Gene Therapy Market Size and Forecast (2021 - 2031), Global and Regional Share, Trend, and Growth Opportunity Analysis Report Coverage: By Vectors (Non-Viral Vectors and Viral Vectors), Indication (Neurological Diseases, Cancer, Duchenne Muscular Dystroph

The gene therapy market size is projected to reach US$ 12.92 billion by 2031 from US$ 5.06 billion in 2024. The market is expected to register a CAGR of 14.5% during 2025-2031. Growing burden of genetic diseases and the advancements in genetic engineering techniques are major factors driving the market growth. Further, the advancements in in vivo gene delivery and nucleic acid is likely to boost the market during the forecast period. However, high costs and affordability issues are among the market deterrents.

The development and use of treatments can be accelerated by precise, effective, and safer modifications to the human genome made possible by advances in genetic engineering technologies, especially CRISPR/Cas9, base editing, and enhanced viral vectors. Clinical trials using CRISPR have significantly increased, progressing past their early stages to treat a variety of illnesses, such as blood disorders like sickle cell disease and beta-thalassemia, different kinds of cancer, and cardiovascular diseases. Precision medicine has new opportunities thanks to the development of CRISPR genome editing. According to the Innovative Genomics Institute, as of 2022, clinical trials are being conducted in seven treatment areas: blood disorders, cancers, inherited eye diseases, diabetes, infectious diseases, inflammatory diseases, and protein-folding disorders.

Therefore, the technological advancements in genetic engineering is propelling the gene therapy market growth.

Further, The adoption of AI, machine learning, and IoT-based technologies is transforming gene therapy medicine. Generative AI models advance personalized medicine by enabling precise treatment predictions and generating patient-specific drug formulations based on genetic profiles. Digital twins, powered by AI and IoT, create virtual patient models for simulating gene therapy responses. These integrations are fostering a shift toward AI-generated personalized regimens, requiring infrastructure adjustments for real-time data processing in gene therapy protocols. Market players such as Gilead Sciences, MeiraGTx, and CRISPR Therapeutics AG are exploring AI-integrated gene therapy solutions. Therefore, the integration of AI and Internet of things in gene therapies are expected to be gene therapy market trend and gain market share during the forecast period.

The comparative company analysis evaluates and categorizes the gene therapy market based on product portfolio (product satisfaction, product features, and availability), recent market developments (merger & acquisition, new product launch & enhancement, investment & funding, award, agreement, collaboration, & partnership, recognition, and expansion), and geographic presence that aids better decision-making and understanding of the competitive landscape. The report profoundly explores the recent significant developments and innovations by the leading vendors in the global gene therapy market. The key market players Novartis AG, Astellas Pharma Inc, Bristol-Myers Squibb Co, Bluebird Bio Inc, Catalent Inc, Sanofi SA, Daiichi Sankyo Co Ltd, CSL Behring LLC, BioMarin Pharmaceutical Inc., and Vertex Pharmaceuticals Inc.

Based on vector, the market is categorized into non-viral vectors and viral vectors. The viral vectors segment held a significant gene therapy market share in 2024 and is projected to register a higher CAGR during the forecast period.

Based on indication, the market is categorized into neurological diseases, Duchenne muscular dystrophy, cancer, hepatological diseases, and other indications. The cancer segment held a significant gene therapy market share in 2024.

Based on delivery mode, the market is bifurcated into in-vivo and ex-vivo. The in-vivo segment held a significant gene therapy market share in 2024 and is expected to register a higher CAGR during 2025-2031. Advancements in delivery technology and the requirement for direct, effective genetic modification of bodily tissues are driving a sharp rise in the demand for in-vivo delivery modes in gene therapy. For instance, approved treatments for spinal muscular atrophy and inherited retinal diseases have been made possible by in-vivo therapies that use adeno-associated virus (AAV) vectors to deliver therapeutic genes directly to the affected tissues or systemically. The development of novel treatments for illnesses that were previously thought to be incurable is made possible by the growing emphasis on in-vivo delivery methods in both industry investment and clinical trial pipelines.

The scope of the gene therapy market report includes an assessment of the market performance in North America, Europe, Asia Pacific, South and Central America, and the Middle East and Africa. As per revenue, North America dominated the gene therapy market share in 2024.

Various organic and inorganic strategies are adopted by companies operating in the gene therapy market. The organic strategies mainly include product launches and product approvals. Inorganic growth strategies witnessed in the market are acquisitions, collaboration, and partnerships. These growth strategies allow the market players to expand their businesses and enhance their geographic presence, along with contributing to the overall market growth. Furthermore, strategies such as acquisitions and partnerships helped strengthen their customer base and extend their product portfolios. A few of the significant developments by key players in the gene therapy market are listed below.

Bluebird bio, Inc. announced new and updated data from LYFGENIA (lovotobegligene autotemcel, or lovo-cel) gene therapy for patients with sickle cell disease who have a history of vaso-occlusive events (VOEs). As of July 2024, 70 patients were treated across the complete lovo-cel clinical development program, with follow-up beyond 9 years in the earliest treated patients.
AviadoBio Ltd. and Astellas Pharma Inc. announced an exclusive option and license agreement for AVB-101, an investigational, AAV-based gene therapy in Phase 1/2 development for patients with frontotemporal dementia with progranulin mutations (FTD-GRN).

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