TIMELINE AND PROGRESS FOR ASO GENE THERAPY

Email from Volunteer Chairman and Founder, Brad Margus:

We’ve been overwhelmed by the generosity with which so many of you have reached out to support our efforts to treat, for the first time in history, a child with ataxia-telangiectasia (A-T) using antisense oligonucleotide gene therapy. This approach could slow or completely stop the disease in its tracks – something we’ve never dared to contemplate in the 25 years since we started the A-T Children’s Project.

This timeline shows the basic steps that we’ll be taking to execute our plan. Recently, we learned that the regulatory authorities will require us to perform additional safety studies, increasing the total cash we need for this project from $1.47 million to nearly $1.8 million. We’ve raised about one-third of this amount in donations and commitments, and so we feel comfortable moving forward, while we continue our incessant fundraising efforts.

Betting almost $1.8 million to rescue one child is admittedly a bold endeavor. And, including just one child in our trial is scary, as various things could go wrong, including factors entirely unrelated to the treatment. Therefore, if we’re able to raise the first $1.8 million soon, we’ll continue our quest for an additional $1.48 million to enable us to design, make and test a second ASO drug that targets a different mutation in the A-T gene. Doing so would enable us to treat two A-T children at different ages at the same time, greatly increasing our probability of success.

I assure you that, if we find in these proof of concept studies that this new type of therapy benefits a child with A-T in any way, we will quickly convene experts from academia and industry to figure out how we can reduce the costs sharply and expand the approach to many more A-T kids.

Your help has been vital in enabling our progress this far. If you’re aware of anyone or any organization that you think might be interested in donating, helping us to reach the finish line and perhaps even add a treatment for a second child to improve our chances, please let us know.

All the best,
Brad

Brad Margus
Volunteer Chairman and Founder

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