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Tongted Das, PhD, MSc, BASc
ISCT Australia & New Zealand Legal & Regulatory Affairs (ANZ LRA) Committee Member
ISCT ESP Leadership Development Program Member
Australia

On behalf of the ISCT ANZ LRA Committee

Summary of Panel Discussion on Malaysia's Leadership in Cell & Gene Therapy

The ISCT Australia New Zealand (ANZ) Legal & Regulatory Affairs Committee (LRA), in collaboration with the ISCT Global Regulatory Task Force, presented a live open-access webinar on ‘Cell and Gene Therapy in Malaysia – Opportunities and Challenges’ which first aired on 17 April 2027 (Malaysia Time) and is now available as open access on demand (registration required) at: https://www.isctglobal.org/webinar/cell-and-gene-in-malaysia

The webinar was championed by Gabrielle O’Sullivan and chaired by three ISCT ANZ LRA members: Tongted Das, Patrick Foong and Domnic Wall. They discussed key issues for cell and gene therapy in Malaysia with seven of Malaysia’s key leaders in cell and gene therapy: Professor(s) Chin Sze Piaw and Cheong Soon-Keng; and Doctor(s), Zubaidah Zakaria, Azizah Ab Ghani, and Firdaus Bin Abdul Aziz; as well as Ms. Asha Thanabalan and Mr. James Then. This article presents some of the many key learnings from the webinar and recommends viewing the webinar for an insight into Malaysia’s fascinating and innovative cell and gene therapy ecosystem. 

Malaysia is emerging as an important hub for cell and gene therapy clinical trials, research, and regulation. This growth is supported by government initiatives, a diverse population, and a strong clinical research network.

Key Strengths Include:

• A diverse population that supports inclusive and representative clinical research.
• An established clinical research ecosystem with experience in complex studies.
• Capacity to conduct highly innovative clinical trials in emerging therapeutic areas.

1. Regulatory and Policy Framework

• Malaysia has a robust regulatory framework for cell and gene therapy products (CGTPs), with guidelines first published in 2016 and updated in 2025.
• CGTPs are classified into Class 1 (minimally manipulated) and Class 2 (more complex, including all gene therapies), with Class 2 products requiring rigorous oversight.
• Malaysia relies on international regulatory standards (e.g., EMA, FDA) for gene therapy assessment and allows for regulatory reliance and abbreviated reviews.

2. Clinical Research Strengths

• Malaysia is a preferred destination for clinical trials due to its supportive ecosystem, strong clinical research networks, and diverse population.
• Clinical Research Malaysia (CRM) provides support for trial planning, investigator/site matching, regulatory navigation, and quality assurance.
• Malaysia has experience with complex, highly innovative trials, including first-in-human and precision medicine studies.

3. Manufacturing and Infrastructure

• Malaysia has 10 CGMP labs approved by NPRA, with at least three capable of gene therapy work.
• Both local and international CROs are accessible in Malaysia.
• Malaysia is building capacity for preclinical work, but some capabilities are still developing and often outsourced.

4. Research and Development Ecosystem

• Malaysia has made significant progress in stem cell research, particularly with mesenchymal stem cells (MSCs) and induced pluripotent stem cells (iPSCs).
• National guidelines and ethical frameworks have evolved, with the third edition of the National Stem Cell Research Guideline published in 2024.
• Non-governmental organisations and academic centres play a key role in supporting research, clinical trials, and patient registries.

5. Training and Capacity Building

• Training is supported through a mix of local postgraduate programs, short-term overseas fellowships, and regional partnerships (notably with Singapore, Japan, and Australia).
• There is a push for hybrid training models combining global exposure, regional attachments, and hands-on GMP manufacturing experience.

6. Barriers and Challenges

• Key barriers include regulatory translation gaps (moving from research to approved therapy), the need for more cell and gene therapy clinical trials, high costs and lack of reimbursement, and insufficient physician awareness/training.
• The presence of unproven stem cell therapies in the private section is a concern, with enforcement complicated by regulatory grey areas, lack of a specific law and evidentiary challenges to tackle this robustly.
• While there are guidelines, they are not legally enforceable. Thus, there is a need for specific legislation to address this area.
• There is a need for better integration between research, clinical practice, and regulatory oversight, as well as improved data transparency and practitioner regulation.
• Regarding Malaysian patients going abroad to seek unproven stem cell treatments (stem cell tourism), there is currently a lack of data. Thus, it is important to explore this area further.

7. Future Directions

• Regulatory Framework: Malaysia is developing a national guideline for regenerative medicine to transition from research to clinical practice, including practitioner certification and training requirements.
• International Collaboration: Malaysia aims to harmonise further with regional standards (ASEAN) and leverage international collaborations to accelerate access and innovation in regenerative medicine.
• Support for Innovators: International sponsors can benefit from expedited review processes by including Malaysian sites in global trials, while local innovators are encouraged to engage regulators early and seek multi-agency support.

Conclusion

Malaysia’s cell and gene therapy landscape is advancing rapidly, supported by strong regulatory foundations, an enabling clinical research environment, and expanding manufacturing and training capacity. Although challenges remain—particularly in access, enforcement, reimbursement, and integration across research, clinical practice, and regulation—Malaysia is well positioned to strengthen its role as a regional leader in advanced therapies. Continued policy development, international collaboration, infrastructure investment, and workforce development will be critical to translating this momentum into broader patient access and sustainable sector growth.

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