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
FDA Accepts Application for CIRM-Funded Program Aimed to Treat Rare Pediatric Disease
The California Institute for Regenerative Medicine (CIRM) congratulates Rocket Pharmaceuticals, Inc, a leading late-stage biotechnology company. The company received U.S. FDA acceptance of the Biologics License Application (BLA) and Priority Review for RP-L201 (marnetegragene autotemcel). CIRM helped support the development of this entiviral vector (LV)-based gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I). What BLA … Continue reading FDA Accepts Application for CIRM-Funded Program Aimed to Treat Rare Pediatric Disease
Why diversity in clinical trials is essential
The California Institute for Regenerative Medicine (CIRM) is proud to join 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 … Continue reading Why diversity in clinical trials is essential
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.
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 … Continue reading A timeless message about stem cells
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
How stem cells helped Veronica fight retinitis pigmentosa and regain her vision
Veronica and Elliott Growing up Veronica McDougall thought everyone saw the world the way she did; blurry, slightly out-of-focus and with tunnel vision. As she got older her sight got worse and even the strongest prescription glasses didn’t help. When she was 15 her brother tried teaching her to drive. One night she got into … Continue reading How stem cells helped Veronica fight retinitis pigmentosa and regain her vision
Creating a ‘bespoke’ approach to rare diseases
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Up until recently the word “bespoke” meant just one thing to me, a hand-made suit, customized and fitted to you. There’s a street in London, Saville Row, that specializes in these suits. They’re gorgeous. They’re also very expensive and so I thought I’d never have a … Continue reading Creating a ‘bespoke’ approach to rare diseases