CTNNB1 Foundation
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.
- Company typePrivate
- Founded2017
- HeadquartersLjubljana, Slovenia
- Headcount1–10
- GTM typeB2B and B2C
- OfferingServices
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.
CTNNB1 Foundation firmographics
Firmographics- 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
CTNNB1 Foundation industry classification
Industry- 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
Where CTNNB1 Foundation is headquartered
LocationHeadquarters
- HQ city
- Ljubljana
- HQ country
- Slovenia
- HQ region
- Europe
Offices1 record
Markets served
CTNNB1 Foundation business model
Business model- GTM type
- B2B and B2C
- Offering type
- Services
- Cost components
- Technology or R&D, Operations, Personnel, Marketing or Sales, Others
Revenue model
- 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.
- 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.
- 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.
- 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
| Model | Billing | Price |
|---|---|---|
| Other | Pay-as-you-go | Donation-based funding model with multiple channels (individual, corporate, peer-to-peer) |
Go-to-market motion3 records
Distribution channels4 records
Marketing channels8 records
CTNNB1 Foundation product offering
Product offeringCore 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
Companies that use CTNNB1 Foundation
Customer profileNamed customers1 record
Segments4 records
Ideal customer profiles4 records
CTNNB1 Foundation technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
CTNNB1 Foundation partnerships and signals
Strategic signalPartnerships
Eleven partnerships are on record, tiered flagship and core.
- Children's Medical Research InstituteflagshipResearch 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 LaboratorycorePerformed preclinical testing for URBAGEN as one of the world-renowned laboratories supporting the gene therapy program.
- Charles RivercorePerformed preclinical testing for URBAGEN.
- National Institute of Chemistry Ljubljana (NIC)corePerformed preclinical testing for URBAGEN and is listed as a partner on the foundation website.
- ViralgenflagshipManufacturing partner; produced the clinical-grade AAV vector for URBAGEN. Manufacturing process initiated November 2023.
- Cima Universidad de Navarra (CIMA)coreCompleted regulatory-enabling toxicology studies for URBAGEN in 2025.
- Czech Centre for PhenogenomicscoreCompleted regulatory-enabling toxicology studies for URBAGEN in 2025.
- LabenacorePerformed biodistribution studies for URBAGEN.
- University of Ljubljana, Faculty of Medicine (UL MF)flagshipListed 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 GenescoreCharitable partner promoting genetic disease awareness; logo featured on the foundation website.
- Global Advocacy AlliancecoreListed as a partner on the foundation website; supports global advocacy for CTNNB1 syndrome.
Scale indicators7 records
Recent moves6 records
Expansion highlights6 records
CTNNB1 Foundation competitors and assessment
Company assessmentDirect 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
CTNNB1 Foundation social profiles
Digital presenceCTNNB1 Foundation compliance and trust
Trust signalCompliance1 record
CTNNB1 Foundation financial estimates
Financial estimateRevenue estimate
Valuation estimate
CTNNB1 Foundation leadership team
Management profileNumber of profiles
Profiles5 records
CTNNB1 Foundation subsidiaries and ownership
Company hierarchySubsidiaries1 record
CTNNB1 Foundation funding detail
Funding detailFunding overview
Funding rounds1 record
Investors
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
CTNNB1 Foundation 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 CTNNB1 Foundation
What does CTNNB1 Foundation do?
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.
Is CTNNB1 Foundation a public or private company?
CTNNB1 Foundation is a private company. It is classified as nonprofit foundation owned and is currently operating.
When was CTNNB1 Foundation founded?
CTNNB1 Foundation was founded in 2017. It employs 1 to 10 people.
Where is CTNNB1 Foundation based?
CTNNB1 Foundation is headquartered in Ljubljana, Slovenia, in the Europe region.
How does CTNNB1 Foundation make money?
Four revenue lines are on record. One-time and recurring donations are the primary driver. The others are government and institutional grants, corporate donations and sponsorships and community peer-to-peer fundraising campaigns.
Who are CTNNB1 Foundation's main competitors?
Direct peers on record are CureSMA, Rett Syndrome Research Trust, Cure Duchenne, Parent Project Muscular Dystrophy, Cure Sanfilippo Foundation, AADC Research Trust and Batten Disease Support & Research Association. Emerging players are Friedreich's Ataxia Research Alliance (FARA) and Passage Bio. REGENXBIO is listed as a broad incumbent.
Does CTNNB1 Foundation have an API?
No public API is recorded for CTNNB1 Foundation.
What industry is CTNNB1 Foundation in?
CTNNB1 Foundation's product category is Rare Disease Gene Therapy Development. Its primary akta.pro industry code is HLAAACAH, Neurology/CNS Gene Therapies, with a secondary code of BPAGACAA, Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS). Its NAICS code is 541714.