California Institute for Regenerative Medicine (CIRM)

For 20 years, CIRM has been at the forefront of regenerative medicine—funding critical research, building infrastructure, and championing patient access to clinical trials. Our 2024 annual report, "Our Journey Forward: How CIRM Catalyzes Regenerative Medicine Today and Beyond," showcases the stories, research, and partnerships that drive our mission. We invite you to explore our annual report here: https://lnkd.in/gbE2M5yJ

When Kurt Gillenberg was diagnosed with cystinosis as a baby, his parents were told that although the condition could be treated, it would ultimately shorten his life. Now, at 32 years old, Kurt has undergone multiple surgeries, kidney transplants, and takes daily medications to manage his condition. However, thanks to a clinical trial led by Stephanie Cherqui, PhD, and funded by CIRM at UC San Diego Health, Kurt's symptoms have been reduced, and his strength has increased. This innovative trial utilizes stem cells and gene therapy to correct the genetic mutation responsible for cystinosis. For Kurt, participating meant more than just a potential lasting treatment—it was also about paving the way for future patients.

CIRM is committed to driving innovative research that supports treating rare diseases. We recently awarded $8 million to Tenaya Therapeutics to advance a clinical trial for a potential gene therapy targeting Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC), a life-threatening heart condition with no current treatment. ARVC affects ~70,000 people in the US, often leading to sudden cardiac death or heart failure. This trial, led by Matthew Pollman,?MD,?MS,?is testing a gene therapy designed to replace the mutated PKP2 gene with a healthy copy—potentially restoring heart function and altering the disease’s course.

Envision a future where treating cystic fibrosis is as easy as taking a deep breath. That’s what a team of pediatric physician-scientists at the UCLA Broad Stem Cell Research Center is working to achieve. Promising preliminary results have since helped the team secure additional support from CIRM, the Cystic Fibrosis Research Institute, and the Cystic Fibrosis Foundation, advancing the project toward preclinical testing. #PreclinicalResearch #RegenerativeMedicine #CysticFibrosis #Nanotech #Biotech #GeneEditing

CIRM awarded $4.6 million to UC Irvine neuroscientist Michael Demetriou, MD, PhD, to support research on a novel CAR T cell therapy designed to target all major cancer types, including solid tumors such as breast, prostate, pancreatic, and ovarian cancer. While CAR T cell therapies have shown significant success in treating certain blood cancers, their application to solid tumors has been limited due to toxicity risks. Dr. Demetriou’s approach, GlyTR CAR T cells, addresses this challenge by targeting abnormal glycans in cancer cells. This technology can potentially improve treatment precision, reduce toxicity, and expand CAR T therapy to a broader range of patients. #CancerResearch #CARTcells #UCIrvine #RegenerativeMedicine

The FDA has granted RMAT (Regenerative Medicine Advanced Therapy) designation to Luxa Biotechnology LLC for its investigational cell therapy targeting dry age-related macular degeneration (AMD). This condition is a leading cause of vision loss, affecting nearly 20 million people in the United States. The RMAT designation is intended to expedite the development of promising regenerative treatments. Supported in part by a $4 million CIRM grant, Luxa’s therapy transplants lab-grown retinal cells to restore vision. This is the first CIRM-funded dry AMD trial to receive RMAT status, highlighting the potential of cell therapies for vision loss. #DryAMD #StemCellTherapy #RMATdesignation #VisionLoss #RegenerativeMedicine #CIRM

Neurona Therapeutics is advancing its cell therapy, NRTX-1001, into a Phase 3 clinical trial for drug-resistant epilepsy—a major milestone for patients with mesial temporal lobe epilepsy (MTLE), the most common form of epilepsy in adults. This potential one-time treatment aims to provide an alternative to existing surgical interventions. With positive early clinical data and an RMAT designation from the FDA, NRTX-1001 has the potential to offer patients with MTLE new treatment options. CIRM supported the Phase 1/2 unilateral MTLE clinical trial with an $8 million grant and has supported Neurona's research from the initial discovery research stage to the ongoing first-in-human clinical trial.

Stuart Lipton, PhD, and his team, is investigating how environmental pollutants contribute to autism spectrum disorder (ASD) and intellectual disability (ID) by inducing redox stress. Using advanced multiomics techniques, researchers aim to uncover treatable targets and develop new therapeutics for these conditions. #ReMIND #NeuroResearch #RegenerativeMedicine #DiscoveryResearch #AutismSpectrumDisorder #Environment #CIRM Scripps Research

Manufacturing challenges continue to pose significant obstacles in delivering cell and gene therapies (CGT) to patients. That's why CIRM held its inaugural Manufacturing Network Symposium, bringing together over 100 experts—including researchers, academic leaders, and industry partners—to advance solutions. With an $18 million investment in the California CGT Manufacturing Network, CIRM is committed to eliminating bottlenecks and accelerating life-changing treatments. #CellTherapy #GeneTherapy #CGTManufacturing #RegenerativeMedicine #CIRM

We are deeply saddened by the passing of John Zaia, MD, a visionary virologist, gene therapy pioneer, and esteemed leader at City of Hope. As director of the CIRM-funded Alpha Stem Cell Clinic, Dr. Zaia dedicated over four decades to advancing regenerative medicine, leaving an indelible impact on HIV research,?COVID response,?pediatric diseases, and cell and gene therapy. Dr. Zaia's groundbreaking work played a key role in one of the world's rare cases of HIV remission following a stem cell transplant. His leadership also helped to shape the future of precision medicine. In addition to his scientific accomplishments, Dr. Zaia was recognized for his wisdom, kindness, and steadfast commitment to patients. His legacy will continue to inspire the field for generations to come. Read more about Dr. Zaia's contributions in this touching tribute that City of Hope shared: https://lnkd.in/gySv4Wcc And read CIRM's tribute to Dr. Zaia: https://lnkd.in/gxxB3pMd Photo courtesy of City of Hope.