UCLA gene therapy offers children with LAD-I new hope

Diagnosed with LAD-I at age eight after years of hospitalizations, Marley Gaskins’ immune system is restored after participating in a CIRM-funded clinical trial.
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CIRM-funded clinical trial gives siblings a second chance at life

The Langenhop children, all diagnosed with LAD-I, now have healthy immune systems after participating in a CIRM-funded clinical trial at UCLA.
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Accelerating Cures for Californians—and Beyond

The California Institute for Regenerative Medicine (CIRM) is a state agency created by California voters to accelerate stem cell and gene therapies for people with unmet medical needs. Since 2004, Californians have entrusted CIRM with $8.5 billion to accelerate promising discoveries through clinical trials, train a regenerative medicine workforce, strengthen the state’s biotechnology economy, and expand access to transformative treatments.

Driving Innovation

Accelerating world class science 

 CIRM is a catalyst for discovery, supporting cutting-edge cell and gene therapies to advance the field of medicine. Through our research and development programs, CIRM continues to accelerate the translation of groundbreaking discoveries into therapies that have the potential to make a real impact on the lives of Californians and patients worldwide.

Building On Discoveries

CIRM supports the full spectrum of research, from early discovery research to clinical trials, while building infrastructure to advance innovation and providing education programs to train the next generation of scientists.

Fueling Growth

Making an impact across California and the world

CIRM drives new possibilities in science and promotes economic growth by creating jobs, funding infrastructure, and growing a diverse workforce to drive progress in the regenerative medicine field.

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Photo Credit: UC Regents

Impacting Communities

Bringing Therapies to Californians 

CIRM is committed to making groundbreaking cell and gene therapy treatments accessible to people who need them.

Funding Progress

To date, CIRM has funded and supported more than 100 cell and gene therapy clinical trials, each aiming to find treatments for diseases and conditions that affect people across California and worldwide.

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CIRM-Funded Clinical Trials

Inspiring The Future

Calling the next generation of scientists

CIRM is passionate about catalyzing growth in regenerative medicine while championing inclusivity. Our education and training programs provide internships and opportunities to inspire scientists and trainees at various levels. 

Stories from The Stem Cellar

CIRM’s official blog

First CA taxpayer-funded therapy approved for rare childhood disorder

The FDA has approved KRESLADI™, a groundbreaking genetic therapy funded by CIRM, to treat severe LAD-I in children, offering a life-saving option where none previously existed.

Closer to Cures: Stanford researcher Crystal Mackall discusses progress on clinical trials for rare cancers of the brain and spine with no known cure.

In 2023, the California Institute for Regenerative Medicine (CIRM) funded a clinical trial led by Stanford researcher Crystal Mackall, MD, to investigate a possible therapy for rare brain and spinal tumors that arise from a specific genetic mutation, known as H3K27M.

CIRM-funded clinical trial gives siblings a second chance at life

The Langenhop children, diagnosed with a rare, life-threatening immune disease, had their immune function successfully restored through a CIRM-funded gene therapy trial by Rocket Pharmaceuticals.