Developing a treatment for CLN1 Batten Disease using enzyme replacement therapy
Official title: Manufacture of an intracerebroventricular Enzyme Replacement Therapy for CLN1 Batten Disease
Funded at Collaborations Pharmaceuticals, INC. · NIH-11051903 · Data updated 2026-11-12
This study is testing a new treatment for young children with CLN1 Batten Disease by delivering a special enzyme directly into their brains to help improve their brain function and overall quality of life.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Collaborations Pharmaceuticals, INC. NIH-funded |
| Lab location | 1 site (Fuquay Varina, United States) |
| Project start | March 8, 2024 |
| Project end | February 28, 2026 |
| Funding year | 2025 |
| Award (this year) | $2,112,893 |
| NIH project number | 5SB1NS135733-02 |
| Project ID | NIH-11051903 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children diagnosed with CLN1 Batten Disease, typically presenting symptoms between 6 and 24 months of age.
✕Less likely to help
Patients with other forms of Batten Disease or those who are not diagnosed with any form of neuronal ceroid lipofuscinosis may not benefit from this research.
What this research studies
This research focuses on creating an enzyme replacement therapy for CLN1 Batten Disease, a severe neurodegenerative disorder affecting young children. The approach involves administering a recombinant enzyme directly into the brain's ventricles to restore enzyme activity that is deficient due to genetic mutations. The study builds on previous findings that showed promising results in animal models, aiming to translate these findings into a potential treatment for affected children. By targeting the underlying cause of the disease, this research seeks to improve neurological function and quality of life for patients.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option that may slow or halt the progression of CLN1 Batten Disease in affected children.
How similar studies have gone: Previous research has shown success with similar enzyme replacement therapies in animal models, indicating potential for effective treatment in humans.
Where this research is happening
-
Collaborations Pharmaceuticals, INC.
Fuquay Varina, United States Active
Researchers
- Principal investigator: Ekins, Sean — Collaborations Pharmaceuticals, INC.
- Study coordinator: Ekins, Sean
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.