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Passage Bio Announces First Patient Dosed in Imagine-1 Study of PBGM01 Gene Therapy for Infantile GM1 Gangliosidosis
– PBGM01 advancement to clinical development represents key milestone for Passage Bio as a clinical-stage company – PHILADELPHIA, April 01, 2021 (GLOBE
About this update from Passage Bio, Inc.
[{"type":"text","content":"– PBGM01 advancement to clinical development represents key milestone for Passage Bio as a clinical-stage company – PHILADELPHIA, April 01, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, announced that the first patient has been dosed in the company’s global Phase 1/2 clinical trial program for PBGM01, an adeno-associated virus (AAV)-delivery gene therapy that is being studied for the treatment of infantile GM1 gangliosidosis (GM1). The patient in the Imagine-1 clinical trial received PBGM01 at The Children’s Hospital at Saint Peter’s University Hospital in New Brunswick, NJ. This officially marks an important milestone in the company’s advancement to a clinical-stage company. “In keeping with our company’s mission to develop life-transforming therapies for rare CNS disorders, we are truly excited to progress the clinical development of PBGM01 with the dosing of our first patient. We look forward to sharing initial safety and biomarker data mid-year,” said Bruce Goldsmith, Ph.D., president and chief executive officer at Passage Bio. “We are also proud of our team at Passage Bio for their outstanding work and the University of Pennsylvania’s Gene Therapy Program for their collaboration in successfully advancing our first investigational therapy into the clinic.” 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 gangliosides in neurons throughout the brain, causing rapidly progressive neurodegeneration. GM1 accumulation also results in progressive damage to other tissues including the heart, liver, and bones and manifests with hypotonia (reduced muscle tone), progressive CNS dysfunction, seizures, and rapid developmental regression. Life expectancy for infants with GM1 ranges from 2-10 years, and infantile GM1 represents approximately 60 percent of the global GM1 incidence of 0.5 to 1 in 100,000 live births. “Our ultimate aim is to develop a transformative gene therapy that will preserve neurological function, and improve developmental potential, survival and quality of life for patients with GM1,” ...