NTSAD is dedicated to advancing the development of effective treatments—and ultimately a cure—for Tay-Sachs, Canavan, GM1 gangliosidosis, and Sandhoff diseases. The NTSAD Research Initiative Program, established in 2002, helps drive this mission forward by funding research that accelerates scientific progress. In addition to grant funding, NTSAD supports researchers by providing letters of recommendation, facilitating scientific connections, and fostering collaboration across the field.

Since its inception, the Research Initiative Program has awarded 76 grants, providing more than $5 million in funding to advance research in these rare neurodegenerative diseases. These high-impact investments have helped generate critical data, sustain essential resources, and accelerate the development of innovative therapeutic approaches.

Many NTSAD-funded studies have produced promising results that strengthened investigators’ ability to secure larger grants from major funders—such as the National Institutes of Health—and, in some cases, to attract support from industry partners. This catalytic effect continues to amplify the reach of NTSAD’s research dollars and propel the field forward.

NTSAD has supported leading researchers and clinicians across the United States, Canada, Israel, and Europe. Their work spans a wide range of research areas, including advancing our understanding of disease pathophysiology, developing and refining gene therapy and other therapeutic strategies, and creating clinical rating scales essential for future trials.

Investigators working on projects aligned with our mission are encouraged to connect with our research team to learn more about opportunities within the Research Initiative Program.

NTSAD is continuously assessing the ultrarare disease landscape. Together with our medical, scientific, and strategic advisors, we identify the gaps and barriers that need to be addressed most urgently in order to advance our mission. Based on this analysis, we determined our current strategic focus areas for research. They consist of the following categories:

• Carrier, prenatal, and newborn screening
• Patient registry

These two categories will be supported by NTSAD staff through advocacy and internal programs. NTSAD awards research grants for projects in the focus areas listed below.

• Clinical trial readiness, for example:
  • Biomarker development
  • Patient-centered outcome measures
• Advancing therapeutic approaches, including genetic medicine, for example:
  • Gene editing
  • Cell therapy
  • Improved delivery systems (LNPs, optimized vectors, BBB crossing)

Grants typically range from $2,000 to $75,000 for one year, and $20,000 to $140,000 for two years. Two-year grants will be reviewed after the first year to assess sufficient progress before a second year of funding is provided.

Our Request for Proposals (RFPs) is by invitation only and open all year. If you consider applying for funding, please contact us at research@ntsad.org and we will set up a meeting to further discuss your research.

Review mechanism:

Full proposals undergo rigorous review by the NTSAD research team, members of our Scientific Advisory Council, and experts in the field as needed. Proposals are evaluated based on alignment with NTSAD’s mission and strategic priorities, scientific merit, and feasibility. All grant funding must be approved by NTSAD leadership and the Executive Committee of the board.

In June 2025, NTSAD awarded its largest single research grant to date—$365,000—to support the second-generation gene therapy study for GM2 at University of Massachusetts/Chan Medical School, led by Principal Investigator Miguel Sena-Esteves, PhD. This significant funding milestone was made possible through the collective generosity of the NTSAD community, including individuals, families, and NTSAD restricted fund holders who believe in the promise of scientific progress.

NTSAD was also joined by three mission-aligned foundations—the Blu Genes Foundation, the Cure Tay-Sachs Foundation, and the Mathew Forbes Romer Foundation—in collectively awarding a total of $870,000 for this critical study.

NTSAD is proud to have made the leading contribution, reinforcing our commitment to driving forward the most promising science on behalf of the families we serve.

A downloadable list of all grants is available here.