INDIANAPOLIS – Akouos, Inc., a wholly owned subsidiary of Eli Lilly and Company (NYSE: LLY), announced positive initial clinical results from the Phase 1/2 AK-OTOF-101 study, which demonstrated pharmacologic hearing restoration within 30 days of AK-OTOF administration in the first participant, an individual with a decade-plus history of profound hearing loss. Results, including initial data from a second participant to …
KIRKLAND – Pfizer Canada ULC announced that Health Canada approved BEQVEZ (fidanacogene elaparvovec), an adeno-associated viral (AAV) vector-based gene therapy for the treatment of adults (aged 18 years or older) with moderately severe to severe hemophilia B (congenital Factor IX (FIX) deficiency) who are negative for neutralizing antibodies to variant AAV serotype Rh74. “Pfizer has more than 30 years of …
LONDON (AP) Britain’s medicines regulator has authorized the world’s first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling disease in the UK. In a statement, the Medicines and Healthcare Regulatory Agency said it approved Casgevy, the first medicine licensed using the gene editing tool CRISPR, which won …
