NTSAD Research Initiative Program

Program Focus: Funding research to advance effective treatments and ultimately a cure for Tay-Sachs, Canavan, GM1 gangliosidosis, and Sandhoff diseases.

Established in 2002, the NTSAD Research Initiative Program is dedicated to advancing scientific progress in rare neurodegenerative diseases. Since inception, the program has awarded 76 grants totaling over $5 million in funding. The program supports researchers not only through grant funding but also through letters of recommendation, scientific connections, and cross-field collaboration facilitation.

Program Impact:

• High-impact investments have generated critical data and sustained essential resources
• Many NTSAD-funded studies have produced results that strengthened investigators' ability to secure larger grants from major funders such as the National Institutes of Health
• Some projects have attracted industry partner support
• Supported leading researchers and clinicians across the United States, Canada, Israel, and Europe

Research Areas Supported:

• Understanding disease pathophysiology
• Developing and refining gene therapy
• Other therapeutic strategies development
• Creating clinical rating scales for future trials

Focus Areas for Funded Research:

1. Clinical Trial Readiness
   • Biomarker development
   • Patient-centered outcome measures
2. Advancing Therapeutic Approaches (including genetic medicine)
   • Gene editing
   • Cell therapy
   • Improved delivery systems (LNPs, optimized vectors, BBB crossing)
3. Strategic Priority Areas (supported through advocacy and internal programs)
   • Carrier, prenatal, and newborn screening
   • Patient registry

Eligible Diseases:

• Tay-Sachs Disease
• Canavan Disease
• GM1 Gangliosidosis
• Sandhoff Disease

Grant Award Range:

• One-year grants: $2,000 to $75,000
• Two-year grants: $20,000 to $140,000
• Indirect costs may not exceed 5% of total grant award
• Two-year grants reviewed after first year to assess progress before second-year funding

Geographic Scope: United States, Canada, Israel, and Europe

Recent Major Award (2025):

• In June 2025, NTSAD awarded its largest single research grant to date—$365,000—to support second-generation gene therapy study for GM2 at University of Massachusetts/Chan Medical School, led by Principal Investigator Miguel Sena-Esteves, PhD. This was made possible through collective generosity of NTSAD community and three mission-aligned foundations (Blu Genes Foundation, Cure Tay-Sachs Foundation, and Mathew Forbes Romer Foundation) which collectively awarded $870,000.

Contact Information:

• Research inquiries: research@ntsad.org
• NTSAD Main Address: 2001 Beacon Street, Suite 204, Boston, MA 02135
• Phone: (617) 277-4463
• Email: info@ntsad.org
• Tax ID: #13-1912877

Past Grant Recipients (2022-2025):

• 2024: Amanda Gross (Auburn) & Jessica Larsen (Clemson) - Nanoparticle DIVERT for Tay-Sachs/Sandhoff
• 2024: Angela Gritti (IRCCS) - Enhancing hematopoietic stem cell gene therapy for GM2
• 2023: Amanda Nagy & Florian Eichler (MGH) - Canavan disease neuroimaging characterization
• 2023: Jennifer Kwon & Julie Kissell (UW) - Clinical rating scale for Late-Onset GM2
• 2023: Dominic Gessler & Guangping Gao (UMass) - MRI-based outcome prediction using ML
• 2022: Elise Townsend (MGH) - Infantile GM2 rating scale validation
• 2022: Amanda Gross (Auburn) - Dual site AAV gene therapy for feline GM1
• 2022: Michael Gelb & Hamid Khaledi (GelbChem) - Newborn screening assay development

How to Apply

Application Type: Request for Proposals (RFPs) by invitation only; open year-round

Initial Contact:

1. Contact NTSAD Research Team: Email research@ntsad.org to express interest in applying for funding
2. Schedule Meeting: NTSAD will set up a meeting to discuss your research in detail
3. Discuss Alignment: Explore how your research aligns with NTSAD's mission and strategic priorities

Full Proposal Submission:

1. Prepare Full Proposal: After initial consultation, develop a complete proposal addressing NTSAD's focus areas
2. Submit Proposal: Submit through process discussed with NTSAD research team

Review Process:

• Proposals undergo rigorous review by:
  • NTSAD research team
  • Members of NTSAD's Scientific Advisory Council
  • Expert consultants in the field as needed

Evaluation Criteria:

• Alignment with NTSAD's mission and strategic priorities
• Scientific merit
• Feasibility of proposed research

Approval:

• All grant funding must be approved by NTSAD leadership and the Executive Committee of the board
• Two-year grants reviewed after Year 1 for sufficient progress before Year 2 funding approved

Required Materials:

• Full research proposal with project details
• Alignment statement with NTSAD's mission and strategic priorities
• Budget documentation (indirect costs limited to 5%)
• Curriculum vitae for principal investigator

Contact for Applications:

• Email: research@ntsad.org
• Phone: (617) 277-4463