ISSCR News
The ISSCR Responds to FDA’s Draft Guidance on Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations
On 24 November, the ISSCR submitted comments on the Food and Drug Administration's draft guidance for Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations. ISSCR appreciates FDA’s willingness to consider alternative trial designs for cellular and gene therapy products. To support this effort, ISSCR requests additional guidance on managing manufacturing changes within adaptive or master protocols; decentralized trials for small populations; long-term follow-up; using surrogate endpoints and biomarkers; and trial design selection.
The ISSCR Commends Government of India on Pursuing Strengthened Regulatory Oversight of Cell- and Stem Cell-Derived Products
Last week, the ISSCR sent a letter to India’s Ministry of Health and Family Welfare commending the Government of India for pursuing draft amendments that would strengthen and clarify India’s regulatory framework for cell- and stem cell-derived products.
In the letter, shared in response to draft amendments to India’s Drugs Rules, 1945, the ISSCR notes the importance of rigorous regulatory review and manufacturing standards for cell- and stem cell-based products to protect patients.
The ISSCR Holds Second Meeting With UK Regulators on the Use of AI to Develop Stem Cell Therapies
The International Society for Stem Cell Research (ISSCR) held its second Broader Scope Scientific Advice meeting with the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) on October 29, 2025, focusing on the use of artificial intelligence (AI) in developing stem cell therapies.
The session explored key topics including the current state and challenges of AI, machine learning, and digitalization systems for automation in cell and gene therapy; applications of AI in autologous iPSC-derived RPE cell therapy; and AI-based image analysis for in-process characterization and decision-making during iPSC generation.
The ISSCR Holds Inaugural Regulatory Meeting with Japan’s Pharmaceuticals and Medical Devices Agency and South Korea’s Ministry of Food and Drug Safety
On 7 November 2024, the International Society for Stem Cell Research (ISSCR) held its first meeting with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) and Korea’s Ministry of Food and Drug Safety (MFDS). Held at PMDA’s Tokyo headquarters, the meeting brought together senior officials from PMDA and MFDS alongside ISSCR leaders based in Japan, Korea, and the United States.
At this inaugural meeting, participants convened to discuss key topics related to the:
1) manufacture of human Pluripotent Stem Cell (hPSC) banks as starting materials for allogeneic, PSC-based therapies, and
2) genetic assessment of hPSCs, 3) jurisdictional differences in guidelines for biological Ancillary Materials (AM).
The participants also had the opportunity to gain insights into the regulatory approaches of the PMDA and MFDS regarding PSC-based products.
The ISSCR Responds to ICH’s Q1 Guideline on Stability Testing of Drug Substances and Drug Products
On 28 July, the ISSCR submitted a response to the International Council for Harmonisation’s (ICH) guideline on stability testing of drug substances and drug products. The ISSCR recommends adding specific examples to clarify appropriate applications of certain studies and incorporating considerations for diverse manufacturing models, particularly decentralized manufacturing, where products are administered immediately after production. The ISSCR also advises including guidance on pluripotent stem cell bank stability testing, referencing Tissue Engineered Products (TEP), and clarifying whether ATMP combined products are covered under the term “combination of a drug product with a medical device.”
To request the comments or learn more, contact Denise de Villa.
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