BridgeBio Pharma Launches Biopharm Company for Rare Genetic DisordersBy
BridgeBio Pharma, a Palo Alto, California-based clinical-stage biopharmaceutical company, has launched CoA Therapeutics, a biopharmaceutical company developing small-molecules designed to increase Coenzyme-A (CoA) levels in genetic disorders where CoA deficiency is implicated.
CoA Therapeutics’ lead compound is expected to enter the clinic in 2019 with a planned initial focus on pantothenate kinase-associated neurodegeneration (PKAN).
PKAN is an autosomal recessive genetic disorder caused by mutations in the pantothenate kinase 2 (PANK2) gene. The PANK2 enzyme plays a role in the synthesis of CoA, which is key in energy metabolism and implicated in a large number of developmental disorders, according to information from BridgeBio.