
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
