May 15, 2026 —
The UK Medicines and Healthcare products Regulatory Agency, acting jointly with the Department of Health in Northern Ireland, has launched a consultation on proposed updates to the legal definition of gene therapy medicinal products, or GTMPs. The consultation, open from May 11 to June 22, 2026, aims to ensure that UK regulation keeps pace with advances in gene therapy, synthetic biology, and genome editing technologies.
The current UK definitions were established more than a decade ago, before many of today’s genetic medicine technologies had matured. Since then, the field has evolved rapidly, with new modalities including synthetic nucleic acids, sequence-specific genome editing, and increasingly complex engineered genetic medicines. The MHRA is now proposing a more mechanism-based definition that classifies a product as a gene therapy based on how it works, rather than relying on older wording that may no longer fully reflect the science.
Importantly, the proposed changes would not alter the approval process for gene therapies, change the regulatory classification of products already licensed, or reduce existing standards for safety, quality, or efficacy. Instead, the consultation is intended to provide greater clarity and consistency for developers, researchers, manufacturers, regulatory professionals, and clinical experts working in the gene therapy sector.
The consultation focuses on five key proposals: removing the requirement for gene therapies to be biological in origin; clarifying when synthetic or recombinant nucleic acids bring a product into scope; ensuring sequence-specific genome editing products are clearly regulated as GTMPs regardless of substance type; maintaining the exclusion of vaccines against infectious diseases from GTMP definitions; and updating the Human Medicines Regulations 2012 to support the revised definitions.
The proposed amendments are designed to give developers greater regulatory certainty, ensure consistent regulation of biologically and synthetically created gene therapy products, support innovation with a more future-proof framework, and maintain high standards of patient safety and regulatory oversight. By opening the consultation to industry and academic experts, the MHRA is seeking input to shape a regulatory framework that can better accommodate the next generation of genetic medicines.
Overall, the consultation reflects a broader global regulatory challenge: gene therapy is no longer limited to traditional viral-vector gene addition. As synthetic biology, RNA technologies, and genome editing platforms continue to expand, regulatory definitions must evolve to remain scientifically accurate, clear for developers, and protective for patients.
