Passage Bio
Passage Bio develops genetic medicines for neurodegenerative diseases, with lead programs in GM1 gangliosidosis and frontotemporal dementia. The company collaborates with the University of Pennsylvania and has a robust preclinical pipeline including candidates for ALS and Huntington’s disease.
Company Overview
Passage Bio, headquartered at One Commerce Square, 2005 Market Street, Philadelphia, PA 19103, is focused on developing genetic medicines for neurodegenerative diseases. The company went public in February 2020 and is traded on the Nasdaq Global Select Market under the ticker PASG. Passage Bio's mission is to address unmet medical needs through gene therapy, targeting conditions such as GM1 gangliosidosis and frontotemporal dementia (FTD).
Pipeline and Clinical Programs
Passage Bio is advancing lead clinical programs in both GM1 gangliosidosis and frontotemporal dementia (FTD). The company’s upliFT-D study is a multinational Phase 1/2 clinical trial evaluating the safety and efficacy of PBFT02, a gene therapy for patients with frontotemporal dementia with progranulin gene mutations. Passage Bio’s product candidate PBFT02 utilizes an AAV1 viral vector to deliver modified DNA encoding the granulin genotype (GRN) directly to a patient's cells through a single injection to the cisterna magna. Additionally, the company is investigating PBGM01 for treating GM1 gangliosidosis.
Research and Development
Passage Bio boasts a promising preclinical pipeline that includes candidates for amyotrophic lateral sclerosis (ALS) and Huntington’s disease. The company leverages advanced gene therapy techniques, exploring the therapeutic potential of elevating progranulin (PGRN) levels in the CNS to alter the course of neurodegenerative diseases. Passage Bio's genotype therapy portfolio spans both pediatric and adult central nervous system (CNS) indications.
Strategic Collaborations
Passage Bio has a strategic collaboration with the University of Pennsylvania’s Gene Therapy Program (GTP) and Orphan Disease Center. Additionally, they partner with Catalent for manufacturing capabilities and maintain in-house process development at their Princeton West Innovation Campus in Hopewell, NJ. The company also collaborates with Informed DNA to support a genetic screening and counseling program for frontotemporal disease, and with Invitae to facilitate genetic testing for early identification of children with GM1 gangliosidosis.
Manufacturing and Distribution
Passage Bio has established robust manufacturing and global clinical distribution networks to support their pipeline from lab to clinic and potentially to commercialization. The company partners with Catalent for manufacturing while also having in-house process development capabilities at their Princeton West Innovation Campus. These resources ensure scalable production of their gene therapy candidates to meet clinical and future commercial demands.