REGENXBIO
- Company typePublic
- Founded2009
- HeadquartersRockville, United States
- Headcount251–500
- GTM typeB2B
- OfferingHardware or Manufacturing
REGENXBIO firmographics
Firmographics- Name
- REGENXBIO
- Legal name
- REGENXBIO Inc.
- Website
- https://regenxbio.com
- Company type
- Public
- Founded year
- 2009
- Operating status
- Operating
- Headcount range
- 251–500 employees
- Ownership category
- akta.pro rank
REGENXBIO industry classification
Industry- Product category
- Gene therapy / AAV-based biopharmaceuticals
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714)
- SIC
- Biological Products, (No Disgnostic Substances) (2836)
- akta.pro primary industry
- AAV Vector Gene Therapy Developers (HLAAACAA)
- akta.pro secondary industries
- Neurology/CNS Gene Therapies (HLAAACAH), Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Ophthalmology Gene Therapies (HLAAACAG), Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream) (HLAAACAN)
Keywords
Where REGENXBIO is headquartered
LocationHeadquarters
- HQ city
- Rockville
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
REGENXBIO business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Operations, Personnel, Infrastructure
Revenue model
- Royalty Revenue from ZOLGENSMA: REGENXBIO earns royalties from ZOLGENSMA (onasemnogene abeparvovec), a gene therapy for spinal muscular atrophy using NAV Technology, commercialized by Novartis. Royalty stream from product sales.
- Partnership and Licensing Revenue: Revenue from strategic partnerships including upfront payments, milestone payments, and ongoing royalties from AbbVie (ABBV-RGX-314 for retinal diseases) and Nippon Shinyaku (MPS programs). Includes $72.9 million upfront license payment from Nippon Shinyaku in 2025.
- Milestone Payments: Expected $100 million milestone payment from AbbVie upon first patient dosing in Phase IIb/III NAAVIGATE study for diabetic retinopathy expected Q2 2026. Additional potential milestone payments tied to regulatory approvals and commercial milestones from partners.
- Royalty Bond Financing: In May 2025, REGENXBIO closed a royalty bond agreement with Healthcare Royalty for up to $250 million, monetizing expected royalties and milestones from gene therapy products including ZOLGENSMA and others for rare and retinal diseases.
Go-to-market motion1 record
Distribution channels3 records
Marketing channels5 records
REGENXBIO product offering
Product offeringCore offering
REGENXBIO develops investigational AAV gene therapies for rare genetic and retinal diseases using its proprietary NAV Technology Platform of 100+ novel adeno-associated virus vectors. Its clinical pipeline includes RGX-202 for Duchenne muscular dystrophy, RGX-121 (Navsunli) for MPS II (Hunter syndrome), and ABBV-RGX-314 for wet AMD and diabetic retinopathy, supported by in-house cGMP manufacturing via the NAVXpress process. The platform is also licensed to other developers, including Novartis for the approved ZOLGENSMA therapy.
Product overview
REGENXBIO is a clinical-stage biotechnology company developing gene therapies using its proprietary NAV Technology Platform. The company's portfolio includes multiple therapeutic candidates: NAVSUULI (RGX-121) for MPS II, RGX-202 for Duchenne muscular dystrophy, and ABBV-RGX-314 (sura-vec) for wet AMD and diabetic retinopathy. These programs leverage the NAV AAV vector platform and are supported by the company's NAVXpress manufacturing process and in-house cGMP manufacturing capabilities.
Differentiator
Problem solved
Functional benefit
Products and services
- NAV Technology Platform REGENXBIO's proprietary library of 100+ novel AAV vectors used to develop gene therapies across multiple therapeutic areas and licensed to other gene therapy developers.
- RGX-121 (Navsunli) One-time AAV gene therapy designed to deliver the human iduronate-2-sulfatase (IDS) gene to the CNS for the treatment of patients with Mucopolysaccharidosis Type II (Hunter syndrome).
- RGX-202 One-time AAV gene therapy delivering a novel microdystrophin transgene with a functional C-Terminal domain for the treatment of Duchenne muscular dystrophy.
- ABBV-RGX-314 (sura-vec) One-time AAV gene therapy designed to deliver sustained anti-VEGF antibody expression in the eye for patients with wet age-related macular degeneration and diabetic retinopathy.
- NAVXpress Manufacturing Process Proprietary scalable AAV vector manufacturing process enabling consistent yield and product purity for clinical and commercial cGMP production at REGENXBIO's Manufacturing Innovation Center.
Quantifiable outcome
- 93% of patients achieved primary endpoint of 10% microdystrophin expression at Week 12 in Phase 3 AFFINITY DUCHENNE trial (p<0.0001)
- +3 more outcomes
Companies that use REGENXBIO
Customer profileNamed customers4 records
Segments2 records
Ideal customer profiles3 records
REGENXBIO technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature5 records
REGENXBIO partnerships and signals
Strategic signalPartnerships
Two partnerships are on record, tiered core.
- Nippon Shinyaku / NS PharmacoreStrategic partnership with Nippon Shinyaku for development and commercialization of REGENXBIO's MPS programs including RGX-121/Navsunli for Hunter syndrome (MPS II). REGENXBIO received $110 million upfront payment plus up to $700 million in potential development and commercial milestones. NS Pharma Inc. (subsidiary) holds U.S. commercialization rights for Navsunli.
- AbbViecoreGlobal partnership with AbbVie for development and commercialization of ABBV-RGX-314 (sura-vec) for wet age-related macular degeneration (wet AMD) and diabetic retinopathy. REGENXBIO received upfront payments and is eligible for up to $100 million milestone upon first patient dosing in NAAVIGATE study. AbbVie leads global commercialization with REGENXBIO manufacturing clinical supply.
Scale indicators7 records
Recent moves7 records
Expansion highlights5 records
REGENXBIO competitors and assessment
Company assessmentDirect peers
- uniQure: uniQure is a clinical-stage gene therapy company developing AAV-based therapeutics for hemophilia, Huntington's disease, and other indications, and is also a NAV Technology licensee. Both companies focus on AAV gene therapy platforms targeting rare diseases with high unmet need.
- BioMarin Pharmaceutical: BioMarin developed and commercializes Roctavian, the first FDA-approved AAV gene therapy for hemophilia A. Both companies operate in the AAV gene therapy space targeting rare genetic diseases with one-time curative treatments.
- Adverum Biotechnologies: Adverum is developing ixoberogene soroparvovec (Ixo-vec), an AAV-based gene therapy for wet AMD, directly competing with REGENXBIO's ABBV-RGX-314 in the ophthalmology gene therapy space. Both companies target the wet AMD market with one-time AAV gene therapy alternatives to chronic anti-VEGF injections.
- Sarepta Therapeutics: Sarepta commercializes Elevidys, the only FDA-approved gene therapy for Duchenne muscular dystrophy and REGENXBIO's primary competitive rival in the DMD gene therapy space. Both companies are developing AAV-based microdystrophin therapies, making Sarepta the most direct clinical and commercial comparable.
- 4D Molecular Therapeutics: 4DMT is a clinical-stage gene therapy company developing tailored AAV vectors for ophthalmology and cardiology indications. Both companies develop novel AAV capsid platforms targeting multiple rare disease and ophthalmic indications.
- Spark Therapeutics: Spark Therapeutics (acquired by Roche) developed and commercializes Luxturna, the first FDA-approved gene therapy for an inherited genetic disease (RPE65 mutation). Both companies develop AAV-based therapeutics for rare genetic diseases, with Jean Bennett on REGENXBIO's board having co-founded Spark.
Broad incumbents
- Pfizer: Pfizer commercializes Beqvez, an FDA-approved AAV gene therapy for hemophilia B. As a large pharmaceutical incumbent with gene therapy capabilities, Pfizer competes broadly with REGENXBIO in the AAV gene therapy market while offering a wider portfolio of therapeutics.
- Novartis: Novartis commercializes ZOLGENSMA, the first FDA-approved NAV Technology-based gene therapy for SMA, generating royalty revenue for REGENXBIO. As a global pharmaceutical leader with established gene therapy commercialization, Novartis is both a partner and a broad incumbent in the gene therapy market.
Emerging players
- Solid Biosciences: Solid Biosciences is a clinical-stage gene therapy company developing AAV-based treatments for Duchenne muscular dystrophy. As a direct competitor in the same indication with similar AAV-based microdystrophin approach, Solid is an emerging peer in the DMD gene therapy space.
- Capricor Therapeutics: Capricor is developing cell therapy (deramiocel) and exosome-based treatments for Duchenne muscular dystrophy. While using a different therapeutic modality, Capricor targets the same patient population as RGX-202 and represents an emerging player in the DMD treatment landscape.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat5 records
Key risks7 records
Key highlights7 records
Customer concentration
REGENXBIO social profiles
Digital presenceREGENXBIO financial estimates
Financial estimateRevenue estimate
Valuation estimate
REGENXBIO leadership team
Management profileNumber of profiles
Profiles18 records
REGENXBIO funding detail
Funding detailFunding overview
Funding rounds13 records
Investors19 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
REGENXBIO 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 REGENXBIO
What does REGENXBIO do?
REGENXBIO develops investigational AAV gene therapies for rare genetic and retinal diseases using its proprietary NAV Technology Platform of 100+ novel adeno-associated virus vectors. Its clinical pipeline includes RGX-202 for Duchenne muscular dystrophy, RGX-121 (Navsunli) for MPS II (Hunter syndrome), and ABBV-RGX-314 for wet AMD and diabetic retinopathy, supported by in-house cGMP manufacturing via the NAVXpress process. The platform is also licensed to other developers, including Novartis for the approved ZOLGENSMA therapy.
Is REGENXBIO a public or private company?
REGENXBIO is a public company. It is classified as public and is currently operating.
When was REGENXBIO founded?
REGENXBIO was founded in 2009. It employs 251 to 500 people.
Where is REGENXBIO based?
REGENXBIO is headquartered in Rockville, United States, in the North America region.
How does REGENXBIO make money?
Four revenue lines are on record. Royalty Revenue from ZOLGENSMA is the primary driver. The others are partnership and Licensing Revenue, milestone Payments and royalty Bond Financing.
Who are REGENXBIO's main competitors?
Direct peers on record are uniQure, BioMarin Pharmaceutical, Adverum Biotechnologies, Sarepta Therapeutics, 4D Molecular Therapeutics and Spark Therapeutics. Broad incumbents are Pfizer and Novartis. Emerging players are Solid Biosciences and Capricor Therapeutics.
Does REGENXBIO have an API?
No public API is recorded for REGENXBIO.
What industry is REGENXBIO in?
REGENXBIO's product category is Gene therapy / AAV-based biopharmaceuticals. Its primary akta.pro industry code is HLAAACAA, AAV Vector Gene Therapy Developers, with a secondary code of HLAAACAH, Neurology/CNS Gene Therapies. Its NAICS code is 541714 and its SIC code is 2836.