UK Approves World's First CRISPR Gene Therapy for Blood Disorders

U.S.-based companies Vertex Pharmaceuticals and CRISPR Therapeutics welcomed the approval, with optimism for treating various diseases.

Screenshot 2023 11 26 At 3 26 10 Pm
Reuters

According to a recent Reuters article, the United Kingdom has authorized the use of a gene therapy called Casgevy to treat sickle-cell disease and another inherited blood disorder in patients over the age of 12. The move marks the first instance of approval for a medicine using CRISPR, which was awarded the Nobel Prize in 2020. Sickle cell disease and β-thalassemia result from genetic errors affecting the production of hemoglobin, crucial for transporting oxygen in red blood cells. 

Fresh from the show floor: pharma packaging innovations for 2026
Serialization mandates. Containment demands. Sterile barrier requirements. Our editors found the pharma packaging innovations addressing your biggest challenges at PACK EXPO Las Vegas. Get your free curated report now.
GET YOUR COPY
Fresh from the show floor: pharma packaging innovations for 2026