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