Passage Bio Doses First Patient in Global Clinical Trial for Infantile Krabbe Disease, A Rare Fatal Pediatric Condition
Company expects to report interim safety and biomarker data for Cohort 1 patients in the global phase 1/ 2 clinical trial, GALax-C, by end of 2022
Excerpt from the Press Release:
PHILADELPHIA, March 10, 2022 (GLOBE NEWSWIRE) — Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for central nervous system (CNS) disorders, announced that the first patient with early infantile Krabbe disease has received PBKR03, an adeno-associated virus (AAV)-delivery gene therapy, in its global Phase 1/2 clinical trial, GALax-C.
“It is gratifying to dose the first patient in our GALax-C trial,” said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. “We now have two global clinical trials with patients dosed under way. The advancement of these two clinical trials represents significant progress for our company in our mission to develop transformative medicines for people with devastating CNS disorders. We look forward to reporting initial safety and biomarker data from GALax-C by end of 2022.”
Krabbe disease is a rare pediatric lysosomal storage disorder caused by mutations in the GALC gene, which encodes galactosylceramidase, an enzyme that breaks down galactosylceramide and psychosine. Without adequate levels of galactosylceramidase, psychosine accumulates, causing widespread death of myelin-producing cells and progressive damage to nerves in both the brain and peripheral tissues of affected children. This is characterized in children by loss of acquired milestones, staring episodes, apnea, peripheral neuropathy, severe weakness, unresponsiveness to stimuli, seizures, blindness, and deafness. Life expectancy for early infantile Krabbe disease, the most severe form, is two years.
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