 12.72.1 VBL's Cancer Platform
  • 12.73 Vical Inc.
    • 12.73.1 Poloxamer Delivery System
    • 12.73.2 ASP0113
    • 12.73.3 HSV-2 Therapeutic Vaccine
    • 12.73.4 CyMVectin Prophylactic CMV Vaccine
    • 12.73.5 VL-2397 Antifungal
  • 12.74 ViroMed Co., Ltd.
    • 12.74.1 VM202
    • 12.74.2 Fast Track Designation for VM202
    • 12.74.3 VM501
  • 12.75 Vivebiotech SL
    • 12.75.1 GMP Solutions
    • 12.75.2 Viral Vector Services
    • 12.75.3 ZELIGEN Technology
  • 12.76 Voyager Therapeutics Inc.
    • 12.76.1 Product Pipeline
  • 12.77 Xenon Pharmaceuticals Inc.
    • 12.77.1 Glybera

INDEX OF FIGURES

  • Figure 2.1: Shematic of Gene Therapy using a Virus Vector
  • Figure 2.2: Schematic to Explain Gene Augmentation Therapy
  • Figure 2.3: Schematic to Exlain Gene Inhibition Therapy
  • Figure 2.4: Schematic to Explain Suicide Gene Therapy
  • Figure 2.5: Ex vivo Route of Gene Delivery in Gene Therapy
  • Figure 2.6: In Vivo Route of Gene Delivery in Gene Therapy
  • Figure 7.1: Academic Mettle: Number of Research Papers Published,1975-2016
  • Figure 7.2: Number of Gene Therapy Clinical Trials Approved Worldwide,1989-2016
  • Figure 7.3: Geographical Distribution of Gene Therapy Clinical Trials,2016
  • Figure 7.4: Major Countries Participating in Gene Therapy Clinical Trials,2016
  • Figure 7.5: Major Indications Addressed by Gene Therapy Clinical Trials,2016
  • Figure 7.6: Top Gene Types Transferred
  • Figure 7.7: Phases of Gene Therapy Clinical Trials
  • Figure 7.8: Venture Investment in Gene Therapy by Body System as of2014
  • Figure 8.1: Top Five Vectors used in Gene Therapy
  • Figure 9.1: Global Market for Cystic Fibrosis Therapeutics,20162022
  • Figure 9.2: Global Market for Hemophilia Factor Proteins,2016-2022
  • Figure 9.3: Global Market for Sickle Cell Anemia,2016-2022
  • Figure 9.4: Global Market for HIV Therapeutics,2016-2022
  • Figure 9.5: Global Market for Leukemia Drugs,2016-2022
  • Figure 9.6: Global Market for Ovarian Cancer Therapeutics,2016-2022
  • Figure 9.7: Global Market for Pancreatc Cancer Therapeutics,2016-2022
  • Figure 9.8: Global Market for Head & Neck Cancer Therapeutics,2016-2022
  • Figure 9.9: Global Market for Melanoma Therapeutics,2016-2022
  • Figure 9.10: Global Market for Prostate Therapeutics,2016-2022
  • Figure 9.11: Global Market for Breast Cancer Therapeutics,2016-2022
  • Figure 9.12: Global Market for Cholesterol Lowering Drugs,2016-2022
  • Figure 9.13: U.S. Market for HD Therapeutics,2016-2022
  • Figure 10.1: Global Market for Gene Therapy Technologies, Services and Products,2016-2022
  • Figure 10.2: Market for Technologies, Services and Products by indication,2016-2022
  • Figure 10.3: Percent Share of Gene Therapy Market by Indication,2016
  • Figure 10.4: Global Market for Gene Therapy by Market Segment,2016-2022
  • Figure 10.5: Percent Share of Gene Therapy market by Geography,2016
  • Figure 11.1: Major Ten Companies Developing AAV Vector Candidates
  • Figure 11.2: Diseases and Corresponding Number of Drugs Studied using AAV Vectors
  • Figure 11.3: Major Ten Companies Developing Lentiviral Vector Candidates
  • Figure 11.4: Overview of a Typical Viral Vector Manufacturing Process
  • Figure 11.5: Global Cold Chain and Non-Cold Chain Logistics Spending,2016-2022
  • Figure 11.6: Global Spending on Logistics Related to Clinical Trials,2016-2022
  • Figure 11.7: Estimated Average Per-Patient Clinical Trial Costs by Phase of Study
  • Figure 11.8: Estimated Per-Patient Clinical Trial Expenditure by Indication

INDEX OF TABLES

  • Table 2.1: Differences between Somatic Gene Therapy and Germline Gene Therapy
  • Table 2.2: Few Examples of Suicide Gene Products
  • Table 2.3: Genetic Diseases Treatable by Ex Vivo Gene Therapyusing Bone Marrow Cells
  • Table 2.4: Examples of Diseases Treatable by In Vitro Route of Gene Delivery
  • Table 2.5: Important Gene Therapy Players and Product Candidates
  • Table 3.1: Approved Gene Therapy Products
  • Table 4.1: Number of Non-Cancer Phase III Gene Therapy Candidates as of2016
  • Table 5.1: Key Players with Phase II Product Candidates
  • Table 6.1: Commercialization Status of Gene Therapies in E.U. Member Countries
  • Table 6.2: Prices for Gene Therapy
  • Table 7.1: Approved and Published Clinical Gene Therapy Protocols
  • Table 7.2: Number of Gene Therapy Clinical Trials Approved Worldwide,1989-2016
  • Table 7.3: Geographical Distribution of Gene Therapy Clinical Trials,2016
  • Table 7.4: Gene Therapy Clinical Trials by Country,2016
  • Table 7.5: Gene Therapy Clinical Trials by Indications,2016
  • Table 7.6: Gene Types Transferred in Gene Therapy Clinical Trials
  • Table 7.7: Phases of Gene Therapy Clinical Trials
  • Table 7.8: Timeline of Big Pharma Investment and Licensing in Gene Therapy,2010-2016
  • Table 7.9: Funds Raised by Gene Therapy Companies Since2013
  • Table 8.1: Most Utilized Non-Viral Vectors in Gene Therapy
  • Table 8.2: Advantages and Disadvantages of Four Physical Methods in Gene Delivery
  • Table 8.3: Major Types of Viral Vectors used in Gene Therapy, Advantages and Disadvantages
  • Table 8.4: Key Properties of Viral Vectors
  • Table 8.5: Advantages and Disadvantages of Viral Vectors by Type
  • Table 8.6: Salient Features of Adenovirus Vectors
  • Table 8.7: Advantages and Disadvantages of Adenovirus Vectors
  • Table 8.8: Salient Features of Retrovirus
  • Table 8.9: Advantages and Disadvantages of Retroviral Vectors
  • Table 8.10: Comparison of Retrovirus and Adenovirus
  • Table 8.11: Salient Features of Adeno-Associated Virus Vectors
  • Table 8.12: Advantages and Disadvantages of Adeno-Associated Vectors
  • Table 8.13: AAV Serotypes
  • Table 8.14: Genes Transduced by AAV for Specific Diseases
  • Table 8.15: Salent Features of Lentivirus Vectors
  • Table 8.16: Advantages and Disadvantages of Lentiviral Vectors
  • Table 8.18: Advantages and Disadvantages of Pox/Vaccinia Vectors
  • Table 8.19: Major Features of Viral Vectors by Type
  • Table 8.20: Examples of Clinical Trials Using Retroviruses & Lentiviruses
  • Table 8.21: Examples of Clinical Trials Using Retroviruses & Lentiviruses
  • Table 8.22: Vectors Used in Gene Therapy Clinical Trials,2016
  • Table 8.23: Examples of Virus Vector Manufacturing Systems
  • Table 9.1: Clinical Applications of Gene Therapy
  • Table 9.2: Identifying Number, Intervention and Status of Gene Therapy Trials for Blindness
  • Table 9.3: Gene Therapy Clinical Trial for Eye Disorders
  • Table 9.4: Genes and Loci Underlying Parkinson Disease
  • Table 9.5: Gene Therapy Clinical Trials for Parkinson Disease
  • Table 9.6: U.S. Data on Cystic Fibrosis,2000-2015
  • Table 9.7: Gene Therapy Clinical Trials for Cystic Fibrosis
  • Table 9.8: Gene Therapy Clinical Trials for Pompe Disease
  • Table 9.9: Gene Therapy Clinical Trials for Batten Disease
  • Table 9.10: Gene Therapy Clinical Trials for Immune Disorders
  • Table 9.12: Gene Therapy Clinical Trials for Wiskott-Aldrich Syndrome
  • Table 9.13: Gene Therapy Clinical Trials for Hemophilia
  • Table 9.14: Gene Therapy Product Candidates for Hemophilia A and B
  • Table 9.15: Ongoing Gene Therapy Clinical Trials for Sickle Cell, Fanconi and Thalassemia
  • Table 9.16: Gene Therapy Clinical Trials for Heart Diseases
  • Table 9.17: Gene Therapy for Muscular Dystrophies
  • Table 9.18: Gene Therapy Clinical Trials for HIV
  • Table 9.19: Gene Therapy Clinical Trials for Blood Cancer
  • Table 9.20: Gene Therapy Clinical Trials for Ovarian Cancer
  • Table 9.21: Gene Therapy Clinical Trials for Pancreatic Cancer
  • Table 9.22: Gene Therapy Clinical Trials for Head and Neck Cancers
  • Table 9.23: Gene Therapy Clinical Therapy for Melanoma
  • Table 9.24: Gene Therapy Clinical for Prostate Cancer
  • Table 9.25: Gene Therapy Clinical Trials for Breast Cancer
  • Table 9.26: Gene Therapy Clinical Trials for Fabry Disease
  • Table 9.27: Gene Therapy for Familial Hypercholesterolemia
  • Table 9.28: Gene Therapy Clinical Trials for Tay-Sachs disease
  • Table 10.1: Estimated Market for Five Gene Therapy Products in the U.S. in the Next20 Years
  • Table 10.2: Market for Technologies, Services and Products by Indication,2016-2022
  • Table 10.3: Global Market for Gene Therapy by Market Segment,2016-2022
  • Table 10.4: Percent Share of Gene Therapy market by Geography,2016
  • Table 10.5: The Estimated Drug Market that is Wide Open for Gene Therapy Product Candidates Focusing on13 Diseases
  • Table 11.1: Major Ten Companies Developing AAV Vectors
  • Table 11.2: Diseases and Corresponding Number of Drugs Studied using AAV Vectors
  • Table 11.3: Major Ten Companies Developing Lentiviral Vector Candidates
  • Table 11.4: Global Cold Chain and Non-Cold Chain Logistics Spending,2016-2022
  • Table 11.5: Estimated Average Per-Patient Clinical Trial Costs by Phase of Study
  • Table 11.6: Estimated Per-Patient Clinical Trial Expenditure by Indication
  • Table 11.7: Patient-Per Clinical Trial Expenditure by Disease Area and Phase
  • Table 12.1:4D's Product Pipeline
  • Table 12.2: Abeona's Product Pipeline
  • Table 12.3: Advantagene's Program Overview
  • Table 12.4: Adverum's Pipeline of Gene Therapy Programs
  • Table 12.5: Agilis DNA Therapeutic Programs
  • Table 12.6: Generx Product Pipeline
  • Table 12.7: Potential Economic Opportunity for Generx (Ad5FGF-4)
  • Table 12.8: AGTC's Ophthalmology Development Programs
  • Table 12.9: AnGes' Product Pipeline
  • Table 12.10: Asklepios' Product Pipeline
  • Table 12.11: Audentes' Pipeline
  • Table 12.12: AvroBio's Programs
  • Table 12.13: Benitec's In-House Programs
  • Table 12.14: Benitecs Licensed Programs
  • Table 12.15: Bluebird's Program Pipeline
  • Table 12.16: Product Pipeline from Cellectis
  • Table 12.17: Dimension's Product Pipeline
  • Table 12.18: Fibroblast's Product Pipeline
  • Table 12.19: Genethon's Pipeline
  • Table 12.20: Summary of GenVec's Product Pipeline
  • Table 12.21: Immune Design's Product Pipeline
  • Table 12.22: Inovio's Pipeline of Products
  • Table 12.23: Types of Genome Editing
  • Table 12.24: Intellia's Product Pipeline
  • Table 12.25: Kite's Product Pipeline
  • Table 12.26: Lysogene's Product Pipeline
  • Table 12.27: Medgenics' Product Pipeline
  • Table 12.28: Mologen's Product Pipeline
  • Table 12.29: Oxford BioMedica's Product Pipeline
  • Table 12.30: REGENXBIO's Therapeutic Programs
  • Table 12.31: Renova's Pipeline
  • Table 12.32: Sangamo's Product Pipeline
  • Table 12.33: Sarepta's Product Pipeline for DMD
  • Table 12.34: Sarepta's Programs for Infectious Diseases
  • Table 12.35: Spark Therapeutics' Pipeline
  • Table 12.36: Tocagen's Product Pipeline
  • Table 12.37: Transgene's Product Pipeline
  • Table 12.38: unQure's Pipeline
  • Table 12.39: VBL's Cancer Pipeline
  • Table 12.40: Vical's Product Pipeline
  • Table 12.41: Voyager Therapeutics, Product Pipeline
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