Lead Product(s): AAVrh.10 Expressing Beta-galactosidase
Therapeutic Area: Genetic Disease Product Name: LYS-GM101
Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 31, 2021
LYS-GM101 (‘adeno-associated viral vector serotype rh.10 expressing beta-galactosidase’) received orphan drug designation for the treatment of GM1 gangliosidosis in the European Union and in the US in 2017, as well as the Rare Pediatric Disease designation in the US in 2016.