Phase 2 & 3 CGT Trials in APAC
The Asia-Pacific region is quickly becoming a key player in advanced cell and gene therapy (CGT). Many companies across Australia, China, India, Japan, South Korea and others are moving into important Phase 2 and 3 clinical trials, showing strong progress toward getting treatments approved.
This report shares an overview of the top companies, products, and diseases being targeted in these late-stage trials. From CAR-T and stem cells to gene editing and RNA therapies, APAC innovators are driving new treatments with global potential.
Australia
Cynata
- Product: CYP‑001 (iPSC‑MSC)
- Indication: Acute GvHD
- Phase & Status: Phase 2 first patient treated, results expected late 2025
Mesoblast
- Product: CYP‑001 (iPSC‑MSC)
- Indication: Acute GvHD
- Phase & Status: Phase 2 first patient treated, results expected late 2025
Carina Biotech
- Product: CNA3103 (CAR‑T)
- Indication: Metastatic colorectal cancer
- Phase & Status: Phase 1/2a early stage
Novartis
- Product: DFT383 (lentivirus)
- Indication: Cystinosis
- Phase & Status: Phase 2
Speaking at CGTWA 2025
Deborah Rathjen
Chief Executive Officer, Carina Biotech, Australia
China
CARsgen Therapeutics
- Product / Platform: CT053 (BCMA CAR-T)
- Indication: Relapsed/refractory multiple myeloma
- Phase: Phase 2/3
- Notes: Approved in China 2023
JW Therapeutics
- Product / Platform: Relmacabtagene Autoleucel
- Indication: Lymphoma
- Phase: Phase 2
- Notes: NMPA conditional approval 2021
Gracell Biotechnologies
- Product / Platform: GC012F (CAR-T, BCMA+CD19)
- Indication: Multiple Myeloma
- Phase: Phase 1/2
- Notes: Rapid progress in China
IASO Biotherapeutics
- Product / Platform: Equecabtagene Autoleucel
- Indication: Multiple Myeloma
- Phase: Phase 2/3
- Notes: Approved in China 2023
Fosun Kite Biotechnology
- Product / Platform: Axicabtagene Ciloleucel
- Indication: DLBCL
- Phase: Phase 2
- Notes: Joint venture with Kite Pharma
Legend Biotech
- Product / Platform: Cilta-Cel (CARVYKTI)
- Indication: Multiple Myeloma
- Phase: Phase 2/3
- Notes: Approved in US/China; JNJ partner
Juventas Cell Therapy
- Product / Platform: DFV890
- Indication: Chronic limb-threatening ischemia
- Phase: Phase 2
- Notes: Cell therapy pipeline growing
Eureka Therapeutics
- Product / Platform: ARTEMIS TCR-mimic platform
- Indication: Liver cancer
- Phase: Phase 1/2
- Notes: TCR-like CAR-T tech
EXUMA Biotech
- Product / Platform: Rapid point-of-care CAR-T
- Indication: Solid tumors
- Phase: Phase 1/2
- Notes: Local site production models
PersonGen BioTherapeutics
- Product / Platform: CD19/CD22 Dual CAR-T
- Indication: Lymphoma
- Phase: Phase 2
- Notes: Novel dual-targeting design
Hong Kong
HKU (HKUMed)
- CGT Activity Summary: Administering CAR‑T therapy; Phase 3 lymphoma trial 7 patients; BCMA CAR‑T for MM; GMP facility
- Phase 2/3 Status: Phase 3 lymphoma trial; ongoing clinical CAR‑T treatments
India
Stempeutics Research
- Product: Stempeucel-DFU
- Indication: Diabetic Foot Ulcer
- Phase: Phase 3
- Notes: Completed enrollment May 2023
- Product: Stempeucel-CLI
- Indication: Critical Limb Ischemia
- Phase: Phase 3
- Notes: Label-extension trial started 2018
Speaking at CGTWA 2025
Udaykumar Kolkundkar
Senior Vice President – Technical Operations, Stempeutics Research, India
Japan
AnGes
- Product / Focus: Collategene (HGF gene therapy)
- Phase / Status: Phase 3 Completed
- Notes: First gene therapy approved in Japan
JCR Pharmaceuticals
- Product / Focus: Temcell (allogeneic MSC therapy)
- Phase / Status: Phase 2/3 Market approved in Japan
- Notes: For aGVHD; Japan’s first approved cell therapy
SanBio
- Product / Focus: SB623 (MSC-based neuroregenerative therapy)
- Phase / Status: Phase 2b in US, Japan
- Notes: For stroke-related disabilities
Noile-Immune Biotech
- Product / Focus: CAR-T (NIB102)
- Phase / Status: Phase 1/2 progressing
- Notes: Solid tumor CAR-T trials with Takeda support
Takara Bio
- Product / Focus: HF10 oncolytic virus + CAR-T platform
- Phase / Status: Phase 2 Japan/US
- Notes: Ongoing cancer therapy studies
Takeda
- Product / Focus: Multiple CGT assets
- Phase / Status: Late-stage trials globally Japan included
- Notes: Working on rare disease and oncology CGTs
Osaka University
- Product / Focus: Various (gene/cell therapy)
- Phase / Status: Involved in several phase 1/2 studies
- Notes: Academic CGT hub
Kyoto Univ. CiRA
- Product / Focus: iPSC therapies
- Phase / Status: Phase 1/2
- Notes: Regenerative therapy leadership
Speaking at CGTWA 2025
Ryuichi Morishita
Chief Section of Gene Therapy, Department of Geriatric Medicine, Osaka University, Japan
Cuorips
- Product / Focus: iPSC-derived cardiomyocytes
- Phase / Status: Phase 1/2 Japan
- Notes: Clinical trial underway
South Korea
Helixmith (ViroMed)
- Product / Platform: VM202 (HGF gene therapy)
- Indication: Diabetic Peripheral Neuropathy (DPN)
- Phase: Phase 3
- Notes: Ongoing US and Korea trials
- Product / Platform: VM202-DFU
- Indication: Diabetic Foot Ulcers
- Phase: Phase 3
- Notes: Late-stage trials
Medipost
- Product / Platform: Cartistem (allogeneic stem cell)
- Indication: Knee osteoarthritis
- Phase: Approved in Korea; Phase 3 abroad
- Notes: First approved allogeneic stem cell therapy in Korea
- Product / Platform: Pneumostem
- Indication: BPD in preterm infants
- Phase: Phase 2/3
- Notes: Pediatric clinical trial in progress
Speaking at CGTWA 2025
Antonio Lee
CEO and Managing Director, MEDIPOST, South Korea
Curocell
- Product / Platform: CRC01 (CAR-T)
- Indication: DLBCL (relapsed/refractory)
- Phase: Phase 2
- Notes: First domestic CAR-T therapy in Korea; fast-track designated
Corestem
- Product / Platform: NeuroNata-R (autologous MSC)
- Indication: ALS
- Phase: Phase 3
- Notes: Korea's first stem cell therapy for ALS; approved under conditional license
S. BIOMEDICS
- Product / Platform: SB-01
- Indication: Parkinson’s disease (iPSC therapy)
- Phase: Phase 1/2
- Notes: Supported by KFDA; long-term safety data under review
Pharmicell
- Product / Platform: Cellgram-AMI
- Indication: Acute myocardial infarction
- Phase: Phase 2/3
- Notes: Autologous stem cell therapy
Rznomics
- Product / Platform: RZ-001 (gene editing RNAzyme)
- Indication: Liver cancer
- Phase: Phase 1/2 → Phase 2 expected
- Notes: First RNAzyme therapy; supported by MFDS Korea
Why Attend Cell & Gene Therapy World Asia 2025?
As more Phase 2/3 trials move forward in Asia, CGTWA 2025 (10–11 September, Sheraton Towers Singapore) will be the top place to gather:
- Clinical leaders sharing real trial results and approval strategies
- CROs and CMOs offering help with trial sites, scale-up and testing
- Biotech and pharma teams looking for partners and market plans
- Regulators giving updates on fast-track approvals, GMP and safety rules
Don’t miss your chance to meet the companies in this report and be part of the future of CGT in Asia.
Share With Your Network
