MMA is caused by a mutation in genes responsible for the breakdown of certain proteins and fats, leading to their toxic accumulation in cells. MMA usually presents in infancy and may be life-threatening.
LB-001 was developed using LogicBio’s GeneRide platform, and is designed to use cells’ natural DNA repair process to insert a corrective copy of the gene into the patient’s genome.
“With fast track status, we plan to continue to work closely with the FDA to fully utilize the opportunities presented by this designation to make LB-001 available to patients as quickly as possible,” Daniel Gruskin, M.D., LogicBio’s SVP, head of clinical development, said in a statement.
LogicBio plans to initiate a Phase 1/2 clinical trial of LB-001 in pediatric patients with MMA in early 2021.