Prime Medicine, a biotechnology company committed to delivering a new class of differentiated, one-time curative genetic therapies, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for PM359, submitted on March 29,…
Akouos, a wholly owned subsidiary of Eli Lilly and Company, announced positive initial clinical results from the Phase 1/2 AK-OTOF-101 study, which demonstrated pharmacologic within 30 days of AK-OTOF administration in the first participant, an individual with a decade-plus history…
Regeneron announced preliminary, positive safety and efficacy results from the first patient (<2 years of age) dosed in the Phase 1/2 CHORD trial investigating otoferlin gene therapy (DB-OTO) in children with profound genetic hearing loss due to mutations of the…
Cleveland Clinic has infused a new gene therapy to deliver a working gene to address the leading cause of hypertrophic cardiomyopathy (HCM) in the first patient in the world as part of a clinical trial. Myosin binding protein C3 (MYBPC3)…