Passage Bio

Overview

Passage Bio is a US-based gene therapy company focused on developing transformative treatments for central nervous system disorders. Founded in 2019 and headquartered in Philadelphia, Pennsylvania, Passage Bio leverages strategic partnerships with the University of Pennsylvania's Gene Therapy Program and exclusive licensing of next-generation AAV capsids to develop treatments for rare monogenic CNS diseases and common neurodegenerative disorders[@passage].

The company's lead programs target Parkinson's disease in patients with glucocerebrosidase (GBA1) mutations, a significant genetic risk factor for Parkinson's disease. Their AAVhu68 capsid technology enables enhanced delivery to the brain compared to standard approaches[@aavhu2020].

Company Overview

| Attribute | Value |
|-----------|-------|
| Ticker | NASDAQ: PASG |
| Founded | 2019 |
| Headquarters | Philadelphia, PA |
| CEO | William G. H. Chou |
| Status | Public |
| Market Cap | ~$20 million (2026) |
| Employees | ~70 |

Funding History

| Round | Year | Amount |
|-------|------|--------|
| Series A | 2019 | $55 million |
| Series B | 2020 | $110 million |
| IPO | 2020 | $130 million |

Strategic Partnerships

University of Pennsylvania

Passage Bio has an exclusive collaboration with the University of Pennsylvania's Gene Therapy Program, led by Dr. James M. Wilson. This partnership provides:

• Access to novel AAV capsids (AAVhu68)
• Vector optimization expertise
• Clinical development support
• Manufacturing capabilities

Goldman Sachs

...

Overview

Passage Bio is a US-based gene therapy company focused on developing transformative treatments for central nervous system disorders. Founded in 2019 and headquartered in Philadelphia, Pennsylvania, Passage Bio leverages strategic partnerships with the University of Pennsylvania's Gene Therapy Program and exclusive licensing of next-generation AAV capsids to develop treatments for rare monogenic CNS diseases and common neurodegenerative disorders[@passage].

The company's lead programs target Parkinson's disease in patients with glucocerebrosidase (GBA1) mutations, a significant genetic risk factor for Parkinson's disease. Their AAVhu68 capsid technology enables enhanced delivery to the brain compared to standard approaches[@aavhu2020].

Company Overview

| Attribute | Value |
|-----------|-------|
| Ticker | NASDAQ: PASG |
| Founded | 2019 |
| Headquarters | Philadelphia, PA |
| CEO | William G. H. Chou |
| Status | Public |
| Market Cap | ~$20 million (2026) |
| Employees | ~70 |

Funding History

| Round | Year | Amount |
|-------|------|--------|
| Series A | 2019 | $55 million |
| Series B | 2020 | $110 million |
| IPO | 2020 | $130 million |

Strategic Partnerships

University of Pennsylvania

Passage Bio has an exclusive collaboration with the University of Pennsylvania's Gene Therapy Program, led by Dr. James M. Wilson. This partnership provides:

• Access to novel AAV capsids (AAVhu68)
• Vector optimization expertise
• Clinical development support
• Manufacturing capabilities

Goldman Sachs

Investment banking partnership for financial strategy and capital markets guidance.

Pipeline Programs

GBA" style="color:#e0e0e0;margin:1.2rem 0 0.5rem;font-size:1rem;font-weight:600;border-bottom:1px solid rgba(255,255,255,0.08);padding-bottom:0.3rem">PBFT02 (AAV-GBA)

PBFT02 is Passage Bio's lead clinical program for Parkinson's disease patients with GBA1 mutations[@pbft].

• Mechanism: Adeno-associated virus (AAVhu68) gene therapy delivering functional GBA1 gene
• Target: Glucocerebrosidase (GCase)
• Phase: Phase 1 (IMAGINE trial)
• Indication: Parkinson's disease with GBA1 mutations
• Delivery: Intra-cisterna magna (ICM) administration
• Dose: Single administration

PBGM01 (Galactosemia)

Beyond Parkinson's, Passage Bio is developing treatments for rare genetic disorders:

• Indication: Classic galactosemia (GALT deficiency)
• Mechanism: AAV-GALT gene therapy
• Phase: Preclinical

Additional Programs

Preclinical pipeline includes:

• Krabbe disease (GALC deficiency)
• Frontotemporal dementia (progranulin deficiency)
• Additional CNS indications

Technology Platform

AAVhu68 Capsid

Passage Bio's proprietary AAVhu68 capsid offers significant advantages over standard AAV9[@gene2023]:

Enhanced CNS Tropism: Higher transduction efficiency in neurons and astrocytes

Reduced Off-Target: Lower delivery to peripheral organs

Lower Immunogenicity: Reduced pre-existing antibody prevalence

Dose Flexibility: Allows for lower doses while maintaining efficacy

Manufacturing

Passage Bio has established in-house GMP manufacturing capabilities in Philadelphia, enabling:

• Process development
• Scale-up
• IND-enabling studies
• Commercial supply

Clinical Development

IMAGINE Trial (PBFT02)

The Phase 1 IMAGINE trial is evaluating PBFT02 in Parkinson's disease patients with GBA1 mutations:

Primary Endpoints:

• Safety and tolerability
• Biomarker: CSF GCase activity
• Biomarker: Alpha-synuclein in CSF

Patient Population:

• [Parkinson](/diseases/parkinsons-disease)'s disease diagnosis
• Confirmed GBA1 mutation
• Hoehn & Yahr stage 2-3
• Age 40-75

Status: Enrolling (as of 2024)

Competitive Landscape

| Company | Drug | Mechanism | Indication | Phase |
|--------|------|-----------|------------|-------|
| Prevail/Lilly | PR001 | AAV-GBA | PD-GBA | Phase 1/2 |
| uniQure | AAV-GBA | AAV-GBA | PD-GBA | Phase 1 |
| Spark/Roche | -- | -- | -- | Preclinical |

• [PD Pipeline Companies](/companies/pd-pipeline-companies)
• [Parkinson's Disease](/genes/ar)
• [Prevail Therapeutics](/companies/prevail-therapeutics)
• [GBA1](/proteins/gba1)
• [Alpha-Synuclein](/mechanisms/alpha-synuclein)
• [Gene Therapy](/therapeutics/gene-therapy)
• [Parkinson's Disease — GBA gene therapy](/genes/ar)
• [Alzheimer's Disease — Gene therapy pipeline](/genes/th)
• [Gene Therapy for Neurodegeneration — AAV approaches](/genes/th)

See Also

• [Alzheimer's Disease](/diseases/alzheimers-disease)
• [Parkinson's Disease](/diseases/parkinsons-disease)

External Links

• [PubMed](https://pubmed.ncbi.nlm.nih.gov/)
• [KEGG Pathways](https://www.genome.jp/kegg/pathway.html)

References

Unknown, Passage Bio Corporate Website (n.d.)

[Unknown, AAVhu68 Capsid - Molecular Therapy (2020) (2020)](https://doi.org/10.1016/j.ymthe.2020.08.019)

Unknown, PBFT02 IMAGINE Trial - ClinicalTrials.gov (n.d.)

[Unknown, Gene Therapy for PD-GBA - Nature Reviews Neurology (2023) (2023)](https://doi.org/10.1038/s41582-023-00776-4)