Posted by Michael Wonder on 10 Sep 2024
BridgeBio receives FDA’s regenerative medicine advanced therapy designation for BBP-812 Canavan disease gene therapy program
10 September 2024 - Receipt of RMAT designation is based on preliminary clinical evidence from the CANaspire Phase 1/2 clinical trial, which showed functional improvements in all dosed patients indicating that BBP-812 has potential to address the unmet needs of individuals with Canavan disease.
BridgeBio Pharma today announced that the US FDA has granted regenerative medicine advanced therapy designation to BBP-812, an investigational intravenous adeno-associated virus serotype 9 gene therapy for the treatment of Canavan disease.
