BANGKOK – Southeast Asia is seeing a major shift in modern medicine. The region has long carried a heavy burden of genetic and infectious diseases, and for many families, treatment has often meant lifelong medication with limited relief. Now gene therapy, once available mainly in wealthy countries, is starting to offer real hope of long-term, one-off treatments for millions.
Thailand, which has a clear goal to become a leading medical centre in the region, is stepping forward as a key driver of gene therapy research, manufacturing, and fair access across ASEAN.
The Promise of Gene Therapy
Gene therapy changes the way many people think about treatment. Instead of managing symptoms, it targets the root molecular cause of a disease. Many gene therapies, often grouped under Cell and Gene Therapies (CGTs), are developed as one-time treatments that can offer lasting benefit.
At its core, gene therapy involves changing a patient’s genes to treat or cure an illness. This can be done in several ways, including:
- Gene addition or replacement: A healthy copy of a gene is added to make up for one that does not work properly. This remains the most common method.
- Gene editing (such as CRISPR-Cas9): Faulty DNA sequences are corrected within the patient’s own cells.
- Immunotherapy: Immune cells, such as T cells in CAR T-cell therapy, are modified so they can better detect and kill cancer cells.
Worldwide, clinical trials have shown strong results in a range of conditions, from rare single-gene disorders, such as Spinal Muscular Atrophy (SMA) and some inherited forms of blindness, to more common illnesses like several cancers and inherited blood diseases. One of these is Thalassemia, a major public health concern across Southeast Asia.
Thailand’s Role as a Regional Centre for Gene Therapy
Thailand is quickly building a strong position in this advanced area of medicine. Several advantages support this progress, including a well-regarded healthcare system, firm government backing for life sciences, and partnerships with international organisations. Progress is clear in three main areas: infrastructure, regulation, and patient access.
Building Strong Manufacturing Capacity
A key part of Thailand’s strategy is to host a regional hub for CGT development and manufacturing. One important move is the partnership between UK-based eXmoor Pharma and Thailand’s Siam Bioscience, which plans to build a major centre for Cell and Gene Therapy development and production near Bangkok.
This centre is expected to offer end-to-end services, including:
- Consultancy for therapy design and development
- Process development for new treatments
- Manufacturing that meets Good Manufacturing Practice (GMP) standards
Local CGT manufacturing is more than a badge of honour. It plays a direct role in:
- Cutting complex logistics: Gene therapies are fragile and often need strict transport conditions. Local production shortens supply chains.
- Speeding up access: Patients across ASEAN can receive high-quality therapies more quickly when they are produced closer to home.
- Boosting home-grown research: Thai scientists gain better support for moving their discoveries from the lab into clinical use.
Strengthening Rules and Oversight
Gene therapies are powerful tools, so they require clear, reliable regulation that protects patients and gives confidence to global partners. Thailand has been working to tighten and clarify its rules. Recent changes to the regulation of drugs imported for clinical trials, effective from early 2024, set out a more detailed structure for research activities.
Key regulatory measures include:
- Requiring a Clinical Trial Application (CTA) to be filed with the Thai Food and Drug Administration (Thai FDA) before any trial starts.
- Requiring compliance with International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP) guidelines, which align Thai clinical trial standards with internationally accepted practice.
- Strengthening post-approval oversight, so the Thai FDA can inspect, modify, suspend, or cancel ongoing studies when needed to protect participants.
This more mature regulatory system helps attract global sponsors and researchers. It shows that Thailand offers a stable, ethical setting for advanced clinical trials in gene and cell therapies.
Growing Genomics and Improving Clinical Access
Progress is not limited to labs and factories. Thailand is also weaving Genomic Medicine into its public health services. Siriraj Hospital, together with the National Health Security Office (NHSO), has widened access to cancer gene mutation testing, including BRCA1 and BRCA2, so that all Thai citizens can be tested regardless of income.
This programme, part of the Thailand Genomics Project, is an important step for precision medicine. By identifying people with high-risk mutations, clinicians can offer targeted therapies and closer monitoring, and in some cases, gene-based treatments.
Gene therapies are also starting to reach patients with rare and serious conditions. The Thai FDA has approved treatments for Spinal Muscular Atrophy (SMA), including the gene therapy onasemnogene abeparvovec-xioi.
Cost remains a major barrier for full public coverage, and economic evaluations have raised concerns about affordability. Even so, the fact that these treatments have been registered and can be prescribed in Thailand marks a big step forward for access.
Barriers Ahead: The High Cost of Gene Therapy
Despite the strong momentum, making gene therapy widely available across Southeast Asia is far from simple. The biggest obstacle is cost, followed closely by gaps in the workforce and infrastructure.
Price tags for many gene therapies rank among the highest in modern medicine. Some treatments are priced at over $2 million per dose. For low and middle-income countries such as Thailand, this creates a real strain on public health budgets.
A cost-utility study in Thailand found that high-cost treatments for conditions like SMA overshoot the usual value-for-money threshold for the national healthcare system. The conclusion was that these therapies, while clinically effective, are currently too expensive for broad coverage.
Other important challenges include:
- Workforce gaps: There is a shortage of trained experts, especially bioinformaticians, who are needed to handle and interpret large volumes of genetic data.
- Specialised facilities: Gene therapies require advanced laboratories and certified manufacturing plants, along with clinical centres that can handle complex preparation, delivery, and follow-up.
- Long-term funding: Local research, clinical trials, and patient access all depend on reliable funding from both domestic and international sources. Sustainable models for reimbursement are still under development.
Looking Ahead: Shared Effort and Fair Access
To unlock the full potential of gene therapy for Southeast Asia, cooperation will matter more than individual efforts. Governments, researchers, industry partners, and patient groups all have a role to play.
Some key steps include:
- Regional alignment: ASEAN countries can gain by working towards harmonised regulatory frameworks and similar clinical trial standards. This can support multi-country studies, cut research costs, and speed up approvals across borders.
- New payment models: Policy-makers are starting to explore different ways to pay for high-cost therapies. Ideas include outcomes-based payment schemes, where health systems pay based on how well a treatment works over time, and pooled regional procurement to share costs.
- Focusing on local health needs: Research that targets conditions common in Southeast Asia, such as Thalassemia and certain infectious diseases, will deliver the greatest benefit for the local population.
Thailand’s work across science, industry, and government shows the energy building in this field. As research advances and local manufacturing expands, the idea of a one-time cure is shifting from theory to practice.
If this progress continues, the next decade could see gene therapy move from a rare option to a more routine part of care, offering a real chance at a healthier future for millions of people across Southeast Asia.
