We have now taken one of the most important steps so far in our gene therapy program for Friedreich’s ataxia. The first mice have been dosed, and our in vivo studies have officially begun.

This marks the transition from design and preparation to true biological testing in a living system. It allows us to study how the therapy behaves in vivo, how the frataxin gene is expressed in relevant tissues, and to begin assessing safety and biological effect in a controlled preclinical setting.

At the same time, we know that this is only the beginning. In vivo studies take time, and it will be several months before reliable and complete results can be generated. As the program progresses, we will share updates and insights whenever possible.

This is a critical step on the path toward Proof of Concept. The next major phase will be the planned toxicology studies later this spring, an essential requirement to advance the therapy toward clinical trials. Preparations for this stage are already underway, although full funding has not yet been secured. We therefore continue to gather support and resources to ensure we can move forward without delay.

We move ahead step by step, with humility, determination, and one single focus: the children.
For every child waiting for a chance at life.
This is why we do this.