CIRM-backed gene therapy developed at UCLA receives FDA approval

The FDA approval of a UCLA-tested gene therapy for a rare childhood immune disorder marks a milestone not only for physician-scientist Dr. Donald Kohn and the families who participated in the clinical trial, but also for the California Institute for Regenerative Medicine (CIRM) and the clinical infrastructure it has built to help move promising cell and gene therapies toward patients.

In March 2026, the U.S. Food and Drug Administration approved Kresladi, a one-time gene therapy for children with severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disease that leaves children vulnerable to recurrent, life-threatening infections. The approval was based on results from a global Phase 1/2 clinical trial led at UCLA by Kohn, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

Six of the nine children enrolled in the trial were treated at UCLA. All nine were alive one year after treatment without requiring a bone marrow transplant, according to CIRM.

A milestone for CIRM

The approval represents a particularly important milestone for CIRM: Kresladi is the first FDA-approved therapy to emerge from CIRM-supported research and clinical development.

CIRM invested nearly $5.9 million to support the Kresladi clinical trial site at UCLA Mattel Children’s Hospital. The investment helped Kohn and his collaborators test an approach that uses a patient’s own blood-forming stem cells, adding a healthy copy of the ITGB2 gene before returning the corrected cells to the patient. The goal is to enable the body to produce functioning immune cells capable of fighting infection.

The achievement reflects CIRM’s broader strategy of supporting therapies across the long path from laboratory discovery to clinical testing and, ultimately, regulatory approval.

The UCLA Alpha Clinic: building a pathway to patients

That same goal is at the heart of the UCLA Health Alpha Clinic, which was established in 2015 as one of the inaugural sites in CIRM’s Alpha Clinics Network. The Alpha Clinic provides specialized infrastructure and expertise to help UCLA investigators launch and conduct complex cell and gene therapy clinical trials safely and efficiently. During its first five years, the clinic helped advance 28 stem cell and gene therapies into Phase 1 and/or Phase 2 clinical trials. In 2022, UCLA received additional CIRM funding to expand the clinic’s work, including patient access, education, workforce development and community engagement.

While CIRM’s support for the LAD-I trial was provided specifically through its clinical-stage funding, the approval illustrates the larger mission shared by CIRM and the UCLA Alpha Clinic: building the scientific, clinical and operational pathways needed to turn experimental cell and gene therapies into treatments that can reach patients.

For rare diseases such as LAD-I, that infrastructure is especially important. Small patient populations, highly specialized manufacturing and complex clinical care can make these therapies difficult to develop and commercialize even when the underlying science is promising.

Kresladi’s approval demonstrates what can happen when academic research, public investment, clinical infrastructure, patient participation and industry development come together. It also provides a model for the therapies still moving through that pipeline at UCLA and across CIRM’s statewide Alpha Clinics Network.

For more information about the therapy, Dr. Kohn’s clinical trial and the children who participated, read the full story from the UCLA Broad Stem Cell Research Center: FDA approves gene therapy for severe leukocyte adhesion deficiency-I,a rare immune disorder

Image credit: UCLA Broad Stem Cell Research Center