East Asia in Phase 3: The Region ’s Momentum in Late-Stage Cell and Gene Therapy

East Asia is rapidly becoming a powerhouse in the global cell and gene therapy (CGT) landscape. With strong government support, cutting-edge biotech innovation, and expanding clinical infrastructure, countries like South Korea, Japan, China, and Taiwan are advancing a growing number of CGT programs into late-stage development.

The following table presents a curated snapshot of ongoing Phase 3 trials and status across the region. It highlights the diversity of therapeutic approaches from CAR-T and stem cell therapies to gene-modified products and viruses and their application to both rare and high-burden diseases.

Country	Company	Therapy	Modality	Indication(s)	Phase 3 / Approval Status
South Korea	Helixmith	Engensis (VM202)	Non-viral plasmid DNA gene therapy (HGF)	Diabetic Peripheral Neuropathy, Foot Ulcers	In Phase 3 trials in the U.S.
South Korea	Nature Cell	JointStem	Autologous adipose-derived mesenchymal stem cell(MSC therapy	Severe Knee Osteoarthritis	Phase 3 tria in South Korea and received FDA Breakthrough Therapy Designation and RMAT designation (NCT04427930)
South Korea	Corestem	Neuronata-R (Lenzumestrocel)	Autologous bone marrow-derived MSC therapy	Amyotrophic lateral scleosis(ALS)	In Phase 3 trials in Korea. (NCT04745299)
South Korea	Anterogen	ALLO-ASC-DFU	Allogeneic adipose-derived MSC therapy	Diabetic Foot Ulcers	Phase 3 ongoing in Korea (NCT04569409)
South Korea	GC Cell	GCC4002 (Immuncell-LC)	Cytokine-induced Killer (CIK) cell therapy	Resectable Pancreatic Cancer	Phase 3 trial ongoing in Korea (NCT04969731)
South Korea	Kangstem Biotech	FURESTEM-AD Inj.	Stem cell therapy	Moderate to severe Atopic Dermatitis	Phase 3 ongoing (NCT01927705)
South Korea	Pharmicell	Cellgram-LC	Stem cell therapy	Alcoholic Cirrhosis	Phase 3 ongoing (NCT04689152)
South Korea	Kolon TissueGene	TG-C	Allogeneic cell & gene therapy (TGF-β1)	Osteoarthritis	Phase 3 trial in the U.S. (NCT03291470)
South Korea	MEDIPOST	CARTISTEM®	Allogeneic umbilical cord blood-derived MSC	Traumatic cartilage degeneration including Degenerative Osteoarthritis	Phase 3 clinical trial ongoing in Japan
Japan	Nippon Shinyaku and Capricor Therapeutics	CAP-1002 (deramiocel)	Allogeneic cardiac-derived cell therapy	Duchenne Muscular Dystrophy (DMD)	Phase 3 ongoing (NCT05126758)
Japan	Nippon Shinyaku and REGENXBIO	RGX-121 (clemidsogene lanparvovec)	AAV9-based gene therapy	Mucopolysaccharidosis Type Ⅱ	BLA submitted (NCT03566043)
Japan	Chugai Pharmaceutical and Sarepta	ELEVIDYS® (delandistrogene moxeparvovec)	Microdystrophin gene therapy	DMD	Phase 3 follow-up ongoing (NCT05967351)
Japan	Metcela	JRM-001	Autologous cardiac stem cell therapy	Functional Single Ventricle (Pediatric Congenital Heart Disease)	Phase 3 trial resumed on February 2025 in Japan.
Japan	Fujifilm Toyama Chemical	FF-31501	Human autologous synovial stem cells	Meniscus Tear	Phase 3 ongoing in Japan (NCT05777967)
Japan	Astellas Gene Therapies	AT132 (Resamirigene bilparvovec)	AAV8-Delivered Gene Therapy	X-Linked Myotubular Myopathy	Phase 2/3 study (NCT03199469)
China	JW Therapeutics	JWCAR029/Relmacabtagene Autoleucel (relma-cel)	Autologous anti-CD19 CAR-T cell therapy	2L Large B-Cell Lymphoma (LBCL)	Approved in China for LBCL follicular lymphoma and mantle cell lymphoma.
China	Legend Biotech	Cilta-Cel (CARTITUDE)	BCMA-targeted autologous CAR-T cell therapy	Multiple Myeloma(MM)	Approved in the U.S., EU, and Japan and NDA accepted in China. Additional Phase 3 trials are ongoing (NCT05201781)
China	CARsgen Therapeutics	CT053	BCMA-targeted CAR-T cell therapy	Multiple Myeloma	Launched in China (LUMMICAR-1) Phase II/III ongoing in US/Canada (LUMMICAR-2)
China	IASO BioTherapeutics and Innovent Biologics	FUCASO® (Equecabtagene Autoleucel)	BCMA-targeted CAR-T cell therapy	Multiple Myeloma	Approved in China and NDA accepted in Hong Kong and Singapore -Phase 3 trials ongoing for earlier-line MM treatment (NCT06464991)
China	Neurophth Therapeutics	NR082	AAV-based gene therapy	Leber Hereditary Optic Neuropathy	Phase 2/3 trial in China (NCT04912843)
Taiwan	UnicoCell Biomed	ELIXCYTE®	Adipose-derived stem cell therapy	Knee Osteoarthritis	Phase 3 trial recruiting in Taiwan (NCT05526001)

Key Observations

China is emerging as a global leader in CAR-T and gene therapies, particularly for hematologic malignancies like lymphoma and multiple myeloma, with several therapies already approved and commercially launched.
Japan and South Korea are at the forefront of stem cell-based therapies and next-generation gene therapies, supported by robust regulatory frameworks, accelerated approval pathways, and academic-industry collaboration.
A growing number of Phase 3 trials are being conducted in East Asia, either as local initiatives or in collaboration with global biotech firms, underlining the region's increasing importance as a hub for pivotal CGT development and commercialisation.

East Asia is no longer just catching up but it is now shaping the future of cell and gene therapy. With more global partnerships and pivotal trials happening here, collaboration is key.

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