Gene Therapy Initiative Accelerated with Vertex Acquisition of Exonics Therapeutics!

In 2003 when my son Hawken was diagnosed with Duchenne people were not contemplating a cure. To find a pharmaceutical company working on Duchenne was like finding a needle in the haystack. Only sixteen years later and big pharma and biotech are now competing to be leaders in the Duchenne space. Clinical trials for Duchenne are happening all over the world. And our Duchenne patients are living longer! Each day, we have small successes, delivering more hope to boys and families.

But our work is not done. This is just the beginning of CRISPR. Dr. Olson’s work is promising, but we have to continue funding innovative science and more technologies that will treat all patients, no matter of their mutation, age or ambulatory status. With the ongoing gifts from supporters we will continue to evaluate early stage research that will be impactful and complementary to gene editing and gene therapy, as well as the next generation of gene therapy research. It is our hope and dream that these initiatives will be disease modifying and lead to a cure as well as treat the many side effects of this disease. We have never been more hopeful that we truly can cure Duchenne.

Lastly, are proud to be the first and only Duchenne advocacy organization to believe in Dr. Olson’s work at Exonics and we will continue to raise funds to support the scientists, innovators and mavericks who have the ability to develop the treatments to cure Duchenne.

We thank all you who have supported us and believed in us. We look forward to keeping you informed of our next investments! Read both press releases below for details.

With gratitude — Debra

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