Image Credit: Canva stock images After 21 years (and a few months), the California Institute of Regenerative Medicine (CIRM) has hit a major milestone with its FDA approved therapy. The promise of stem cells In 2004, when California voters created CIRM to fund research with stem cells, which can divide to form many different tissues in the body. Stem cells held great potential, but scientists … Continue reading First therapy supported by CIRM approved by the FDA
FDA
New program to help patients with rare disease
Recently, the California Institute for Regenerative Medicine (CIRM) announced a new approach for how its funding can advance treatments for people living with rare diseases. Individually, few people have any single rare disease, but in aggregate, about 30 million people in the U.S. have one of the roughly 10,000 rare diseases, and 95% have no … Continue reading New program to help patients with rare disease
Two CIRM-funded research projects receive RMAT designation from FDA
The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to two research projects funded by the California Institute for Regenerative Medicine (CIRM). RMAT is a fast‑track designation that speeds the development, review, and potential approval of treatments for serious or life‑threatening diseases. The first RMAT designation went to a … Continue reading Two CIRM-funded research projects receive RMAT designation from FDA
CIRM-Funded Program For Rare Pediatric Disease
The California Institute for Regenerative Medicine (CIRM) congratulates Rocket Pharmaceuticals, Inc., a leading late‑stage biotechnology company. The company recently received U.S. FDA acceptance of the Biologics License Application (BLA) and Priority Review for RP-L201 (marnetegragene autotemcel). CIRM helped support development of this lentiviral‑vector gene therapy for severe Leukocyte Adhesion Deficiency‑I (LAD‑I). What the BLA Means … Continue reading CIRM-Funded Program For Rare Pediatric Disease
Why diversity in clinical trials is essential
The California Institute for Regenerative Medicine (CIRM) joins fellow advocates of clinical trial diversity in applauding a new law that will allow the U.S. Food & Drug Administration (FDA) to require diverse representation in clinical trials. A clinical trial, as defined by the FDA, tests potential treatments in human volunteers to see whether they should … Continue reading Why diversity in clinical trials is essential
A timeless message about stem cells
Dr. Daniel Kota The world of stem cell research is advancing rapidly, with new findings and discoveries seemingly every week. And yet some things that we knew years ago are still every bit as relevant today as they were then. Take, for example, a TEDx talk by Dr. Daniel Kota, a stem cell researcher and the Director, … Continue reading A timeless message about stem cells
Clinical trial for SCID
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. SCID therapy … Continue reading Clinical trial for SCID
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, life is extremely challenging. SCID leaves them without a functioning immune system, so even minor infections can be life‑threatening. Without treatment, many children die within the first few years of life. CIRM-funded clinical trials CIRM‑funded stem cell and gene therapy trials at UCLA and UCSF/St. … Continue reading Fast Track designation for a therapy making transplants safer for children with a fatal immune disorder
The power of the patient advocate: how a quick visit led to an $11M grant to fund a clinical trial
Members of NFOSD visiting UC Davis in 2013 At CIRM, we have the resources to fund promising research through clinical trials. These projects are expensive—often costing tens of millions of dollars. Phase 2 clinical trial But many programs that reach our Board begin years earlier, in much humbler circumstances, powered by patient advocates, grassroots fundraising, … Continue reading The power of the patient advocate: how a quick visit led to an $11M grant to fund a clinical trial
Stem cell-derived retinal patch continues to show promising results two years post-implantation
Earlier this year, we wrote about the promising results of a phase 1 clinical trial aimed at replacing deteriorating retinal cells. These would be used for people suffering from age-related macular degeneration- one of the leading causes of blindness worldwide for people over 50. Now there’s even more good news! Highlighted in a news story on the … Continue reading Stem cell-derived retinal patch continues to show promising results two years post-implantation