Gene Therapy Pioneer to Headline Global Talk on Curing Immune Disorders

NEW YORK, NY – February 11, 2026 – The Jeffrey Modell Foundation (JMF) is set to spotlight the cutting edge of genetic medicine, announcing that renowned immunologist Dr. Donald B. Kohn will lead a virtual discussion on gene therapy for Primary Immunodeficiency (PI) on March 25, 2026. The event, part of the foundation's global JMF Speaker Series, aims to bring revolutionary scientific insights directly to the clinicians, researchers, and patient families on the front lines of this life-threatening condition.

Dr. Kohn, a Distinguished Professor at the University of California, Los Angeles (UCLA), is a leading figure in the development of treatments that rewrite the very code of disease. His upcoming talk will focus on gene therapy for PI and Inborn Errors of Immunity (IEI), a group of over 450 rare, chronic genetic disorders where a part of the body's immune system is missing or functions improperly, leaving individuals vulnerable to severe and recurrent infections.

A New Horizon in Treatment

For decades, the primary treatments for severe forms of PI were limited to lifelong antibiotic regimens, immunoglobulin replacement therapy, or high-risk bone marrow transplants from a matching donor. While life-saving, these approaches come with significant drawbacks, including the risk of graft-versus-host disease (GVHD) from transplants and the burden of continuous medical intervention. Gene therapy represents a paradigm shift, offering the potential for a one-time, permanent cure.

Dr. Kohn’s work is at the forefront of this revolution. He is best known for developing a successful gene therapy for adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID), a devastating condition often called "bubble baby disease." Infants born with ADA-SCID have virtually no immune system and, without intervention, rarely survive past their first two years.

The process pioneered by Dr. Kohn and his colleagues involves harvesting a patient's own blood stem cells. In the lab, these cells are modified using a disabled lentiviral vector—a sophisticated biological tool—to deliver a healthy, functional copy of the missing or faulty gene. These corrected cells are then infused back into the patient. Because the cells are the patient's own, the risk of rejection and GVHD is eliminated. The corrected stem cells engraft in the bone marrow and begin producing a continuous supply of healthy immune cells, effectively building a new, functional immune system from within. The success of this approach has been profound, with studies reporting that dozens of children with ADA-SCID have been effectively cured, now living healthy, normal lives.

Amplifying Expertise on a Global Scale

The virtual talk is a key component of the JMF Speaker Series, an educational initiative launched in 2025 to connect the global PI community with world-leading experts. By making the event free and accessible worldwide, the foundation aims to democratize knowledge and accelerate the adoption of innovative treatments.

"We're thrilled to have Dr. Kohn as the next expert for our JMF Speaker Series," said Vanessa Tenembaum, Chief Executive Officer of the Jeffrey Modell Foundation, in a statement. "With this event we're amplifying his expertise to clinicians, researchers, patients, and advocates across the globe to transform outcomes and improve quality of life for people living with Primary Immunodeficiency."

The foundation, established in 1987 by Fred and the late Vicki Modell in memory of their son Jeffrey, has been a tireless advocate for the PI community. Its work spans from promoting newborn screening for early diagnosis to funding the research that fuels breakthroughs like those being developed by Dr. Kohn. This speaker series extends that mission, ensuring that the latest scientific advancements are not confined to academic journals but are shared widely to inspire hope and inform care.

A Collaboration of Leaders

The significance of the event is further underscored by the collaboration of several top-tier institutions. Dr. Kohn's groundbreaking research is a product of the robust scientific environment at UCLA's Broad Stem Cell Research Center and Jonsson Comprehensive Cancer Center.

The educational integrity of the talk is certified through a joint providership between the Jeffrey Modell Foundation and The Columbia University Vagelos College of Physicians and Surgeons, which provides Continuing Medical Education (ACCME) accreditation. This ensures that participating physicians and healthcare professionals receive recognized credit for their learning, encouraging broad clinical engagement.

Adding to the depth of expertise, the session will be moderated by Dr. Jordan S. Orange, a leading pediatric immunologist who serves as Pediatrician-in-Chief and Chair of the Department of Pediatrics at Children's Hospital of Philadelphia (CHOP). Dr. Orange's role as moderator will bridge the gap between pioneering research and clinical application, ensuring the discussion addresses the practical questions and concerns of those who treat and live with PI every day. This convergence of research, clinical leadership, education, and patient advocacy highlights a powerful, unified strategy for confronting rare diseases.

While the success in treating conditions like ADA-SCID marks a monumental achievement, the field of gene therapy continues to evolve. Researchers like Dr. Kohn are now applying similar techniques to other immunodeficiencies, such as X-linked SCID and chronic granulomatous disease, and exploring next-generation technologies like CRISPR for even more precise gene editing. The upcoming talk offers a unique opportunity for a global audience to hear directly from a pioneer about the current state of the art and the future promise of a cure for these devastating diseases.