The Hyper IgM Foundation Blog 

NEW YORK, New York — The Hyper IgM Foundation is proud to announce the awarding of a $100,000 research grant to Dr. Caroline Kuo, M.D., at the Regents of the University of California, Los Angeles (UCLA). This major seed funding will support pioneering research titled “Preclinical Development of a Prime Editing-Based Therapy for X-linked Hyper IgM,” aiming to accelerate the development of a safe, durable, and curative treatment for patients living with this severe inherited immunodeficiency.

This award marks a significant milestone, building upon foundational gene-editing research supported by the Foundation since 2017. Dr. Kuo’s newest research utilizes prime editing—a next-generation genome engineering platform capable of correcting disease-causing mutations without introducing double-strand DNA breaks or requiring viral donor templates. By operating without these disruptions, prime editing drastically reduces genotoxic risk, better preserves long-term hematopoietic stem cells, and restores physiological gene regulation under native controls.

Hyper IgM Foundation President Akiva Zablocki, MPH, stated:

“We are thrilled to back Dr. Kuo’s visionary work at UCLA as she transitions toward next-generation prime editing. This grant reflects the Foundation’s critical role as a catalyst for high-impact science at a translational stage. By funding the collection of essential preclinical data, we are helping to pave a clear regulatory path toward first-in-human clinical trials. This is exactly the type of precise, mutation-directed science that can scale to benefit a broad spectrum of our patient community.”

The one-year grant period will fund direct research expenses to generate a critical preclinical data package required for FDA engagement and Investigational New Drug (IND)-enabling activities. Crucially, the project will screen and optimize prime editing reagents for a panel of nine distinct, patient-specific CD40L mutations, making future clinical options adaptable and scalable for a wider patient cohort.

For individuals living with X-Linked Hyper IgM Syndrome (X-HIGM), traditional choices remain dangerously limited. Allogeneic stem cell transplantation is currently the only known curative option, but it poses substantial morbidity and mortality risks and is frequently unfeasible for patients who are older, lack a matching donor, or suffer from pre-existing chronic infections. Most patients face a lifelong reliance on supportive measures like immunoglobulin replacement therapy and prophylactic antibiotics, which do not address the underlying genetic cause.

The Hyper IgM Foundation’s investment represents strategic seed funding designed to bridge mechanistic discovery and clinical application. By building a powerful translational pipeline, this research aims to unlock future substantial support from federal agencies and industry partners, moving promising laboratory concepts directly to patients.

About the Hyper IgM Foundation

The Hyper IgM Foundation’s mission is to improve the treatment, quality of life, and long-term outlook for children and adults living with Hyper IgM Syndrome. In addition to funding cutting-edge research, the Foundation connects families affected by Hyper IgM with vital education, advocacy, and peer support. Since its inception, the Hyper IgM Foundation has awarded more than $430,000 in research grants to advance the global understanding and cure of this rare immunodeficiency.

To learn more about Hyper IgM Syndrome or to support the Foundation’s ongoing research initiatives, please visit www.hyperigm.org/donate.

For media inquiries or scientific questions about this grant, please contact info@hyperigm.org.