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Passage Bio Receives FDA Clearance of IND Application for Lead Gene Therapy Candidate PBGM01 for Treatment of Infantile GM1 Gangliosidosis
FDA clearance marks second regulatory authorization for global Phase 1/2 clinical trial, Imagine-1 study, expected to dose first patient in first quarter 2021
About this update from Passage Bio, Inc.
[{"type":"text","content":"FDA clearance marks second regulatory authorization for global Phase 1/2 clinical trial, Imagine-1 study, expected to dose first patient in first quarter 2021 PHILADELPHIA, Jan. 04, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today 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. “FDA clearance of our IND represents a significant milestone that supports the transition of Passage Bio to a clinical-stage company,” said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. “It is an achievement that reflects the potential of our bold science, the deep value of our partnership with the University of Pennsylvania’s Gene Therapy Program, and the dedication of our entire team to our shared mission to deliver one-time gene therapies that improve the lives of patients with rare CNS disorders.” 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 percent of the global GM1 incidence of 0.5 to 1 in 100,000 live births. “I am pleased that Passage Bio has FDA clearance to proceed with this study,” said James Wilson, M.D., Ph.D., director of the Gene Therapy Program at the University of Pennsylvania (Penn) and chief scientific advisor of Passage Bio. “We look forward to Passage Bio’s clinical advancement of this therapy that aims to slow...