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Over the past year, an incredible amount of work has taken place behind the scenes in our gene therapy program for FA at UT Southwestern Medical Center.

Today, our researchers are evaluating two therapeutic DNA constructs using our first-generation FYHR vector.

Multiple groups of mice have been treated, behavioural testing is ongoing, and tissues from the brain, heart and other organs are being analysed to evaluate efficacy and safety.

The goal is to select a lead DNA construct for the next stage of development.

At the same time, we are already preparing what comes next.

In parallel with the ongoing FYHR studies, the team has begun generating a human transferrin receptor (hTfR1) FA mouse model to prepare for evaluation of our next-generation CapX vector.

By starting this work now, we hope to avoid unnecessary delays.

Ultimately, the data will help guide an important decision:

Continue with FYHR – or advance the selected DNA construct using next-generation CapX.

From research to patients

Taking a gene therapy from preclinical research toward patients requires more than promising science. It requires capital, expertise, manufacturing, regulatory strategy, clinical development, strategic partnerships – and people willing to open the right doors.

We are building this program with the intention of taking it all the way.

As the science progresses, I would be very interested in connecting with people and organizations across gene therapy, biotech, rare disease, investment, foundations and clinical development who may be able to contribute to what comes next.

Whether through funding, expertise, partnerships or introductions, there are many ways to become part of this journey.

Our objective remains simple:

Turn rigorous science into a safe and effective treatment for people living with FA – and bring that science closer to the children and adults waiting for it.

If this program speaks to you, I would be very happy to connect.

Thank you to Dr. Butler, Dr. Gray, Dr Napierala and the entire team at UT Southwestern Medical Center, and to every family, donor and supporter who has helped make this possible.

Let’s take it all the way.

Zardasht Rad
Founder, cure FA children

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.

This video shows part of the AAV virus production workflow in the lab, where the virus is generated and prepared for downstream processing. This is the platform used to deliver the functional FXN gene to cells.

Thank you to everyone who has stood by us in this fight – you are our heroes. You’ve carried us this far. Every single contribution has made a difference. You’ve helped us fight for a treatment not only for Tom & Lev – but for all children with Friedreich’s Ataxia. You’ve reached out your hand and shown what humanity truly means. Because this isn’t just a medical issue. It’s a humanitarian one.

Thanks to you, we’ve raised SEK 2.5 million – through GoFundMe, Swish, and cureFAchildren.com.
We now move forward with cell testing and AAV production. But the fight continues. The next phase is safety & efficacy studies in mice. That’s why we’re raising the GoFundMe goal.

Please share and help us show what we can do – together.

With love, hope, and deep gratitude,
Zardasht, Helena, Tom & Lev ❤️‍