The United Kingdom’s medicines regulator has launched a consultation to overhaul how gene therapies are legally defined, in a move aimed at keeping pace with rapid advances in gene editing, synthetic biology and next-generation genetic medicines.
The proposed changes by the Medicines and Healthcare products Regulatory Agency (MHRA), announced jointly with the Department of Health in Northern Ireland, seek to modernise the regulatory framework governing gene therapy medicinal products (GTMPs). The consultation will run until 22 June 2026.
The changes matter beyond the UK because global regulatory definitions increasingly shape how advanced therapies move through international supply chains, clinical trials and approvals. For Indian biotechnology firms and researchers working on mRNA therapies, gene editing and synthetic nucleic acid platforms, the evolving regulatory landscape in the UK and Europe could influence future collaborations and market access.
Gene therapies have evolved sharply since the current UK definitions were framed more than a decade ago. According to the consultation document, existing legislation was developed when treatments relied on a narrower set of technologies. Newer therapies now include synthetic genetic material, chemically produced mRNA constructs and genome-editing systems that do not fit neatly within existing legal categories.
At the centre of the consultation is a proposal to classify gene therapies based on how they work rather than whether they originate from biological material. The MHRA said the revised definitions are intended to provide regulatory certainty for developers while ensuring consistent oversight of both biologically derived and synthetic therapies.
The regulator has proposed removing the requirement that gene therapies must be biological in origin. Under the proposed framework, synthetic or chemically produced nucleic acid therapies could also be classified as GTMPs if they meet other regulatory criteria.
The consultation also seeks to bring newer genome-editing technologies within the formal gene therapy framework, regardless of whether they rely on recombinant nucleic acids. Current rules, the MHRA noted, do not adequately account for emerging non-recombinant and non-nucleic-acid-based gene editing approaches.
The proposed amendments would classify products involving sequence-specific genome editing as gene therapies even if the active substance is not itself a recombinant nucleic acid. The changes would also cover products containing recombinant or synthetic nucleic acids that mediate their effect through transcription or translation.
Importantly, the MHRA said the updated definitions would not alter existing approval pathways or reduce current standards for safety, quality or efficacy. Products already licensed would also retain their regulatory classification.
Jon Beaman, MHRA Deputy Director for Innovative Medicines, said the proposals were designed to ensure the 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,” Beaman said.
The consultation identifies several problems with the current framework. The existing definition excludes synthetic nucleic acid constructs even when they function identically to biologically derived therapies. It also does not adequately address newer genome-editing technologies or products where genetic modification serves manufacturing or safety functions rather than directly driving therapeutic action.
The regulator warned that such gaps could create classification uncertainty, inconsistent oversight and barriers for developers, potentially affecting patient access to innovative medicines.
Another key proposal is to maintain the exclusion of vaccines against infectious diseases from GTMP classification, irrespective of the technology used. The MHRA said the existing vaccine regulatory framework remains appropriate for preventive vaccines administered to healthy individuals.
The consultation also reflects broader international regulatory discussions. The MHRA noted that the European Union has separately proposed amendments to its own definition of gene therapy medicinal products. However, the UK proposal differs in certain areas, including the use of the phrase “target genome” instead of “host genome”, and the decision not to limit GTMP classification only to products involving “long lasting” transcription or translation.
The MHRA said it is particularly seeking feedback from developers, manufacturers, researchers, regulatory professionals and academic experts, although the consultation is open to all stakeholders.
The broader objective, according to the consultation document, is to create a future-proof regulatory framework that supports innovation while maintaining high standards of patient safety and oversight. The regulator said clearer and technologically neutral definitions could strengthen international alignment and help ensure patients gain access to safe and effective advanced therapies.
