Therapy Development Fund

Therapy Development Fund

Funder: Kabuki Syndrome Foundation

Geographic Scope: International (academic and industry investigators welcome)

Status: Active

Overview

The Therapy Development Fund is designed to bridge the gap between basic science and clinical trials for Kabuki syndrome treatments. Strategic investments target therapies already in development and aim to advance potential treatments from proof-of-concept to clinical trials.

Funding Focus

• Studies to better understand the safety and efficacy of potential treatments
• Research advancing therapies toward clinical trials
• Translation of promising discoveries from lab to patients

Eligibility

• Academic investigators
• Industry investigators

What They Fund

• Safety and efficacy studies of potential treatments
• Proof-of-concept to clinical trial advancement

Background

Rare disease research faces significant hurdles in bridging the gap between basic science and clinical trials. The Therapy Development Fund addresses this critical funding gap. Over $2,000,000 has been secured to support LEAP (the roadmap to Kabuki syndrome treatments) so far. The KSF is the only organization funding the discovery and development of therapeutics for Kabuki syndrome.

Contact

Email: info@kabukisyndromefoundation.org Address: P.O. Box 214, Northbrook, IL 60065 Website: kabukisyndromefoundation.org