News Hub

Fajar Dumadi, BSc
ISCT
Canada

Summary

This article highlights pivotal regulatory events and legislative updates from around the world that are influencing the future of cell and gene therapy. Highlights include MHRA webinars on modular and decentralized manufacturing, ISCT’s Global Regulators Summit, and a joint call for a 10-year moratorium on Heritable Human Genome Editing. Workshops and roundtables hosted by FDA, EMA, PMDA, and CAT addressed pressing scientific and regulatory challenges, with a strong focus on accelerating access and harmonizing standards.

Legislative updates across regions introduced new guidance and consultation papers, from adaptive trial designs (ICH E20) and external controls (EMA) to post-approval data strategies and expedited pathways for regenerative therapies (FDA). These developments reflect a global push toward more flexible, risk-based frameworks for CGT innovation.

Key Regulatory Events

• UK MHRA
  • MHRA Modular Manufacture and Point of Care Regulations Webinar – January 28, 2025 (1)
  • Decentralised Manufacturing Regulations Update Webinar - June 17, 2025 (2) 
    
• ISCT/ARM/ASGCT Joint conference on Heritable Human Genome Editing - 26 March 2025
  • A conference co-organized by ISCT, ARM, and ASGCT brought together scientists, academic centers, biotechnology organizations, patients, and service providers closest to the cell and gene therapy field to discuss the controversial and unresolved issues surrounding Heritable Human Genome Editing (HHGE). Resulted in a call for a 10-year moratorium on HHGE (published May 21, 2025) (3). 
• ISCT 3rd Annual Global Regulators Summit - 6 May 2025
  • Attended by regulators from 15+ agencies worldwide, focused on accelerated pathways for cell and gene therapies.
• FDA Cell and Gene Therapy Roundtable - 5 June 2025
  • A roundtable that involved panelists from various leading cell and gene therapy organizations, including ISCT, to discuss the most pressing issues and challenges in the field. The roundtable concluded with an emphasis on collaborating across stakeholders to improve access to rare disease therapies (4)
• EMA 14th Industry Stakeholder Platform Meeting - 23 June 2025
  • This fourteenth meeting between regulators and representatives of industry stakeholder organisations focused on the operation of the centralised procedure for human medicines, in particular the implementation of the variation regulation and guideline; along with ongoing initiatives concerning the centralised procedure (5)
• PMDA-ATC Review of CGTPs Seminar 2025 for South-East Asian countries - 15-17 July 2025
  • The seminar was attended by 25 regulators from Southeast Asian regulatory agencies and focused on key aspects of the review process for Cell and Gene Therapy Products (CGTPs). Topics included an overview of regulatory review procedures, quality control, non-clinical and clinical data evaluation, and environmental risk assessment. The program also featured industry perspectives on CGTP development. Participants engaged in case study discussions and a roundtable session, during which each representative shared the current status of CGTP reviews in their respective countries (6). 
• CAT-EMA Workshop on Gene Editing - 16 September 2025 (7)
  • Workshop organised by CAT with the support of the Alliance for Regenerative Medicines (ARM), aims to identify current scientific and regulatory challenges of in vivo and in vitro gene editing (GE) medicines.
• FDA CBER OTP Public Listening Meeting: Leveraging Knowledge for Facilitating the Development and Review of Cell and Gene Therapies - 18 September 2025 (8)
  • Virtual public listening meeting hosted by FDA CBER OTP to solicit perspectives from cell and gene therapy (CGT) manufacturers and other stakeholders on leveraging prior knowledge and experience to facilitate product development and application review.

Global Legislative and Framework Updates

• New Zealand Medicines Amendment Bill (9)
  • Proposal to streamline the verification pathway and update the prescribing settings
  • Public consultation: April 29-May 19, 2025
• EMA concept paper on Guideline on GMP Practice specific to ATMPs  (10)
  • Updates to Part IV of Eudralex Volume 4
  • Public consultation: May 8 – July 8, 2025
• FDA guidance on approaches to assessment of overall survival in oncology clinical trials (11)
  • Provides recommendations to sponsors on the assessment of overall survival in randomized oncology clinical trials, with an emphasis on the analysis of overall survival as a pre-specified safety endpoint. 
  • Public consultation: August 20 – October 20, 2025
• ICH E20 guideline on adaptive designs for clinical trials (12)
  • Provides direction for confirmatory clinical trials using adaptive designs to evaluate treatments within the context of their overall development programs.
  • Public consultation: August 20 – October 20, 2025 
    
• EMA draft concept paper on the Development of a Reflection Paper on the Use of External Controls for Evidence Generation in Regulatory Decision-Making (13)
  • Outline the main challenges with external controls and further discuss the circumstances and methodological constraints under which the use of external controls could be considered appropriate for generating pivotal or supportive evidence, either for efficacy, safety or other relevant regulatory decision-making objectives. 
  • Public consultation opened from 25 July – 31 October 2025
• TGA proposed annotations to ICH E6(R3) Guideline for Good Clinical Practice (GCP) (14)
  • TGA annotations to the ICH E6(R3) Guideline for GCP will ensure consistency with Australian legislative requirements for clinical trials of unapproved medicines and biologicals under the Clinical Trial Notification (CTN) and Clinical Trial Approval (CTA) schemes.
  • Public consultation: August 20 – October 20, 2025
    
• FDA Draft Guidance on Expedited Programs for Regenerative Medicine Therapies for Serious Conditions (15)
  • This guidance provides sponsors engaged in the development of regenerative medicine therapies for serious or life-threatening diseases or conditions with our recommendations on the expedited development and review of these therapies, including as provided under section 506(g) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), as added by section 3033 of the 21st Century Cures Act (Cures Act). This draft guidance, when finalized, will supersede the final guidance of the same title dated February 2019.
  • Public consultation: September 24 – November 24, 2025
    
• FDA Draft Guidance on Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations (16)
  • This guidance provides recommendations to sponsors who are planning clinical trials of cell and gene therapy (CGT) products intended for use in a disease or condition that affects a small population—generally one that meets the definition of a rare disease or condition under section 526(a)(2) of the FD&C Act (21 U.S.C. 360bb(a)(2)).
  • Public consultation: September 24 – November 24, 2025
    
• FDA Draft Guidance on Post-approval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy Products (17)
  • The purpose of this guidance is to discuss methods and approaches for capturing post-approval safety and efficacy data for cell and gene therapy (CGT) products. important for gathering data on product safety and effectiveness over time. This guidance does not address data collected for the purpose of expanding clinical indications.
  • Public consultation: September 24 – November 24, 2025
    

REFERENCES

1. MHRA Modular Manufacture and Point of Care Regulations Webinar - YouTube [Internet]. 2025 [cited 2025 Sep 2]. Available from: https://www.youtube.com/watch?v=HhYGERVex8g
2. MHRA. Decentralised Manufacturing Regulations Update Webinar - YouTube [Internet]. 2025 [cited 2025 Sep 2]. Available from: https://www.youtube.com/watch?v=yXh7J9M5p6o
3. Barrett D, Cannon P, Forte M, Hunt T, Jang Q, Smith D. International Call for a 10-Year Moratorium on Heritable Human Genome Editing: A multi-stakeholder initiative to ensure safe and responsible use of genetic technologies May 2025. Cytotherapy [Internet]. 2025 Aug 1 [cited 2025 Sep 2];27(8):885–7. Available from: https://www.isct-cytotherapy.org/action/showFullText?pii=S1465324925007182
4. FDA CBER. Cell and Gene Therapy Roundtable [Internet]. 2025 [cited 2025 Sep 2]. Available from: https://www.fda.gov/vaccines-blood-biologics/workshops-meetings-conferences-biologics/cell-and-gene-therapy-roundtable-06052025
5. European Medicines Agency (EMA). Fourteenth meeting of the industry stakeholder platform on the operation of the centralised procedure for human medicines [Internet]. 2025 [cited 2025 Sep 2]. Available from: https://www.ema.europa.eu/en/events/fourteenth-meeting-industry-stakeholder-platform-operation-centralised-procedure-human-medicines
6. Pharmaceuticals and Medical Devices Agency (PMDA). PMDA-ATC Review of CGTPs Seminar 2025 for South-East Asian countries [Internet]. 2025 [cited 2025 Sep 3]. Available from: https://www.pmda.go.jp/english/symposia/0338.html
7. European Medicines Agency (EMA). Committee for Advanced Therapies (CAT) workshop on gene editing [Internet]. 2025 [cited 2025 Sep 2]. Available from: https://www.ema.europa.eu/en/events/committee-advanced-therapies-cat-workshop-gene-editing
8. FDA CBER. FDA CBER OTP Public Listening Meeting: Leveraging Knowledge for Facilitating the Development and Review of Cell and Gene Therapies [Internet]. 2025 [cited 2025 Sep 2]. Available from: https://www.fda.gov/news-events/otp-events-meetings-and-workshops/fda-cber-otp-public-listening-meeting-leveraging-knowledge-facilitating-development-and-review-cell
9. NZ Ministry of Health. Medicines Amendment Bill [Internet]. 2025 [cited 2025 Sep 2]. Available from: https://www.legislation.govt.nz/bill/government/2025/0134/5.0/LMS1035396.html
10. European Medicines Agency (EMA). Concept paper on the revision of Part IV Guidelines on Good Manufacturing Practice specific to Advanced Therapy Medicinal Products. 2025;
11. FDA. Draft Guidance - Approaches to Assessment of Overall Survival in Oncology Clinical Trials [Internet]. 2025 [cited 2025 Sep 2]. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/approaches-assessment-overall-survival-oncology-clinical-trials
12. European Medicines Agency (EMA). ICH E20 Guideline on adaptive designs for clinical trials. 2025 [cited 2025 Sep 2]; Available from: www.ema.europa.eu/contact
13. European Medicines Agency (EMA). Draft Concept Paper on the Development of a Reflection Paper on the Use of External Controls for Evidence Generation in Regulatory Decision-Making. 2025 [cited 2025 Sep 2]; Available from: www.ema.europa.eu/contact
14. Therapeutic Goods Administration (TGA). Proposed Annotations to ICH E6(R3) Guideline for Good Clinical Practice (GCP). 2025;
15. FDA. Draft Guidance - Expedited Programs for Regenerative Medicine Therapies for Serious Conditions [Internet]. 2025 [cited 2025 Oct 2]. Available from: https://www.fda.gov/media/188874/download
16. FDA. Draft Guidance - Innovative Designs for Clinical Trials  of Cellular and Gene Therapy  Products in Small Populations [Internet]. 2025 [cited 2025 Oct 2]. Available from: https://www.fda.gov/media/188892/download
17. FDA. Draft Guidance - Postapproval Methods to Capture  Safety and Efficacy Data for Cell and  Gene Therapy Products [Internet]. 2025 [cited 2025 Oct 2]. Available from: https://www.fda.gov/media/188891/download