Passage Bio
Passage Bio works on 2 rare diseases tracked on Trial Friend, including Cerebral Adrenoleukodystrophy, Waldenström Macroglobulinemia, with 2 recruiting clinical trials.
Passage Bio is a gene therapy company focused on AAV-delivered therapeutics for rare central nervous system diseases. The company's lead active program is PBFT02 for frontotemporal dementia with GRN mutations. In August 2024, Passage Bio out-licensed its pediatric programs PBGM01 (GM1 gangliosidosis) and PBKR03 (Krabbe disease) to GEMMA Biotherapeutics.
Focus areas at Passage Bio
As a rare disease specialist, Passage Bio has active clinical trial programs and drug development efforts across 2 rare diseases, including Cerebral Adrenoleukodystrophy, Waldenström Macroglobulinemia. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows all active and recruiting studies sponsored by Passage Bio, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Passage Bio is headquartered in Philadelphia, United States, founded in 2019. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Passage Bio Drug Pipeline
Passage Bio has 2 active clinical trials across 2 development stages, with 2 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline includes all of Passage Bio's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
Passage Bio Clinical Trials (2)
Active and recruiting clinical trials sponsored by Passage Bio, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
FDA-Approved Drugs
Passage Bio Trial Locations
Passage Bio clinical trials are running at 33 sites in 5 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (2)
Diseases targeted by Passage Bio's clinical trial and drug development programs
Cerebral adrenoleukodystrophy is the most severe form of X-linked adrenoleukodystrophy, caused by mutations in the ABCD1 gene that impair breakdown of very-long-chain fatty acids. These accumulate in ...
Waldenström Macroglobulinemia is a rare blood cancer in which malignant B-lymphocytes produce excessive amounts of a protein called macroglobulin (immunoglobulin M or IgM). This excess protein thicken...
Patient Resources
Organizations and resources related to Passage Bio's rare disease focus areas
Frequently Asked Questions About Passage Bio
Common questions about Passage Bio's rare disease programs, clinical trials, and treatments.