Interested in Applying for Funding to Research Sanfilippo Syndrome?
Are you a researcher who is interested in collaborating with and/or applying for research funding from Cure Sanfilippo Foundation to study Sanfilippo syndrome?
Cure Sanfilippo Foundation seeks to support research that fills critical gaps in current knowledge and access to treatment across basic and translational science, clinical care, and therapeutic interventions in clinical trials. Our overarching goal is to advance a pipeline of scientific discovery which will improve the lives of children with Sanfilippo syndrome. Scientist-clinician-patient collaborations are highly encouraged and an area of active engagement for the Foundation.
Dissemination of research findings through publications and presentations are required responsibilities of funded applicants. Sharing of data for broad scientific advancement is especially critical in the context of ultra-rare diseases like Sanfilippo syndromes. Applicants are to include a data sharing plan and informed consent language which enables data/biological sample sharing for all clinical study proposals. You may also learn more about the Foundation’s efforts around data-sharing.
Funding Mechanisms
Cure Sanfilippo Foundation held an open call for research proposals. Letters of Intent (LOI) were open for submission from Feb. 14 to March 28, 2025.
Proposals were submitted under the following mechanisms:
- Pilot Awards
- ADVANCE Awards
All funding awards are contingent upon alignment with the Foundation’s goals and priorities as informed by recognized gaps in clinical care and/or scientific knowledge which present significant barriers to the development of beneficial therapies and improved quality of life for affected individuals. Funding levels and award categories may vary over time to adapt to the ongoing funding commitments by the Foundation and availability of resources. Award funding is milestone driven and proposals should be written with clear milestones.
Who could apply:
- Academic investigators
- For-profit entity investigators
Priority areas
Priority areas of interest for this submission period include proposals seeking collaborative research with Cure Sanfilippo Foundation to:
- Advance the clinical understanding, diagnosis, measurement tools and management of movement disorders in individuals with Sanfilippo syndromes.
- Evaluate the feasibility of biomarkers related to mitochondrial function, oxidative stress, neurotransmitter profiles, autophagy, mitophagy functions in humans with Sanfilippo syndromes. Preference for clinically-available laboratory tests.
- Increase the evidence base of behavioral therapy (often referred to as Applied Behavioral Analysis) in individuals with Sanfilippo syndromes. May include case series which report on longitudinal data and validated assessment tools.
- Case studies comparing outcomes and/or biomarkers between siblings who have received enzyme restorative therapy vs an untreated sibling.
- Advance innovative translational research for Sanfilippo syndrome which include a plan to begin a clinical trial within 2 years. May include combination therapies. May be subtype specific or basket studies.
Proposals outside these priority areas are also welcome.
Indirect policy
The maximum allowable indirect costs/overhead can equal no more than 5% of the total approved grant amount, and are not to exceed $10,000 regardless of total approved funding amount.
If you would like to consult with the Foundation’s Chief Science Officer about your research ideas, we welcome you to reach out regardless of grant round timing. Please email Dr. O’Neill at Research@CureSanfilippoFoundation.org.
General Funding Mechanisms
Pilot Funding Mechanism
Up to $50,000 USD per year (total costs), up to two years
Year 2 funding will be dependent upon progress demonstrated in Year 1. Funding is milestone driven.
ADVANCE Sanfilippo Funding Mechanism
$51,000 USD or more per year (total costs)
This funding opportunity is intended for proposals with a larger scope of work and funding needs. Funding is milestone driven.
Fellowship Award Mechanism
$60,000 USD per year (total costs), for up to two years. Renewal application is required for Year 2 funding, if applicable.
This award is highly competitive and need-based.
The Foundation’s aim in supporting trainees is to build a pipeline of talent among early career scientists and clinicians focused on Sanfilippo syndrome from within and outside of the field of lysosomal diseases.
A Letter of Intent is not required in order to apply.
For Questions
If you have questions about the Foundation’s funding opportunities through Cure Sanfilippo Foundation, please email us at Research@CureSanfilippoFoundation.org.