This approach recently earned FDA approval for a gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder in children. It also underlies a growing portfolio of trials for blood and immune diseases at UCLA. But of all the diseases Kohn has worked on, sickle cell disease has been the most challenging — and most instructive. Results from a phase 1–2 clinical ... This approach recently earned FDA approval for a gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder in children. It also underlies a growing portfolio of trials for blood and immune diseases at UCLA. But of all the diseases Kohn has worked on, sickle cell disease has been the most challenging — and most instructive. Results from a phase 1–2 clinical trial for sickle cell disease that Kohn led over nearly a decade are detailed in a new study in Blood Advances. Kohn and Prueksapraopong present the abstract for the sickle cell clinical trial at the ASTCT/CIBMTR Tandem Meeting in 2025. Until recently, the only established cure was a bone marrow transplant from a matched donor — a treatment unavailable to most patients, and that carries the risk of graft-versus-host disease. "Because gene therapy uses the patient's own stem cells and modifies them to correct the disease, patients don't need to be on lifelong immunosuppressants," said first author Dr. Clinical trial yields insights about the role of chemotherapy, inflammation and trial design that could help shape next-gen curative therapies. Results from a UCLA-led clinical trial for sickle cell disease reveal the unique challenges of treating blood disorders with gene therapy and provide crucial insights into improved clinical trial design for blood diseases.

This approach recently earned FDA approval for a gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder in children. It also underlies a growing portfolio of trials for blood and immune diseases at UCLA. But of all the diseases Kohn has worked on, sickle cell disease has been the most challenging — and most instructive. Results from a phase 1–2 clinical ... This approach recently earned FDA approval for a gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder in children. It also underlies a growing portfolio of trials for blood and immune diseases at UCLA. But of all the diseases Kohn has worked on, sickle cell disease has been the most challenging — and most instructive. Results from a phase 1–2 clinical trial for sickle cell disease that Kohn led over nearly a decade are detailed in a new study in Blood Advances. Kohn and Prueksapraopong present the abstract for the sickle cell clinical trial at the ASTCT/CIBMTR Tandem Meeting in 2025. | Courtesy of Chattip Prueksapraopong, M.D. Until recently, the only established cure was a bone marrow transplant from a matched donor — a treatment unavailable to most patients, and that carries the risk of graft-versus-host disease. "Because gene therapy uses the patient's own stem cells and modifies them to correct the disease, patients don't need to be on lifelong immunosuppressants," said first author Dr. Results from a UCLA-led clinical trial for sickle cell disease reveal the unique challenges of treating blood disorders with gene therapy and provide crucial insights into improved clinical trial design for blood diseases. Clinical trial yields hard-won insights about the role of chemotherapy, inflammation and trial design — which could help shape next-generation curative therapies · Left: An AI-generated illustration of red blood cells, including sickled forms, to represent a blood sample from a patient with sickle cell disease.

FDA approves Kresladi, a one-time gene therapy for leukocyte adhesion deficiency type I (LAD-I), developed by Rocket Pharmaceuticals. This marks the first gene therapy for this rare inherited immune disorder caused by mutations in the ITGB2 gene, addressing a critical unmet need in pediatric care.

Established in 2004 to accelerate ... clinical trials for the gene therapy in collaboration with Rocket and is now celebrating the first-ever FDA approval made possible through its support. Kohn is optimistic that this approval will encourage more companies to develop treatments for other rare diseases... Established in 2004 to accelerate stem cell therapies, CIRM co-funded clinical trials for the gene therapy in collaboration with Rocket and is now celebrating the first-ever FDA approval made possible through its support. Kohn is optimistic that this approval will encourage more companies to develop treatments for other rare diseases. Severe leukocyte adhesion deficiency-I is a rare genetic disease that prevents white blood cells from functioning normally, leaving affected children susceptible to recurrent, life-threatening infections. In a UCLA clinical trial, Dr. Donald Kohn treated young patients using a gene therapy that adds a healthy copy of the gene to the patients’ own blood stem cells. The therapy, now approved by the FDA under the accelerated approval pathway, could provide a blueprint for developing and commercializing therapies for other rare diseases. Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role in a clinical trial culminated in the first-ever U.S. Without treatment, survival beyond childhood is rare. Approval of the therapy, marketed under the name Kresladi, was based on the results of a clinical trial led at UCLA by Kohn, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research.