Last year, CIRM awarded $5.53 million to Rosa Bacchetta, M.D. at Stanford University to complete the work necessary to conduct a clinical trial for IPEX syndrome, a rare pediatric disease.

This rare disease is caused by mutations in the FOXP3 gene, leaving people with the condition vulnerable to immune system attacks on their organs and tissues. These attacks can be devastating, even fatal.

Orphan Drug

CIRM President and CEO Dr. Maria Millan said this therapy meets a real need. “CIRM supported this project from an early stage, showing our commitment to moving promising research into people. Lymphoma is the most common blood cancer and the sixth most diagnosed cancer in California. Many patients still face serious complications from chemotherapy, so treatments that reduce those complications can improve quality of life and, we hope, long‑term outcomes.”

In a news release, Dr. Paul Finnegan, Angiocrine’s CEO, welcomed the RMAT designation. “It reflects the promising efficacy and safety data from our Phase 1b/2 study. This is an important step toward the first approval of AB‑205. We appreciate the FDA’s assessment and the support from CIRM.”

This investment marked CIRM’s 50th funded clinical trial—a milestone worth celebrating. The company hopes to begin a Phase 3 trial in the U.S. and Europe next year.

You can access the full transcript of his talk in a previous blog post.