Passage Bio Receives FDA Clearance of IND Application for Lead Gene Therapy Candidate PBGM01 for Treatment of Infantile GM1 Gangliosidosis
January 04, 2021 at 07:00 am EST
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Passage Bio, Inc. announced that U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for the company's lead product candidate, PBGM01, an adeno-associated virus (AAV)-delivery gene therapy that is being studied for the treatment of infantile GM1 gangliosidosis (GM1). GM1 is a rare and often life-threatening CNS disorder with no approved disease-modifying therapies available. The company expects to dose the first patient for the global PBGM01 clinical trial program in the first quarter of 2021. GM1, a rare monogenic lysosomal storage disease, is caused by mutations in the GLB1 gene, which encodes the lysosomal enzyme beta -galactosidase (β-gal). Reduced β-gal activity results in the accumulation of toxic levels of GM1 in neurons throughout the brain, causing rapidly progressive neurodegeneration. GM1 manifests with hypotonia (reduced muscle tone), progressive CNS dysfunction, and rapid developmental regression. Life expectancy for infants with GM1 is two to four years, and infantile GM1 represents approximately 60% of the global GM1 incidence of 0.5 to 1 in 100,000 live births.
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 Alzheimerâs 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.