Posted by Michael Wonder on 22 May 2020
Passage Bio receives rare paediatric disease designation for PBGM01 for patients with GM1 gangliosidosis
21 May 2020 - PBGM01 to enter the clinic in fourth quarter of 2020.
Passage Bio today announced the U.S. FDA has granted rare paediatric disease designation to PBGM01 broadly for the treatment of GM1 gangliosidosis.
PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1 gangliosidosis and has previously been granted orphan drug designation.
