Passage BIO(US:PASG)2022-03-04 19:38Company Overview - Passage Bio is dedicated to developing genetic medicines for central nervous system (CNS) disorders[3, 70] - The company leverages a partnership with Penn's Gene Therapy Program (GTP) for AAV gene therapy research[3, 4, 70] - Passage Bio has dedicated manufacturing and analytics capabilities to ensure product supply[3, 8, 70] Pipeline and Portfolio - The company has a diversified portfolio strategy across rare and large, monogenic and non-monogenic CNS indications[5, 6, 70] - The pipeline includes 3 clinical-stage therapies and 6 research-stage pipeline candidates[5] - The company is advancing programs for Amyotrophic Lateral Sclerosis (ALS), Charcot-Marie-Tooth Type 2A (CMT2A), Parkinson's, Canavan and Huntington's diseases[68] Clinical Programs - PBGM01 (GM1 Gangliosidosis): Ongoing Phase 1/2 Imagine-1 trial showed a positive safety profile, functional transgene expression, and developmental gains in Cohort 1[5, 15, 18] - Interim Cohort 1 data showed increases in CSF β-gal activity of +0.6 nmol/mL/3hr and serum β-gal activity of +6.0 nmol/mL/3hr in Patient 1[21, 23] - Patient 1 demonstrated meaningful gains in developmental milestones, advancing to 9 months developmental age[29] - Patient 2 regained previously lost developmental milestones at month 4[36] - PBKR03 (Krabbe Disease): Phase 1/2 GALax-C trial is focused on early infantile Krabbe, with first patient dosing expected in early 2022[5, 43, 44] - The trial includes dose escalation and confirmatory cohorts with doses of 1.5e11 GC/g and 5.0e11 GC/g[44] - PBFT02 (Frontotemporal Dementia - GRN): Phase 1/2 upliFT-D trial is focused on early symptomatic FTD-GRN, with first patient dosing expected in early 2022[5, 48, 49] - The trial includes dose escalation cohorts with doses of 3.3e10 GC/g and 1.1e11 GC/g[49] Financials - The company had a cash balance of $316 million as of December 31, 2021[69] - The cash on hand is expected to fund operations to year-end 2023[69]