Japan Gene Therapy Market - 2025-2033

Japan Gene Therapy Market – Industry Trends & Outlook

The Japan gene therapy market 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.

Gene therapy is a medical approach that involves modifying, replacing, or supplementing a patient's genetic material to treat or cure diseases caused by genetic mutations or dysfunctions. It works by introducing functional genes, editing defective ones (e.g., using CRISPR-Cas9), or deactivating harmful genes to address conditions like genetic disorders, cancers, and chronic diseases at their root cause. This innovative method can employ viral vectors (e.g., adeno-associated viruses) or non-viral techniques to deliver therapeutic genes, offering potential cures rather than temporary symptom management.

One of the major market drivers is the rising product development activities and product approvals, advancements in gene editing and delivery technologies, and high unmet needs in oncology and rare diseases.

Opportunities in Japan are abundant, growing focus on personalized medicine, especially given the nation’s aging population and high prevalence of genetic and rare diseases. There is a strong demand for curative therapies targeting conditions such as hemophilia, spinal muscular atrophy, and certain inherited retinal disorders. Additionally, Japan’s government is investing in precision medicine and fostering collaborations between academia, industry, and government agencies, creating a fertile environment for research and commercialization.

Key trends shaping the market include a surge in partnerships between Japanese pharmaceutical companies and global biotech firms, increased adoption of advanced gene editing technologies like CRISPR, and a growing pipeline of both ex vivo and in vivo gene therapies.

Japan Gene Therapy Market Dynamics: Drivers

Rising research and development activities and product approvals

Japan has long been a leader in biotechnology and pharmaceutical innovation, and this extends to the gene therapy sector. The rapid development of gene editing techniques such as CRISPR-Cas9 and viral vector-based gene delivery systems has opened up new avenues for gene therapy research. Japanese companies and research institutions are leveraging these technologies to explore potential treatments for a wide range of genetic diseases, including rare conditions and those with no current treatment options.

As gene therapies continue to demonstrate their efficacy and safety in clinical trials, Japan's regulatory body (the PMDA, Pharmaceuticals and Medical Devices Agency) has been approving more gene therapy products. These approvals provide a significant boost to the market.

Also, in August 2024, Chugai Pharmaceutical submitted Elevidys (delandistrogene moxeparvovec/SRP-9001) for approval in Japan as a gene therapy for Duchenne muscular dystrophy (DMD). This regulatory filing is supported by data from a global phase III study (likely including the EMBARK trial), which demonstrated significant functional and clinical benefits in ambulatory patients.

Japan Gene Therapy Market Dynamics: Restraints

High cost of gene therapies

The high developmental and treatment costs of gene therapies are expected to be a major barrier to the growth of the gene therapy market in Japan, despite the significant potential health benefits these treatments offer. The costs of developing, manufacturing, and administering gene therapies are often exorbitantly high, making them inaccessible for many patients, especially in countries like Japan, where economic constraints are more pronounced.

Gene therapies are cutting-edge treatments that typically involve one-time procedures to correct or replace defective genes in a patient's cells. However, the development process for these therapies is expensive, involving complex research, manufacturing, and regulatory approvals. As a result, the cost of cell and gene therapies can range from USD 300,000 to USD 4 million per patient, depending on the specific therapy and condition being treated. One of the most well-known examples is Zolgensma, a gene therapy for spinal muscular atrophy (SMA). Zolgensma is priced at around USD 2.1 million per patient, which makes it one of the most expensive treatments in the world.

Japan Gene Therapy Market Segment Analysis

The Japan gene therapy market is segmented based on approach, vector type, technique, and application.

Approach: The in vivo segment was valued at US$ 131.77 million in 2024 and is estimated to reach US$ 832.54 million by 2033, growing at a CAGR of 22.7% during the forecast period from 2025-2033

In vivo gene therapy involves directly delivering the therapeutic gene into the patient’s body, typically using a viral vector to transport the gene to the target cells. Unlike ex vivo gene therapy, which involves extracting cells from the patient, editing them in a lab, and then reintroducing them into the body, in vivo therapy works by directly modifying genes within the patient’s body.

In vivo gene therapy offers the advantage of treating the disease directly within the patient’s body, without the need for complex cell collection and manipulation outside the body. This makes the process more efficient and minimizes the risk of complications that could arise from the ex vivo approach.

Japan is known for its strong scientific research capabilities and technological innovation, making it a prime location for cutting-edge gene therapy advancements. The continued improvements in vector technology and delivery methods make in vivo gene therapy a highly attractive option for both researchers and healthcare providers in the country.

Japan has a well-established healthcare system that prioritizes minimally invasive treatments and patient comfort, making in vivo therapy an appealing choice due to its simpler administration process. For instance, in March 2024, YolTech Therapeutics is a biotech company focused on developing in vivo gene editing therapies, which involve directly editing genes inside the body to treat genetic disorders. The company recently announced its participation in TIDES Asia 2024, a significant conference on drug discovery, development, and manufacturing in the field of biologics and gene therapies in Japan.

Japan Gene Therapy Market Major Players

The major players in the Japan gene therapy market include 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, and Sangamo Therapeutics, among others.

Japan Gene Therapy Market – Key Developments

In July 2023, the Japan Pharmaceutical and Medical Devices Agency (PMDA) granted approval to Novartis’ Luxturna for the treatment of inherited retinal dystrophies (IRDs) resulting from biallelic mutations in the RPE65 gene. This gene therapy, developed by Novartis, is now available in Japan for patients with this rare genetic condition.