CTNNB1 Foundation

Who are we fighting for?

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.

CONFERENCE 2024

Between 14 and 16 June, 83 of our CTNNB1 syndrome children travelled to Slovenia to attend our Second International CTNNB1 syndrome conference.

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.

Download Product Overview

Download ASGCT 2024 Poster

Thanks to our incredible donors, we have raised 2.5 million euros in the last four years. We are just a few months away from initiating our first CTNNB1 community clinical trial with the gene replacement therapy product URBAGEN.

Download Operating (2021-2024) & Forecasted Budget (2024-2025)

Based on the CTNNB1 Cost Timeline plan for the development of URBAGEN, we have a funding gap of EUR 1.2 million. This will support toxicology manufacturing, regulatory support, toxicology, GMP manufacturing and clinical trial preparation.