California Institute for Regenerative Medicine

02/18/2026

Leukocyte adhesion deficiency type 1 (LAD-1) is a rare and devastating pediatric disorder that severely impacts the immune system, leading to life-threatening infections and delayed healing. For children like Marley, diagnosed at age 8 after years of hospitalizations, the options were limited and risky.

However, a clinical trial led by Dr. Donald Kohn at UCLA, sponsored by Rocket Pharmaceuticals and CIRM, is changing the landscape of treatment. This gene therapy approach involves correcting a child's own faulty stem cells in a lab and then reintroducing them, eliminating the risks associated with traditional bone marrow transplants.

Marley, now a healthy kid enjoying camping and everyday life, was the first LAD-1 patient to receive this therapy in 2019. Recent reports indicate that Marley and the other patients treated in the trial remain healthy and disease-free years after therapy. This one-time treatment offers the promise of a normal, healthy life for LAD-1 patients.

Read Marley's inspiring story: https://bit.ly/4aBvHZT

02/10/2026

For people with rare diseases, new treatments have always come too slowly. Each therapy takes years and a lot of resources. CIRM is working to change that by investing $100 million over two years with a new program called RAPID. The goal is to bring hope to millions living with rare conditions faster.

CIRM is moving to a platform model to make gene therapies reach more people, more quickly. This new funding program builds on the success of personalized treatments, like the one that helped baby KJ Muldoon. The goal is to bring that kind of hope to many more families facing rare diseases.

The funding will support projects that develop genetic therapies, from early-stage ideas to those already in clinical trials. This investment aims to open a new era for gene therapy and bring more treatments to patients who need them.

Read this article by Genetic Engineering & Biotechnology News about CIRM's RAPID program:

Over the next two years, the planned funding will be allocated to projects that have received some FDA feedback as well as emerging efforts.

02/06/2026

CIRM is adopting a platform approach for rare disease therapies.

This new funding program draws parallels to how car manufacturers build multiple models on a single foundation—enabling faster development and delivery of multiple genetic therapies for a wider range of rare diseases.

With $100 million allocated over two years, CIRM is committed to turning hope into reality for rare disease patients and families facing these challenges.

Learn more about this program on our blog: https://bit.ly/4qYLvNd

01/30/2026

Millions in the U.S. are affected by rare diseases, yet 95% have no available therapies. This is especially heartbreaking for children, who often lose their lives far too young.

CIRM is tackling this challenge with a new funding program to accelerate treatments for rare diseases. By leveraging advances in genetic therapies and a new FDA pathway, CIRM is allocating $100 million over two years to support platform technologies.

This approach aims to deliver more therapies to more patients more quickly, offering hope where options are limited.

Learn more about this initiative: https://bit.ly/4qYLvNd

01/28/2026

Learn more about a major advance in pediatric cancer treatment at our upcoming speaker series, Closer to Cures, on January 29 at 10:30 AM (PST).

Hear from Dr. Crystal Mackall of Stanford University about the CAR-T cell therapy trial for aggressive brain and spinal cord tumors, including a childhood brainstem cancer that her and her team have been researching and developing.

Livestreaming on our YouTube channel at youtube.com/cirmtv

01/28/2026

Join us for a Closer to Cures event where you can hear about progress of CIRM grantees toward FDA approval on January 29 at 10:30 AM (PST).

The livestreamed event is part of the full CIRM board meeting: https://www.youtube.com/

Featured speaker, Dr. Cory Nicholas of Neurona Therapeutics, will share some of the latest news on the cell therapy they are developing to treat epilepsy.

Learn how this research is already helping some patients in clinical trials and is moving toward a potential future FDA approval.

01/27/2026

Join us for our upcoming speaker series Closer to Cures on Thursday, January 29 at 10:30 AM (PST).

Hear from CIRM awardees Dr. Cory Nicholas of Neurona Therapeutics and Dr. Crystal Mackall of Stanford University about the progress of their clinical trials for epilepsy and brain cancer research in regenerative medicine.

These presentations are part of the CIRM governing board meeting, which is open to the public.

01/27/2026

CIRM is funding over $7.5 million in two early-stage research projects at the Gladstone Institutes to advance regenerative medicine.

$5.1 million will support Dr. Bruce Conklin’s team as they develop a gene-editing platform designed to overcome one of the biggest barriers in gene therapy—making treatments accessible and effective for more patients across hundreds of genetic diseases.

$2.4 million will fund Dr. Benoit Bruneau’s work using stem cells and AI to uncover how genetic mutations disrupt heart development, paving the way for next-generation heart and organ regeneration therapies.

These grants are part of CIRM’s Discovery (DISC0) Program, which supports foundational research to turn bold ideas into future cures.

Read full story: https://bit.ly/3W2e7HB

Two ambitious research projects led by Gladstone investigators are boosted by funds from the 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