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 (ß-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). Its third product candidate, PBKR03, utilizes a next-generation AAVhu68 capsid to deliver to the brain and peripheral tissues a functional gene encoding the hydrolytic enzyme galactosyl ceramidase, (GALC) for Krabbe disease. The Company also has six programs in the research stage: PBML04 for metachromatic leukodystrophy; PBAL05 for amyotrophic lateral sclerosis; PBCM06 for Charcot-Marie-Tooth Type 2A; and programs for Canavan disease, and others.
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