California Institute for Regenerative Medicine

08/12/2025

Cristian Rodriguez, a California State University, Northridge graduate and CIRM Bridges trainee, once planned to become a firefighter. Now, he's breaking ground in spinal cord research at UCLA.

Cristian discovered his passion for stem cell science through the CIRM-funded CSUN-UCLA Bridges to Stem Cell Research Program. His work focuses on restoring sensation for people with spinal cord injuries, and he’s already helping develop protocols that could one day restore bladder control.

Cristian is proving that career paths aren’t linear and impact begins with opportunity.

Cristian Rodriguez is advancing stem cell-based approaches that could one day restore sensation to people with spinal cord injuries.

08/05/2025

Every advance toward new therapies relies on a team of skilled scientists, and CIRM has supported the training of thousands of them.

As we celebrate CIRM’s 20th anniversary, we’re revisiting one of our first and most impactful investments: building a skilled biomedical workforce. Since 2006, CIRM has committed over $447 million to educational and work training programs, creating life-changing research opportunities for more than 4,600 biomedical students from across California.

These programs open doors for students from all backgrounds to access cutting-edge labs and build the skills needed to develop tomorrow’s treatments and therapies.

In celebration of the California Institute for Regenerative Medicine (CIRM)’s 20th anniversary, we have been reflecting on some of the early projects we supported and their outcomes.Today, we’re lo…

08/01/2025

Gene therapy is transforming the outlook for people with sickle cell disease—but what does that mean for patients now?

The UCSF Sickle Cell Center of Excellence is hosting a virtual town hall on August 16 from 11 AM - 12:15 PM, featuring Mark Walters, MD, an expert in sickle cell disease and cell/gene therapy, along with a sickle cell warrior who recently received gene therapy. Attendees will learn about current treatment options, risks, and what’s on the horizon.

Register to attend by August 15 at https://www.eventbrite.com/e/sickle-cell-gene-therapy-updates-virtual-town-hall-tickets-1520347623669?aff=oddtdtcreator

07/21/2025

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://bit.ly/4eWpwRI

In celebration of the California Institute for Regenerative Medicine (CIRM)’s 20th anniversary, we have been reflecting on some of the early projects we supported and their outcomes.Today, we’re lo…

07/18/2025

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 Broad Stem Cell Research Center at UCLA, Gerald 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:

Nothing could have prepared Dr. Gerald Lipshutz for what he experienced at a small patient-physician symposium in 2022 in Utah.

07/09/2025

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

07/08/2025

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 Genascence, 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://bit.ly/4nzlpzb

07/02/2025

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.

Research Investigator Julia Kaye, PhD, stands for a portrait in a lab at Gladstone Institutes. Photo courtesy of Gladstone Institutes. Before there can be any therapies for disease, scientists need…

06/27/2025

After a childhood treatment left his legs weak and numb, Dongwei Sun made it his mission to help others by developing stem cell and gene therapy technologies.

As a CIRM Scholar at UC 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.

CIRM Scholar Dongwei Sun, PhD (left), with his mentor Huinan Liu, PhD, professor of bioengineering at the University of California, Riverside. As a child, Dongwei Sun underwent a medical treatment …

06/26/2025

A clinical trial developed by Rocket Pharmaceuticals and co-led with UCLA, supported by a $5.8 million CIRM grant, has delivered positive results for children with severe leukocyte adhesion deficiency-I (LAD-I), a rare and life-threatening genetic immune disorder.

All nine children who received the investigational gene therapy responded positively and are living without any disease symptoms.

The therapy works by correcting the CD18 gene in the patient’s own blood stem cells—eliminating the need for a donor and reducing the risks associated with bone marrow transplants. It represents a major advancement in the treatment of rare pediatric diseases and reflects the growing impact of gene and cell therapy approaches.

An investigational gene therapy has successfully restored immune function in all nine children treated with the rare and life-threatening immune disorder called severe leukocyte adhesion deficiency-I, or LAD-I, in an international clinical trial co-led by UCLA.

06/20/2025

Epilepsy affects more than 3 million Americans－ including 500,000 Californians. Although many people with epilepsy benefit from anti-seizure medications, for about one-third of patients, these medications don't work.

With support from a $14 million award from CIRM, Neurona Therapeutics has reached a major milestone in developing its stem cell approach to treating epilepsy. The company announced recently that the first patient has been dosed in a Phase 1/2 clinical trial evaluating NRTX-1001, a cell therapy for adults whose epilepsy affects both sides of the brain and for which anti-seizure drugs have not been effective. CIRM has supported this research from its early discovery stage at UCSF to the current first-in-human clinical trials.

Current treatments for drug-resistant epilepsy usually involve invasive surgeries that destroy brain tissue and carry serious, irreversible risks. Patients with epilepsy on both sides of the brain are typically not eligible for these surgeries due to the risk of profound and permanent memory loss.

The cell therapy is composed of neurons derived from human embryonic stem cells that inhibit excitatory neurons. When injected into the brain, these cells are designed to calm overactive neurons and reduce seizures without damaging healthy brain tissue. In this trial, patients receive a one-time injection of NRTX-1001 into both sides of the brain.

If successful, data from these trials—along with an upcoming Phase 3 study—could support future FDA approval of NRTX-1001 for drug-resistant epilepsy, offering a potential treatment for patients and families affected by this life-altering condition.

Read the full blog at https://blog.cirm.ca.gov/2025/06/20/neurona-doses-first-patient-in-cirm-funded-bilateral-epilepsy-cell-therapy-trial/

06/11/2025

Eric Adler, MD, a professor of medicine at UC San Diego and Chief Medical Officer at Lexeo Therapeutics, is developing gene therapy approaches to treat heart failure- one of the leading causes of death and hospitalization in the U.S.

Supported by over $8 million in CIRM funding, his research focuses on correcting genetic mutations that cause cardiac dysfunction, intending to improve outcomes for patients who otherwise face transplants or life-threatening complications.

Image Credit: The Adler Lab at the University of California, San Diego In the fall of 2024, the California Institute for Regenerative Medicine (CIRM) helped sponsor the Gene Therapy Initiative symp…