Why Gene Therapy forFriedreich’s Ataxia (FA)?
Currently, there is no cure for FA. However, gene therapy offers a revolutionary approach by delivering a functional copy of the FXN gene directly into affected cells, potentially stopping or even reversing the disease’s progression. Gene therapy has already transformed the treatment of several rare diseases. FA is a strong candidate for this breakthrough.
We’re deeply grateful to everyone who has helped us get this far!
Our gene therapy progress (2025)
- Optimized the FXN gene
- Built DNA plasmids
- Bacterial DNA production
- Verification & quality control
- First successful cloning
- Expert collaboration
- AAV-vector manufacturing
- Next step
Next Steps in the Program
Pre-Clinical Phase (in progress → Proof of Concept)
- Complete in-vivo mouse studies for safety and efficacy.
- Analyze frataxin levels, cell function, and symptom improvement.
- Achieve Proof of Concept to show the treatment works in an animal model.
Preparing for Clinical Trials (IND Preparation)
- Arrange a pre-IND meeting with the FDA
- Manufacture larger GMP-grade batches of the AAV vector.
- Conduct required safety and toxicology studies in animals (FDA/EMA standards).
- Compile data for regulatory submissions.
- File IND (FDA) and CTA (EMA) applications to begin human trials.
Human Clinical Trials
- Phase I/IIa: small patient groups, focusing on safety and early effects.
- Phase IIb/III: larger cohorts to confirm efficacy, determine dosing, and assess long-term safety.
Approval & Patient Access
- Submit marketing applications to the FDA and EMA
- Make the treatment available for children and adults with FA.
Support the research
Your support makes the gene-therapy program possible and gives hope to families everywhere.
Every donation goes directly to research.
Donate now