Gene therapy is allowed, but clinical trials require a license from the Medicines and Healthcare Devices Regulatory Agency (MHRA), as well as oversight from the Human Tissue Authority (HTA) and the Health Research Authority (HRA). Legally, all requests for gene therapy must be submitted to a GTAC that can be transferred to other designated RECs. The United Kingdom has become the first country in the world to approve genetic editing as a possible cure for two inherited blood disorders. To help us improve GOV, UK, we would like to know more about your visit today. Complete this survey (opens in a new tab).
Genome editing acts like a pair of molecular scissors that can cut a strand of DNA at a specific site, allowing scientists to alter the structure of a gene, a form of manipulation that doesn't involve introducing DNA from other organisms. In the United Kingdom, as in most countries around the world, it is illegal to edit the genome of embryos that lead to pregnancy. However, clinical trials of genome-editing treatments are progressing in many countries, and a citizen jury recently met at the Wellcome Genome Campus, near Cambridge, to discuss under what circumstances the British government should consider changing that law. All 21 jurors had personal experience with a genetic condition.
Some were parents of children who died from a genetic condition and others had an inherited condition, such as cystic fibrosis. Practical advice for centers that implement gene therapies, detailing governance requirements and optimal preparation, venue, decision-making support. For the first time in the world, the UK drug regulator has approved a therapy that uses the CRISPR—Cas9 genetic editing tool as a treatment. The decision marks another highlight for a biotechnology that has been hailed as revolutionary in the decade since its discovery.
All requests for authorization of clinical trials related to gene therapies and cell therapies will be evaluated by the MHRA and, where appropriate, will now be submitted to the MHRA Clinical Trial Expert Advisory Group for review. The following document is designed to advise centers that wish to conduct gene therapy trials or introduce commercialized gene therapy. If you are seeking ethical approval of a gene therapy clinical trial, you should contact the Gene Therapy Advisory Committee (GTAC).). Advanced therapy drugs (ATMP), such as new cell and gene therapies (CGT), offer life-changing health benefits for patients with different types of cancer and rare diseases, but there is increasing evidence of the challenges that companies face in developing such treatments and making them accessible to patients.
