The Batten Disease Global Research Initiative Grant Program aims to invest in the most promising research worldwide that addresses key research questions and areas of unmet need in Batten disease.

This may include, but is not limited to, one or more of the Top Research Priority questions for Batten disease, as identified in the worldwide Research Priority Setting Partnership survey conducted by the Batten Disease Clinical Research Consortium and published in The Lancet Neurology: Research priorities for the neuronal ceroid lipofuscinoses. Augustine, E., et al. 2025 Oct;24(10):811-812..

Top Research Priorities for Batten Disease

1. What are the best gene-targeted therapy options for Batten disease? How can this research be accelerated?
2. What are valid, reliable biomarkers that can inform Batten disease diagnosis, prognosis, or treatment response?
3. What impact does Batten disease have on organs and body systems outside the central nervous system?
4. Can disease progression be halted or reversed?
5. How does Batten disease impact cognition and behavior?  What are the best treatment strategies for these symptoms?
6. What strategies can be used to enhance early diagnosis (including newborn screening)? What are the impacts of early detection and diagnosis?
7. What interventions are best for maintaining function in Batten disease (including communication, learning, motor skills, mobility, etc.)?
8. Which therapies have the potential for the fastest translation from bench to bedside? How do we accelerate therapy development?
9. What can be learned about Batten disease from similar lysosomal disorders? Which disease processes represent shared therapeutic targets?
10. Which complementary health approaches (including diet, supplements, exercise, etc.) are helpful for improving symptoms or slowing progression in Batten disease?
11. What are the common causes and signs of pain and distress in the NCLs, and how do we reliably measure, report, and treat pain and distress?
12. What are the optimal clinical trial designs for NCLs?

Grants of up to USD $50,000 will be awarded for one year, with the potential of renewal based on outcomes in the first year. Exceptional applications may be funded at a higher amount if the budget request is justified.

Funding will be provided by the Global Research Initiative, with financial contributions made by core member organizations BDSRA Foundation, BDSRA Canada, and BDSRA Australia, with in-kind support from BDFA. In addition to the collective funding provided by these organizations, co-funding support may be sought from other interested parties. Additional organizations or individuals who choose to co-fund specific projects under the Global Research Initiative Grant Program will be recognized as Global Research Initiative Funding Partners (find out more under FUNDING PARTNERS below).

The deadline for Expressions of Interest (EOI) for the second BDGRI Grant Round has passed. Sign up below to stay informed and receive updates as they become available.

The Batten Disease Global Research Initiative is proud to showcase the results of its inaugural round, announced in March 2025. Read more about the funded projects here.