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Passage Bio Announces Presentation of Data from Animal Models of Krabbe Disease at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting
– Preclinical data from University of Pennsylvania’s Gene Therapy Program demonstrate single injection of AAVhu68 carrying a functional GALC gene resulted in
About this update from Passage Bio, Inc.
[{"type":"text","content":"– Preclinical data from University of Pennsylvania’s Gene Therapy Program demonstrate single injection of AAVhu68 carrying a functional GALC gene resulted in normalization of GALC enzyme activity and improved all parameters in animal models of Krabbe disease –\n – Treated Krabbe dogs showed nerve conduction normalization, CSF psychosine levels normalization, improved brain histopathology, phenotypic correction and increased survival – – Twitcher mouse model demonstrated peripheral nerve correction, a dose-dependent correction of phenotype and increased survival – PHILADELPHIA, May 12, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced the presentation of preclinical data for its Krabbe disease program. This data was presented today in an virtual oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 23rd Annual Meeting by Juliette Hordeaux, D.V.M., Ph.D., senior director of translational research at the University of Pennsylvania’s Gene Therapy Program. “The data presented today showcase the promising potential of pairing CSF administration with high potency vectors to achieve robust, scalable effects utilizing cross-correction on central and peripheral nerve function,” said James Wilson, M.D., Ph.D., director of the Gene Therapy Program at the University of Pennsylvania and chief scientific advisor of Passage Bio. “The marked improvements on critical markers of disease such as myelination and neuroinflammation as well as, and perhaps more importantly, phenotypic improvements in function and ultimately prolonged survival suggest that ICM gene therapy may be incredibly efficacious for rare CNS indications such as Krabbe disease.” “We are extremely excited about this data for our Krabbe program. We believe it demonstrates the potential of the AAVhu68 GALC therapeutic PBKR03 as a life-altering therapy for patients with infantile Krabbe disease, and we look forward to advancing this program into the clinic,” said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. “These findings suggest that PBKR03 may be able to normalize GAL-C activity and restore myelination and nerve function in both the brain and key peripheral tissues that would h...