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- Urgency for effective treatment underscored by European Commission designation for investigational gene therapy PBKR03 - Global Phase 1/2 trial – GALax-C – PBKR03 planned to initiate in first half of 2021 PHILADELPHIA, April 05, 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, today announced that the European Commission has granted Orphan designation for PBKR03, an adeno-associated virus (AAV)-delivery gene therapy for the treatment of Krabbe disease (Globoid Cell Leukodystrophy). The designation was based on a positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products. Currently, there are no approved disease-modifying therapies available for Krabbe disease, a rare lysosomal storage disease that most often presents early in a child’s life. The U.S. Food and Drug Administration (FDA)

University of pennsylvaniaUnited statesGwen fisherStuart hendersonBruce goldsmithExchange commissionUniversity of pennsylvania gene therapy programEuropean unionEuropean commissionDrug administrationPassage bio incGloboid cell leukodystrophyEuropean medicines agency committeeOrphan medicinalFast trackOrphan drug