Japan Gene Therapy Market Growth: $148.06M in 2024 to $904.94M by 2033 | CAGR 20.3%

AUSTIN, TX, UNITED STATES, June 20, 2025 /EINPresswire.com/ -- Japan Gene Therapy Market Overview 2025

In 2024, the Japan Gene Therapy Market Size was valued at US$ 148.06 Million in 2024 and is forecasted to see substantial growth, reaching around US$ 904.94 Million by 2033, with an anticipated CAGR of 20.3% during the period from 2025 to 2033.

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Regional Outlook

Gene therapy is gaining ground across Japan’s major regions:
Kanto (Tokyo area) is home to the country’s top biotech startups and major pharmaceutical headquarters, where most clinical trials and regulatory activities are coordinated.

Kansai (Osaka, Kyoto) is becoming a center for regenerative medicine and gene editing research.

Kyushu and Tohoku are witnessing the emergence of research hubs focused on neurological and inherited diseases.

Chubu and Hokkaido are showing interest in gene therapy logistics and delivery systems, including ultra-cold storage and transportation networks.
This regional expansion minimizes bottlenecks and encourages localized innovation, positioning Japan as a leader in the APAC gene therapy market.

Company Landscape

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

Market Segmentation:

By Approach: In-Vivo, Ex vivo

By Vector Type: Viral Vectors, Adeno-Associated Virus , Herpes Simplex Virus, Lentivirus, 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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Latest Developments

In July 2023, Japan's Pharmaceuticals and Medical Devices Agency (PMDA) approved Novartis' Luxturna to treat inherited retinal dystrophies (IRDs) caused by biallelic mutations in the RPE65 gene. This gene therapy is now accessible in Japan for individuals affected by this rare genetic disorder.

Latest News from Japan

New Approval for Rare Disease Treatment

Japan’s drug regulators recently approved a new gene therapy for children suffering from a rare form of muscular dystrophy. This treatment is designed for a specific subset of patients and has been approved with a limited license for ongoing data collection. Japan’s conditional approval process allows patients faster access to innovative therapies while ensuring long-term safety and effectiveness.

Expansion of Clinical Trials

More gene therapy clinical trials are being launched in Japan than ever before. Hospitals and research institutions are now working with pharmaceutical partners to test new therapies for inherited blood disorders, eye diseases, and neurological conditions. This increase in trials is helping to build a stronger evidence base and more local data for regulatory decisions.

Cold Chain Innovations

Japanese companies are also leading the way in solving one of gene therapy’s toughest logistical challenges: maintaining ultra-cold storage conditions during transit. A successful long-distance delivery of sensitive gene therapy products, maintained at sub-zero temperatures, was recently completed, demonstrating Japan’s readiness for global therapy distribution.

Experts Thoughts:

The gene therapy market in Japan is no longer a future vision it’s a thriving, rapidly growing field that holds promise for transforming how chronic and rare diseases are treated. With strong government support, world-class researchers, and an eager medical community, Japan is uniquely positioned to become a global leader in gene therapy development.

The parallel developments in the U.S. reinforce the idea that gene therapy is going global, and Japan is playing a crucial role in this transformation. From regulatory speed to scientific rigor and infrastructure readiness, Japan is laying the foundation for a healthier, gene-powered future.

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Sai Kiran
DataM Intelligence 4Market Research
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