Passage Bio, Inc. announced that the company’s lead program PBGM01 is entering the clinic to study its safety and efficacy in addressing infantile GM1, a rare and often life-threatening CNS disorder with no approved disease-modifying therapies. The global Phase 1/2 study, Imagine-1, is expected to enroll the first patient in the first quarter of 2021 and is expected to report initial 30-day safety and biomarker data mid-year 2021. PBGM01 has received regulatory agency clearance from FDA and MHRA. The investigational therapy has also received orphan drug and rare pediatric disease designations from FDA, as well as orphan drug designation from the European Medicines Agency for the treatment of GM1. The company has submitted investigational new drug (IND) applications to FDA for Phase 1/2 clinical studies of PBFT02 in FTD-GRN, a devastating form of early onset dementia; and PBKR03 in Krabbe disease, a pediatric disease with rapid progression, typically resulting in death by 2 years old. The company recently received orphan drug designation from FDA for PBFT02 for FTD-GRN. PBKRO3 has orphan drug and rare pediatric disease designations from FDA for treatment of Krabbe disease. Passage Bio anticipates the start of both clinical programs in the first half of 2021. Initial data from these trials are anticipated to potentially readout in late 2021 or early 2022, depending on the timing of first patient treated in each study. In parallel with pipeline advancement, the company has focused on establishing manufacturing and global distribution from clinical development through initial commercialization. Clinical supply for PBGM01 for phase 1 /2 global trial, manufactured through partnership with Catalent, is already in place. The company have also manufactured clinical supplies to initiate clinical trials for next two most advanced programs – PBKRO3 for Krabbe disease and PBFT02 for FTD-GRN. In December, Passage Bio announced that it had completed construction and started production at its dedicated Current Good Manufacturing Practices (CGMP) manufacturing suite at Catalent Cell & Gene Therapy’s facility in Maryland. The company also announced the planned opening in the second quarter of 2021 of a gene therapy manufacturing research and development site at the Princeton West Innovation Campus in Hopewell, New Jersey, for Chemistry, Manufacturing and Controls (CMC) laboratory operations to support analytics, assay development, and product testing for the company’s gene therapy programs.