United States Gene Therapy Market is expected to reach US$ 35.91 Billion by 2033 | Top key players - Novartis AG, Amgen, Inc., CSL Behring LLC.

Market Size and Growth:

The Gene Therapy Market size reached US$ 4.80 Billion in 2024 and is expected to reach US$ 35.91 Billion by 2033, growing at a CAGR of 23.1% during the forecast period 2025-2033. The Market is driven by increasing approvals of innovative therapies, rising prevalence of genetic disorders, and advancements in viral vector technologies. According to DataM Intelligence

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The Gene Therapy Market involves the development and commercialization of treatments that modify or replace defective genes to cure or prevent diseases. It focuses on delivering functional genes into patients' cells using viral or non-viral vectors. This market is driven by advances in genetic engineering, personalized medicine, and the rising prevalence of genetic disorders.

Industry Recent Developments:

United States:

✅ October 2025: The U.S. gene therapy market size reached USD 11.4 billion in 2025 with a projected CAGR of 20% through 2034. This growth is driven by increased financing, adoption of viral vectors, and collaboration among market players to spur innovation and cost savings. Regulatory advancements continue to accelerate approvals for gene therapies.

✅ October 2025: Cell and gene therapy clinical trials in the U.S. are rapidly expanding with a market value growth from USD 4.64 billion in 2024 to a projected USD 17.03 billion by 2033, driven by increased R&D funding, a favorable regulatory environment, and expanded applications in oncology and personalized therapies.

✅ September 2025: The FDA has continued robust support for gene therapy approvals, including accelerated pathways like RMAT designations, speeding development and access to innovative treatments for genetic and rare diseases.

Japan:

✅ October 2025: Japan's gene therapy market is projected to grow with a CAGR of 22.79% from 2025 to 2032. Growth drivers include rising cases of genetic disorders, increased research collaborations, government support, and regulatory reforms enabling faster product approvals under the Pharmaceutical and Medical Devices Act.

✅ September 2025: Japanese authorities have updated regulations to foster cell and gene therapy development, including accelerated approval processes for regenerative medicines, facilitating quicker commercialization of novel therapies.

✅ September 2025: Notable advancements in Japan include approval expansions for CAR-T therapies targeting refractory multiple myeloma by Bristol-Myers Squibb K.K., reflecting industry progress and supportive regulatory framework in the country.

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FDA Approvals & Regulatory Actions:

✅ ENCELTO (revakinagene taroretcel-lwey) - FDA biologics license approved early March 2025 for MacTel type 2; first approved therapy for this rare retinal disease. (See FDA approval letter and Summary Basis for Regulatory Action.)

✅ Regulatory oversight & safety signals - FDA continued to scrutinize marketed gene therapies and suspend or place holds when safety signals emerged (example: actions/requests involving Sarepta's Elevidys programs in 2025). This highlights the continuing emphasis on long-term safety monitoring for gene therapies.

Mergers & Acquisitions (2025):

✅ Bristol Myers Squibb → 2seventy bio - BMS moved to acquire 2seventy bio (cancer cell therapy partner) in a deal announced in March 2025 to consolidate CAR-T economics and rights.

✅ AstraZeneca → EsoBiotec - AstraZeneca agreed to acquire EsoBiotec (cell therapy biotech) in a deal (up to ~$1B) announced in 2025 as part of expanding cell/gene oncology investments.

✅ Sector trend: Q2/H1 2025 saw many mid-sized biopharma deals (gene/cell platforms included), with total biopharma M&A value concentrated in a handful of strategic platform buys - the market favored platform & ophthalmology/rare-disease plays.

EU / EMA Regulatory Highlights:

✅ Vyjuvek (beremagene geperpavec) - the EMA/Committee for Advanced Therapies and European Commission completed regulatory steps in 2025 (marketing authorisation issued 23 April 2025) for a topical gene therapy for dystrophic epidermolysis bullosa - an example of active ATMP approvals in the EU in 2025.

✅ Regulatory caution & trial pauses - the EMA continued to pause or request additional oversight on gene therapy trials where safety concerns appeared (e.g., actions tied to Sarepta's DMD program in 2025). This underscores parallel caution from both FDA and EMA on long-term safety.

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Major Key Players:

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

Segments Covered in the Gene Therapy Market:

By Approach: Ex-Vivo, In-Vivo.

By Vector Type: Viral Vectors, Non-Viral Vectors.

By Technique: Gene Addition, Gene Silencing, Gene Editing.

By Application: Oncology, Musculoskeletal Conditions, Blood Disorders, Rare Diseases, Ophthalmology, Others.

Regional Analysis for Gene Therapy Market:

⇥ North America (U.S., Canada, Mexico)

⇥ Europe (U.K., Italy, Germany, Russia, France, Spain, The Netherlands and Rest of Europe)

⇥ Asia-Pacific (India, Japan, China, South Korea, Australia, Indonesia Rest of Asia Pacific)

⇥ South America (Colombia, Brazil, Argentina, Rest of South America)

⇥ Middle East & Africa (Saudi Arabia, U.A.E., South Africa, Rest of Middle East & Africa)

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Frequently asked questions:

➠ What is the global sales value, production value, consumption value, import and export of Gene Therapy market?

➠ Who are the global key manufacturers of the Gene Therapy Industry? How is their operating situation (capacity, production, sales, price, cost, gross, and revenue)?

➠ What are the Gene Therapy market opportunities and threats faced by the vendors in the global Gene Therapy Industry?

➠ Which application/end-user or product type may seek incremental growth prospects? What is the market share of each type and application?

➠ What focused approach and constraints are holding the Gene Therapy market?

➠ What are the different sales, marketing, and distribution channels in the global industry?

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