MHRA opens UK-wide consultation on redefining gene therapies

The Medicines and Healthcare products Regulatory Agency (MHRA), acting jointly with the Department of Health in Northern Ireland, has today launched a consultation calling to update the legal definition of gene therapy medicinal products (GTMPs) in the UK. The proposed changes aim to ensure UK regulation keeps up with advances in gene therapy, synthetic biology, and gene editing technologies.

Gene therapies have evolved significantly since the current definitions were established, more than a decade ago. Since then, advances in science and manufacturing technologies have prompted consideration on whether the wording remains appropriate for use.

The MHRA is consulting on updated definitions that would classify a medicinal product as a gene therapy, based on how it works.

The updated definition will not alter the approval process for gene therapies, the regulatory classification of products already licensed, or change any existing safety, quality, or efficacy standards.

The proposed amendments intend to:

• Provide greater certainty for developers working on gene therapy products.
• Ensure consistent regulation across all gene therapy products, whether these are created biologically or synthetically.
• Support innovation across the sector, giving researchers and companies a future-proof regulatory framework.
• Maintain high standards of patient safety and regulatory oversight.

Jon Beaman, MHRA Deputy Director Innovative Medicines, said:

“This proposal reflects how fast-paced scientific progress can be and ensures our regulatory framework remains robust, clear and fit for the future.

“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 input provided by our industry colleagues will help to shape the final approach and ensure the proposals deliver the best possible outcome for patients and the wider health sector.”

Given the technical nature of the proposal, the MHRA is primarily interested in encouraging feedback from developers, manufacturers, researchers, regulatory professionals, scientific advisers, and clinical and academic experts. However, anyone is welcome to respond.

The consultation seeks views 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 products involving sequence specific genome editing are clearly regulated as GTMPs, regardless of substance type.
• Maintaining the exclusion of vaccines against infectious diseases from GTMP definitions.
• Proposing updates to the Human Medicines Regulations 2012 to support these revised definitions.

The consultation opens on 11 May 2026 and will run until 22 June 2026.

To read the consultation and submit a response, visit: Consultation on the regulation of Gene therapy medicinal products

Notes

• The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe. All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks.
• The MHRA is an executive agency of the Department of Health and Social Care.

SOURCE: GOV