California Institute for Regenerative Medicine

03/27/2026

Today marks a milestone for the California Institute for Regenerative Medicine: Our first therapy approved by the U.S. Food and Drug Administration. This moment wouldn’t have been possible without the vision of the people of California, who created CIRM, and the scientists, patient advocates, clinical trial participants, and countless others who all worked to make this therapy a reality.

This new gene therapy, called KRESLADI™, developed by Rocket Pharmaceuticals, Inc., treats the rare childhood disease Leukocyte Adhesion Deficiency-1 (LAD-1). LAD-I is a genetic disease where a child’s immune system can’t properly fight infections, putting them at risk for serious illness. Few kids with severe, untreated LAD-1 survive to adulthood.

“With the first FDA-approved therapy from CIRM funding, we are delivering on our commitment to Californians to support the development of new stem cell and gene therapies that save lives,” said Jonathan Thomas, PhD, JD, CIRM President and CEO.

Previously, children with severe LAD-1 were treated with bone marrow transplants, but this therapy does not always work, and it can have serious long-term complications.

“The FDA-approved treatment for LAD-1 is a major step forward for Californians who supported investing in the promise of regenerative medicine,” said Rosa Canet-Avilés, PhD, CIRM Chief Science Officer. “We are thrilled that children with LAD-1 have this new option available to them. In the future, we expect to see many more FDA-approved stem cell and gene therapies that will benefit people in California and around the world who have no other options.”

Read full story: https://bit.ly/4ta5sRP

03/25/2026

California is at the forefront of gene therapy for rare diseases. Inspired by families like the Langenhops, whose children are thriving after a CIRM-funded trial for LAD-1, we're accelerating hope.

As highlighted by the Sonoma Valley Sun, our new RAPID program is revolutionizing the development of treatments for the millions affected by rare conditions.

Read full article: https://bit.ly/4bSeNHb

Ava, Olivia, and Landon Langenhop were all born with a rare and life-threatening immune disorder called severe leukocyte adhesion deficiency-I, or LAD-I. This rare genetic condition makes them at risk of near-constant bacterial and fungal infections, and survival beyond childhood is rare without tre...

03/19/2026

CIRM is funding a clinical trial led by Stanford Medicine researcher Crystal Mackall that shows promise as a possible therapy for rare childhood cancers, which are so aggressive and devastating that the current treatment typically only prolongs the patient’s life a few months.

The CIRM-funded clinical trial is still underway, but preliminary results show some patients had tumors that shrank by more than half, and one patient had their tumor completely disappear.

Recently, Mackall discussed the preliminary results of this clinical trial as part of CIRM’s Closer to Cures speaker series and discussed next steps for this research.

Read the full story and watch the presentation:

Written by Holly Alyssa MacCormick In 2023, the California Institute for Regenerative Medicine (CIRM) funded a clinical trial led by Stanford researcher Crystal Mackall, MD, to investigate a possib…

03/17/2026

In 2019, Jon and Alicia Langenhop learned that all three of their children had a rare, life-threatening immune disease called leukocyte adhesion deficiency-1 (LAD-1). A CIRM-funded clinical trial of a genetic therapy developed by Rocket Pharmaceuticals to treat LAD-1 successfully restored immune function in all three children and all nine participants.

The disease is caused by gene mutations that limit or block the production of a protein that white blood cells need to access and work in infection sites. This protein is so critical for healthy immune function that few kids with severe and untreated LAD-1 survive to adulthood.

The experimental LAD-1 gene therapy was so successful that it restored immune function to all nine participants in the clinical trial.

Read the full story: https://bit.ly/4uP7EQy

03/06/2026

For kids and young adults diagnosed with rare brain and spinal tumors that arise from a specific genetic mutation, known as H3K27M, there is no known cure or effective therapy on the market. But this could soon change.

CIRM is funding a clinical trial led by Stanford Medicine researcher Crystal Mackall that uses a technique called CAR-T therapy to “train” the patient’s own immune system to identify and destroy cancer cells.

Preliminary results of this phase 1 clinical trial show several patients had significant improvement in the disabilities caused by their disease, some patients had tumors that shrank by more than half, and one patient had their tumor completely disappear.

Mackall discusses the results of the clinical trial as part of the Closer to Cures speaker series. Learn more: https://bit.ly/4b4epWV

02/26/2026

CIRM clinical trial update: For people living with epilepsy and their families, seizures—even if they are rare—can be debilitating and profoundly disruptive.

Epileptic seizures can impair cognition and memory, and not knowing when the next seizure may strike can force people living with this disease to give up certain activities, including driving, working, and hobbies.

CIRM has funded epilepsy research since the early 2000s, leading to a series of discoveries that turn embryonic stem cells into a type of brain cell that calms surrounding neurons.

Preliminary results from two CIRM-funded clinical trials led by Neurona Therapeutics show that a new possible therapy, called NRTX-1001, has minimal side effects and patients have fewer seizures.

Some of the patients treated are now seizure-free.

Recently, Cory Nicholas, PhD, the CEO and co-founder of Neurona Therapeutics, discussed the results of Neurona’s clinicals on epilepsy as part of the Closer to Cures speaker series, highlighting progress of CIRM-funded clinical trials.

Read the full story: https://bit.ly/4tWzidS

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.