Passage Bio

What Passage Bio does

Passage Bio is a clinical-stage genetic medicines company developing AAV-based gene therapies targeting monogenic central nervous system disorders, with its lead candidate PBFT02 designed to elevate progranulin levels in the CNS as a treatment for frontotemporal dementia caused by GRN gene mutations. The company's core technology platform uses an AAV1 viral vector delivered via a single intra-cisterna magna (ICM) injection directly into the cerebrospinal fluid, an approach intended to achieve broad CNS distribution at lower vector doses with reduced immune response compared with systemic AAV delivery. Pipeline assets include the ongoing upliFT-D Phase 1/2 trial in FTD-GRN and FTD-C9orf72 cohorts, potential expansion of the progranulin-elevation mechanism into ALS and Alzheimer's disease (rs5848 SNP), a preclinical Huntington's disease program using AAV-delivered miRNA to suppress MSH3, and out-licensed pediatric programs (GM1 gangliosidosis, Krabbe disease, metachromatic leukodystrophy) now held by GEMMA Biotherapeutics.

The company is pre-revenue with no approved products or disclosed pricing; revenue mechanics, if achieved, would rely on future one-time sale or licensing of approved gene therapy products to specialist neurology centers treating rare neurodegenerative indications. The company has raised approximately $379.5M in private financing prior to its February 2020 Nasdaq IPO and reported $46.3M in cash and a $45.5M net loss for FY2025.

In April 2026, the FDA required a randomized controlled registrational trial for PBFT02 in FTD-GRN rather than the proposed single-arm design, triggering a 75% workforce reduction (from 24 to roughly 6 employees) and a strategic review. In June 2026, Passage Bio announced an all-stock reverse merger with Remix Therapeutics in which pre-merger Passage Bio shareholders will retain approximately 7% of the combined entity (to be operated as Remix, ticker RMTX), supported by a concurrent $100M private placement led by Decheng Capital. The transaction is expected to close in Q4 2026.

Name

Passage Bio

Legal name

Passage Bio, Inc.

Website

https://passagebio.com

Company type

Public

Founded year

2018

Operating status

Operating

Headcount range

51–100 employees

Short description

Passage Bio is a clinical-stage gene therapy company developing AAV1-based treatments delivered intra-cisternally to elevate progranulin in the CNS, with lead candidate PBFT02 in a Phase 1/2 trial for frontotemporal dementia, now merging into Remix Therapeutics.

Ownership category

akta.pro rank

Product category

Gene Therapy

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834)

akta.pro primary industry

Neurology/CNS Gene Therapies (HLAAACAH)

akta.pro secondary industries

AAV Vector Gene Therapy Developers (HLAAACAA), Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream) (HLAAACAN)

Keywords

• Gene therapy development
• AAV-based therapeutics
• CNS disease treatment
• Neurodegenerative disease therapy
• Clinical-stage biotechnology

Headquarters

HQ city

Philadelphia

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales

Revenue model

1. Gene Therapy Development and Commercialization: Pre-revenue clinical stage biotechnology company with no currently approved products. The company is developing gene therapies for neurodegenerative diseases with PBFT02 as the lead candidate targeting frontotemporal dementia. Revenue generation would come from future product sales upon successful FDA approval and commercialization.

Go-to-market motion1 record

Distribution channels3 records

Marketing channels4 records

Core offering

Passage Bio is a clinical-stage genetic medicines company developing AAV-based gene therapies for monogenic central nervous system diseases. Its lead candidate PBFT02 is an AAV1 viral vector delivering the granulin gene (GRN) via intra-cisterna magna (ICM) injection to elevate progranulin levels in the CNS, currently in Phase 1/2 clinical trials for frontotemporal dementia with expansion plans into ALS, Alzheimer's, and Huntington's disease.

Product overview

Passage Bio is a clinical-stage genetic medicines company developing a focused pipeline of optimized AAV-based gene therapies for neurodegenerative diseases. The company's core technology platform uses adeno-associated virus (AAV1 serotype) vectors delivered via intra-cisterna magna (ICM) administration to target the CNS. Lead product PBFT02 is an AAV1 viral vector delivering the granulin gene to elevate progranulin levels, currently in Phase 1/2 clinical trials for multiple FTD indications (FTD-GRN, FTD-C9orf72), with expansion into ALS and Alzheimer's disease. The company is also advancing a preclinical Huntington's disease program targeting MSH3 suppression. All programs utilize the same AAV-based genetic medicine platform with direct CSF delivery approach.

Differentiator

Problem solved

Functional benefit

Products and services

• PBFT02

Quantifiable outcome

• 64% reduction in whole brain atrophy at 12 months in FTD-GRN patients with CDR score of 1 at baseline compared to natural history data
• +3 more outcomes

Named customers1 record

Segments3 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Five partnerships are on record, tiered flagship and core.

• Remix TherapeuticsflagshipStrategic or Co-development Partner · 24 June 2026Passage Bio agreed to an all-stock reverse merger with Remix Therapeutics, a private RNA specialist. Upon closing expected in Q4 2026, pre-merger Passage Bio shareholders will retain approximately 7% of the combined company operating as Remix Therapeutics, trading under ticker RMTX. Remix CEO Peter Smith will lead the combined entity.
• GEMMA BiotherapeuticscoreStrategic or Co-development Partner · 1 August 2024In August 2024, Passage Bio partnered with GEMMA Biotherapeutics to license three pediatric gene therapy candidates including PBGM01 for GM1 gangliosidosis, along with candidates for Krabbe disease and metachromatic leukodystrophy.
• InformedDNAcoreStrategic or Co-development PartnerPartnership with InformedDNA to design and support a genetic testing and counseling program that is free of charge to patients in the US with frontotemporal disease, supporting clinical trial recruitment for the upliFT-D study.
• Prevention GeneticscoreStrategic or Co-development PartnerPartnership with Prevention Genetics to provide healthcare providers access to no-cost genetic testing for patients who are suspected of having FTD in all markets, supporting clinical trial recruitment.
• Wedbush PacGrowcoreImplementation/ SI/ Consulting PartnerEngaged Wedbush PacGrow as financial advisor to assist in the strategic review process initiated in April 2026 following FDA feedback requiring a randomized controlled registrational trial for PBFT02.

Scale indicators9 records

Recent moves7 records

Expansion highlights5 records

Direct peers

• Voyager Therapeutics: Clinical-stage gene therapy company developing AAV-based therapies for CNS diseases including Alzheimer's, Parkinson's, and Huntington's. Directly comparable as a CNS-focused AAV gene therapy developer with similar pipeline breadth and delivery mechanism innovation.
• Taysha Gene Therapies: Clinical-stage AAV gene therapy company focused on monogenic CNS diseases, with a pipeline spanning Rett syndrome, GM2 gangliosidosis, and SURF1 deficiency. Closely comparable in target indication (rare monogenic CNS), platform (AAV), and clinical stage.
• REGENXBIO: Clinical and commercial-stage AAV gene therapy company with proprietary NAV platform technology used across CNS, retinal, and metabolic diseases. Directly comparable as an AAV platform developer with CNS pipeline assets (RGX-121 for MPS II, RGX-111 for MPS I).
• Prevail Therapeutics (Eli Lilly): AAV9-based gene therapy developer acquired by Eli Lilly in 2020, focused on CNS diseases including frontotemporal dementia (GRN) and Parkinson's disease with GBA1 mutations. Highly comparable target indication (FTD-GRN) and approach (AAV gene therapy for neurodegenerative diseases).
• uniQure: Clinical and commercial-stage gene therapy company developing AAV-based therapies for CNS (Huntington's with AMT-130) and metabolic diseases. Comparable as an AAV platform developer with a CNS pipeline asset directly competitive in the Huntington's indication.

Broad incumbents

• Sarepta Therapeutics: Commercial-stage genetic medicines company with approved AAV gene therapies (Elevidys for DMD) and an RNA-based pipeline. Comparable as a leader in genetic medicines targeting rare neurological diseases with both AAV and alternative modality approaches.
• BioMarin Pharmaceutical: Commercial-stage rare disease biopharma with a CNS gene therapy program (Roctavian for hemophilia A and an AAV-based CLN2 Batten disease program). Comparable as an established player developing AAV gene therapies for rare neurological conditions with regulatory and commercialization experience.
• Spark Therapeutics (Roche): Gene therapy pioneer and Roche subsidiary, developer of the first FDA-approved AAV gene therapy (Luxturna for RPE65-mediated inherited retinal disease). Comparable as an established AAV gene therapy platform within a larger biopharma portfolio.
• Novartis Gene Therapies: Commercial-stage biopharma with multiple gene therapy franchises including Zolgensma (AAV9 for SMA) and Kymriah (CAR-T). CEO Will Chou previously led Kymriah's global launch at Novartis. Comparable as an established commercial-stage gene therapy leader with CNS and rare disease focus.
• Pfizer (Gene Therapy Division): Large biopharma with an active gene therapy pipeline including AAV-based programs for CNS and rare diseases (DMD, hemophilia). Comparable as a broad incumbent investing in AAV gene therapy across multiple CNS and rare disease indications.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xx.com/passage_bio
• Facebookfacebook.com/PassageBio
• LinkedInlinkedin.com/company/passage-bio

Revenue estimate

Valuation estimate

Number of profiles

Profiles9 records

Funding overview

Funding rounds3 records

Investors11 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Passage Bio