WIEDEMANN-STEINER SYNDROME FOUNDATION 2025 GRANT PROGRAM - LOI
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The Wiedemann-Steiner Syndrome Foundation will provide 2-year grants to support research related to Wiedemann-Steiner Syndrome. At least 3 awards will be granted.
Background
Wiedemann-Steiner Syndrome (WSS) is a rare genetic disorder resulting from mutations in the MLL gene (also known as KMT2A) on the long arm of chromosome 11. The syndrome’s genetic underpinnings were clarified in 2012 by a group of researchers led by Dr. Wendy Jones. The gene encodes a histone methyl transferase which helps modify the expression of many other genes. The condition is autosomal dominant, and in most cases, the mutation occurred de novo. From an unpublished survey of 76 WSS families, the symptom determined to be of “Very High Impact” by most families, was disruptive behaviors. This included aggressive behaviors, impulsivity, and behaviors outside of age-appropriate societal norms and the most important therapeutic goals were to reduce these behaviors and improve cognitive functioning.
The Orphan Disease Center, in collaboration with the WSS Foundation, is seeking grant applications that aim to further progress the understanding of the disease:
- Two $90,000 grants to support the development and characterization, for the advancement toward therapeutic goal, of patient-derived cell models of WSS Syndrome (ex. iPSCs, neurons, reporter lines). These cells model will be deposited into a publicly accessible biobank.
- One $90,000 grant to support a Natural History Study. Proposals for this grant should focus on understanding the progression of the condition over time, often to support product development, especially for rare diseases. These studies are observational and aim to identify factors that influence disease development and outcomes.
Eligibility
All individuals holding a faculty-level appointment at an academic institution or a senior position at a non-profit institution or foundation are eligible to respond to this RFA.
Letter of Interest Instructions
All applicants must first submit a Letter of Interest (LOI) to be reviewed for consideration of a full application submission. Please submit your LOI by August 8th, 2025.
Format for the 1‐page LOI:
- Project Title
- PI and Co‐PIs, and associated institutions or organizations
- Overall goal of the project
- Why application is responsive to the RFA: please note that the applicant will not be invited to submit a full application if relevance to the RFA is not clearly described in the LOI
- Brief background including relevant literature.
- Specific Aims provided as a brief list
- Specify the variant(s) and/or cell type(s) to be studied, a high-level project timeline and budget
- Requested resources should be in terms of up to 2 years of funding and total award amount – up to $90,000 (including direct and indirect costs). It is strongly preferred that applicant institutions waive indirect costs allowing the total amount to go towards research, but indirect costs up to a maximum of 10% could be accepted.
LOI Due Date: LOI document is to be uploaded no later than 5pm (ET) on August 8, 2025.
Applicants will be notified via email with a decision regarding their LOI, which, if successful, will invite the applicant to submit a full application.
Please review RFA here before applying.