遺伝子治療の市場規模、シェア、動向、予測：遺伝子タイプ、ベクタータイプ、送達方法、用途、地域別、2025-2033年

世界の遺伝子治療の市場規模は2024年に58億米ドルとなりました。今後、IMARC Groupは、2033年には179億米ドルに達し、2025年から2033年にかけて13.3%のCAGRを示すと予測しています。現在、北米が市場を独占しており、2024年の市場シェアは56%を超えます。北米市場を牽引しているのは、強固なヘルスケアインフラ、研究事業への投資の増加、先進的な規制枠組み、イノベーションを促進する大手バイオテクノロジー企業の存在です。

遺伝子治療の市場分析：

市場の成長と規模：遺伝子異常や希少疾患の有病率の上昇に後押しされ、業界は急速に拡大しています。また、製薬企業の研究開発投資も活発化しており、市場にプラスの影響を与えています。

技術の進歩：ウイルスベクター技術の開発により、遺伝子治療のデリバリーの有効性と安全性が向上しています。さらに、CRISPR-Cas9技術は遺伝子編集能力を向上させ、遺伝子治療の市場統計をさらに加速させています。

産業への応用遺伝子治療のは、がん、遺伝的問題、自己免疫疾患など様々な疾患の治療に使用されています。体外で細胞を操作するEx vivo遺伝子治療のは、特定の遺伝性疾患の治療に人気を集めています。

地理的動向：IMARCによる遺伝子治療の市場統計によると、北米は強力なヘルスケアインフラと発達したバイオテクノロジー部門により市場を独占しています。しかし、アジア太平洋地域は患者数の多さに後押しされ、急速に拡大しつつあります。

競合情勢：大手開発企業は、希少遺伝子異常やがんを含む様々な疾患を対象とした新規遺伝子治療法の開発および規制当局の認可取得のため、包括的な臨床研究を実施しています。市場の主要企業には、Abeona Therapeutics Inc.、Adaptimmune Therapeutics plc、Adverum Biotechnologies Inc.、Amgen Inc.、Astellas Pharma Inc.、Biogen Inc.、Bluebird Bio Inc.、Mustang Bio Inc. Fortress Biotech Inc.、Novartis AG、Orchard Therapeutics plc、UniQure N.V.、Voyager Therapeutics Inc.などがあります。

課題と機会：業界は、規制上の制限や高い研究コストといった制約に直面する一方で、希少疾患以外の用途の拡大や新しい送達方法の研究といった可能性にも期待しています。

将来の展望遺伝子治療の市場の見通しは、遺伝子異常や希少疾患に対する新規治療に対する需要の高まりにより、明るいものになると予想されます。さらに、患者の特性に合わせた個別化遺伝子治療のが市場拡大の原動力になると予測されます。

遺伝子治療の市場動向：

ウイルスベクター技術の進歩

ウイルスベクター技術の絶え間ない進歩と革新は、市場成長を促進する重要な要因です。ウイルスベクターは、治療用遺伝子を患者の細胞に導入するための送達手段として使用されます。この技術における最近の進歩は、遺伝子治療の療法の安全性と有効性を大幅に向上させています。これらの開発には、アデノ随伴ウイルス（AAV）やレンチウイルスなど、より特異的で集中的なウイルスベクターの開発が含まれ、AAVは免疫反応や損傷を最小限に抑えながら遺伝物質を輸送する能力により人気を博しています。さらに、遺伝子治療の市場概要にあるように、研究者はより大きな遺伝子ペイロードを運ぶ可能性のあるウイルスベクターの開発に注力しており、治療可能な疾患の範囲を広げています。こうした技術革新は、遺伝子治療の有効性を高めるだけでなく、副作用の可能性を最小限に抑え、患者にとってより安全な治療法となります。その結果、ウイルスベクター技術の進歩は、より広範な医療問題に対する遺伝子治療の治療法の受容と有効性を加速させています。例えば、MERCK KGaAのVirusExpress 293 Adeno-Associated Virus（AAV）Production Platformは、AAVやレンチウイルスベクターを含む完全なウイルスベクター製造を提供しています。さらに、キャタレント社はアデノ随伴ウイルス（AAV）ベクターの開発と製造のためのUpTempo Virtuosoプラットフォーム・プロセスを立ち上げました。

研究開発への投資と資金調達

遺伝子治療の研究開発（R&D）への投資と資金調達の増大が市場開拓の原動力となっています。製薬企業、バイオテクノロジー企業、政府機関は、遺伝子治療の技術と治療薬の開発に大きな資源を投入しています。遺伝子治療はアンメット・メディカル・ニーズに対応し、長期的な利益をもたらす可能性が高く、大きな資金を集めています。大手製薬会社は研究を加速させるために、遺伝子治療の新興企業を買収したり、独自の遺伝子治療のセクションを構築したりしており、遺伝子治療の市場の見通しにプラスの影響を与えています。さらに、政府からの補助金やインセンティブは、学術機関や研究会社が遺伝子治療の実験を行う動機付けとなっています。米国の希少疾病用医薬品法（Orphan Drug Act）、および他国の同種の法律は、希少疾病用医薬品の開発にインセンティブを与えています。例えば、嚢胞性線維症財団（Cystic Fibrosis Foundation）は、サリオジェン・セラピューティクス社（SalioGen Therapeutics）への投資を報告し、同社の嚢胞性線維症に対する新規遺伝子治療の前臨床研究を支援しています。サロネンのジーン・コーディング・アプローチは、ゲノム上のあらゆる遺伝子の機能をオン、オフ、または変更するように設計されています。

遺伝子異常と希少疾患の有病率の上昇

遺伝子異常や希少疾患の有病率の上昇が市場の成長を後押ししています。これらの疾患の多くは有効な治療法がほとんどない、あるいは全くないため、遺伝子治療のは魅力的な選択肢となっています。遺伝学の理解が進むにつれて、診断される人が増え、新薬の需要が高まっています。さらに、遺伝子治療のは、欠陥のある遺伝子を修復したり置き換えたりすることで、多くの疾患の根本的な原因を治療する能力を持っています。これは、筋ジストロフィー、嚢胞性線維症、その他の遺伝性代謝障害を含む疾患です。製薬会社やバイオテクノロジー企業は、この拡大する医療需要を満たすために調査に投資しており、その結果、市場が拡大し、この分野が改善されています。例えば、米国疾病管理予防センターの脊髄性筋萎縮症（SMA）に関する記事によると、SMAは遺伝性疾患で、約1万人に1人が罹患しています。したがって、最も一般的な希少疾患のひとつです。

協力的な規制環境

特に米国食品医薬品局（FDA）や欧州医薬品庁（EMA）など、いくつかの国の規制機関は、遺伝子治療のが本質的な医療ニーズに対応できる可能性があることを認識しています。その結果、遺伝子治療の研究と承認のための体制を整え、加速させています。さらに、ファスト・トラック指定、画期的治療指定、オーファンドラッグ指定は、遺伝子治療の候補が利用できる規制措置の一つです。これらの指定は、臨床試験手続きを容易にし、開発スケジュールを短縮し、企業の遺伝子治療の研究への参加を促します。例えば、米国FDAは、成人および小児のβ-サラセミア患者を治療する初の細胞ベースの遺伝子治療のであるZynteglo（betibeglogene autotemcel）を承認しました。このような承認は、遺伝子治療の開発増加により市場の成長を促進すると予想されます。

目次

第1章 序文

第2章 調査範囲と調査手法

• 調査の目的
• ステークホルダー
• データソース
  • 一次情報
  • 二次情報
• 市場推定
  • ボトムアップアプローチ
  • トップダウンアプローチ
• 調査手法

第3章 エグゼクティブサマリー

第4章 イントロダクション

• 概要
• 主要業界動向

第5章 世界の遺伝子治療の市場

• 市場概要
• 市場実績
• COVID-19の影響
• 市場予測

第6章 市場内訳：遺伝子タイプ別

• 抗原
• サイトカイン
• 腫瘍抑制剤
• 自殺遺伝子
• 欠乏
• 成長因子
• 受容体
• その他

第7章 市場内訳：ベクタータイプ別

• ウイルスベクター
  • 主要セグメント
    • アデノウイルス
    • レンチウイルス
    • レトロウイルス
    • アデノ随伴ウイルス
    • 単純ヘルペスウイルス
    • ポックスウイルス
    • ワクシニアウイルス
    • その他
• 非ウイルス技術
  • 主要セグメント
    • 裸ベクターとプラスミドベクター
    • 遺伝子銃
    • エレクトロポレーション
    • リポフェクション
    • その他

第8章 市場内訳：送達方法別

• 生体内
• Ex-Vivo

第9章 市場内訳：用途別

• 腫瘍性疾患
• 希少疾患
• 心血管疾患
• 神経疾患
• 感染症
• その他

第10章 市場内訳：地域別

• 北米
  • 米国
  • カナダ
• アジア太平洋地域
  • 中国
  • 日本
  • インド
  • 韓国
  • オーストラリア
  • インドネシア
  • その他
• 欧州
  • ドイツ
  • フランス
  • 英国
  • イタリア
  • スペイン
  • ロシア
  • その他
• ラテンアメリカ
  • ブラジル
  • メキシコ
  • その他
• 中東・アフリカ
  • 市場内訳：国別

第11章 SWOT分析

• 概要
• 強み
• 弱み
• 機会
• 脅威

第12章 バリューチェーン分析

第13章 ポーターのファイブフォース分析

• 概要
• 買い手の交渉力
• 供給企業の交渉力
• 競合の程度
• 新規参入業者の脅威
• 代替品の脅威

第14章 価格分析

第15章 競合情勢

• 市場構造
• 主要企業
• 主要企業のプロファイル
  • Abeona Therapeutics Inc.
  • Adaptimmune Therapeutics plc
  • Adverum Biotechnologies Inc.
  • Amgen Inc.
  • Astellas Pharma Inc.
  • Biogen Inc.
  • Bluebird Bio Inc.
  • Mustang Bio Inc.（Fortress Biotech Inc.）
  • Novartis AG
  • Orchard Therapeutics plc
  • UniQure N.V.
  • Voyager Therapeutics Inc.

List of Figures

• Figure 1: Global: Gene Therapy Market: Major Drivers and Challenges
• Figure 2: Global: Gene Therapy Market: Sales Value (in Billion USD), 2019-2024
• Figure 3: Global: Gene Therapy Market Forecast: Sales Value (in Billion USD), 2025-2033
• Figure 4: Global: Gene Therapy Market: Breakup by Gene Type (in %), 2024
• Figure 5: Global: Gene Therapy Market: Breakup by Vector Type (in %), 2024
• Figure 6: Global: Gene Therapy Market: Breakup by Delivery Method (in %), 2024
• Figure 7: Global: Gene Therapy Market: Breakup by Application (in %), 2024
• Figure 8: Global: Gene Therapy Market: Breakup by Region (in %), 2024
• Figure 9: Global: Gene Therapy (Antigen) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 10: Global: Gene Therapy (Antigen) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 11: Global: Gene Therapy (Cytokine) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 12: Global: Gene Therapy (Cytokine) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 13: Global: Gene Therapy (Tumor Suppressor) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 14: Global: Gene Therapy (Tumor Suppressor) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 15: Global: Gene Therapy (Suicide Gene) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 16: Global: Gene Therapy (Suicide Gene) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 17: Global: Gene Therapy (Deficiency) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 18: Global: Gene Therapy (Deficiency) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 19: Global: Gene Therapy (Growth Factors) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 20: Global: Gene Therapy (Growth Factors) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 21: Global: Gene Therapy (Receptors) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 22: Global: Gene Therapy (Receptors) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 23: Global: Gene Therapy (Other Gene Types) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 24: Global: Gene Therapy (Other Gene Types) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 25: Global: Gene Therapy (Viral Vector) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 26: Global: Gene Therapy (Viral Vector) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 27: Global: Gene Therapy (Non-Viral Techniques) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 28: Global: Gene Therapy (Non-Viral Techniques) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 29: Global: Gene Therapy (In-Vivo Gene Therapy) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 30: Global: Gene Therapy (In-Vivo Gene Therapy) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 31: Global: Gene Therapy (Ex-Vivo Gene Therapy) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 32: Global: Gene Therapy (Ex-Vivo Gene Therapy) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 33: Global: Gene Therapy (Oncological Disorders) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 34: Global: Gene Therapy (Oncological Disorders) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 35: Global: Gene Therapy (Rare Diseases) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 36: Global: Gene Therapy (Rare Diseases) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 37: Global: Gene Therapy (Cardiovascular Diseases) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 38: Global: Gene Therapy (Cardiovascular Diseases) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 39: Global: Gene Therapy (Neurological Disorders) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 40: Global: Gene Therapy (Neurological Disorders) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 41: Global: Gene Therapy (Infectious Disease) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 42: Global: Gene Therapy (Infectious Disease) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 43: Global: Gene Therapy (Other Applications) Market: Sales Value (in Million USD), 2019 & 2024
• Figure 44: Global: Gene Therapy (Other Applications) Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 45: North America: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 46: North America: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 47: United States: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 48: United States: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 49: Canada: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 50: Canada: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 51: Asia-Pacific: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 52: Asia-Pacific: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 53: China: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 54: China: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 55: Japan: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 56: Japan: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 57: India: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 58: India: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 59: South Korea: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 60: South Korea: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 61: Australia: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 62: Australia: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 63: Indonesia: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 64: Indonesia: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 65: Others: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 66: Others: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 67: Europe: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 68: Europe: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 69: Germany: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 70: Germany: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 71: France: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 72: France: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 73: United Kingdom: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 74: United Kingdom: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 75: Italy: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 76: Italy: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 77: Spain: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 78: Spain: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 79: Russia: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 80: Russia: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 81: Others: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 82: Others: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 83: Latin America: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 84: Latin America: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 85: Brazil: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 86: Brazil: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 87: Mexico: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 88: Mexico: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 89: Others: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 90: Others: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 91: Middle East and Africa: Gene Therapy Market: Sales Value (in Million USD), 2019 & 2024
• Figure 92: Middle East and Africa: Gene Therapy Market: Breakup by Country (in %), 2024
• Figure 93: Middle East and Africa: Gene Therapy Market Forecast: Sales Value (in Million USD), 2025-2033
• Figure 94: Global: Gene Therapy Industry: SWOT Analysis
• Figure 95: Global: Gene Therapy Industry: Value Chain Analysis
• Figure 96: Global: Gene Therapy Industry: Porter's Five Forces Analysis

List of Tables

• Table 1: Global: Gene Therapy Market: Key Industry Highlights, 2024 and 2033
• Table 2: Global: Gene Therapy Market Forecast: Breakup by Gene Type (in Million USD), 2025-2033
• Table 3: Global: Gene Therapy Market Forecast: Breakup by Vector Type (in Million USD), 2025-2033
• Table 4: Global: Gene Therapy Market Forecast: Breakup by Delivery Method (in Million USD), 2025-2033
• Table 5: Global: Gene Therapy Market Forecast: Breakup by Application (in Million USD), 2025-2033
• Table 6: Global: Gene Therapy Market Forecast: Breakup by Region (in Million USD), 2025-2033
• Table 7: Global: Gene Therapy Market: Competitive Structure
• Table 8: Global: Gene Therapy Market: Key Players

The global gene therapy market size was valued at USD 5.8 Billion in 2024. Looking forward, IMARC Group estimates the market to reach USD 17.9 Billion by 2033, exhibiting a CAGR of 13.3% from 2025-2033. North America currently dominates the market, holding a market share of over 56% in 2024. The North American market is driven by robust healthcare infrastructure, rising investments in research operations, advanced regulatory frameworks, and the presence of leading biotechnology firms fostering innovation.

Gene Therapy Market Analysis:

Market Growth and Size: The industry is expanding rapidly, fueled by the rising prevalence of genetic abnormalities and rare diseases. Additionally, pharmaceutical businesses are increasingly investing in research and development (R&D) activities, which are positively impacting the market.

Technological Advancements: Developments in viral vector technology are enhancing the efficacy and safety of gene therapy delivery. Moreover, CRISPR-Cas9 technology improves gene editing capabilities, which further accelerates gene therapy market statistics.

Industry Applications: Gene therapy is used to treat a variety of ailments, including cancer, genetic problems, and autoimmune conditions. Ex vivo gene therapy, in which cells are manipulated outside the body, is gaining popularity for treating specific genetic illnesses.

Geographical Trends: As per the gene therapy market statistics by IMARC, North America dominates the market, owing to its strong healthcare infrastructure and developed biotechnology sector. However, Asia Pacific is emerging as a rapidly expanding market, propelled by a big patient population.

Competitive Landscape: Leading players are conducting comprehensive clinical studies to develop and get regulatory clearance for novel gene treatments aimed at a variety of ailments, including rare genetic abnormalities and cancer. Some of the key players in the market include Abeona Therapeutics Inc., Adaptimmune Therapeutics plc, Adverum Biotechnologies Inc., Amgen Inc., Astellas Pharma Inc., Biogen Inc., Bluebird Bio Inc., Mustang Bio Inc. (Fortress Biotech Inc.), Novartis AG, Orchard Therapeutics plc, UniQure N.V., Voyager Therapeutics Inc., etc.

Challenges and Opportunities: While the industry confronts constraints like regulatory limitations and high research costs, it also expects possibilities for increasing uses beyond rare disorders and investigating novel delivery modalities.

Future Outlook: The gene therapy market outlook is expected to be positive, with rising demand for novel therapies for genetic abnormalities and uncommon illnesses. Furthermore, individualized gene therapy tailored to unique patient characteristics is projected to drive market expansion.

Gene Therapy Market Trends:

Advancements in Viral Vector Technology

Continuous progress and innovation in viral vector technology are important factors driving market growth. Viral vectors are used as delivery vehicles to introduce therapeutic genes into the cells of patients. Recent advances in this technique have greatly improved the safety and efficacy of gene therapy therapies. These developments include the creation of more specific and focused viral vectors, such as adeno-associated viruses (AAVs) and lentiviruses, with AAVs gaining popularity due to their ability to transport genetic material with minimal immune reaction and damage. Furthermore, as per the gene therapy market overview, researchers are focusing on developing viral vectors that may carry bigger genetic payloads, broadening the range of curable disorders. These technical innovations not only increase the efficacy of gene treatments but also minimize the likelihood of adverse effects, making them safer for patients. As a result, the advancement of viral vector technology is accelerating the acceptance and efficacy of gene therapy treatments for a broader range of medical problems. For instance, MERCK KGaA, the VirusExpress 293 Adeno-Associated Virus (AAV) Production Platform, offers full viral vector manufacturing, including AAV and lentiviral vectors. Additionally, Catalent Inc. launched UpTempo Virtuoso platform process for the development and manufacturing of adeno-associated viral (AAV) vectors.

Investment And Funding in Research and Development

The growing investments and funding for gene therapy research and development (R&D) efforts are driving market expansion. Pharmaceutical businesses, biotechnology firms, and government agencies are putting major resources into developing gene therapy technologies and therapeutics. Gene treatments have a strong potential for addressing unmet medical needs and providing long-term benefits, which is drawing significant funding. Major pharmaceutical corporations are either purchasing gene therapy startups or building their own gene therapy sections to speed up research, which is positively impacting the gene therapy market outlook. Furthermore, government subsidies and incentives motivate academic institutions and research firms to conduct gene therapy experiments. The Orphan Drug Act in the United States, as well as comparable legislation in other countries, gives incentives for the development of rare illness medicines. For instance, the Cystic Fibrosis Foundation reported investing in SalioGen Therapeutics to support the company's preclinical research into novel gene therapy for cystic fibrosis. Salonen's Gene Coding approach is designed to turn on, turn off, or modify the function of any gene in the genome.

Rising Prevalence of Genetic Disorders and Rare Diseases

The rising prevalence of genetic abnormalities and rare illnesses is propelling the market growth. Many of these diseases have little or no effective treatment choices, making gene therapy an intriguing alternative. As our understanding of genetics advances, more people are diagnosed, and the demand for novel medicines grows. Moreover, gene therapy has the ability to treat the underlying causes of many disorders by repairing or replacing defective genes. This comprises illnesses including muscular dystrophy, cystic fibrosis, and other hereditary metabolic disorders. Pharmaceutical and biotechnology businesses are investing in gene therapy research to fulfill this expanding medical demand, resulting in market expansion and improvements in the area. For instance, as per the United States Center for Disease Control and Prevention article on spinal muscular atrophy (SMA), SMA is a genetic disorder that affects around 1 in every 10,000 people. Therefore, it is one of the most common rare diseases.

Supportive Regulatory Environment

Regulatory organizations in several countries, especially the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), recognize the potential of gene therapy to address essential medical needs. Consequently, they have created structures and accelerated the research and approval of gene treatments. Additionally, fast-track designations, breakthrough treatment designations, and orphan drug status are among the regulatory measures available to gene therapy candidates. These designations ease the clinical trial procedure, shorten development schedules, and encourage corporations to participate in gene therapy research. For instance, the U.S. FDA approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia. Such approvals are expected to drive market growth due to the rise in the development of gene therapies.

Gene Therapy Market Segmentation:

Breakup by Gene Type:

Antigen

Cytokine

Tumor Suppressor

Suicide Gene

Deficiency

Growth Factors

Receptors

Others

Antigen genes are a critical segment in gene therapy, especially in the context of cancer immunotherapy. These genes code for tumor-specific antigens or antigens associated with infectious agents. By introducing antigen genes into the cells of the patients, the immune system can be activated to recognize and target cancer cells or pathogens. They also enable personalized cancer treatments, where patient-specific antigens can be targeted for a more effective and targeted immune response. For instance, Imugene reported a new clinical trial collaboration and supply agreement with MSD, a tradename of Merck & Co., Inc., Kenilworth, NJ, USA, to evaluate the safety and efficacy of Imugene's HER-Vaxx, a B-cell activating immunotherapy, in combination with MSD's anti-PD-1 therapy, pembrolizumab (KEYTRUDA), in patients with HER-2 positive gastric cancer.

Breakup by Vector Type:

Viral Vector

Adenoviruses

Lentiviruses

Retroviruses

Adeno-Associated Virus

Herpes Simplex Virus

Poxvirus

Vaccinia Virus

Others

Non-Viral Techniques

Naked and Plasmid Vectors

Gene Gun

Electroporation

Lipofection

Others

Viral vector holds the largest share in the industry.

Viral vectors, such as adeno-associated viruses (AAVs) and lentiviruses, are gaining traction due to their efficient delivery of therapeutic genes into target cells. AAVs are favored for their safety profile and ability to integrate genetic material into the host genome. They are widely used for a variety of gene therapy applications, including treating genetic disorders, rare diseases, and certain types of cancer. Ongoing research and advancements in viral vector technology are driving the growth of this segment, with numerous clinical trials and approved therapies utilizing viral vectors. For instance, the U.S. FDA granted approval at an accelerated pace for SKYSONA- a lentiviral vector gene therapy shown to slow the progression of neurodegenerative dysfunction, or active cerebral adrenoleukodystrophy in boys with the age range of 4-17.

Breakup by Delivery Method:

In-Vivo Gene Therapy

Ex-Vivo Gene Therapy

In-vivo gene therapy involves the direct delivery of therapeutic genes or genetic material into the body of the patient. This approach aims to modify or correct genetic defects within the tissues or organs of patients in their natural physiological environment. In-vivo gene therapy methods typically utilize viral vectors, such as adeno-associated viruses (AAVs) or lentiviruses, to transport the therapeutic genes. This segment of gene therapy is often used for diseases that require systemic treatment, such as certain types of inherited disorders or conditions where the target tissue is easily accessible within the body. For instance, recent advancements in gene supplementation therapy are expanding the options for the treatment of neurological disorders. Among the available delivery vehicles, adeno-associated virus (AAV) is often the favored vector.

Breakup by Application:

Oncological Disorders

Rare Diseases

Cardiovascular Diseases

Neurological Disorders

Infectious Disease

Others

Oncological disorders account for the majority of the market share.

Gene therapy is effective in the treatment of various cancers, including solid tumors and hematological malignancies. Approaches range from introducing tumor-suppressing genes to activating the immune system against cancer cells. CAR-T cell therapy, a type of gene therapy, is gaining traction for its success in treating certain types of leukemia and lymphoma. The ongoing research and development (R&D) activities of gene therapies for oncological disorders are impelling the market growth. For instance, the U.S. FDA approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple myeloma who have not responded to or whose disease has returned after at least four last lines (different types) of treatment. Abecma is the first cell-based gene therapy approved by the FDA for treating multiple myeloma.

Breakup by Region:

North America

United States

Canada

Asia-Pacific

China

Japan

India

South Korea

Australia

Indonesia

Others

Europe

Germany

France

United Kingdom

Italy

Spain

Russia

Others

Latin America

Brazil

Mexico

Others

Middle East and Africa

North America leads the market, accounting for the largest gene therapy market share.

The market research report has also provided a comprehensive analysis of all the major regional markets, which include North America (the United States and Canada); Asia Pacific (China, Japan, India, South Korea, Australia, Indonesia, and others); Europe (Germany, France, the United Kingdom, Italy, Spain, Russia, and others); Latin America (Brazil, Mexico, and others); and the Middle East and Africa. According to the report, North America accounted for the largest market share.

North America has a robust healthcare infrastructure, a well-established biotechnology industry, and significant investment in gene therapy research and development (R&D). Favorable regulatory policies, including fast-track designations and orphan drug status, are accelerating the approval and commercialization of gene therapies. Key players in the gene therapy market are headquartered in North America, contributing to the leadership of the region in gene therapy innovations. For instance, as per an update by the National Institutes of Health (NIH), the NIH, USFDA, ten pharmaceutical companies, and five non-profit organizations have partnered to accelerate the development of gene therapies for the 30 million Americans who suffer from a rare disease. Such initiatives are anticipated to increase the demand for gene therapy, thereby contributing to market growth.

Competitive Landscape:

Key industry competitors are currently involved in a number of strategic initiatives. These firms are constantly growing their gene therapy portfolios via research, acquisitions, and collaborations. They are undertaking comprehensive clinical studies to develop and get regulatory clearance for novel gene treatments that will treat a variety of ailments, including rare genetic abnormalities and cancer. In addition, they are investing in manufacturing facilities to increase output and ensure gene treatments are available to a larger patient population. Furthermore, these industry heavyweights are pursuing collaborations with academic institutions and smaller biotech businesses to promote innovation and improve gene therapy technology.

The market research report has provided a comprehensive analysis of the competitive landscape. Detailed profiles of all major companies have also been provided. Some of the key players in the market include:

Abeona Therapeutics Inc.

Adaptimmune Therapeutics plc

Adverum Biotechnologies Inc.

Amgen Inc.

Astellas Pharma Inc.

Biogen Inc.

Bluebird Bio Inc.

Mustang Bio Inc. (Fortress Biotech Inc.)

Novartis AG

Orchard Therapeutics plc

UniQure N.V.

Voyager Therapeutics Inc.

(Please note that this is only a partial list of the key players, and the complete list is provided in the report.)

Gene Therapy Market News:

January 2023: Spark Therapeutics and Neurochase established a strategic collaboration to develop Neurochase's unique delivery technology for use with selected gene treatments for rare disorders in the CNS. In this agreement, Neurochase will contribute its extensive knowledge in direct drug delivery technology to Spark's premier AAV platform.

January 2023: Voyager Therapeutics and Neurocrine Biosciences entered into a strategic collaboration for the commercialization & development of Voyager's GBA1 program and other next-generation gene therapies for neurological diseases.

June 2023: The U.S. FDA granted approval to Sarepta for ELEVIDYS gene therapy to treat DMD in children of age 4-5 years.

May 2023: Krystal Biotech was granted approval for VYJUVEK gene therapy for Dystrophic Epidermolysis Bullosa treatment.

December 2023: Swiss Agency for Therapeutic Products granted approval to Libmeldy for the treatment of early-onset metachromatic leukodystrophy.

January 2024: Biogen and Ginkgo Bioworks announced that they have completed the gene therapy collaboration involving AAV-based vectors. This is expected to fuel the demand for gene therapies in the coming years.

Key Questions Answered in This Report

• 1.What is gene therapy?
• 2.How big is the global gene therapy market?
• 3.What is the expected growth rate of the global gene therapy market during 2025-2033?
• 4.What are the key factors driving the global gene therapy market?
• 5.What is the leading segment of the global gene therapy market based on gene type?
• 6.What is the leading segment of the global gene therapy market based on vector type?
• 7.What is the leading segment of the global gene therapy market based on delivery method?
• 8.What is the leading segment of the global gene therapy market based on application?
• 9.What are the key regions in the global gene therapy market?
• 10.Who are the key players/companies in the global gene therapy market?

Table of Contents

1 Preface

2 Scope and Methodology

• 2.1 Objectives of the Study
• 2.2 Stakeholders
• 2.3 Data Sources
  • 2.3.1 Primary Sources
  • 2.3.2 Secondary Sources
• 2.4 Market Estimation
  • 2.4.1 Bottom-Up Approach
  • 2.4.2 Top-Down Approach
• 2.5 Forecasting Methodology

3 Executive Summary

4 Introduction

• 4.1 Overview
• 4.2 Key Industry Trends

5 Global Gene Therapy Market

• 5.1 Market Overview
• 5.2 Market Performance
• 5.3 Impact of COVID-19
• 5.4 Market Forecast

6 Market Breakup by Gene Type

• 6.1 Antigen
  • 6.1.1 Market Trends
  • 6.1.2 Market Forecast
• 6.2 Cytokine
  • 6.2.1 Market Trends
  • 6.2.2 Market Forecast
• 6.3 Tumor Suppressor
  • 6.3.1 Market Trends
  • 6.3.2 Market Forecast
• 6.4 Suicide Gene
  • 6.4.1 Market Trends
  • 6.4.2 Market Forecast
• 6.5 Deficiency
  • 6.5.1 Market Trends
  • 6.5.2 Market Forecast
• 6.6 Growth Factors
  • 6.6.1 Market Trends
  • 6.6.2 Market Forecast
• 6.7 Receptors
  • 6.7.1 Market Trends
  • 6.7.2 Market Forecast
• 6.8 Others
  • 6.8.1 Market Trends
  • 6.8.2 Market Forecast

7 Market Breakup by Vector Type

• 7.1 Viral Vector
  • 7.1.1 Market Trends
  • 7.1.2 Key Segments
    • 7.1.2.1 Adenoviruses
    • 7.1.2.2 Lentiviruses
    • 7.1.2.3 Retroviruses
    • 7.1.2.4 Adeno-Associated Virus
    • 7.1.2.5 Herpes Simplex Virus
    • 7.1.2.6 Poxvirus
    • 7.1.2.7 Vaccinia Virus
    • 7.1.2.8 Others
  • 7.1.3 Market Forecast
• 7.2 Non-Viral Techniques
  • 7.2.1 Market Trends
  • 7.2.2 Key Segments
    • 7.2.2.1 Naked and Plasmid Vectors
    • 7.2.2.2 Gene Gun
    • 7.2.2.3 Electroporation
    • 7.2.2.4 Lipofection
    • 7.2.2.5 Others
  • 7.2.3 Market Forecast

8 Market Breakup by Delivery Method

• 8.1 In-Vivo Gene Therapy
  • 8.1.1 Market Trends
  • 8.1.2 Market Forecast
• 8.2 Ex-Vivo Gene Therapy
  • 8.2.1 Market Trends
  • 8.2.2 Market Forecast

9 Market Breakup by Application

• 9.1 Oncological Disorders
  • 9.1.1 Market Trends
  • 9.1.2 Market Forecast
• 9.2 Rare Diseases
  • 9.2.1 Market Trends
  • 9.2.2 Market Forecast
• 9.3 Cardiovascular Diseases
  • 9.3.1 Market Trends
  • 9.3.2 Market Forecast
• 9.4 Neurological Disorders
  • 9.4.1 Market Trends
  • 9.4.2 Market Forecast
• 9.5 Infectious Disease
  • 9.5.1 Market Trends
  • 9.5.2 Market Forecast
• 9.6 Others
  • 9.6.1 Market Trends
  • 9.6.2 Market Forecast

10 Market Breakup by Region

• 10.1 North America
  • 10.1.1 United States
    • 10.1.1.1 Market Trends
    • 10.1.1.2 Market Forecast
  • 10.1.2 Canada
    • 10.1.2.1 Market Trends
    • 10.1.2.2 Market Forecast
• 10.2 Asia-Pacific
  • 10.2.1 China
    • 10.2.1.1 Market Trends
    • 10.2.1.2 Market Forecast
  • 10.2.2 Japan
    • 10.2.2.1 Market Trends
    • 10.2.2.2 Market Forecast
  • 10.2.3 India
    • 10.2.3.1 Market Trends
    • 10.2.3.2 Market Forecast
  • 10.2.4 South Korea
    • 10.2.4.1 Market Trends
    • 10.2.4.2 Market Forecast
  • 10.2.5 Australia
    • 10.2.5.1 Market Trends
    • 10.2.5.2 Market Forecast
  • 10.2.6 Indonesia
    • 10.2.6.1 Market Trends
    • 10.2.6.2 Market Forecast
  • 10.2.7 Others
    • 10.2.7.1 Market Trends
    • 10.2.7.2 Market Forecast
• 10.3 Europe
  • 10.3.1 Germany
    • 10.3.1.1 Market Trends
    • 10.3.1.2 Market Forecast
  • 10.3.2 France
    • 10.3.2.1 Market Trends
    • 10.3.2.2 Market Forecast
  • 10.3.3 United Kingdom
    • 10.3.3.1 Market Trends
    • 10.3.3.2 Market Forecast
  • 10.3.4 Italy
    • 10.3.4.1 Market Trends
    • 10.3.4.2 Market Forecast
  • 10.3.5 Spain
    • 10.3.5.1 Market Trends
    • 10.3.5.2 Market Forecast
  • 10.3.6 Russia
    • 10.3.6.1 Market Trends
    • 10.3.6.2 Market Forecast
  • 10.3.7 Others
    • 10.3.7.1 Market Trends
    • 10.3.7.2 Market Forecast
• 10.4 Latin America
  • 10.4.1 Brazil
    • 10.4.1.1 Market Trends
    • 10.4.1.2 Market Forecast
  • 10.4.2 Mexico
    • 10.4.2.1 Market Trends
    • 10.4.2.2 Market Forecast
  • 10.4.3 Others
    • 10.4.3.1 Market Trends
    • 10.4.3.2 Market Forecast
• 10.5 Middle East and Africa
  • 10.5.1 Market Trends
  • 10.5.2 Market Breakup by Country
  • 10.5.3 Market Forecast

11 SWOT Analysis

• 11.1 Overview
• 11.2 Strengths
• 11.3 Weaknesses
• 11.4 Opportunities
• 11.5 Threats

12 Value Chain Analysis

13 Porters Five Forces Analysis

• 13.1 Overview
• 13.2 Bargaining Power of Buyers
• 13.3 Bargaining Power of Suppliers
• 13.4 Degree of Competition
• 13.5 Threat of New Entrants
• 13.6 Threat of Substitutes

14 Price Analysis

15 Competitive Landscape

• 15.1 Market Structure
• 15.2 Key Players
• 15.3 Profiles of Key Players
  • 15.3.1 Abeona Therapeutics Inc.
    • 15.3.1.1 Company Overview
    • 15.3.1.2 Product Portfolio
  • 15.3.2 Adaptimmune Therapeutics plc
    • 15.3.2.1 Company Overview
    • 15.3.2.2 Product Portfolio
  • 15.3.3 Adverum Biotechnologies Inc.
    • 15.3.3.1 Company Overview
    • 15.3.3.2 Product Portfolio
    • 15.3.3.3 Financials
  • 15.3.4 Amgen Inc.
    • 15.3.4.1 Company Overview
    • 15.3.4.2 Product Portfolio
    • 15.3.4.3 Financials
    • 15.3.4.4 SWOT Analysis
  • 15.3.5 Astellas Pharma Inc.
    • 15.3.5.1 Company Overview
    • 15.3.5.2 Product Portfolio
    • 15.3.5.3 Financials
    • 15.3.5.4 SWOT Analysis
  • 15.3.6 Biogen Inc.
    • 15.3.6.1 Company Overview
    • 15.3.6.2 Product Portfolio
    • 15.3.6.3 Financials
    • 15.3.6.4 SWOT Analysis
  • 15.3.7 Bluebird Bio Inc.
    • 15.3.7.1 Company Overview
    • 15.3.7.2 Product Portfolio
    • 15.3.7.3 Financials
    • 15.3.7.4 SWOT Analysis
  • 15.3.8 Mustang Bio Inc. (Fortress Biotech Inc.)
    • 15.3.8.1 Company Overview
    • 15.3.8.2 Product Portfolio
  • 15.3.9 Novartis AG
    • 15.3.9.1 Company Overview
    • 15.3.9.2 Product Portfolio
    • 15.3.9.3 Financials
    • 15.3.9.4 SWOT Analysis
  • 15.3.10 Orchard Therapeutics plc
    • 15.3.10.1 Company Overview
    • 15.3.10.2 Product Portfolio
    • 15.3.10.3 Financials
  • 15.3.11 UniQure N.V.
    • 15.3.11.1 Company Overview
    • 15.3.11.2 Product Portfolio
    • 15.3.11.3 Financials
  • 15.3.12 Voyager Therapeutics Inc.
    • 15.3.12.1 Company Overview
    • 15.3.12.2 Product Portfolio
    • 15.3.12.3 Financials