Stem Cell Agency Board Approves Funding for Rare Immune Disorder

THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Taylor Lookofsky (center), a person with IPEX syndrome, with his father Brian and Dr. Rosa Bacchetta IPEX syndrome is a rare condition where the body can’t control or restrain an immune response, so the person’s immune cells attack their own healthy tissue. The syndrome mostly affects … Continue reading Stem Cell Agency Board Approves Funding for Rare Immune Disorder

Looking back and looking forward: good news for two CIRM-supported studies

https://open.spotify.com/episode/4IimCtQ8STpFikwZRXqSAB Dr. Rosa Bacchetta on the right with Brian Lookofsky (left) and Taylor Lookofsky after CIRM funded Dr. Bacchetta's work in October 2019. Taylor has IPEX syndrome It’s always lovely to end the week on a bright note and that’s certainly the case this week, thanks to some encouraging news about CIRM-funded research targeting blood … Continue reading Looking back and looking forward: good news for two CIRM-supported studies

CIRM-funded treatment gets orphan drug and rare pediatric disease designations from FDA

From left to right: Brian Lookofsky , Taylor Lookofsky, and Rosa Bacchetta, M.D. Picture taken October 2019 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. This is a rare disease caused by mutations in the FOXP3 gene, which leaves people … Continue reading CIRM-funded treatment gets orphan drug and rare pediatric disease designations from FDA

What is IPEX syndrome? A deeper dive into a CIRM funded award

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 advocate Taylor Lookofsky, a young man with IPEX syndrome. In his speech, he talked about the impact the condition has had on … Continue reading What is IPEX syndrome? A deeper dive into a CIRM funded award

The challenges of living with IPEX

Last week the CIRM Board awarded $5.53 million to Dr. Rosa Bacchetta at Stanford to complete the work necessary to conduct a clinical trial for IPEX syndrome. This is a rare disease caused by mutations in the FOXP3 gene which leaves people with the condition vulnerable to immune system attacks on their organs and tissues. … Continue reading The challenges of living with IPEX

Stem Cell Agency Approves Funding for Clinical Trials Targeting Parkinson’s Disease and Blindness

The governing Board of the California Institute for Regenerative Medicine (CIRM) yesterday invested $32.92 million to fund the Stem Cell Agency’s first clinical trial in Parkinson’s disease (PD), and to support three clinical trials targeting different forms of vision loss. This brings the total number of clinical trials funded by CIRM to 60. The PD … Continue reading Stem Cell Agency Approves Funding for Clinical Trials Targeting Parkinson’s Disease and Blindness