PASG

Passage Bio, Inc. is a clinical-stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases. Its clinical product candidate, PBFT02, seeks to elevate progranulin levels to restore lysosomal function and slow disease progression across a variety of neurodegenerative diseases. PBFT02 utilizes an adeno-associated virus capsid to deliver a functional granulin gene, or GRN, encoding progranulin to the brain via intra cisterna magna administration. The lead indication for PBFT02 is frontotemporal dementia, or FTD, caused by progranulin deficiency, or FTD-GRN. Its development programs consist of PBFT02 for the treatment of FTD-GRN, PBFT02 for the treatment of FTD-C9orf72 and amyotrophic lateral sclerosis, and PBFT02 for the treatment of Alzheimer’s disease. Its clinical product candidates include PBGM01, PBKR03 and PBML04. It has one unnamed preclinical research program that explores multiple potential treatment targets for Huntington's disease.