LogicBio Therapeutics submits LB-001 IND for MMA
LogicBio Therapeutics announced it has submitted an Investigational New Drug application with the U.S. Food and Drug Administration to initiate a Phase 1/2 trial of LB-001, a recombinant adeno-associated viral vector with human methylmalonyl-COA mutase gene for the treatment of methylmalonic acidemia. LB-001 leverages LogicBio's proprietary, promoterless, nuclease-free genome editing technology, GeneRide, and has previously received both orphan drug and rare pediatric disease designations from the FDA. LogicBio intends to disclose additional details regarding the planned Phase 1/2 trial, including trial size, endpoints, and timelines, once the FDA accepts the IND. LogicBio plans to initiate a Phase 1/2 trial in pediatric MMA patients in the first half of 2020, with preliminary data expected in the second half of 2020.