Japan Gene Therapy Market is expected to reach US$ 904.94 Million by 2033 | Major Companies - Alnylam Pharmaceuticals, Inc., NOVARTIS AG, Sarepta Therapeutics, Inc., Krystal Biotech, Inc.

Market Size and Growth:

The Japan Gene Therapy Market size reached US$ 148.06 Million in 2024 and is expected to reach US$ 904.94 Million by 2033, growing at a CAGR of 20.3 % during the forecast period 2025-2033.

The Japan Gene Therapy Market report, published by DataM Intelligence, provides in-depth insights and analysis on key market trends, growth opportunities, and emerging challenges. Committed to delivering actionable intelligence, DataM Intelligence empowers businesses to make informed decisions and stay ahead of the competition. Through a combination of qualitative and quantitative research methods, it offers comprehensive reports that help clients navigate complex market landscapes, drive strategic growth, and seize new opportunities in an ever-evolving global market.

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The Japan Gene Therapy Market encompasses the development, commercialization, and application of therapies that modify or manipulate genes to treat or prevent diseases. It includes viral and non-viral vectors, CAR-T therapies, and genome-editing technologies, targeting conditions such as cancer, genetic disorders, and rare diseases. Growth is driven by increasing R&D, regulatory support, and advancements in personalized medicine.

Recent Key Developments of Japan:

✅ December 2025: AskBio's AB-1005 and AB-1002 received Pioneering Regenerative Medical Product designation from Japan's MHLW for Parkinson's disease and congestive heart failure.

✅ October 2025: Otsuka Pharmaceutical entered a licensing agreement for 4D-150, an ophthalmic gene therapy drug candidate targeting anti-VEGF for eye diseases.

✅ September 2025: Japan launched the JEAP program to accelerate regenerative medicine and cell/gene therapy market entry for startups, offering mentorship and connections to CDMOs and pharma.

Recent Key Developments of Europe:

✅ November 2025: First European patients were randomized in the REGENERATE-PD Phase II trial for AB-1005, an investigational gene therapy for moderate-stage Parkinson's disease.

✅ September 2025: ExCellThera's Zemcelpro, a non-genetically modified cell therapy for hematological malignancies, received EU authorization.

✅ September 2025: Cell Therapies and Teijin announced a strategic collaboration to expand access to advanced cell and gene therapies across Japan and the Asia Pacific region, with implications for European supply chains.

List of the Key Players in the Japan Gene Therapy Market:

Alnylam Pharmaceuticals, Inc.
NOVARTIS AG
Sarepta Therapeutics, Inc.
Krystal Biotech, Inc.
CSL
Bluebird Bio, Inc.
SPARK THERAPEUTICS, INC.
Ferring
Vertex Pharmaceuticals Incorporated
Amgen, Inc
Orchard Therapeutics Plc
Gensight Biologics
Ultragenyx Pharmaceutical Inc.
REGENXBIO Inc.
Oxford Biomedica PLC
Benitec Biopharma Inc.
Transgene
Sangamo Therapeutics

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This Report Covers:

✔ Go-to-market Strategy.

✔ Neutral perspective on the market performance.

✔Development trends, competitive landscape analysis, supply side analysis, demand side analysis, year-on-year growth, competitive benchmarking, vendor identification, and other significant analysis, as well as development status.

✔Customized regional/country reports as per request and country level analysis.

✔ Potential & niche segments and regions exhibiting promising growth covered.

✔ Analysis of Market Size (historical and forecast), Total Addressable Market (TAM), Serviceable Available Market (SAM), Serviceable Obtainable Market (SOM), Market Growth, Technological Trends, Market Share, Market Dynamics, Competitive Landscape and Major Players (Innovators, Start-ups, Laggard, and Pioneer).

Segments Covered in the Japan Gene Therapy Market:

By Approach: In-Vivo, Ex vivo.

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

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

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

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Chapter Outline:

⏩ Market Overview: It contains five chapters, as well as information about the research scope, major manufacturers covered, market segments, Japan Gene Therapy market segments, study objectives, and years considered.

⏩ Market Landscape: The competition in the Japan Gene Therapy Market is evaluated here in terms of value, turnover, revenues, and market share by organization, as well as market rate, competitive landscape, and recent developments, transaction, growth, sale, and market shares of top companies.

⏩ Companies Profiles: The Japan Gene Therapy market's leading players are studied based on sales, main products, gross profit margin, revenue, price, and growth production.

⏩ Market Outlook by Region: The report goes through gross margin, sales, income, supply, market share, CAGR, and market size by region in this segment. North America, Europe, Asia Pacific, Middle East & Africa, and South America are among the regions and countries studied in depth in this study.

⏩ Market Segments: It contains the deep research study which interprets how different end-user/application/type segments contribute to the Japan Gene Therapy Market.

⏩ Market Forecast: Production Side: In this part of the report, the authors have focused on production and production value forecast, key producers forecast, and production and production value forecast by type.

⏩ Research Findings: This section of the report showcases the findings and analysis of the report.

⏩ Conclusion: This portion of the report is the last section of the report where the conclusion of the research study is provided.

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