Today, February 28th, is Rare Disease Day. It’s a day to remind ourselves of the millions of people, and their families, struggling with these diseases. These conditions are also called or orphan diseases because, in many cases, drug companies were not interested in adopting them to develop treatments. At the California Institute for Regenerative Medicine (CIRM), we have no such reservations. In fact last Friday our governing Board voted to invest almost $12 million to support a clinical trial for IPEX syndrome.

Dr. Bacchetta has received approval from the Food and Drug Administration (FDA) to test this approach in a Phase 1 clinical trial for patients suffering with IPEX syndrome.

Two young brothers beat the odds against successful stem cell transplants to halt the symptoms of a rare genetic disease called IPEX syndrome.

Learn about new cell, gene, and regenerative medicine discoveries, pre-clinical development and clinical trials of curative medicines and challenges in bringing these cures to market at this one-day conference for clinicians, basic scientists, patient advocates, translational investigators and biotech and pharmaceutical experts.

Brian Lookofsky (left) and his son Taylor Lookofsky (right) at the CIRM Board meeting on October 31, 2019. Taylor is living with IPEX syndrome. Last week we shared a powerful story of patient advoc…