MHRA Opens UK-Wide Consultation on Redefining Gene Therapies

The United Kingdom’s Medicines and Health care products Regulatory Agency (MHRA) launched a public consultation on proposed updates to the legal definition of gene therapy medicinal products (GTMPs), aiming to modernize regulations in response to advances in synthetic biology, genome editing and engineered cell therapies.

MHRA stated that the current GTMP definitions, originally developed in 2007, were created when therapies relied on a narrower range of technologies. According to the agency, newer therapies may involve synthetic genetic material, novel genome-editing approaches and highly engineered cells and tissues that do not fit neatly within the U.K.’s existing regulatory categories.

MHRA is proposing a technology-neutral framework that would classify products based on their mechanism of action rather than whether they are biologically derived.

The consultation outlines several proposed changes, including:

• 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.
• Updating the Human Medicines Regulations 2012 to support the revised definitions.

The proposal would not change existing approval pathways or safety, quality and efficacy standards for currently licensed products.

The consultation will remain open through June 22.