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遺伝子治療の世界市場：2025年～2033年

Global Gene Therapy Market - 2025-2033

出版日
ページ情報
英文 180 Pages
納期
即日から翌営業日
カスタマイズ可能
適宜更新あり
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=144.06円
遺伝子治療の世界市場：2025年～2033年
出版日: 2025年03月25日
発行: DataM Intelligence
ページ情報: 英文 180 Pages
納期: 即日から翌営業日
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概要

世界の遺伝子治療の市場規模は、2024年に47億9,000万米ドルに達し、2033年には359億1,000万米ドルに達すると予測され、予測期の2025年～2033年のCAGRは23.1％と見込まれています。

遺伝子治療は、患者の細胞に遺伝物質を改変または導入することにより、遺伝性疾患の治療や予防を目指す最新の医療技術です。これは、ウイルスなどのベクターを用いて機能的遺伝子や遺伝子編集ツールを患者の体内に送り込むことで達成されます。病気の原因となる特定の遺伝子に焦点を当てることで、科学者たちは欠陥のある遺伝情報を修正したり、置き換えたりすることを目指しています。

遺伝子治療は、遺伝性疾患、心血管疾患、感染症、がんなどに対処する計り知れない可能性を秘めています。遺伝子治療は、病気の原因となる遺伝子を置き換えたり、不活性化したりすることによって行われます。場合によっては、特定の疾患を治療するために新しい遺伝子を体内に導入することもあります。遺伝子治療では、ヘルスケアプロバイダーが健康な遺伝子のコピーを体内の細胞に送り込みます。この健康な遺伝子は、損傷した遺伝子を置き換えたり、変異した遺伝子を不活性化したり、全く新しい遺伝子を導入したりします。

先進デリバリーベクターの開発と利用は、遺伝子治療市場の成長を牽引する最も重要な要因のひとつです。デリバリーベクターは、治療用遺伝子を体内の特定の細胞に送達するために不可欠です。これらのベクターの効率、精度、安全性は、遺伝子治療の有効性とその普及に大きく影響します。

より効率的なベクターの開発により、固形がん、神経疾患、心血管疾患など、これまでは送達の課題から治療が困難であった幅広い疾患への遺伝子治療の適用が可能になりつつあります。

例えば、2024年10月、RocheはDyno Therapeuticsのアデノ随伴ウイルス（AAV）キャプシド・プラットフォームを応用し、Dyno Therapeuticsに10億米ドル以上の利益をもたらす可能性のある提携を通じて、不特定の神経疾患をターゲットとした遺伝子治療用の次世代AAVベクターを開発しました。

アデノ随伴ウイルス（AAV）ベクターは、肝臓、筋肉、眼球など様々な組織を標的にできることから、最も一般的に使用されているデリバリーシステムのひとつです。脊髄性筋萎縮症（SMA）に対するゾルゲンマのようなAAVベースの遺伝子治療は、すでに高い効果が証明されています。

遺伝子治療のコストが高いことは、遺伝子治療市場の成長とアクセシビリティに影響を与える最も大きな課題のひとつです。遺伝子治療は多くの重篤な疾患に対して治癒の可能性を示していますが、その高価な価格が普及の障壁となっています。

例えば、遺伝子治療薬ZyntegloとSkysonaの価格は、1回投与あたり280万米ドルと300万米ドルです。さらに、ゾルゲンマの定価は210万米ドルと報告されています。遺伝子治療の開発には高価で長いプロセスが必要です。遺伝子治療の臨床試験は数年に及ぶことが多く、患者の監視が厳しく、規制当局の承認手続きも高額です。

例えば、レーバー先天性黒内障の遺伝子治療薬Luxturnaは、開発から承認まで10年以上を要し、その費用は患者一人当たり85万米ドルです。遺伝子治療の多くは、個々の患者の遺伝子構成に合わせた個別化治療であるため、症例ごとにオーダーメイドの製造が必要となります。この個別化により、コストはさらに増加します。

例えば、YescartaやKymriahのようなCAR-T療法は、患者のT細胞を収集し、改変することによって患者ごとにカスタマイズされますが、このプロセスには複雑な製造と品質管理対策が含まれます。これらの治療法には、患者一人当たり30万米ドルから40万米ドル以上の費用がかかります。

当レポートでは、世界の遺伝子治療市場について調査し、市場の概要とともに、アプローチ別、ベクタータイプ別、技術別、用途別、地域別動向、競合情勢、および市場に参入する企業のプロファイルなどを提供しています。

目次

第1章 市場のイントロダクションと範囲

第2章 エグゼクティブの洞察と重要なポイント

第3章 市場のハイライトと戦略的ポイント

  • 主な動向と将来の予測

第4章 アプローチ別

  • ベクタータイプ別
  • 技術別
  • 用途別
  • 地域別

第5章 市場力学

  • 影響要因
    • 促進要因
    • 抑制要因
    • 機会
    • 影響分析

第6章 戦略的洞察と業界展望

  • 市場のリーダーと先駆者
  • CXOの視点
  • 最新の開発とブレークスルー
  • ケーススタディ/進行中の調査
  • 規制と償還の情勢
  • ポーターのファイブフォース分析
  • サプライチェーン分析
  • 特許分析
  • SWOT分析
  • アンメットニーズとギャップ
  • 市場参入と拡大のための推奨戦略
  • シナリオ分析ベストケース、ベースケース、ワーストケースの予測
  • 価格分析と価格動向
  • キーオピニオンリーダー

第7章 遺伝子治療市場、アプローチ別

  • 生体外
  • 生体内

第8章 遺伝子治療市場、ベクタータイプ別

  • ウイルスベクター
    • アデノ随伴ウイルス
    • 単純ヘルペスウイルス
    • レンチウイルス
  • 非ウイルスベクター

第9章 遺伝子治療市場、技術別

  • 遺伝子追加
  • 遺伝子サイレンシング
  • 遺伝子編集

第10章 遺伝子治療市場、用途別

  • 腫瘍学
  • 筋骨格系疾患
  • 血液疾患
  • 希少疾患
  • 眼科
  • その他

第11章 遺伝子治療市場、地域別市場分析と成長機会

  • 北米
    • 米国
    • カナダ
  • 欧州
    • ドイツ
    • 英国
    • フランス
    • スペイン
    • イタリア
    • その他
  • ラテンアメリカ
    • メキシコ
    • ブラジル
    • アルゼンチン
    • その他
  • アジア太平洋
    • 中国
    • インド
    • 日本
    • 韓国
    • その他
  • 中東・アフリカ

第12章 競合情勢と市場ポジショニング

  • 競合状況の概要と主要な市場参入企業
  • 市場シェア分析とポジショニングマトリックス
  • 戦略的パートナーシップ、合併、買収
  • 製品ポートフォリオとイノベーションにおける主な発展
  • 企業ベンチマーク

第13章 企業プロファイル

  • Alnylam Pharmaceuticals, Inc.
  • Spark Therapeutics, Inc.
  • Novartis AG
  • bluebird bio, Inc.
  • Ferring Pharmaceuticals Inc.
  • Vertex Pharmaceuticals Incorporated
  • Sarepta Therapeutics, Inc.
  • CSL Behring LLC
  • Amgen, Inc.
  • Orchard Therapeutics group
  • Krystal Biotech, Inc.

第14章 仮定と調査手法

第15章 付録

目次
Product Code: BT1341

The global gene therapy market reached US$ 4.79 billion in 2024 and is expected to reach US$ 35.91 by 2033, growing at a CAGR of 23.1% during the forecast period 2025-2033.

Gene therapy is a modern medical technique that seeks to cure and prevent genetic disorders by modifying or introducing genetic material into the cells of a patient. This is achieved by using vectors, such as viruses, to deliver functional genes or gene-editing tools to the patient's body. By focusing on the specific genes that cause diseases, scientists aim to rectify or replace faulty genetic information, which could offer enduring solutions.

Gene therapy has immense potential for addressing inherited disorders, cardiovascular diseases, infectious diseases, and cancer. Gene therapy works by replacing or inactivating disease-causing genes. In some cases, gene therapy introduces new genes into the body to treat a specific disease. With gene therapy, healthcare providers deliver a healthy copy of a gene to cells inside the body. This healthy gene replaces a damaged gene, inactivates a mutated gene or introduces an entirely new gene.

Market Dynamics: Drivers & Restraints

Growing Utilization of Advanced Delivery Vectors

The development and utilization of advanced delivery vectors are one of the most significant factors driving the growth of the gene therapy market. Delivery vectors are essential for delivering therapeutic genes to specific cells in the body. The efficiency, precision, and safety of these vectors can significantly influence the effectiveness of gene therapies and their widespread adoption.

The development of more efficient vectors is enabling gene therapies to be applied to a wider range of diseases, including solid tumors, neurological disorders, and cardiovascular diseases, which were previously difficult to treat due to delivery challenges.

For instance, in October 2024, Roche applied Dyno Therapeutics' engineered adeno-associated virus (AAV) capsid platform to develop next-generation AAV vectors for gene therapies targeting unspecified neurological diseases, through a collaboration that could generate more than $1 billion for Dyno.

Adeno-associated virus (AAV) vectors are among the most commonly used delivery systems due to their ability to target a variety of tissues, including the liver, muscle, and eye. AAV-based gene therapies, such as Zolgensma for Spinal Muscular Atrophy (SMA), are already proving highly effective.

High Cost associated with the Gene Therapies

The high cost of gene therapies is one of the most significant challenges impacting the growth and accessibility of the gene therapy market. While gene therapies have shown curative potential for many serious diseases, their expensive price tags pose barriers to widespread adoption.

For instance, the gene therapies Zynteglo and Skysona are priced at US$ 2.8 million and US$ 3 million per dose. Moreover, Zolgensma has a reported list price of US$ 2.1 million. The development of gene therapies is an expensive, lengthy process. Clinical trials for gene therapies often span several years and involve high patient monitoring and expensive regulatory approval procedures.

For instance, Luxturna, a gene therapy for Leber's congenital amaurosis, took over 10 years from development to approval, and its cost is $850,000 per patient. Many gene therapies are personalized treatments that are tailored to the genetic makeup of individual patients, requiring bespoke production for each case. This individualization further increases costs.

For instance, CAR-T therapies such as Yescarta and Kymriah are customized for each patient by collecting and modifying the patient's T-cells, a process that involves complex manufacturing and quality control measures. These therapies can cost over $300,000 to $400,000 per patient.

Segment Analysis

The global gene therapy market is segmented based on the approach, vector type, technique, application, and region.

Gene addition in the technique segment is expected to dominate the gene therapy market.

Gene Addition therapy involves introducing a functional gene into a patient's cells to compensate for deficiencies caused by loss-of-function mutations. This approach is particularly effective in addressing recessive monogenic diseases, where a faulty gene fails to produce a necessary protein.

According to John Wiley & Sons, Inc.'s research study in August 2024, stated that while various vectors can be used to deliver the functional gene, adeno-associated virus (AAV) vectors are often favored for gene therapy due to their small size, lack of human pathogenicity, weakened post-injection immune responses, and ease of engineering. However, Gene Addition cannot address all gene mutations, including dominant mutations, polygenic mutation conditions, and large genes that are incompatible with delivery vectors.

Furthermore, key players in the industry are product approvals that would drive this segment's growth. For instance, in November 2024, PTC Therapeutics, Inc. received accelerated approval from the U.S. Food and Drug Administration (FDA) for its gene therapy to treat Aromatic L-Amino Acid Decarboxylase (AADC) deficiency.

This marks the first gene therapy approved in the U.S. that is directly administered to the brain. The gene therapy will be marketed in the U.S. under the brand name Kebilidi and is approved for both children and adults with AADC deficiency, covering the full spectrum of disease severity. KEBILIDI is a Gene Addition therapy administered directly into the putamen region of the brain using a precise neurosurgical procedure called stereotactic surgery. It works by replacing a faulty gene.

Also, in August 2022, Bluebird Bio, Inc. received FDA approval for ZYNTEGLO (betibeglogene autotemcel) is a one-time, custom-made gene therapy that uses Gene Silencing to address the underlying genetic cause of beta-thalassemia in patients who require regular red blood cell (RBC) transfusions. Zynteglo addresses the underlying genetic cause of the disease.

Geographical Analysis

North America is expected to dominate the Gene Therapy market

The gene therapy market in the North America region is expected to be driven by various factors like the increasing prevalence of cancer, genetic disorders, and other rare diseases. As per clinical Case Reports research publication in February 2024, SMA, a genetic disorder resulting from mutations in both copies of the SMN1 gene in cells, affects 1 in 6,000 to 1 in 10,000 children.

According to the National Heart, Lung, and Blood Institute data in September 2024, Sickle cell disease (SCD) affects approximately 100,000 people in the United States, with over 90% being non-Hispanic Black or African American, and 8 million people worldwide. The demand for gene-based treatments is expected to accelerate, driving the overall growth of the gene therapy market.

Furthermore, in this region, a major number of key players' presence, a well-advanced healthcare infrastructure, technological advances, product launches, approvals, and increasing research activities are driving the gene therapy market in the North American region.

For instance, in September 2024, Vertex Pharmaceuticals Incorporated announced that Health Canada granted marketing authorization for CASGEVY (exagamglogene autotemcel) for individuals 12 years and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta-thalassemia (TDT). CASGEVY is an autologous genome-edited hematopoietic stem cell-based therapy. It is estimated that approximately 2,000 patients in Canada are eligible for this treatment, with the majority having SCD.

Similarly, in March 2024, Orchard Therapeutics, acquired by Kyowa Kirin, announced its U.S. launch plans for Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene therapy for children with early-onset metachromatic leukodystrophy (MLD). MLD is a rare, rapidly progressive, and ultimately fatal neurometabolic disease affecting roughly one in 100,000 live births.

Competitive Landscape

The global market players in the gene therapy market are Alnylam Pharmaceuticals, Inc., Spark Therapeutics, Inc., Novartis AG, bluebird bio, Inc., Ferring Pharmaceuticals Inc., Vertex Pharmaceuticals Incorporated, Sarepta Therapeutics, Inc., CSL Behring LLC, Amgen, Inc., Orchard Therapeutics group, Krystal Biotech, Inc. among others.

Why Purchase the Report?

  • Pipeline & Innovations: Reviews ongoing clinical trials, product pipelines, and forecasts upcoming pharmaceutical advancements.
  • Type Performance & Market Positioning: Analyzes product performance, market positioning, and growth potential to optimize strategies.
  • Real-World Evidence: Integrates patient feedback and data into product development for improved outcomes.
  • Physician Preferences & Health System Impact: Examines healthcare provider behaviors and the impact of health system mergers on adoption strategies.
  • Market Updates & Industry Changes: Covers recent regulatory changes, new policies, and emerging technologies.
  • Competitive Strategies: Analyzes competitor strategies, market share, and emerging players.
  • Pricing & Market Access: Reviews pricing models, reimbursement trends, and market access strategies.
  • Market Entry & Expansion: Identifies optimal strategies for entering new markets and partnerships.
  • Regional Growth & Investment: Highlights high-growth regions and investment opportunities.
  • Supply Chain Optimization: Assesses supply chain risks and distribution strategies for efficient Type delivery.
  • Sustainability & Regulatory Impact: Focuses on eco-friendly practices and evolving regulations in healthcare.
  • Post-market Surveillance: Uses post-market data to enhance product safety and access.
  • Pharmacoeconomics & Value-Based Pricing: Analyzes the shift to value-based pricing and data-driven decision-making in R&D.

The global gene therapy market report would provide approximately 45 tables, 46 figures, and 180 pages.

Target Audience 2024

  • Manufacturers: Pharmaceutical, Biotech Companies, Contract Manufacturers, Distributors, Hospitals.
  • Regulatory & Policy: Compliance Officers, Government, Health Economists, Market Access Specialists.
  • Technology & Innovation: R&D Professionals, Clinical Trial Managers, Pharmacovigilance Experts.
  • Investors: Healthcare Investors, Venture Fund Investors, Pharma Marketing & Sales.
  • Consulting & Advisory: Healthcare Consultants, Industry Associations, Analysts.
  • Supply Chain: Distribution and Supply Chain Managers.
  • Consumers & Advocacy: Patients, Advocacy Groups, Insurance Companies.
  • Academic & Research: Academic Institutions.

Table of Contents

1. Market Introduction and Scope

  • 1.1. Objectives of the Report
  • 1.2. Report Coverage & Definitions
  • 1.3. Report Scope

2. Executive Insights and Key Takeaways

3. Market Highlights and Strategic Takeaways

  • 3.1. Key Trends and Future Projections

4. Snippet by Approach

  • 4.1. Snippet by Vector Type
  • 4.2. Snippet by Technique
  • 4.3. Snippet by Application
  • 4.4. Snippet by Region

5. Dynamics

  • 5.1. Impacting Factors
    • 5.1.1. Drivers
      • 5.1.1.1. Growing Utilization of Advanced Delivery Vectors
      • 5.1.1.2. Expansion into the Rare Diseases
    • 5.1.2. Restraints
      • 5.1.2.1. High Cost Associated with the Gene Therapies
      • 5.1.2.2. Safety and Long-Term Efficacy Concerns
    • 5.1.3. Opportunity
      • 5.1.3.1. Personalized Cancer Gene Therapies
    • 5.1.4. Impact Analysis

6. Strategic Insights and Industry Outlook

  • 6.1. Market Leaders and Pioneers
    • 6.1.1. Emerging Pioneers and Prominent Players
    • 6.1.2. Established leaders with the largest selling Brand
    • 6.1.3. Market leaders with established Product
  • 6.2. CXO Perspectives
  • 6.3. Latest Developments and Breakthroughs
  • 6.4. Case Studies/Ongoing Research
  • 6.5. Regulatory and Reimbursement Landscape
    • 6.5.1. North America
    • 6.5.2. Europe
    • 6.5.3. Asia Pacific
    • 6.5.4. Latin America
    • 6.5.5. Middle East & Africa
  • 6.6. Porter's Five Force Analysis
  • 6.7. Supply Chain Analysis
  • 6.8. Patent Analysis
  • 6.9. SWOT Analysis
  • 6.10. Unmet Needs and Gaps
  • 6.11. Recommended Strategies for Market Entry and Expansion
  • 6.12. Scenario Analysis: Best-Case, Base-Case, and Worst-Case Forecasts
  • 6.13. Pricing Analysis and Price Dynamics
  • 6.14. Key Opinion Leaders

7. Gene Therapy Market, By Approach

  • 7.1. Introduction
    • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Approach
    • 7.1.2. Market Attractiveness Index, By Approach
  • 7.2. Ex-Vivo*
    • 7.2.1. Introduction
    • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 7.3. In-Vivo

8. Gene Therapy Market, By Vector Type

  • 8.1. Introduction
    • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Vector Type
    • 8.1.2. Market Attractiveness Index, By Vector Type
  • 8.2. Viral Vectors*
    • 8.2.1. Introduction
    • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
    • 8.2.3. Adeno-Associated Virus
    • 8.2.4. Herpes Simplex Virus
    • 8.2.5. Lentivirus
  • 8.3. Non-Viral Vectors

9. Gene Therapy Market, By Technique

  • 9.1. Introduction
    • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Technique
    • 9.1.2. Market Attractiveness Index, By Technique
  • 9.2. Gene Addition*
    • 9.2.1. Introduction
    • 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 9.3. Gene Silencing
  • 9.4. Gene Editing

10. Gene Therapy Market, By Application

  • 10.1. Introduction
    • 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
    • 10.1.2. Market Attractiveness Index, By Application
  • 10.2. Oncology*
    • 10.2.1. Introduction
    • 10.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 10.3. Musculoskeletal Conditions
  • 10.4. Blood Disorders
  • 10.5. Rare Diseases
  • 10.6. Ophthalmology
  • 10.7. Others

11. Gene Therapy Market, By Regional Market Analysis and Growth Opportunities

  • 11.1. Introduction
    • 11.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
      • 11.1.1.1. Market Attractiveness Index, By Region
  • 11.2. North America
    • 11.2.1. Introduction
    • 11.2.2. Key Region-Specific Dynamics
    • 11.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Approach
    • 11.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Vector Type
    • 11.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Technique
    • 11.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
    • 11.2.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 11.2.7.1. U.S.
      • 11.2.7.2. Canada
  • 11.3. Europe
    • 11.3.1. Introduction
    • 11.3.2. Key Region-Specific Dynamics
    • 11.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Approach
    • 11.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Vector Type
    • 11.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Technique
    • 11.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
    • 11.3.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 11.3.7.1. Germany
      • 11.3.7.2. U.K.
      • 11.3.7.3. France
      • 11.3.7.4. Spain
      • 11.3.7.5. Italy
      • 11.3.7.6. Rest of Europe
  • 11.4. Latin America
    • 11.4.1. Introduction
    • 11.4.2. Key Region-Specific Dynamics
    • 11.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Approach
    • 11.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Vector Type
    • 11.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Technique
    • 11.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
    • 11.4.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 11.4.7.1. Mexico
      • 11.4.7.2. Brazil
      • 11.4.7.3. Argentina
      • 11.4.7.4. Rest of Latin America
  • 11.5. Asia-Pacific
    • 11.5.1. Introduction
    • 11.5.2. Key Region-Specific Dynamics
    • 11.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Approach
    • 11.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Vector Type
    • 11.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Technique
    • 11.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
    • 11.5.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 11.5.7.1. China
      • 11.5.7.2. India
      • 11.5.7.3. Japan
      • 11.5.7.4. South Korea
      • 11.5.7.5. Rest of Asia-Pacific
  • 11.6. Middle East and Africa
    • 11.6.1. Introduction
    • 11.6.2. Key Region-Specific Dynamics
    • 11.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Approach
    • 11.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Vector Type
    • 11.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Technique
    • 11.6.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application

12. Competitive Landscape and Market Positioning

  • 12.1. Competitive Overview and Key Market Players
  • 12.2. Market Share Analysis and Positioning Matrix
  • 12.3. Strategic Partnerships, Mergers & Acquisitions
  • 12.4. Key Developments in Product Portfolios and Innovations
  • 12.5. Company Benchmarking

13. Company Profiles

  • 13.1. Alnylam Pharmaceuticals, Inc.*
    • 13.1.1. Company Overview
    • 13.1.2. Product Portfolio
      • 13.1.2.1. Product Description
      • 13.1.2.2. Product Key Performance Indicators (KPIs)
      • 13.1.2.3. Historic and Forecasted Product Sales
      • 13.1.2.4. Product Sales Volume
    • 13.1.3. Financial Overview
    • 13.1.4. Company Revenue
      • 13.1.4.1. Geographical Revenue Shares
      • 13.1.4.1.1. Revenue Forecasts
      • 13.1.4.2. Key Developments
      • 13.1.4.2.1. Mergers & Acquisitions
      • 13.1.4.2.2. Key Product Development Activities
      • 13.1.4.2.3. Regulatory Approvals, etc.
      • 13.1.4.3. SWOT Analysis
  • 13.2. Spark Therapeutics, Inc.
  • 13.3. Novartis AG
  • 13.4. bluebird bio, Inc.
  • 13.5. Ferring Pharmaceuticals Inc.
  • 13.6. Vertex Pharmaceuticals Incorporated
  • 13.7. Sarepta Therapeutics, Inc.
  • 13.8. CSL Behring LLC
  • 13.9. Amgen, Inc.
  • 13.10. Orchard Therapeutics group
  • 13.11. Krystal Biotech, Inc. (LIST NOT EXHAUSTIVE)

14. Assumptions and Research Methodology

  • 14.1. Data Collection Methods
  • 14.2. Data Triangulation
  • 14.3. Forecasting Techniques
  • 14.4. Data Verification and Validation

15. Appendix

  • 15.1. About Us and Services
  • 15.2. Contact Us