Passage Bio Inc
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Passage Bio, Inc. is a clinical-stage genetic medicines company. The Company is focused on developing transformative therapies for central nervous system (CNS) disorders. Its first product candidate, PBGM01, utilizes a AAVhu68 capsid to deliver to the brain and peripheral tissues a functional GLB1 gene encoding lysosomal beta-galactosidase (B-gal), for GM1. Its second product candidate, PBFT02, utilizes an AAV1 capsid to deliver to the brain a functional GRN gene encoding progranulin (PGRN), for FTD caused by progranulin deficiency (FTD-GRN). The Company has two programs in the research stage: PBAL05 for amyotrophic lateral sclerosis (ALS), and an unnamed program for Huntington’s disease. PBAL05 is targeting patients with ALS who have a gain-of-function mutation in the C9orf72 gene. It also has an exploratory research program for larger non-monogenic indications, which is focused on temporal lobe epilepsy (TLE).
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