California Institute for Regenerative Medicine

08/28/2026

When Marguerite Casillas was diagnosed with Multiple Sclerosis (MS) in 2003, she faced the fear and uncertainty that so many newly diagnosed patients feel. But instead of letting MS define her limits, she spent 18 years continuing her corporate career before dedicating her retirement to a new purpose: advocacy.

Now serving on CIRM’s board, Marguerite is urging scientists to remember the human beings behind the data points.

Why is patient advocacy crucial for scientific breakthroughs?

🔵 Patients are the experts: Nobody understands a condition better than those living with it daily.

🔵 Accessible research: Marguerite’s feedback helped researchers break an exhausting 90-minute interview into shorter, manageable sessions—making the study accessible for people dealing with MS fatigue.

🔵 "Nothing about us without us": True progress happens when patients have an active seat at the table.

Marguerite’s message to future researchers is clear: Listen to patients, involve them early, and never lose sight of why the science matters.

Read Marguerite's whole story: https://bit.ly/4qEGXfu

08/27/2026

The human immune system is designed to protect us from harmful cells and foreign substances, and with a little help from cell and gene therapy, it can do even more.

CAR T-cell therapy is a form of genetic engineering that gives the immune system a powerful boost by leveraging the body’s own defenses to fight disease. Scientists collect a patient’s own T-cells, modify them in a lab so they can recognize specific harmful cells, and then return those modified cells to the patient’s body.

Once inside the body, these CAR T-cells act like a “living drug,” searching for and destroying targeted cells. Today, CAR T-cell therapies are primarily used to treat certain blood cancers, including leukemia, lymphoma, and multiple myeloma.

Researchers are also exploring how this approach could be used to treat other diseases, including autoimmune diseases, HIV, aggressive brain cancers, and kidney cancer.

CIRM is supporting CAR T-cell research and clinical trials that aim to expand the potential of this promising therapy and bring new options to patients.

Learn how CAR T-cell therapy works and why scientists are excited about its future: https://bit.ly/46o7Fjb

08/20/2026

The families and patient advocates who live with autism every day are helping scientists make advances in autism research.

Researcher Matthew State, MD, PhD, chief scientific advisor for the Aligning Research to Impact Autism (ARIA) initiative and chair of psychiatry and behavioral sciences at UCSF, has worked alongside patient advocates including Allyson Berent of Foundation for Angelman Syndrome Therapeutics and Alison Singer of the Autism Science Foundation for years.

Their alliance helps foster research that can have the greatest positive impact for patients with schizophrenia, profound autism, and other severe neurodevelopmental disorders.

How CIRM supports this momentum:

🔵 The ReMIND Initiative: Funding multi-investigator projects to help researchers make breakthroughs faster together, which is important for complex neurological research projects, such as studies related to schizophrenia, profound autism, and other severe neurodevelopmental disorders.

🔵 Advocates at the Table: CIRM includes patient advocates on its governing board, giving them a direct voice in funding decisions. When research teams treat families as partners, we break down barriers, reduce stigma, and clear the path toward life-changing therapies.

Read more: https://bit.ly/4xb2YVR

08/14/2026

Knee osteoarthritis affects millions of people and remains a major cause of pain, disability, and reduced mobility.

Tara Ghiglieri, a former Broadway dancer and Radio City Rockette, participated in an early clinical trial at UC San Diego testing GNSC-001, an investigational gene therapy developed by Genascence. Funded in part by CIRM, the therapy is being studied as a potential way to provide longer-lasting relief by targeting inflammation and cartilage damage directly in the joint.

Explore an innovative experimental treatment for knee osteoarthritis to alleviate pain and improve mobility after injuries.

08/13/2026

We marveled at the first sparks of innovation coming from the next generation of California scientists this summer in Berkeley, where more than 100 high school interns converged for the 2026 CIRM SPARK conference.

The students got the chance to show off some of what they learned about cell and gene therapy while working in labs, research institutions, and universities across the state. The high school intern conference came on the heels of another CIRM conference the week prior in Sacramento for undergraduate, graduate, and postdoctoral students.

Over the last 20 years, CIRM has supported more than 4,600 students, including high school, undergraduate, graduate, and postdoctoral fellows, through internships and training programs across the state. Many CIRM student alumni are now part of the science infrastructure that makes California a world leader in biotechnology. The students we saw this summer will help grow that workforce in the future and keep the state at the forefront of regenerative medicine.

Thank you to Biocom California Institute and Generation STEAM for organizing the event — and to our incredible speakers for sharing your insights, stories, and encouragement.

08/04/2026

A CIRM-funded early-stage clinical trial is exploring whether a single infusion of modified CAR-T cells could offer a new approach to treating HIV.

Dr. Steven Deeks of UCSF presented the preliminary findings that showed promising signs of viral control in some participants after stopping standard HIV treatment. The study remains ongoing, but the results add to growing interest in whether cell and gene therapy strategies could one day reduce or eliminate the need for lifelong antiretroviral therapy.

Read full story: https://bit.ly/3UdghpZ

08/03/2026

KPBS wrote a wonderful update on Connor Dalby, a patient who received treatment from Dr. Olivia Kim-McManus' CIRM-funded clinical trial.

Could a treatment designed for one San Diego County teenager with a rare genetic disease one day help other children like him? That’s the question UCSD researchers are asking after a personalized treatment helped one patient walk for the first time in his life.

07/31/2026

California didn't just bet on science. It bet on scientists.

This week at the 2026 CIRM Trainee Networking Conference in Sacramento, nearly 500 attendees, including undergraduate, graduate, and postdoctoral trainees, came together to share their research, swap career strategies, and hear from patient advocates about how their lab work is changing lives.

From poster sessions featuring research on cell and gene therapy to panel discussions on biotech careers to the patient stories, this group of scientists was reminded why their work matters.

Programs like Bridges, COMPASS, and CIRM Scholars represent something bigger than training. They're California's investment in a generation of brilliant scientists who will carry regenerative medicine from the lab bench to the clinic.

Thank you to the trainees, program directors, patient advocates, and speakers who made this more than a conference. Most of all, thank you to the people of California, who invested in building the science infrastructure and invested in the people who have made the state a world leader in regenerative medicine.

The future of science in our state is in good hands.

07/24/2026

This is a meaningful milestone for the cystinosis community.

These preliminary results reflect years of dedication from families, advocates, researchers, and organizations committed to advancing this work, including early funding from CIRM that helped move the trial forward from its beginning stages.

07/21/2026

A Phase 1 clinical trial at Stanford Medicine points to a promising path toward safer stem cell transplants for children with Fanconi anemia, replacing radiation or high-risk chemotherapy with a targeted antibody approach. The early results are encouraging, with all three patients doing well at least two years after treatment.

The work reflects years of translational research aimed at reducing toxicity while preserving the life-saving potential of stem cell transplantation. CIRM funded more than $10 million to this research that is helping advance innovative approaches that could improve outcomes for patients with rare and fragile conditions.

Learn more:

Researchers are pioneering a safer stem cell transplant for children with Fanconi anemia, reducing toxicity and improving outcomes.