UK regulator launches consultation on redefining gene therapy

The Medicines and Healthcare products Regulatory Agency (MHRA) has opened a UK-wide consultation on proposed changes to the legal definition of gene therapy medicinal products (GTMPs).

The MHRA is the UK government agency responsible for regulating medicines and medical devices, and ensuring their safety, quality, and efficacy. It operates as an executive agency of the Department of Health and Social Care. The consultation, run jointly with the Department of Health in Northern Ireland, is open from 11 May 2026 to 22 June 2026. The proposed revisions would update the definition of GTMPs to reflect recent innovations in gene therapies, synthetic biology and genome editing technologies.

Jon Beaman, MHRA deputy director of innovative medicines, said: 'Our teams have carefully designed these updates to ensure that patient safety remains at the centre of any change, while also giving developers, researchers and clinicians greater regulatory certainty, while supporting innovation across the industry.' The changes are intended to clarify whether newer therapies made using synthetic genetic material or genome editing fall within existing GTMP rules.

The consultation covers five proposed changes to the criteria used to classify GTMPs, without altering the underlying regulatory principles that apply to them. These changes are intended to ensure that synthetically manufactured products and genome editing technologies are appropriately captured within the current regulatory framework.

Under the proposed updates, there will be no changes to the regulatory status of currently licensed products, and vaccines against infectious diseases will continue to be excluded from GTMP definitions. The MHRA said there would be no reduction in safety, quality or efficacy requirements. Beaman said the revisions aim to 'deliver the best possible outcome for patients and the wider health sector.'

Responses are encouraged from developers, manufacturers, researchers, regulatory professionals, scientific advisors, and clinical and academic experts who Beaman said 'will help to shape the final approach'. Stakeholders and interested parties are also welcome to respond to the consultation despite the technical nature of the proposal.

The definition of GTMPs was originally developed in 2007, and their regulation is primarily governed by the Human Medicines Regulations 2012. This existing framework excludes therapies involving certain synthetic genetic materials, newer forms of genome editing, and highly engineered cells and tissues.

Should the proposed revisions be accepted, GTMP classification would include active substances containing recombinant or synthetic nucleic acid components that undergo transcription or translation, as well as substances whose mechanism of action involves sequence-specific genome editing.