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
Rare Diseases
Turning Hope into Reality: How a Genetic Therapy Helped Connor
Photo credit: n-Lorem Connor Dalby is a 15-year-old teenager who loves music and the beach, but his journey has been far from ordinary. Diagnosed with a rare mutation in the SCN2A gene, his life has been filled with challenges. From the time he was just a few weeks old, he had trouble sleeping and struggled … Continue reading Turning Hope into Reality: How a Genetic Therapy Helped Connor
Bringing a community together at the 2nd Annual ALSP Conference
In addition to funding innovative programs and research that accelerate the development of stem cell and gene therapy treatments, the California Institute for Regenerative Medicine (CIRM) provides funding to support valuable mission-specific scientific conferences where people can meet to exchange scientific information to meet goals relevant to CIRM’s mission. Recently, CIRM supported the 2nd Annual … Continue reading Bringing a community together at the 2nd Annual ALSP Conference
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
Making transplants easier for kids, and charting a new approach to fighting solid tumors.
Every year California performs around 100 kidney transplants in children but, on average, around 50 of these patients will have their body reject the transplant. These children then have to undergo regular dialysis while waiting for a new organ. Even the successful transplants require a lifetime of immunosuppression medications. These medications can prevent rejection but … Continue reading Making transplants easier for kids, and charting a new approach to fighting solid tumors.
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
A better, faster, more effective way to edit genes
Clinical fellow Brian Shy talks with postdoctoral scholar Tori Yamamoto in the Marson Lab at Gladstone Institutes on June 8th, 2022. Photo courtesy Gladstone Institutes. For years scientists have been touting the potential of CRISPR, a gene editing tool that allows you to target a specific mutation and either cut it out or replace it … Continue reading A better, faster, more effective way to edit genes
The Stem Cellar 