Business
Passage Bio Announces Publication of Preclinical Data that Support Ongoing Clinical Study of PBKR03 in Krabbe Disease
Preclinical studies conducted with University of Pennsylvania’s Gene Therapy Program demonstrate biologic effects of vector-optimized gene therapy PBKR03 for
About this update from Passage Bio, Inc.
[{"type":"text","content":"Preclinical studies conducted with University of Pennsylvania’s Gene Therapy Program demonstrate biologic effects of vector-optimized gene therapy PBKR03 for Krabbe diseasePBKR03 showed marked improvements in safety, disease progression and key biomarkers for Krabbe disease in large and small animal modelsCompany is enrolling patients for cohort 1 in global clinical trial, GALax-C, with PBKR03 for infantile Krabbe disease, a rare, fatal pediatric condition PHILADELPHIA, March 29, 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, today announced publication of the robust pre-clinical studies that supported clinical study initiation for PBKR03 gene therapy for infantile Krabbe disease. In the paper reporting these preclinical results, Juliette Hordeaux, D.V.M., Ph.D., D.E.C.V.P., and colleagues from University of Pennsylvania’s Gene Therapy Program (GTP), report marked improvements in both disease progression and key biomarkers in large and small animal models of Krabbe disease following a single administration of PBKR03, with no observed dose-limiting toxicities. The paper is published online ahead of print in the May issue of Human Gene Therapy. 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 neurons 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 infantile Krabbe disease, the most severe form, is two years. “The benefits of our strategic collaboration with GTP are apparent when you see the robust preclinical data underpinning our decision to move forward with PBKR03 for infantile Krabbe disease,” said Bruce Goldsmith, Ph.D., chief executive officer and president, Passage Bio. “These preclinical studies support our belief that gene replacement with PBKR03 has the poten...