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://xmrrwallet.com/cmx.plnkd.in/gbE2M5yJ

Starting off Day 2 of the #CIRMTraineeConference with an inspiring and insightful career panel. Trainees are learning about the various career paths in the regenerative medicine field— from R&D and lab work to policy and science communication. Thank you to our moderator, Keau Wong, and discussion panelists: Tisho Tefera, Ph.D., Ying-Tsu Loh, Tita Tavares, Tepring Piquado, PhD, Anthony Castro, Joseph Gold, and Vignesh Nadar for sharing their personal experiences and insights.

These programs are among the many reasons why I’m enjoying being back at CIRM. I’m looking forward to meeting many of these students at the upcoming trainee conference.

In California’s booming life sciences industry, opportunity begins with access to training. CIRM has built a robust pipeline of training programs that have provided more than 4,600 students at every level of education with hands-on experience in cutting-edge biomedical research. These programs don’t just build careers—they help power California’s $414 billion life sciences industry and ensure new treatments reach the patients who need them. Learn more about how we’re investing in people, and not just science: https://xmrrwallet.com/cmx.plnkd.in/gJDA8YqN

Creatine Transporter Deficiency (CTD) is a rare brain disorder with no cure—yet. But families like Jeffrey Randall Allen’s are fighting for a future where gene therapy changes everything. At the UCLA Broad Stem Cell Research Center, Gerry Lipshutz, MD, and his team, with a $2.2 million CIRM award, are developing a one-time gene therapy to treat CTD at its source. Jeffrey, who has raised global awareness through the Prime Video show Beast Games, will share his advocacy journey at CIRM’s Trainee Networking Conference, inspiring the next generation of scientists advancing cell and gene therapy research. Read the full story: https://xmrrwallet.com/cmx.plnkd.in/gpe7xkrk

New Discovery research funding opportunity: the DISC5 Awards. The DISC5 Awards support exploratory, innovative research studies led by collaborative pairs of investigators who employ human stem cells and/or genetic approaches to address knowledge gaps or bottlenecks in the field of stem cell science and regenerative medicine. The DISC5 program promotes interdisciplinary collaboration and fosters high-risk, high-reward studies that can advance our understanding of human biology and disease. This program contributes to CIRM’s broader goal of identifying novel disease targets and biomarkers that can become a foundation for future therapies while driving knowledge sharing and data transparency to increase the value of these outcomes to the field. For full funding program details, visit this page: cirm.ca.gov/disc5

Osteoarthritis affects nearly 600 million people worldwide, causing pain and limiting mobility. While treatments help manage symptoms, there is no cure. CIRM is funding four teams that are working on solutions, including the Genascence Corporation, which recently reported promising results from their DONATELLO trial. Their gene therapy targets the molecule IL-1, which drives osteoarthritis, aiming to slow or reverse disease progression. "These results reflect the kind of innovation CIRM was created to support," said Lisa Kadyk, PhD, CIRM Fellow, Clinical Development. "By harnessing gene therapy, this approach offers hope for a more effective, long-term treatment for knee OA." Read the full blog at https://xmrrwallet.com/cmx.pbit.ly/44BQPwa

Most ALS cases have no known genetic cause, making it challenging to develop effective treatments. With support from CIRM, principal investigator Julia Kaye, PhD, at Gladstone Institutes, is working to change that. She is uncovering key disease mechanisms and testing potential therapies using induced pluripotent stem cells from patients with sporadic ALS. By creating more accurate disease models, Dr. Kaye's work aims to identify new therapeutic targets that could benefit a broader range of patients. https://xmrrwallet.com/cmx.plnkd.in/gJQQVBeB

CIRM was honored to attend and have a booth at the National Organization for Rare Disorders (NORD) Living Rare Living Stronger Patient and Family event in Atlanta today! CIRM Community Outreach Manager Aditi Desai MPH, CPH, CYT, met with and had conversations with patients, caregivers, families, advocates, and rare disease medical professionals about their experiences and clinical trial access.

After a childhood treatment left his legs weak and numb, David (Dongwei) Sun made it his mission to help others by developing stem cell and gene therapy technologies. As a CIRM Scholar at University of California, Riverside and now a scientist at Cedars-Sinai, Dongwei is engineering materials to help stem cells repair the heart and blood vessels. His story is a powerful example of how early experiences and a little of CIRM’s support can shape a career in advancing stem cell science. https://xmrrwallet.com/cmx.plnkd.in/gJYe54FR