LB-401

LB-401 is a preclinical gene therapy candidate developed by LogicBio Therapeutics that applies the company’s GeneRide genome editing platform as a next-generation approach for in vivo treatment of an undisclosed monogenic liver disease. It is designed to achieve site-specific integration of a therapeutic transgene at a designated genomic locus in hepatocytes using homologous recombination, enabling durable expression without exogenous nucleases. The program represents an expansion of LogicBio’s genome editing pipeline beyond its lead candidate LB-001 for methylmalonic acidemia and is being advanced toward IND-enabling studies as a systemic, liver-directed gene-editing therapy.