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Passage Bio

Rare disease drugmaker · based in Philadelphia, United States · founded 2019 · passagebio.com

Passage Bio works on Cerebral Adrenoleukodystrophy and Waldenström Macroglobulinemia, 2 of the rare diseases we track.

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.

Passage Bio clinical trials

We did not find active Passage Bio trials for the rare diseases we track on ClinicalTrials.gov right now.

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More about 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 active and recruiting rare disease 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.

Rare diseases Passage Bio works on

Each disease page lists every open trial, not only Passage Bio's, plus approved treatments.

Cerebral Adrenoleukodystrophy

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.

How common: 1 in 20,000 males worldwide; cerebral form develops in approximately 35-40% of boys with X-ALD

Waldenström Macroglobulinemia

Waldenström Macroglobulinemia is a rare blood cancer in which malignant B-lymphocytes produce excessive amounts of a protein called macroglobulin (immunoglobulin M or IgM).

How common: Approximately 1,500 new cases per year in the United States; median age of diagnosis is 68 years

Where to get support

Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Passage Bio's own site is passagebio.com, and our help paying for treatment page lists drugmaker programs and charity funds.

Questions about Passage Bio