The Medicines and Healthcare products Regulatory Agency (MHRA) has launched a consultation on proposed changes to how gene therapies are defined in UK legislation, aiming to ensure regulation keeps pace with rapid advances in science and technology.
The consultation, launched jointly with the Department of Health in Northern Ireland, seeks views on updates to the legal definition of gene therapy medicinal products (GTMPs) to reflect developments in gene editing, synthetic biology and advanced manufacturing.
Gene therapies have evolved significantly since the current legal definitions were introduced more than a decade ago.
Advances in areas such as genome editing technologies, synthetic nucleic acids and new delivery methods have raised questions about whether existing definitions remain fit for purpose.
The MHRA says the proposed changes will ensure that regulation reflects how gene therapies work in practice, rather than relying solely on older classifications based on how they are produced.
A key change under consideration is redefining gene therapies based on their mechanism of action, rather than their biological origin.
This would mean that treatments would be classified as gene therapies depending on how they interact with genetic material, regardless of whether they are created using biological or synthetic processes.
The move is intended to provide greater clarity and consistency in how new therapies are regulated.
The MHRA says the updated definitions are designed to:
- Provide greater certainty for developers and manufacturers
- Ensure consistent regulation across all gene therapy products
- Support innovation in emerging technologies
- Maintain high standards of patient safety and oversight
By clarifying regulatory boundaries, the agency aims to help researchers and companies bring new treatments forward more efficiently.
Importantly, the MHRA emphasised that the proposed changes will not alter the approval process for gene therapies.
There will be:
- No changes to the regulatory status of currently licensed products
- No reduction in safety, quality or efficacy requirements
The consultation focuses purely on updating legal definitions to ensure they remain relevant in a changing scientific landscape.
The consultation seeks feedback on five main proposals:
- Removing the requirement for gene therapies to be biological in origin
- Clarifying when synthetic or recombinant nucleic acids bring a product within scope
- Ensuring genome editing technologies are clearly regulated as gene therapies
- Maintaining the exclusion of vaccines against infectious diseases from gene therapy definitions
- Updating the Human Medicines Regulations 2012 to reflect these changes
These proposals aim to create a more flexible and future‑proof regulatory framework.
Given the technical nature of the proposals, the MHRA is particularly encouraging responses from developers and manufacturers, academic researchers and clinicians, and regulatory professionals and scientific advisers. However, the consultation is open to all stakeholders and interested parties, reflecting the broad impact of gene therapy innovation.
Gene therapies represent one of the fastest‑growing areas of modern medicine, offering potential treatments for a wide range of conditions, including rare diseases, cancers and genetic disorders.
By updating its regulatory framework, the UK aims to remain at the forefront of life sciences innovation, supporting research, investment and the development of new treatments.
Jon Beaman, MHRA Deputy Director for Innovative Medicines, commented:
“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.”
This is particularly important in fields such as gene editing, where technologies are evolving rapidly and may not fit neatly into existing categories.
The consultation will remain open until 22 June 2026, after which the MHRA will review responses and consider next steps.
Any changes to legislation would be introduced following this process, ensuring that updates are informed by expert feedback and aligned with international best practice.
The MHRA said the consultation reflects its broader commitment to supporting innovation while maintaining robust regulatory standards.
By clarifying definitions and ensuring consistent oversight, the agency aims to create a framework that enables new therapies to reach patients safely, efficiently and with confidence.
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