AAV-delivery Gene Therapy for GM1 Gangliosidosis Granted Rare Pediatric Disease Designation

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease (RPD) designation to PBGM01, an AAV-delivery gene therapy, for the treatment of GM1 gangliosidosis, according to a press release.

PBGM01 has previously been granted orphan drug designation.

“This is the second regulatory designation we have received from the FDA for our lead program in GM1 and reflects the high unmet need in this patient population,” said Bruce Goldsmith, PhD president and chief executive officer of Passage Bio, in the press release. “As a company we are committed to developing therapies that transform the lives of patients suffering from serious life-threatening CNS disorders. We believe that PBGM01 has the potential to restore developmental progression, enabling patients to achieve additional milestones and improve quality of life. We look forward to advancing PBGM01 into clinical testing later this year.”

Read the full press release here.