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Passage Bio is focused on developing gene therapies for the treatment of rare neurological diseases. The company utilizes an adeno-associated virus (AAV) platform to deliver therapeutic genes directly to the central nervous system. This technology aims to address the underlying genetic causes of these diseases by providing a means to correct or replace defective genes, thereby potentially halting or reversing disease progression.

In addition to its AAV platform, Passage Bio is engaged in advancing its pipeline of product candidates through rigorous preclinical and clinical development. The company emphasizes a strong commitment to scientific innovation and collaboration, leveraging insights from genetic research to inform its therapeutic strategies. This approach positions Passage Bio at the forefront of gene therapy, particularly in the context of neurological disorders, where traditional treatment options may be limited.

United States

Passage Bio is a genetic medicines company based in Philadelphia, PA that is developing a deep pipeline of adeno-associated virus (AAV) delivered therapeutics for the treatment of rare monogenic central nervous system disorders (CNS).

We seek to deliver transformative therapies to patients by leveraging our team’s decades of experience, including our founders, as well as the transformative potential genetic medicine technology to develop treatments that improve outcomes for patients with serious, life-threatening, rare CNS disorders.

Central Nervous System (CNS)

Genetic Diseases

Metabolic Diseases

Passage Bio

Frontotemporal dementia

Neurological disorder

We (PASG) are developing PBFT02 to treat FTD-GRN with a single dose of PBFT02 by intra-cisterna magna injection. PBFT02 is a gene therapy that utilizes an AAV1 viral vector to deliver a modified DNA encoding the granulin (GRN) gene to a patient's cells. The goal of this vector and delivery approach is to provide higher than normal levels of progranulin (PGRN) to the central nervous system (CNS) to overcome the progranulin deficiency in GRN mutation carriers, who have been observed to have reduced cerebrospinal fluid PGRN levels ranging from 30% to 50% of the PGRN levels observed in normal, mutation non-carriers.
Gene therapy

PBFT02 (AAV1)

Rare genetic disease

We (PASG) &nbsp;are developing PBGM01 to treat infantile GM1 with a single dose of PBGM01 by intra-cisterna magna injection. PBGM01 utilizes a next-generation AAVhu68 viral vector to deliver modified DNA encoding the b-gal enzyme to a patient's cells. The goal of this vector and delivery approach is to increase levels of the b-gal enzyme in both the central nervous system (CNS) and the peripheral nervous system (PNS).

Passage Bio

Company Details