Image Credit: Canva Stock Photo Neurona Therapeutics has reached a major milestone in the development of its stem cell approach to treating epilepsy. 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 affects both sides of the brain … Continue reading Neurona Doses First Patient in CIRM-Funded Bilateral Epilepsy Cell Therapy Trial
Clinical Trials
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. That’s a lot of people with painful knees, hips, and other joints. Although pain medication … Continue reading Gene Therapy for Osteoarthritis Shows Positive Results in Early Trial
20 Years of CIRM: The evolution of a CIRM-funded therapy for macular degeneration
Image Credit: University of Southern California (USC) Eye Institute In celebration of CIRM’s 20th anniversary, we are reflecting on some of the early projects we supported that have since advanced to clinical trials. One such trial for a form of blindness originated with a 2007 SEED grant to David Hinton, MD, of the University of … Continue reading 20 Years of CIRM: The evolution of a CIRM-funded therapy for macular degeneration
New gene therapy aims to restore function in 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. To date, the California Institute for Regenerative Medicine (CIRM) has invested more than $109 million in … Continue reading New gene therapy aims to restore function in early-onset Parkinson’s
Diving into a new era of treatment: 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." Justin had to give up diving as well as … Continue reading Diving into a new era of treatment: 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. One of those projects was an $8 million award to Matthew Pollman, MD, MS, Senior Vice President of Clinical Development at Tenaya … Continue reading Advancing Heart Health: CIRM Funds Tenaya Therapeutics’ Gene Therapy Trial for ARVC
FDA Grants RMAT Designation to Luxa Biotechnology’s CIRM-funded Dry AMD Therapy
Credit: Shutterstock The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation for a project focused on vision loss that was funded in part by the California Institute for Regenerative Medicine (CIRM). This designation is given to highly promising regenerative medicine therapies that treat serious or life-threatening diseases with the goal of accelerating the path … Continue reading FDA Grants RMAT Designation to Luxa Biotechnology’s CIRM-funded Dry AMD Therapy
Gene Therapy Brings Hope to Families Affected by Rare Immune Disorder
Photo Courtesy of Andrea Fernandez de Soto Imagine a life where every time you step outside, everything in your environment poses a life-threatening risk. For children born with ADA-SCID (adenosine deaminase severe combined immunodeficiency), this is a reality. Their immune systems are so compromised that a common cold or a simple scrape on the knee … Continue reading Gene Therapy Brings Hope to Families Affected by Rare Immune Disorder
A Step Forward in the Fight Against Advanced Kidney Cancer
Photo Courtesy: Allogene Therapeutics Metastatic renal cell carcinoma (RCC) is an advanced form of kidney cancer with few treatment options and a five-year survival rate of less than 17%. With funding support from CIRM, Allogene Therapeutics is developing ALLO-316, an allogeneic CAR-T cell therapy which targets CD70—a protein highly expressed on clear cell renal cell … Continue reading A Step Forward in the Fight Against Advanced Kidney Cancer
The Stem Cellar 