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
clinical trials
Neurona Doses First Patient in CIRM-Funded Bilateral Epilepsy Cell Therapy Trial
Image Credit: Canva Stock Photo Neurona Therapeutics has reached a major milestone in the development of its stem cell approach to treating epilepsy. A new phase in clinical trial The company recently announced that the first patient has been dosed in a Phase 1/2 clinical trial evaluating NRTX-1001, a cell therapy for adults whose epilepsy … Continue reading Neurona Doses First Patient in CIRM-Funded Bilateral Epilepsy Cell Therapy Trial
Gene Therapy for Osteoarthritis Shows Positive Results in Early Trial
Image Credit: Shutterstock Here’s a shocking statistic: in 2020, 7.6% of the world’s population was living with the most common form of arthritis – osteoarthritis (OA). That amounts to 595 million people globally and 32.5 million adults in the U.S. Still no cure That’s a lot of people with painful knees, hips, and other joints. … Continue reading Gene Therapy for Osteoarthritis Shows Positive Results in Early Trial
New gene therapy for early-onset Parkinson’s
Image source: Shutterstock Nearly one million people in the U.S. are living with Parkinson’s disease, which causes progressive symptoms including tremors, stiffness, and gait and balance issues. Although there are medications to treat symptoms, the disease has no cure. CIRM's support for potential Parkinson's treatments To date, the California Institute for Regenerative Medicine (CIRM) has … Continue reading New gene therapy for early-onset Parkinson’s
A patient’s journey with an epilepsy stem cell therapy
Justin Graves diving by the Grand Cayman Islands In 2007, Justin Graves was working at a SCUBA shop in Louisville, Kentucky, when he had his first epileptic seizure. "SCUBA diving has just been something I've loved my whole life," he said. "My whole family loves it." Medication not always effective Justin had to give up … Continue reading A patient’s journey with an epilepsy stem cell therapy
Breaking barriers in rare disease: A gene therapy transforms cystinosis treatment
A 12 year-old Kurt Gillenberg. Photo courtesy of Kurt Gillenberg. When Kurt Gillenberg was 10 months old, his parents knew something wasn’t right. But it wasn’t until he reached 18 months that they found a doctor at the University of California, San Diego, who diagnosed Kurt with cystinosis – a rare genetic, metabolic defect that … Continue reading Breaking barriers in rare disease: A gene therapy transforms cystinosis treatment
Advancing Heart Health: CIRM Funds Tenaya Therapeutics’ Gene Therapy Trial for ARVC
Histology of human cardiac muscle under microscope. Credit: Shutterstock The California Institute for Regenerative Medicine (CIRM) recently announced funding a series of projects spanning the earliest stages of research through to clinical trials. A rare heart condition One of those projects was an $8 million award to Matthew Pollman, MD, MS, Senior Vice President of … Continue reading Advancing Heart Health: CIRM Funds Tenaya Therapeutics’ Gene Therapy Trial for ARVC
Luxa Biotechnology’s CIRM-funded Dry AMD Therapy Milestone
Credit: Shutterstock The U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to a promising new treatment for dry age-related macular degeneration (AMD)—a leading cause of vision loss. The therapy is being developed by Luxa Biotechnology and supported in part by the California Institute for Regenerative Medicine (CIRM). What RMAT Designation Means RMAT designation … Continue reading Luxa Biotechnology’s CIRM-funded Dry AMD Therapy Milestone
Potential Gene Therapy for Children with ADA-SCID
Photo Courtesy of Andrea Fernandez de Soto Imagine stepping outside and facing life‑threatening danger. For children born with ADA‑SCID (adenosine deaminase severe combined immunodeficiency), this is daily life. Their immune systems are so weak that a cold or a scraped knee can cause a dangerous infection. Donald Kohn, MD, a leading researcher at the UCLA … Continue reading Potential Gene Therapy for Children with ADA-SCID
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