CTNNB1 Foundation

What CTNNB1 Foundation does

CTNNB1 Foundation is a Slovenia-based, research-driven non-profit (headquartered at Dalmatinova 5, 1000 Ljubljana) founded around 2017 and formally launched into gene therapy research in 2021. Its mission is to develop a disease-modifying treatment for CTNNB1 syndrome, a severe neurodevelopmental disorder for which no approved therapy exists. The foundation serves an ultra-rare disease population — affected children and their families — alongside the clinicians and researchers who treat and study the condition.

The foundation's core product is URBAGEN, an AAV-based gene replacement therapy that delivers a functional copy of the CTNNB1 gene directly into the brain. URBAGEN was granted EU Orphan Drug Designation (EU/3/25/3101) by the EMA and is being evaluated clinically in the GAIN-CTNNB1 trial, which began recruiting pediatric participants aged 2–12 in late 2025 and dosed its second patient in May 2026. The program is supported by a vertically integrated set of capabilities: research partnership with the Children's Medical Research Institute (signed 2021), preclinical testing through Jackson Laboratory, Charles River, and the National Institute of Chemistry Ljubljana, clinical-grade AAV manufacturing with Viralgen (initiated November 2023), and regulatory-enabling toxicology (CIMA Universidad de Navarra, Czech Centre for Phenogenomics) and biodistribution (Labena) work. Complementing the therapy, the foundation runs the Dragonfly Natural History Study to collect longitudinal patient data, and convenes the global community through the annual International CTNNB1 Syndrome Conference (4th edition, Barcelona, June 2026).

CTNNB1 Foundation's business model is donation-driven rather than commercial: it generates no product revenue. Cumulative funding of more than €4 million was raised across 2021–2024 across individual donations, peer-to-peer campaigns (GiveButter-powered "Walking with Urban" campaigns raised €1,462,531), corporate donations (Sartorius, GaletaBio, Agilent, Elpida, Mediasi, Columbus Fundacion, 3D-PXC), patient-association contributions (CTNNB1 Association Spain €235,000; Slovenian Palčica Pomagalčica €580,000), founder personal donations (€325,000), and government grants (Slovenian government €1,000,000 in late 2024). The top five contributors provided 86% of total funding, indicating a concentrated donor base. The foundation sells nothing; it funds, develops, and ultimately seeks to make available a curative gene therapy for an ultra-rare pediatric population.

Name

CTNNB1 Foundation

Legal name

CTNNB1 Foundation

Website

https://ctnnb1-foundation.org

Company type

Private

Founded year

2017

Operating status

Operating

Headcount range

1–10 employees

Short description

CTNNB1 Foundation is a Slovenia-based non-profit developing URBAGEN, a first-in-class AAV gene replacement therapy for CTNNB1 syndrome, currently in EMA-approved GAIN-CTNNB1 pediatric clinical trials (ages 2–12). It funds operations via donations, peer-to-peer campaigns, corporate sponsors, and grants, serving affected children and their families globally.

Ownership category

akta.pro rank

Product category

Rare Disease Gene Therapy Development

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

akta.pro primary industry

Neurology/CNS Gene Therapies (HLAAACAH)

akta.pro secondary industry

Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS) (BPAGACAA)

Keywords

• Gene replacement therapy
• Rare disease research
• Clinical trial development
• Natural history study
• Patient advocacy foundation

Headquarters

HQ city

Ljubljana

HQ country

Slovenia

HQ region

Europe

Offices1 record

Markets served

GTM type

B2B and B2C

Offering type

Services

Cost components

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

Revenue model

1. One-time and recurring donations: Direct donations from individuals and families through the foundation website, PayPal, and GiveButter peer fundraising pages; over €4 million raised across 2021–2024 via multiple fundraising campaigns. CTNNB1 Spain contributed €235,000 in targeted fundraising, and founders personally donated €325,000.
2. Government and institutional grants: Public-sector grant funding; the Slovenian government contributed €1,000,000 in late 2024 to enable clinical trial development in Slovenia. Other institutional funding supports basic and proof-of-concept gene therapy studies.
3. Corporate donations and sponsorships: Direct corporate contributions and sponsorship via donation agreements; recent contributions include €40,000 from CTNNB1 Association Spain and €15,000 jointly from GaletaBio and Sartorius to lead the final fundraising campaign. Supporters/partners include Sartorius, GaletaBio, Agilent, Elpida, Mediasi, Columbus Fundacion, 3D-PXC, and Jeans for Genes.
4. Community peer-to-peer fundraising campaigns: Family-driven campaigns — most notably the 'Walking with Urban' Slovenian campaigns — collectively raised €1,462,531 to fund basic and proof-of-concept gene therapy studies; the Slovenian association 'Palčica Pomagalčica' contributed €580,000 in support.

Pricing tiers

Go-to-market motion3 records

Distribution channels4 records

Marketing channels8 records

Core offering

CTNNB1 Foundation is a research-driven non-profit that develops and supports URBAGEN, a first-in-class AAV-based gene replacement therapy delivering a functional CTNNB1 gene directly into the brain to treat CTNNB1 syndrome. The therapy is being clinically evaluated through the GAIN-CTNNB1 trial and is supported by the global Dragonfly Natural History Study, with all activities funded via donations, corporate sponsorships, and government grants.

Product overview

CTNNB1 Foundation is a research-driven non-profit organization built around a single core investigational product — the URBAGEN gene replacement therapy — supported by a portfolio of complementary programs and modules. The flagship URBAGEN therapy (granted EMA Orphan Drug Designation EU/3/25/3101) is being evaluated clinically through the GAIN-CTNNB1 clinical trial, while the Dragonfly NHS Natural History Study generates the longitudinal patient data that informs URBAGEN's development. The Treatment Strategies and Research Activities & Publications programs document and advance the underlying science, the annual International CTNNB1 Syndrome Conference convenes the global community of families, researchers, and clinicians, and the CTNNB1 Foundation Membership Program keeps supporters informed of research, clinical, and event updates.

Differentiator

Problem solved

Functional benefit

Brands

• URBAGEN: CTNNB1 Foundation's investigational gene replacement therapy product for CTNNB1 syndrome; granted Orphan Drug Designation by the EMA (EU/3/25/3101).
• GAIN-CTNNB1
• Dragonfly NHS

Products and services

• URBAGEN Gene Replacement Therapy Investigational AAV-based gene replacement therapy that delivers a functional copy of the CTNNB1 gene directly into the brain to treat CTNNB1 syndrome at its root cause; first CTNNB1 gene therapy showing functional restoration in preclinical models and granted EMA Orphan Drug Designation (EU/3/25/3101). Not commercially sold.
• GAIN-CTNNB1 Clinical Trial First-in-human clinical trial evaluating the URBAGEN gene therapy for CTNNB1 syndrome; approved in 2025 and open for recruitment of pediatric participants aged 2–12 years. Clinical trial application was officially submitted to the European Medicines Agency (EMA).
• Dragonfly Natural History Study (NHS) Global longitudinal natural history study for CTNNB1 syndrome that collects essential clinical data from patients worldwide to support research, family support, patient care, and clinical trial design for URBAGEN and future treatments.
• International CTNNB1 Syndrome Conference Annual international conference series bringing together the CTNNB1 community, families, researchers, and clinicians to share research progress, clinical trial updates, and conduct clinical examinations. The 4th edition is held in Barcelona, June 18–19, 2026.
• CTNNB1 Foundation Membership Program Membership subscription program providing supporters with research and clinical trial updates, the monthly newsletter, and notifications about upcoming events and participation opportunities.

Quantifiable outcome

• Over €4 million raised across 2021–2024 to fund gene therapy development
• +2 more outcomes

Named customers1 record

Segments4 records

Ideal customer profiles4 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Eleven partnerships are on record, tiered flagship and core.

• Children's Medical Research InstituteflagshipStrategic or Co-development PartnerResearch agreement signed in 2021 to begin development of different constructs of the URBAGEN gene replacement therapy. Foundation describes this as the starting point of the gene therapy program.
• The Jackson LaboratorycoreImplementation/ SI/ Consulting PartnerPerformed preclinical testing for URBAGEN as one of the world-renowned laboratories supporting the gene therapy program.
• Charles RivercoreImplementation/ SI/ Consulting PartnerPerformed preclinical testing for URBAGEN.
• National Institute of Chemistry Ljubljana (NIC)coreImplementation/ SI/ Consulting PartnerPerformed preclinical testing for URBAGEN and is listed as a partner on the foundation website.
• ViralgenflagshipStrategic or Co-development PartnerManufacturing partner; produced the clinical-grade AAV vector for URBAGEN. Manufacturing process initiated November 2023.
• Cima Universidad de Navarra (CIMA)coreImplementation/ SI/ Consulting PartnerCompleted regulatory-enabling toxicology studies for URBAGEN in 2025.
• Czech Centre for PhenogenomicscoreImplementation/ SI/ Consulting PartnerCompleted regulatory-enabling toxicology studies for URBAGEN in 2025.
• LabenacoreImplementation/ SI/ Consulting PartnerPerformed biodistribution studies for URBAGEN.
• University of Ljubljana, Faculty of Medicine (UL MF)flagshipStrategic or Co-development PartnerListed as a primary partner on the foundation website; key collaborators include Dr. Damjan Osredkar (clinical trial protocol), Nina Žakelj, and Špela Miroševič who present at ASGCT and other scientific meetings.
• Jeans for GenescoreGTM or Marketing PartnerCharitable partner promoting genetic disease awareness; logo featured on the foundation website.
• Global Advocacy AlliancecoreStrategic or Co-development PartnerListed as a partner on the foundation website; supports global advocacy for CTNNB1 syndrome.

Scale indicators7 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• CureSMA: Patient-driven non-profit foundation that funded and led the development of the first FDA-approved AAV gene therapy for spinal muscular atrophy (Zolgensma). Closely comparable model: rare pediatric neurodevelopmental disease, parent-led non-profit driving AAV gene therapy from preclinical through clinical trials via community fundraising and academic/CRO partnerships.
• Rett Syndrome Research Trust: Non-profit research foundation funding gene therapy development for Rett syndrome, another severe neurodevelopmental disorder. Highly comparable in disease profile (rare pediatric CNS disorder), funding model (community-driven donations), and therapeutic modality (AAV-based gene replacement).
• Cure Duchenne: Patient-founded non-profit funding Duchenne muscular dystrophy gene therapy research. Comparable in mission (rare pediatric genetic disease), structure (foundation-led drug development), and go-to-market (community fundraising, annual conferences, scientific conference participation).
• Parent Project Muscular Dystrophy: Largest non-profit organization focused on Duchenne muscular dystrophy, funding and accelerating gene therapy development. Comparable as a rare disease parent-driven foundation that runs its own research programs, annual conferences, and patient registries analogous to CTNNB1 Foundation's Dragonfly NHS.
• Cure Sanfilippo Foundation: Parent-led non-profit driving AAV gene therapy development for Sanfilippo syndrome (MPS III), an ultra-rare pediatric neurodevelopmental disorder. Highly analogous in disease profile (ultra-rare CNS), therapeutic modality (AAV gene replacement), and foundation-led clinical translation model.
• AADC Research Trust: Rare disease foundation that funded and supported development of an AAV gene therapy for AADC deficiency. Closely comparable ultra-rare pediatric CNS disorder, foundation-led clinical translation, and AAV delivery modality.
• Batten Disease Support & Research Association: Patient advocacy foundation funding gene therapy and other treatments for Batten disease, an ultra-rare pediatric neurodevelopmental disorder. Comparable in disease profile, foundation-led translational research model, and community-driven funding approach.

Emerging players

• Friedreich's Ataxia Research Alliance (FARA): Non-profit research foundation funding gene therapy and other treatments for Friedreich's ataxia. Comparable as a rare pediatric neurogenetic disease foundation driving translational research, but FA is slightly less ultra-rare than CTNNB1 syndrome.
• Passage Bio: Clinical-stage gene therapy biotech developing AAV-based treatments for rare pediatric CNS disorders. Comparable as a peer AAV-CNS gene therapy developer, though operating commercially rather than as a non-profit foundation; represents a potential commercial partner or licensee for the URBAGEN program.

Broad incumbents

• REGENXBIO: Clinical-stage gene therapy biotech developing AAV-based therapies for multiple rare diseases, including CNS indications. Relevant as an established AAV platform developer with manufacturing infrastructure and a broader pipeline; not a direct competitor but an obvious potential partner or acquirer for CTNNB1 Foundation's URBAGEN program.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat5 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xtwitter.com/MirosevicSpela
• YouTubeyoutube.com/channel/UCs_2vyHvIBV071uBtWpgb8g/featured
• Facebookfacebook.com/ctnnb1foundation.org
• Instagraminstagram.com/walking_with_urban
• LinkedInlinkedin.com/company/ctnnb1-foundation

Compliance1 record

Revenue estimate

Valuation estimate

Number of profiles

Profiles5 records

Subsidiaries1 record

Funding overview

Funding rounds1 record

Investors

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M&A

Investments

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Frequently asked questions about CTNNB1 Foundation