True heroes
continue to smile,
proving that courage
is their superpower.
CTNNB1 SYNDROME
CTNNB1 syndrome is a severe neurodevelopmental disorder affecting every aspect of a patient's functioning, including their ability to walk, speak and live independent.
GENE THERAPY
In November 2023, we initiated manufacturing our gene therapy product, URBAGEN, which delivers a healthy CTNNB1 gene via injection into the brain's ventricles.
Join us for the 3rd International CTNNB1 Syndrome Conference and Clinical Examination for the Gene Therapy Program.
16 - 20 Jun 2025 - Bilbao, Spain
2021 - 2024 FINANCIAL REPORT
This report provides a clear summary of what we’ve been up to in the past few years, how we managed to raise funds, what we allocated them to, and what we have planned for the future.
WHAT WE DO?
The CTNNB1 Foundation is a research-driven non-profit organization focused on developing and supporting gene-related treatments with the potential to benefit patients with CTNNB1 syndrome.
CTNNB1 syndrome will not cure itself. Our team has been working tirelessly to develop a gene replacement therapy and make it available to our young patients as soon as possible.
In 2021, we signed a research agreement with the Children’s Medical Research Institute to start developing different constructs of the gene replacement therapy. Initial preclinical testing was performed with world-renowned laboratories, including The Jackson Laboratory, Charles River, and the National Institute of Chemistry Ljubljana. In November 2023, we initiated the manufacturing process with our amazing partner Viralgen, which will produce the product for the clinical trial expected to occur in 2025.
Over the years, the CTNNB1 Foundation significantly increased in income, all together more than 4 million euros have been raised or received through many fundraising campaigns and funding opportunities. However, expenses have also risen dramatically, particularly in 2024, due to manufacturing and preclinical testing costs.
Our five largest contributors (2021–2024) have provided 86% of the total funding that is critical to advancing our gene therapy program towards clinical trials.
The “Walking with Urban” campaigns raised €1,462,531 through many Slovenian campaigns, funding mainly basic and proof-of-concept gene therapy studies. The Slovenian government contributed €1,000,000 in late 2024, to enable clinical trial development in Slovenia.
The Slovenian association “Palčica Pomagalčica” raised €580,000, while the founders personally donated €325,000, underlining their strong commitment. CTNNB1 Spain contributed €235,000 through targeted fundraising.
We are very grateful for all this support, which has enabled us to progress with translating gene therapy program to the clinics. However, we are not done yet. Now is more than ever important to finalize funding and bring this therapy to our children who are waiting for this therapy for years.
There are many ways you can help, please go here and consider donating or helping us fundraise to reach our first CTNNB1 clinical trial.
Help us reach this goal faster.
BECOME A CTNNB1 FOUNDATION MEMBER
As we move closer to the clinical trial – the moment we have all been waiting for – we want to ensure you stay informed and connected.
By becoming a CTNNB1 Foundation Member, you will:
- Receive updates on research and clinical trials
- Stay informed through our monthly newsletter
- Get notified about upcoming events and opportunities to participate
UPCOMING EVENTS
ASGCT, New Orleans (USA), May 13-17: https://annualmeeting.asgct.org
Oral presentation: Preclinical advances for the translation of an AAV-mediated gene therapy for CTNNB1 syndrome (Dr. Andrea Perez-Iturralde)
ESHG, Milan (Italy), May 24-27: https://2025.eshg.org
Workshop: Setting up your gene therapy clinical site: dos and don’ts! (Dr. Špela Miroševič)
Gene4Neuro, Pamplona (Spain), June 3-5: https://www.broadreach-global.com/gene4neuro/2025
Keynote speaker: When science becomes personal: a mother’s journey to gene therapy
3rd International CTNNB1 syndrome conference, Bilbao (Spain), 16-20 June: https://ctnnb1-foundation.org/conference-2025/
CTNNB1 Connect&Cure conference, Boston (USA), 10-12 July: https://curectnnb1.org/2025-ctnnb1-conference-program/
CTNNB1 gene therapy: update on development and clinical translation (Dr. Špela Miroševič)
OUR LATEST NEWS
Read our recent posts and stay tuned!
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