Business
Passage Bio Announces Plan to Deliver on Multiple Meaningful Catalysts in 2021
– Investigational New Drug Applications (IND) submitted for PBFT02 for Frontotemporal Dementia with Granulin Mutation (FTD-GRN) and PBKR03 for Krabbe Disease
About this update from Passage Bio, Inc.
[{"type":"text","content":"– Investigational New Drug Applications (IND) submitted for PBFT02 for Frontotemporal Dementia with Granulin Mutation (FTD-GRN) and PBKR03 for Krabbe Disease – – Three clinical programs to begin in 1H21 – – Metachromatic leukodystrophy program, PBML04, advanced to IND-enabling phase – – Additional option exercised from Penn’s world-class Gene Therapy Program for undisclosed program focused on an adult CNS disorder – – New manufacturing laboratory to support analytical capabilities, clinical product testing and assay validation slated to open in 2Q21 – – PBFT02 for FTD-GRN becomes latest of company’s three most advanced programs to receive FDA orphan drug designation – PHILADELPHIA, Jan. 11, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today provided a corporate outlook detailing the expansion of the company’s pipeline, new regulatory filings, projected program milestones, and research and development progress. Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio, will highlight the company’s recent progress and plans for the future in his presentation today at the 39th Annual J.P. Morgan Healthcare Conference. In his remarks, Dr. Goldsmith will highlight several components of the Company’s progress and timelines, including: Planned initiation of patient dosing in clinical studies for three novel adeno-associated virus (AAV) gene therapies:- Received FDA and MHRA clearance for PBGM01 for infantile GM1 gangliosidosis (GM1)- Submitted IND for PBKR03 for the treatment of Krabbe disease- Submitted IND for PBFT02 for treatment of Frontotemporal Dementia (FTD) with granulin (GRN) mutation.Advancement of PBML04 for the treatment of metachromatic leukodystrophy (MLD) into IND-enabling studiesLicense of a seventh program from the University of Pennsylvania (Penn) for the treatment of an adult rare monogenic CNS disorderPlanned opening of a manufacturing laboratory to support analytical capabilities, clinical product testing and assay validation “It is widely reported that there are approximately 7,000 rare diseases. Less discussed is that out of these 7,000 rare diseases, there are more than 790 rare monogenic CNS diseases, the area where we are focused,” said Dr. Gold...