California Institute for Regenerative Medicine

Infrastructure is essential to moving promising research toward patients. The UCLA Health Center for Advanced Biotherapies will expand on-campus manufacturing capacity for cell and gene therapies, supporting a wider range of personalized treatments and helping researchers move therapies into early clinical trials more efficiently.

CIRM invested $2 million in this significant initiative, underscoring the commitment to advancing regenerative medicine and gene therapy in California. These investments not only build essential capacity for innovative treatments but also nurture the next generation of scientific leaders.

UCLA Health Broad Stem Cell Research Center at UCLA UCLA Jonsson Comprehensive Cancer Center

UCLA opens Center for Advanced Biotherapies, expanding capacity to develop and deliver cell and gene therapies For 30 years, the UCLA Human Gene and Cell Therapy Facility has been the quiet engine behind some of the university’s most ambitious clinical research, supporting more than 25 clinical trials and producing over 300 personalized therapy products for patients with cancer, HIV/AIDS, sickle cell disea...

At UCLA Health California Center for Rare Diseases, “rare” represents more than 7,000 conditions affecting 3 million Californians. Through multidisciplinary care, genetic diagnosis, registries, clinical trials, and novel therapies, UCLA is advancing rare disease research and treatment in real time.

Recent milestones reflect that momentum, from the launch of the nation’s first Multidisciplinary Bloom Syndrome Clinic to advances in gene therapy for Duchenne muscular dystrophy and other genetic conditions. Among these efforts is promising investigational gene therapy research led by Dr. Donald Kohn, whose work, funded by CIRM, is helping expand what may be possible for families affected by rare diseases.

Stories like these really highlight how important it is to keep investing in research that translates into real treatments, and how working together in teams can help bring new therapies to patients.

Read more: https://bit.ly/4tUh4Z5

UCLA research prioritizes hard-to-treat patients through rare disease innovation At the UCLA California Center for Rare Diseases (CCRD), “rare” is a word that includes over 7,000 conditions and affects 3 million Californians. With hundreds of thousands of patients and families touched by rare diseases nationwide, CCRD, directed by Stanley Nelson, MD, has leveraged UCLA’s l...

How do we get better therapies to patients faster?

On the Sanford Stem Cell Institute's reGEN Podcast, City of Hope's Dr. Leo Wang shares how the CIRM Alpha Clinics Network is helping expand access to cutting-edge therapies across California, not just in major research hubs.

He highlights the network’s collaborative model, its focus on implementation and dissemination, and the importance of building infrastructure to enable more patients to access life-saving treatments closer to home.

Listen to the full episode:

Novel Therapeutics and Discoveries | Leo D. Wang, MD, PhD Our conversation with Leo D. Wang, MD, PhD, includes unique insight...

Like many at CIRM, Maria Gonzalez Bonneville's work is personal.

CIRM's vice chair joined the agency in 2011, not long after her father's Parkinson's diagnosis. The job offered her a chance to combine her deep commitment to public service in California with her desire to support this voter-supported effort to accelerate treatments and cures for conditions like her father's.

Maria's role at CIRM now extends to serving as Chair of CIRM's Accessibility and Affordability Working Group, where she's helping ensure that the potentially life-changing therapies that come from CIRM-supported research and clinical trials are accessible to all Californians who need them.

CIRM supports stem cell and genetic research and clinical trials into treatments for hundreds of different conditions, including Parkinson's disease, which affects about a million people in the United States alone.

Maria's work reflects a broader commitment that her work and CIRM's aren't just about finding breakthroughs but about ensuring that patients can access them.

"It isn't just about funding the science," she says. "It's about making sure it gets to people."

Read Maria's story: https://bit.ly/3QZj052