Passage Bio
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.
- Company typePublic
- Founded2018
- HeadquartersPhiladelphia, United States
- Headcount51–100
- GTM typeB2B
- OfferingHardware or Manufacturing
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.
Passage Bio firmographics
Firmographics- 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
Passage Bio industry classification
Industry- 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
Where Passage Bio is headquartered
LocationHeadquarters
- HQ city
- Philadelphia
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
Passage Bio business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations, Marketing or Sales
Revenue model
- 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
Passage Bio product offering
Product offeringCore 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
Companies that use Passage Bio
Customer profileNamed customers1 record
Segments3 records
Ideal customer profiles2 records
Passage Bio technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
Passage Bio partnerships and signals
Strategic signalPartnerships
Five partnerships are on record, tiered flagship and core.
- Remix TherapeuticsflagshipPassage 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 BiotherapeuticscoreIn 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.
- InformedDNAcorePartnership 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 GeneticscorePartnership 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 PacGrowcoreEngaged 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
Passage Bio competitors and assessment
Company assessmentDirect 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
Passage Bio social profiles
Digital presencePassage Bio financial estimates
Financial estimateRevenue estimate
Valuation estimate
Passage Bio leadership team
Management profileNumber of profiles
Profiles9 records
Passage Bio funding detail
Funding detailFunding overview
Funding rounds3 records
Investors11 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Passage Bio M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Passage Bio
What does Passage Bio do?
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.
Is Passage Bio a public or private company?
Passage Bio is a public company. It is classified as public and is currently operating.
When was Passage Bio founded?
Passage Bio was founded in 2018. It employs 51 to 100 people.
Where is Passage Bio based?
Passage Bio is headquartered in Philadelphia, United States, in the North America region.
How does Passage Bio make money?
One revenue line is on record: gene Therapy Development and Commercialization.
Who are Passage Bio's main competitors?
Direct peers on record are Voyager Therapeutics, Taysha Gene Therapies, REGENXBIO, Prevail Therapeutics (Eli Lilly) and uniQure. Broad incumbents are Sarepta Therapeutics, BioMarin Pharmaceutical, Spark Therapeutics (Roche), Novartis Gene Therapies and Pfizer (Gene Therapy Division).
Does Passage Bio have an API?
No public API is recorded for Passage Bio.
What industry is Passage Bio in?
Passage Bio's product category is Gene Therapy. Its primary akta.pro industry code is HLAAACAH, Neurology/CNS Gene Therapies, with a secondary code of HLAAACAA, AAV Vector Gene Therapy Developers. Its NAICS code is 541714 and its SIC code is 2836.