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LogicBio Therapeutics Announces Submission of Investigational New Drug Application (IND) for LB-001 for Methylmalonic Acidemia (MMA) and Highlights Key Corporate Milestones

a?? Filed IND for lead GeneRidea?? candidate LB-001 in pediatric MMA patients a??
a?? Phase 1/2 trial initiation planned for H1 2020, with preliminary data in H2 2020 a??
a?? Established research collaboration with Takeda for GeneRide in Crigler-Najjar Syndrome (CN) a??CAMBRIDGE, Mass., Jan. 10, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients,A today announced itA has submitted an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) to initiate a Phase 1/2 trial of LB-001, a recombinant adeno-associated viral vector with human methylmalonyl-COA mutase (MMUT) gene for the treatment of methylmalonic acidemia (MMA). LB-001 leverages LogicBioa??s proprietary, promoterless, nuclease-free genome editing technology, GeneRidea??, 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.a??We founded LogicBio with the mission of bringing genetic medicines to children with rare diseases. Both the IND submission and the nomination of our second indication represent significant steps in advancing our goal,a?? said Fred Chereau, CEO of LogicBio. a??MMA and CN are devastating early onset diseases with no approved pharmacological therapies, and we are committed to developing novel medicines based on our GeneRide platform for pediatric patients.A  We look forward to a transformational year for LogicBio as we work to advance our programs, validate our platform, and expand our pipeline.a??Today, LogicBio also highlighted key recent and upcoming milestones.
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