It has been just revealed the fact that the UK is the first country to approve CRISPR gene editing therapy. Check out the latest reports about this below.
UK to approve CRISPR gene editing therapy
The UK has made history by becoming the first country in the world to regulate the authorization of a gene-editing therapy based on CRISPR.
The government announced on Thursday that a new treatment for sickle-cell disease and transfusion-dependent β-thalassemia has been authorized by the Medicines and Healthcare Products Regulatory Agency (MHRA) for patients aged 12 and over.
This treatment, known as Casgevy, is the first medicine to be licensed that uses the gene-editing tool CRISPR.
CRISPR is a technology that edits genes by precisely cutting DNA and then utilizing natural DNA repair processes to modify the gene in the desired manner.
According to the government, sickle cell disease and β-thalassemia are genetic conditions that result from errors in the genes for hemoglobin.
Haemoglobin is utilized by red blood cells to transport oxygen throughout the body. Sickle cell disease is prevalent among individuals with African or Caribbean ancestry, while β-thalassemia commonly affects people of Mediterranean, South Asian, Southeast Asian, and Middle Eastern descent.
Casgevy is a therapy that aims to treat a genetic disorder by fixing the faulty gene in a patient’s bone marrow stem cells.
This process involves removing stem cells from the bone marrow, editing them in a laboratory, and then returning them back to the patient.
The results of this therapy can potentially last for the patient’s lifetime.
Casgevy was granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) by the Medicines and Healthcare Products Regulatory Agency (MHRA), which is now responsible for regulating drugs in the UK after Brexit.
The ILAP is an initiative that aims to speed up the marketing authorization process for new drugs.
Vertex Pharmaceuticals and CRISPR Therapeutics, the partners behind Casgevy, welcomed the authorization of this therapy.