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Pivotal study demonstrated a clinically meaningful rate of tumor shrinkage (overall response rate) and duration of response in patients with previously-treated advanced cholangiocarcinoma (CCA)
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Passage Bio collaborator University of Pennsylvania’s Gene Therapy Program to present poster regarding development of a novel mouse model for evaluating efficacy of adeno-associated virus gene therapy for metachromatic leukodystrophy (MLD)
Passage Bio gene therapy candidate, PBML04, showed preliminary signs of efficacy in novel mouse model of MLD
PHILADELPHIA, May 11, 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 today the presentation by its collaborator University of Pennsylvania’s Gene Therapy Program (GTP) of a digital poster at the 24th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). Gourav Roy Choudhury, Ph.D., a senior research investigator at GTP, will report on the development and utility of a novel Arylsulfatase A-deficient mouse model in reprodu
- 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)