NtBuHA therapy for CLN1 Batten disease
Official title: Development of N-tert-(butyl)hydroxylamine (NtBuHA) as a Therapeutic Agent for Treating CLN1 Batten Disease
Funded at Circumvent Pharmaceuticals, INC. · NIH-11399162 · Data updated 2026-11-12
A new drug candidate, NtBuHA/CIRC827, is being developed to treat CLN1 Batten disease in children and teens.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Circumvent Pharmaceuticals, INC. NIH-funded |
| Lab location | 1 site (Portland, United States) |
| Project start | September 20, 2021 |
| Project end | June 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $1,219,273 |
| NIH project number | 5R44NS120360-04 |
| Project ID | NIH-11399162 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and adolescents diagnosed with CLN1 Batten disease who could be eligible for future clinical trials.
✕Less likely to help
Patients without Batten disease or those not eligible for future trials may not benefit from this specific therapy development.
What this research studies
This project aims to move NtBuHA from the lab toward clinical use for CLN1 Batten disease by completing safety studies and regulatory filings. Researchers will test how the drug is absorbed, distributed, metabolized, and excreted, plus its safety in animals and models that mirror the human condition. The team will prepare IND and CTA submissions so doctors can study the therapy in people in the future. If successful, this could shift care from palliative approaches to a disease-modifying option for Batten patients.
Why it matters
Potential benefit: If successful, the research could lead to a disease-modifying treatment that targets the underlying cause of CLN1 Batten disease.
How similar studies have gone: Prior drug development efforts in Batten disease have shown promise in preclinical stages but require human trials to confirm benefit.
Where this research is happening
-
Circumvent Pharmaceuticals, INC.
Portland, United States Active
Researchers
- Principal investigator: Chelsky, Daniel — Circumvent Pharmaceuticals, INC.
- Study coordinator: Chelsky, Daniel
Related research
Other NIH-funded projects on the same topics.
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CIRC827 (NtBuHA) — a potential treatment for CLN1 Batten disease
This project develops CIRC827 (NtBuHA), a new medicine aimed at treating children with CLN1 Batten disease.
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Developing a new treatment for Infantile Neuronal Ceroid Lipofuscinosis (INCL)
This study is testing a new treatment called NtBuHA for children with Infantile Neuronal Ceroid Lipofuscinosis (INCL), a serious brain disorder, to see if it can help improve their health and quality of life.
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.