ASC-601

ASC-601 is an investigational dual-function gene therapy designed for the treatment of Maple Syrup Urine Disease (MSUD) types 1A and 1B. Developed by ASC Therapeutics, the therapy utilizes a recombinant adeno-associated virus serotype 9 (rAAV9) vector to deliver codon-optimized copies of both the BCKDHA and BCKDHB genes. These genes encode the E1α and E1β subunits of the branched-chain α-ketoacid dehydrogenase (BCKDH) enzyme complex, which is responsible for breaking down branched-chain amino acids (BCAAs). In MSUD, mutations in these genes lead to toxic accumulation of BCAAs and their ketoacid derivatives. ASC-601 is designed as a one-time systemic administration that targets the liver, skeletal muscle, heart, and brain to restore enzyme activity and stabilize metabolic biomarkers, potentially offering a curative alternative to restrictive diets and liver transplantation.

Other names

BCKDHA + BCKDHB gene therapyrAAV9-BCKDHA-BCKDHBrAAV-9-BCKDHA-BCKDHBrAAV 9-BCKDHA-BCKDHBMSUD gene therapy