Consultation on the regulation of Gene therapy medicinal products
We are seeking views on proposed updates to the definition of Gene therapy medicinal products to better reflect advances in science and manufacturing.
Advanced Therapy Medicinal Products (ATMPs) represent a category of medicinal products for human use that are based on genes, tissues, or cells. These medicines may be used to treat serious health conditions, including cancer and genetic disorders. In the UK, the definition, and regulation of ATMPs are primarily governed by the Human Medicines Regulations 2012. Gene therapy medicinal products (GTMPs) are a type of ATMP.
Advances in gene therapies increasingly challenge the suitability of the current UK definitions of GTMPs in the Human Medicines Regulations 2012. These definitions, originally developed in 2007, were created at a time when treatments were based on a smaller range of technologies. Today, innovative therapies can involve synthetic genetic material, new forms of genome editing, and highly engineered cells and tissues. Some of these products do not fit neatly into the categories set out in the existing legislation.
Key limitations in the current legislative definition of GTMPs have become apparent. The requirement for GTMPs to be biological in origin excludes synthetic nucleic acid constructs that function identically to biologically derived therapies. The focus in the existing legislation on recombinant nucleic acids was also unable to anticipate both emerging gene editing approaches delivered through non-recombinant nucleic acid systems and potential non nucleic acid platforms. The requirement that the therapeutic effect of a GTMP must derive directly from the recombinant sequence excludes products where genetic modification provides a crucial safety or manufacturing function. This mismatch between regulatory terminology and modern science can lead to classification uncertainty, inconsistent oversight, and potential barriers for developers, ultimately affecting patient access.
This consultation outlines proposed revisions to the current legal definitions to adopt a more technologically neutral approach. Under the proposed revisions, GTMP classification encompass active substances which contain a recombinant or synthetic nucleic acid moiety that undergoes transcription or translation, as well as active substances with a mechanism of action that involves sequence specific genome editing. While these changes update the definitional criteria for classification, they do not alter the underlying regulatory principles applicable to GTMPs, and ensure that synthetically manufactured products, and gene editing systems are appropriately captured within the framework.
This consultation seeks views from stakeholders on proposed amendments to the GTMP definitions. The objective is to modernise the GTMP definitions to reflect current scientific practice, ensure consistent risk-based oversight and maintain the UK’s position as a supportive environment for advanced therapy development. A modernised, future-proofed framework will give developers greater clarity, strengthen international alignment and help ensure that patients benefit from safe, effective and innovative treatments.