PASG - Passage Bio's lead gene therapy an Orphan Drug in Europe for rare nerve cell disease

The European Commission has designated Orphan Drug status to Passage Bio's (PASG) PBGM01 for the treatment of GM1 gangliosidosis (GM1)PBGM01, an adeno-associated virus-delivery gene therapy, and has previously been granted Orphan Drug and Rare Pediatric Disease designation in the U.S. for the same indicationThe company expects to initiate dosing of PBGM01 in a Phase 1/2 trial by end of this year or early 2021, with initial 30-day safety and biomarker data expected in 1H of 2021. GM1 is a rare inherited lysosomal storage disorder caused by mutations in the GLB1 gene which leads to impaired production of an enzyme that plays an essential role in the breakdown of sugar molecules. The condition, which typically presents in infants, gradually destroys nerve cells in the brain and spinal cord.

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Passage Bio's lead gene therapy an Orphan Drug in Europe for rare nerve cell disease