LB-001

LB-001 is an investigational gene editing therapy developed for the treatment of methylmalonic acidemia (MMA) caused by mutations in the methylmalonyl-CoA mutase (MMUT) gene. It utilizes LogicBio's proprietary GeneRide platform, which employs homologous recombination to insert a functional copy of the MMUT gene into the albumin locus of hepatocytes without using exogenous nucleases or promoters. This approach aims to achieve lifelong therapeutic expression of MMUT in the liver following a single intravenous administration. The therapy is delivered via a recombinant adeno-associated virus vector (rAAV-LK03). Clinical development focused on pediatric patients with MMA; however, trials were terminated due to limited clinical benefit and safety concerns such as thrombotic microangiopathy observed in some participants[1][2][3][4][5].

Other names

LB 001LB001LB-001