California Institute for Regenerative Medicine

01/07/2026

When the heart is injured — like after a heart attack — it can’t repair itself the way skin does after a cut. But a new UCLA discovery could change that.

A research team led by Dr. Arjun Deb, supported in part by CIRM funding, has developed a monoclonal antibody called AD-NP1 that boosts the heart’s ability to heal. The drug works by blocking a protein called ENPP1, which interferes with the body’s natural repair process.

In animal studies, AD-NP1 improved heart function, reduced scar tissue, and even restored energy to damaged cells. Now, after FDA approval, the team is preparing for human clinical trials — a major step toward a new class of drugs that could help hearts, kidneys, and other organs recover after injury.

This breakthrough marks a rare achievement: a new drug developed entirely within a university lab, powered by public research funding.

Learn more about how taxpayer-funded science is driving innovation in regenerative medicine: https://bit.ly/48WqZqr

Funded entirely by taxpayer dollars, this first-of-its-kind drug targets cellular metabolism to promote tissue repair of multiple organs.

01/02/2026

Developing new regenerative medicine treatments is only the first step; ensuring that Californians have access to these treatments is just as important.

To put clinical trials and therapies within reach of Californians, CIRM has approved an Access & Affordability strategy that outlines how CIRM will deliver cell and gene therapies to Californians. Many patients, especially those in underserved or remote areas, face barriers to care and affordability. This strategy focuses on improving accessibility at clinical sites, collaborating with payers to support favorable policies, and planning early in research to reduce patient costs.

CIRM's Patient Support Program is also expanding with a $15.6 million Patient Assistance Fund to help cover costs for patients participating in California-based clinical trials.

Maria Gonzalez Bonneville, Chair of the Access & Affordability Working Group, said, "A patient's home address and income should not determine their treatment options. CIRM is committed to expanding equitable access to clinical trials, prioritizing underserved communities statewide."

Read full release: https://bit.ly/4sgVAWP

12/30/2025

CIRM is extending training programs that fund high school, undergraduate, and masters-level students working in labs and industry today, and that will create a regenerative medicine workforce for the future.

These programs — which include SPARK, Bridges, and Compass -- offer enhanced training, hands-on research, and mentorship. These opportunities equip students from California universities, community colleges, and high schools with the critical skills needed to help develop and deliver regenerative medicine therapies.

Kelly Shepard, PhD, Director of Discovery & Education programs, emphasizes the need for this training: "Training students and early career scientists so they have the skills and resources they need to work in the fields of stem cell research is a critical part of developing and delivering regenerative medicine therapies."

Read full release: https://bit.ly/4sgVAWP

12/29/2025

CIRM recently funded over $160 million toward 16 research projects intended to move discoveries into clinical trials and ultimately to patients in California.

"CIRM recognizes that making a medical breakthrough is only the beginning of the complex process of delivering new regenerative medicine therapies to the people of California," said Rosa Canet-Aviles, CIRM's chief scientific officer.

Approved clinical projects include those testing approaches for treating Parkinson’s disease and Huntington’s disease, which both involve injecting stem cell-derived neural cells into the brain of patients to replace damaged cells. Programs targeting the rare genetic diseases Dravet Syndrome and CMTJ4 (Charcot-Marie-Tooth disease type 4J) are both testing genetic therapies intended to fix the mutated genes that underlie those conditions.

Preclinical projects (those intended to move a project toward clinical trials) span a wide range of diseases and therapeutic approaches, including several neurological diseases as well as diseases of the heart, cartilage, lung, and muscle.

All funded projects are intended to bring new therapies for people who otherwise have no options.

Read full press release: https://bit.ly/4sgVAWP

12/22/2025

Fueled by hope for her son Calvin's future, Audrey Davidow tirelessly advocates for research on Pitt-Hopkins Syndrome.

CIRM supports early-stage research, such as a study conducted by Iris Medicine, Inc. This research aims to understand gene regulation for conditions like Pitt-Hopkins Syndrome. This vital work will lay the groundwork for future clinical trials and potential cures.

Calvin's story highlights the urgent need for continued investment in scientific exploration.

Read the full story: https://bit.ly/3KKXoq3

12/17/2025

Stories like Evangelina's highlight why CIRM funds research. Supporting the scientific path to clinical trials helps us achieve life-changing treatments and cures. Watch her inspiring speech: https://youtu.be/SofJVAi19_Y?si=6dU-dHyrbramdtM6

🎥 : Broad Stem Cell Research Center at UCLA

At the UCLA Broad Stem Cell Research Center's 20th Anniversary Celebration, Evangelina Vaccaro shares her extraordinary story. Born with adenosine deaminase–...

12/10/2025

Audrey Davidow's journey with her son Calvin, who has Pitt Hopkins Syndrome, started when he was first diagnosed at 13 months old. Since then she has advocated for the disease, including for research recently funded by CIRM.

The project Davidow supported focuses on developing gene therapy approaches to treating diseases like Pitt Hopkins Syndrome and similar genetic disorders.

Calvin, who struggles to communicate, wrote, “Thank you, CIRM, Please hurry."

Read the full story here: https://bit.ly/3KKXoq3

12/05/2025

From a Fulbright scholar in musicology to a combat surgeon, state cabinet secretary, and ultimately Chair of CIRM’s governing board, Colonel Vito Imbasciani’s career is anything but ordinary.

Throughout every chapter, his mission remained the same: to serve others.

His leadership has helped guide CIRM’s efforts to accelerate cell and gene therapy research, support trainees, and expand access to cutting-edge science.

Learn more on our blog: https://bit.ly/3XCsMdm

Written by Holly Alyssa MacCormick If you ask Vito Imbasciani to tell you about his career, you won’t get a simple answer, but you will get a great story about service, both to his country and the …

12/01/2025

"I don't ever want to think about what it would've meant if Cora didn't have this opportunity," said Chelsea Ferrier, whose daughter Cora participated in a CIRM-funded clinical trial for a rare immune disorder.

Cora, and other kids like her, is now able to fight off all the infections of childhood－infections that, without participating in the gene therapy clinical trial, might have killed her.

Cora is one of many children born with "bubble boy disease" (SCID) who are now able to live full and healthy lives after participating in the clinical trial, supported by decades of sustained funding from CIRM.

Stories like Cora's and those of other children in the trial don't happen by accident. They are the result of choices. Choosing to invest in research in California, choosing to support life-saving clinical trials, and making the family's brave choice to participate in a clinical trial.

Read the full article: https://nyti.ms/48IXtUl

“Bubble boy disease” was once a death sentence. A scientific breakthrough changed that.

11/28/2025

Discovery research takes time — but it’s the foundation for every breakthrough that reaches patients.

Over the years, CIRM has funded millions to advance a potential new cell therapy for drug-resistant temporal lobe epilepsy.

Developed by Neurona Therapeutics, NRTX-1001 is derived from stem cells and aims to provide a less invasive, regenerative alternative to surgery or laser ablation.

This approach is the result of decades of foundational work that began in labs and grew through collaboration, persistence, and consistent support.

Read more on our blog: https://bit.ly/4rkqoFF

11/26/2025

Epilepsy affects more than 3 million people in the U.S., and for about 30% of them, medications don’t work. For Justin Graves, that meant losing his ability to drive and his lifelong passion for diving after his first seizure in 2007.

Justin’s life changed again when he joined a CIRM-supported clinical trial testing a stem cell–based therapy developed by Neurona Therapeutics.

Supported by over $14 million from CIRM grants starting in 2007, the therapy is designed to calm overactive brain circuits without damaging healthy tissue. Within a month of receiving an injection of the cells at UC San Diego, Justin stopped experiencing grand mal seizures.

“I love the idea of being able to help not just a few people with some issues, but the entire world,” Justin said.

His story is a powerful reminder of how CIRM’s long-term investment in research and clinical trials changes lives.

Read Justin's Story on our blog: https://bit.ly/4rqrOPa

11/25/2025

Epilepsy affects more than 3 million people in the US, including over 500,000 Californians, and around one-third of patients don’t respond to medications.

For many, this means losing the ability to drive, work, or maintain their independence. Current options often involve invasive surgeries that permanently destroy brain tissue and aren’t viable for those with seizures on both sides of the brain.

With $14 million of CIRM funding, Neurona Therapeutics is now in the Phase 1/2 stage of the NRTX-1001 clinical trial and dosed the first patient this year.

NRTX-1001, a stem cell-derived neuron therapy designed to calm overactive brain circuits without damaging healthy tissue—a potential new option for people with the most severe and treatment-resistant epilepsy.

This progress builds on promising early results from Neurona’s ongoing trial, which showed a 92% reduction in seizures and meaningful improvements in quality of life.

These advances highlight why long-term investment in early research matters and how patient stories continue to drive the work forward.

Read the full story on our blog: https://bit.ly/3KkYSHk