California Institute for Regenerative Medicine

05/22/2026

Like many at CIRM, Maria Gonzalez Bonneville's work is personal.

CIRM's vice chair joined the agency in 2011, not long after her father's Parkinson's diagnosis. The job offered her a chance to combine her deep commitment to public service in California with her desire to support this voter-supported effort to accelerate treatments and cures for conditions like her father's.

Maria's role at CIRM now extends to serving as Chair of CIRM's Accessibility and Affordability Working Group, where she's helping ensure that the potentially life-changing therapies that come from CIRM-supported research and clinical trials are accessible to all Californians who need them.

CIRM supports stem cell and genetic research and clinical trials into treatments for hundreds of different conditions, including Parkinson's disease, which affects about a million people in the United States alone.

Maria's work reflects a broader commitment that her work and CIRM's aren't just about finding breakthroughs but about ensuring that patients can access them.

"It isn't just about funding the science," she says. "It's about making sure it gets to people."

Read Maria's story: https://bit.ly/3QZj052

05/20/2026

Clinical trials assess the safety and efficacy of new treatments through a series of phased studies, meticulously monitored and subject to regulatory oversight.

Learn more on our blog: https://bit.ly/4wL1oua

05/19/2026

It is hard to create or identify a life-saving therapy if you are not trained to recognize one. James DeKloe, PhD, a distinguished professor of biotechnology and biomanufacturing at Solano Community College, knows this firsthand, and it is one reason why he spoke so passionately about the importance of the Creating Opportunities through Mentorship and Partnership Across Stem Cell Science (COMPASS) program at Solano in a recent presentation for CIRM’s "Closer to Cures" speaker series.

“I have saved dozens of lives in this year and a half,” said Joel Saenz, a graduate of the COMPASS program at Solano Community College who now works at the CIRM-funded UC Davis Good Manufacturing Practice Facility, which manufactures cell, gene, and drug products, including CAR-T cell therapies for cancer.

“Whether it's someone's mom, dad, brother, sister, whoever it may be, I know that I am able to help with that,” said Joel. “And if it wasn't for CIRM, I can't say that I would have that opportunity and be in the position that I'm in today.”

16 California colleges and universities received COMPASS program grants. This education program pairs undergraduate students with mentors who are experts in stem cell research and related fields to help students gain hands-on experience in the lab.

Students in Solano’s COMPASS program learn about the sciences, work in the lab, and participate in outreach opportunities, including volunteering with local K-12 and adult schools and the NMDP. They also learn about FDA regulations and the business part of the industry.

“Our goal is to make sure that every kid knows that if they want to pursue something more, here is a great opportunity for them to do it, and the lives that you save won't just be the patients that you serve,” said another Solano COMPASS trainee alum, Michael Silva, EdD.

Read the full recap of the presentation on our blog: https://bit.ly/4uivoeW

05/14/2026

Hear how California leads the way in scientific research funding for vital stem cell and gene therapies on the Podcast.

This episode features CIRM's President and CEO, Jonathan Thomas (JT). Beyond highlighting California's unique approach to supporting research, JT highlights CIRM's Alpha Clinics Network, which gives the people most in need of these new therapies access to important clinical trials across the state.

Listen to the full podcast episode: https://bit.ly/4u84YfQ

This episode features our conversation with Jonathan Thomas, PhD, J...

05/11/2026

Helping the people of California live healthier lives is a core part of Maria Gonzalez Bonneville’s work as vice-chair of the CIRM’s governing board and as chair of CIRM’s Accessibility and Affordability Working Group (AAWG).

A critical part of her work is crafting and guiding the AAWG's recommendations to CIRM’s governing board to increase the affordability and accessibility of CIRM-funded treatments and clinical trials.

“This is top of mind for us now across the organization,” said Bonneville. Our goal “isn't just to fund the science, it is to fund the science because it needs to get to people.”

Creating a framework to help deliver newly developed stem cell therapies to the people of California is an important part of CIRM’s mission and legacy as a founder in the field.

“CIRM has been an important part of stem cell research for over 20 years,” said Bonneville. “It helped create an ecosystem in California that otherwise would not have existed… California is amazing. We have the economy and the infrastructure to be able to support something like this. So, if it was going to happen anywhere, it was going to happen here."

Read more of Maria's story: https://bit.ly/3QZj052

04/23/2026

In 2021, CIRM helped fund a clinical trial that involved an experimental stem cell treatment for a frequently paralyzing form of spina bifida.

This clinical trial, called the CuRe Trial, is co-led by Diana Farmer, MD, the surgeon-in-chief at UC Davis Health Children’s Hospital and the chief of pediatric surgery at Shriners Children's Northern California.

Recently, Farmer provided an update on the progress of CIRM-funded clinical trials for an experimental stem cell therapy to treat severe forms of spina bifida.

Currently, the CuRe trial is enrolling participants in a clinical trial that will follow the progress of infants treated with the stem cell therapy from birth to six-years of age. This study will help the researchers better understand the stem cell therapy’s safety and long-term effects.

“We don't [yet] know that wiggling your toes when you're born means you're going to walk when you're two-and-a-half,” said Farmer. “But we are, incredibly optimistic that we might be able to get all these kids out of a wheelchair. And that would be pretty exciting.”

Read the full story: https://bit.ly/496XIIGhttps://bit.ly/496XIIG

04/15/2026

Twenty-one years ago, California taxpayers established CIRM with the idea that investing in regenerative medicine could bring new therapies for untreatable diseases.

CIRM is celebrating the first therapy funded by California taxpayer dollars approved by the FDA.

This new gene therapy, called KRESLADI™, is a one-time treatment developed by Rocket Pharmaceuticals, Inc. to treat the rare childhood disease Leukocyte Adhesion Deficiency-1 (LAD-1).

LAD-1 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.

Previously, children with severe LAD-1 were treated with bone marrow transplants. But matching bone marrow donors can be challenging to find quickly, and the therapy can have serious long-term complications. KRESLADI™ corrects the defective gene in the patient’s own blood-forming stem cells, restoring their body’s ability to make healthy white blood cells that fight infections.

In the clinical trial of KRESLADI™, the therapy was so successful that it restored immune function in all nine clinical trial participants.

Learn more: https://bit.ly/4ta5sRP

04/13/2026

Join us April 24 at 10 AM (PT) for the webinar Transforming Rare Disease Research: Hope and Impact. Discover recent advances in the field, as well as CIRM programs intended to accelerate the development of new rare disease therapies.

Learn about CIRM’s new RAPID program and hear from Stephanie Cherqui, PhD, of the Gene Therapy Initiative at UCSD, on breakthroughs that change lives. Don’t miss this inspiring event.

Register here: https://cirm-ca.zoom.us/webinar/register/WN_6EhPvYYfR6WNdf5KkblTLQ #/registration

04/03/2026

CIRM's governing board approved over $111 million to support advances in discovery and clinical research for children's rare diseases, vision loss, and treatments for neurodegenerative conditions.

Rosa Canet-Avilés, PhD, CIRM's Chief Science Officer, said, "CIRM helps bridge the chasm between a promising scientific discovery and a powerful therapy for treating disease. That’s why CIRM’s funding and support of the full process from discovery research to clinical trials to delivering the therapy to patients is so vital."

Learn more about the recently approved funding: https://bit.ly/4dtnLwY

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...