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
