CIRM awards $26 million to boost clinical trials for cancer, heart, and rare diseases

The California Institute for Regenerative Medicine (CIRM) is proud to announce the allocation of $26 million to support clinical research targeting a range of cancers, heart conditions, and rare diseases. This significant funding underscores CIRM’s commitment to advancing innovative stem cell and gene therapy-based treatments through all stages of clinical trial development. This latest round … Continue reading CIRM awards $26 million to boost clinical trials for cancer, heart, and rare diseases

How CIRM-funded research is bringing a lifesaving gene therapy to a 5-year-old

For her first year of life, Seersha Sulack stayed mostly in her bedroom because something as simple as a common cold could have killed her. The five-year-old was born with ADA-SCID, a condition so rare that only eight babies a year are born with it in the United States.  Now, thanks to the work of … Continue reading How CIRM-funded research is bringing a lifesaving gene therapy to a 5-year-old

Join the movement to fight rare diseases

Tomorrow, 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 orphan diseases because, in many cases, drug companies were not interested in adopting them to develop treatments. Here at the California Institute for Regenerative Medicine (CIRM), we … Continue reading Join the movement to fight rare diseases

Update on SCID patient enrolled in CIRM-funded gene therapy trial

Photo credit: UCSF Hataalii Tiisyatonii Begay (HT) is paving the road for newborns with SCID. When HT was born in 2018 in a remote part of the Navajo nation, he was quickly diagnosed with a rare and -usually fatal- condition. Today, thanks to a therapy developed at UCSF and funded by CIRM, he’s a healthy … Continue reading Update on SCID patient enrolled in CIRM-funded gene therapy trial

Bubble baby treatment cleared to restart clinical trial

Evie Vaccaro: Photo courtesy Nancy Ramos Three families battling a life-threatening immune disorder got some great news last week. A clinical trial that could save the life of their child has once again been given the go-ahead by the US Food and Drug Administration (FDA). The clinical trial is the work of UCLA’s Dr. Don … Continue reading Bubble baby treatment cleared to restart clinical trial

Sweet 16 and counting for stem cell clinical trial

Dr. Judy Shizuru: Photo courtesy Jasper Therapeutics Over the years the California Institute for Regenerative Medicine (CIRM) has invested a lot in helping children born with severe combined immunodeficiency (SCID), a fatal immune disorder. And we have seen great results with some researchers reporting a 95 percent success rate in curing these children. Now there’s … Continue reading Sweet 16 and counting for stem cell clinical trial

Fast Track Designation for a therapy making transplants safer for children with a fatal immune disorder

Bone marrow transplant For children born with severe combined immunodeficiency (SCID) life can be very challenging. SCID means they have no functioning immune system, so even a simple infection can prove life threatening. Left untreated, children with SCID often die in the first few years of life. There are stem cell/gene therapies funded by the … Continue reading Fast Track Designation for a therapy making transplants safer for children with a fatal immune disorder

Joining the movement to fight rare diseases

THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST It’s hard to think of something as being rare when it affects up to 30 million Americans and 300 million people worldwide. But the truth is there are more than 6,000 conditions – those affecting 200,000 people or fewer – that are considered rare.   Today, … Continue reading Joining the movement to fight rare diseases

CIRM-funded stem cell clinical trial patients: Where are they now?

Ronnie with his parents Pawash Priyank and Upasana Thakur. Since its launch in 2004, the California Institute for Regenerative Medicine (CIRM) has been a leader in growing the stem cell and regenerative medicine field while keeping the needs of patients at the core of its mission.  To date, CIRM has:   Advanced stem cell research and therapy development for … Continue reading CIRM-funded stem cell clinical trial patients: Where are they now?

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