Kabuki Syndrome Foundation Announces 2026 Discovery Program to Enable Gene-Targeting Therapies

The Kabuki Syndrome Foundation (KSF) is excited to announce the launch of the 2026 Discovery Program, a new research funding initiative designed to generate the foundational scientific evidence needed to enable gene-targeting therapies for Kabuki syndrome.

The Discovery Program represents a strategic investment in foundational research that will help bridge the gap between basic science and future treatments. The 2026 program focuses on answering the key biological questions that must be resolved before gene-targeting approaches can be selected and designed.

Particular areas of interest include:

• Dosage threshold — What biological effect is needed?
  What is the minimum increase in gene function required for meaningful rescue in disease-relevant cell types, and what is the upper safety boundary? How long must restoration be maintained for durable rescue, and which deficits remain reversible at postnatal timepoints?
• Variant mechanism — Is upregulation safe?
  Do pathogenic variants act purely through haploinsufficiency, or do some variant classes (particularly missense variants) exert dominant-negative effects on COMPASS complex activity.

Applications that address these will be given priority consideration, although other innovative projects that directly advance gene-targeting readiness are also eligible.

To promote collaboration and accelerate progress, applicants must form multidisciplinary teams representing two or more institutions or organizations. Funded investigators will also have access to KSF’s growing collection of induced pluripotent stem cell (iPSC) models, providing valuable resources to support their research.

The Discovery Program will provide up to $250,000 over two years for projects with clearly defined milestones and translational outcomes.

Application Timeline

The 2026 Discovery Program will follow a two-stage application process:

• Letter of Intent (LOI) deadline: August 3, 2026
• Full application deadline: September 21, 2026
• Funding decisions announced: November 2026

View the Request for Applications (RFA).

Researchers interested in applying are encouraged to begin forming collaborative teams and preparing their Letters of Intent.  

By investing in the foundational science needed to enable gene targeting therapies, KSF continues its commitment to building the research ecosystem that will bring meaningful treatments to individuals and families affected by Kabuki syndrome.

We look forward to partnering with the research community to accelerate the path toward transformative therapies.