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

Diana Farmer, MD, of UC Davis Health, is transforming the future of fetal surgery and regenerative medicine. This week, she received the prestigious Edward H. Ahrens, Jr. Award for Outstanding Achievement in Patient-Oriented Research from the Association for Clinical and Translational Science. The award recognizes her first-of-its-kind work combining fetal surgery with stem cells to treat spina bifida and her leadership in translating research from the lab to the clinic. As one of the world’s first fetal surgeons, Dr. Farmer pioneered techniques to treat birth defects before birth. Her groundbreaking CuRe Trial, supported by CIRM, is the first to use stem cells to treat spina bifida in utero. Four years into the trial, early results are promising, with treated children reaching key developmental milestones. Dr. Farmer envisions a future where regenerative medicine can address more birth defects and chronic diseases, changing lives before they even begin. https://lnkd.in/g3-Rp74j

CIRM’s Director of Discovery and Education, Kelly Shepard, PhD, was proud to present and attend the annual University of California, Merced's Training Undergraduates in Stem Cell Engineering and Biology (TUSCEB) Program Symposium. At CIRM, we’re committed to supporting the next generation of scientists. Through programs like COMPASS, we create hands-on research and mentorship opportunities that prepare a diverse group of undergraduates for impactful careers in regenerative medicine.

CIRM is excited to announce a new funding opportunity called the DISC4 Awards! DISC4 is a new funding opportunity designed to support collaborative research teams in California. This program aims to facilitate cross-disciplinary groups of researchers who are working to discover new disease mechanisms, expand research across all affected populations, and identify promising therapeutic targets, strategies, or biomarkers. Explore our website to learn more and sign up for email updates on funding opportunities at https://lnkd.in/gt-Q7uua.

For the past 20 years, CIRM has been vital in advancing scientific discovery by funding basic research that might otherwise lack financial support. This mission remains crucial today, particularly as federal funding for biomedical research faces significant challenges. Recent cuts to scientific research funding and the impending expiration of the 21st Century Cures Act are leading to a decline in support for neuroscience research, including the BRAIN Initiative. CIRM is committed to maintaining its leadership in neuroscience research through initiatives like ReMIND and DISC4, which support collaborative, multidisciplinary projects to advance innovative discoveries. To date, CIRM has awarded over $1 billion to various stages of neuroscience research, effectively bridging critical funding gaps and accelerating the development of transformative therapies.

Neurona Therapeutics was recently featured in Endpoints News for its progress in developing NRTX-1001, an innovative cell therapy for drug-resistant epilepsy. With additional funding, Neurona is preparing for a Phase 3 trial—an important step toward bringing this therapy to patients. CIRM has long supported this research, funding NRTX-1001 from its early discovery phase to the ongoing first-in-human trial. Most recently, CIRM awarded Neurona $14 million to expand clinical testing for patients with drug-resistant bilateral temporal lobe epilepsy, an area with significant unmet medical needs. Current treatments for drug-resistant epilepsy often require invasive surgeries with serious risks. NRTX-1001 offers an alternative by using inhibitory neurons to restore brain circuit balance, potentially suppressing seizures without damaging healthy tissue. Early results suggest it may significantly reduce seizures while preserving cognitive function. https://lnkd.in/gpnWTxBr

For the past 20 years, CIRM has driven progress in regenerative medicine, transforming scientific potential into real-world impact. Over 50 new businesses have emerged from CIRM-funded research, and more than 110 clinical trials are advancing therapies that have the potential to change lives. When CIRM was established in 2004, the field faced major barriers: limited funding, few available stem cell lines, and a lack of infrastructure. Early investments focused on overcoming these challenges—supporting researchers, training future scientists, and funding discoveries that laid the groundwork for today’s breakthroughs.

Understanding disease at the cellular level is a critical step toward developing effective therapies. Julia Kaye, PhD, at Gladstone Institutes, is using stem cell models to study amyotrophic lateral sclerosis (ALS). By transforming patient-derived cells into motor neurons, she can observe disease progression and identify potential treatment targets. Dr. Kaye's work also explores how machine learning can reveal key differences between healthy and diseased neurons, advancing our understanding of ALS and paving the way for new therapeutic approaches.

  CIRM has awarded $26.5 million to fund three promising research projects targeting Angelman syndrome, drug-resistant epilepsy, and graft-versus-host disease. These awards bring the total number of CIRM-funded clinical trials to 115, reinforcing our commitment to advancing therapies for patients with serious unmet medical needs. “We are particularly excited about these new awards because they underscore our strategic commitment to accelerating groundbreaking therapies from laboratory to patient care," said Rosa Canet-Aviles, PhD, Chief Science Officer at CIRM. Read full press release: https://lnkd.in/gSzE3w5g

The updated funding concepts include expanded support for large collaborative teams and pairs of investigators, pre-Investigational New Drug (pre-IND) or IND-enabling research, and late-stage clinical trials. With the approval of the new funding and amended programs, CIRM is deepening its commitment to advancing stem cell and gene therapy research in California. “These funding decisions exemplify CIRM’s dedication to driving impactful, patient-focused research that addresses critical unmet medical needs," said CIRM Chief Science Officer, Rosa Canet-Aviles, PhD. CIRM will post the final program announcements and details on its website throughout the spring and fall. Researchers can learn more and sign up for email updates on our website. Read full press release: https://lnkd.in/gSzE3w5g Sign up for email updates: https://lnkd.in/g5SUaFei