Passage BIO, Inc.

Passage BIO, Inc. is a U.S.-based clinical-stage genetic medicines company focused on developing one-time gene therapies for neurodegenerative diseases. The company was incorporated in Delaware and is centered on advancing licensed technology, conducting clinical trials, and manufacturing clinical supply for its product candidates.

2.07

2.07

— Passage BIO, Inc.
%
Clinical-stage gene therapy candidates0% One-time genetic medicines designed to treat neurodegenerative diseases, including PBFT02.
Preclinical and translational development0% Research, assay development, and preclinical studies supporting pipeline advancement.
Clinical trial supply and manufacturing0% Manufacturing of clinical-grade material and supply for ongoing trials.
Licensed technology development0% Development and use of in-licensed intellectual property for CNS gene therapy programs.

Passage BIO’s direct customers are not commercial end-users today; its work is aimed at regulators, clinical...

  • Clinical investigators and trial sitesprimary

    They run studies of PBFT02 and related programs and need clinical supply, protocols, and support.

  • Regulatorsprimary

    FDA and other agencies evaluate the company’s clinical and manufacturing data for approval pathways.

  • Future patients with neurodegenerative diseaseprimary

    The eventual end users of approved gene therapies for CNS disorders such as frontotemporal dementia.

  • Potential strategic partnerssecondary

    Biopharma collaborators or licensors that may support development, funding, or commercialization.

Passage BIO is incorporated in Delaware and operates as a U.S.-based development-stage biotechnology company...

  • •Incorporated in Delaware, United States
  • •Clinical development and operations are U.S.-based
  • •Manufacturing supply currently relies on third-party U.S. facilities
  • •Future market access would depend on U.S. and international reimbursement
  • •No country revenue disclosure is available because the company is pre-commercial

The company’s strategy is to advance PBFT02 and other gene therapy programs through clinical development, with a focus...

01
Advance PBFT02 clinical developmentshort-term

Clinical data are the main driver of value for a pre-commercial gene therapy company.

02
Secure additional capitalshort-term

Development-stage operations require external funding before product revenue exists.

03
Strengthen manufacturing and assay capabilitiesmedium-term

Gene therapies require validated, reproducible manufacturing and release testing.

Passage BIO faces the typical risks of a clinical-stage biotechnology company: clinical failure, regulatory delay,...

high

Single-program concentration

PBFT02 is currently the only clinical-stage product candidate, so setbacks would have outsized impact.

Scope
Pipeline concentration
Materiality
high
high

Clinical development failure

Gene therapy programs can fail on safety, efficacy, dosing, or delivery even after early promise.

Scope
PBFT02 and CNS gene therapy platform
Materiality
high
high

Manufacturing and supply chain disruption

The company relies on third parties for clinical supply, testing, and process development.

Scope
Catalent Maryland and external labs
Materiality
high
high

Financing risk

Operations are funded through external capital until product revenue, if any, is generated.

Scope
Equity, debt, collaborations
Materiality
high
medium

Reimbursement and market access risk

Even if approved, payors may limit coverage or pricing for high-cost gene therapies.

Scope
U.S. and international commercialization
Materiality
medium
Research and development expense recognition
Clinical trial, manufacturing, and development costs
Accruals for clinical and manufacturing vendors
Operating expenses and liabilities
Impairment of licensed technology or intangibles
Balance sheet carrying values and earnings
Stock-based compensation
Operating expense and diluted share count

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: 29/04/2026