THE ACTO TIMES: 2025 AUTUMN EDITION

Dear ACTO Members and Readers of The ACTO Times,

Time flies, and it is now December 2025. This year, we have made significant advances in Asian cell and gene therapy (Asian CGT), highlighted by the great success of the 2025 ACTO Singapore Annual Meeting. However, it is with deep sadness that we share the news of the passing of Vice President Prof. Yao Chan Chen, a key figure in ACTO and The ACTO Times, in November. We extend our heartfelt condolences to his family and hope that Prof. YC Chen rests in peace.

The 2025 Singapore ACTO Annual Meeting brought together regulatory representatives from the EU and various Asian countries to engage in comprehensive reports and discussions on the regulations, clinical practices, and industry aspects of global CGT. A particular focus was placed on regulatory dialogues concerning CGT products and processes, which are driving the rapid alignment of regulations across different countries.

Globally, regulations are aligning, recognizing CGT as “pharmaceuticals.” As of the first half of 2025, there are about 120 CGT products worldwide, with the U.S. having the most at 45, followed by the EU and countries like Japan and South Korea with over 20 each, China with 12, India with 7, and Taiwan and Singapore with 6 each.

Taiwan’s dual-track regenerative medicine law balances patient rights and CGT precision medicine, complemented by health insurance policies, earning international praise. Japan leads in iPSC clinical translation and allogeneic cell sheets, with conditional product approvals expected. Singapore has strong R&D capabilities, while Indonesia, with its large population, is focusing on stem cell clinical trials under HALAL regulations. India, a major pharmaceutical producer, has 7 approved CGT products, showcasing significant potential. Thailand has approved its first gene therapy product, Zolgensma, and is considering CAR-T Kymriah.

China has made significant progress in the CAR-T field. Although it approved its first CD19 CAR-T product Axi-cell in 2021, four years after the U.S., by 2025, China has approved 10 domestically developed CAR-T products, priced at $140,000-$180,000, about one-third of the U.S. product Kymriah. The next generation of CAR-T products is expected to be priced around $40,000, making CGT more accessible. Additionally, China’s NMPA has approved the first gene therapy product for hemophilia B and an allogeneic umbilical cord mesenchymal stem cell product for SR-GvHD.

Gene therapy is proving effective in rare disease treatment. In 2020, Jimi Olaghere successfully underwent CRISPR gene editing for sickle cell disease, leading to FDA approval of Casgevy and Lyfgenia in 2023 for sickle cell disease and β-thalassemia. In May 2025, CRISPR technology achieved another breakthrough by treating an infant with CPS1 deficiency, a rare metabolic disorder；Zolgensma, approved by the FDA in 2019 for spinal muscular atrophy (SMA), has shown remarkable efficacy. By early 2025, over 3,700 patients have received Zolgensma treatment, with significant results. In September, the FDA introduced the Rare Disease Evidence Principles (RDEP) to expedite drug approval for rare diseases affecting fewer than 1,000 patients, using single-arm clinical trial data.

The global CGT market funding reached $5.1 billion in the first quarter of 2025. North America leads with $4.4 billion, followed by the EU with $800 million, and the Asia-Pacific region with the lowest at $500 million, accounting for one-tenth of the total funding. Despite this, Asia is demonstrating strong resilience and ambition in the CGT field. Out of 2,070 CGT companies worldwide, Asia has 750, closely following North America’s 770. The number of clinical trials in Asia has reached 840, tying with North America for the global lead.

The rise of CGT in Asia is poised to remove barriers for underserved patients, offering new hope and opportunities.

Sincerely,

Yen Hua Huang, PhD  

Distinguished Professor, Taipei Medical University  

Editor-in-Chief, The ACTO Times  

ACTO, Asian Cellular Therapy Organization