Knee joint pain rarely gets counted among major public‑health burdens. Yet it affects more than 3 million Californians and 34 million people across the U.S., with both massive economic and health consequences. Costly Impact In California alone, the cost of knee replacement surgeries exceeds $8 billion annually, according to Thomas Chalberg, PhD, founder and CEO … Continue reading Genascence discusses gene therapy for knee osteoarthritis
Only three years ago, the FDA approved the first gene therapies for sickle cell disease (Lyfgenia and Casvegy), historic breakthroughs decades in the making. Yet on World Sickle Cell Awareness Day this year, the reality is that high cost, limited availability, and complex logistics keep these treatments out of reach for most people who need them. Yet these breakthroughs offer tremendous hope. For many years, the … Continue reading Progress on Sickle Cell Disease Treatments
To speed promising new stem cell or gene therapies to patients who need them, the California Institute for Regenerative Medicine (CIRM) supports work at every phase of clinical trials, from early discovery research through clinical testing. CIRM currently supports more than 100 clinical trials in California, conditions that range from rare diseases to cancers and … Continue reading Understanding the Phases of Clinical Trials: How Success Builds at Every Step
Image Credit: Canva - studioroman Often, after decades of intensive scientific research, even the most promising potential new therapies come down to a critical final phase: courageous people stepping forward to participate in a clinical trial. For Clinical Trials Day this year, we're celebrating a few of the many patients who inspire us, while highlighting … Continue reading What We Wish People Knew About Clinical Trials
written by Holly Alyssa MacCormick The California Institute for Regenerative Medicine (CIRM) helped fund a clinical trial that involved an experimental stem cell treatment for a frequently paralyzing form of spina bifida. Today, several of those babies are now able to walk and run unassisted.Babies with severe spina bifida are often treated with surgery after … Continue reading Closer to Cures: Diana Farmer discusses clinical trials for spina bifida
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
written by Holly Alyssa MacCormick Image Credit: The Langenhop Family Alicia Langenhop was seven months pregnant with her third child when she and her husband, Jon, learned that their two toddlers had a life-threatening immune disease called leukocyte adhesion deficiency-1 (LAD-1). One in a million LAD-1 is extremely rare—about one in a million births—but because … Continue reading CIRM funded clinical trial gives siblings a second chance at life
Written by Holly Alyssa MacCormick In 2023, the California Institute for Regenerative Medicine (CIRM) funded a clinical trial led by Stanford researcher Crystal Mackall, MD, to investigate a possible therapy for rare brain and spinal tumors. Devastating cancers These cancers, that arise from a specific genetic mutation, known as H3K27M, are devastating. Each year, about … Continue reading Stanford researcher Crystal Mackall discusses progress on rare cancers clinical trials
Written by Holly Alyssa MacCormick Cory R. Nicholas presenting at the January 29 ICOC board meeting About 3 million adults in the United States have epilepsy, including roughly 400,000 in California, according to the CDC. About one-third to one-half continue to have seizures that do not respond to available medications. Hope for those with epilepsy … Continue reading Cory R. Nicholas shares update on epilepsy clinical trials
Written by Holly Alyssa MacCormick South San Francisco, CA, January 30, 2026 – As many as 1 in 10 people in the U.S. has a rare disease, many of which have no known cure. A new program by the California Institute for Regenerative Medicine (CIRM) could change this by funding novel ways to develop multiple … Continue reading CIRM board supports a rare disease funding plan, administrative changes