CIRC827 (NtBuHA) — a potential treatment 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-11253743 · Data updated 2026-11-12
This project develops CIRC827 (NtBuHA), a new medicine aimed at treating children with CLN1 Batten disease.
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 | 2025 |
| Award (this year) | $1,244,790 |
| NIH project number | 2R44NS120360-03 |
| Project ID | NIH-11253743 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and adolescents with genetically confirmed CLN1 (infantile Batten disease) would be the primary candidates for future clinical trials.
✕Less likely to help
People without CLN1 mutations or those with very advanced, irreversible brain damage may not benefit from this therapy.
What this research studies
Researchers are completing laboratory and animal safety studies to prepare the drug CIRC827 (NtBuHA) for testing in people. They are measuring how the drug is absorbed and processed (ADME/PK), running GLP toxicology and safety pharmacology studies, and using human liver cells to check metabolism and possible drug interactions. If those tests are successful, the team will file regulatory applications (an IND in the US and a CTA in the EU) to start clinical trials. The overall aim is to move care away from only supportive measures toward a treatment that addresses the underlying cause of CLN1.
Why it matters
Potential benefit: If successful, CIRC827 could become the first therapy that targets the underlying cause of CLN1 Batten disease rather than only treating symptoms.
How similar studies have gone: There are currently no approved disease-modifying therapies for CLN1, and CIRC827 is a novel candidate that has completed preclinical work but has not yet been tested in people.
Where this research is happening
-
Circumvent Pharmaceuticals, INC.
Portland, United States Active
Researchers
- Principal investigator: Chelsky, Daniel — Circumvent Pharmaceuticals, INC.
- Study coordinator: Chelsky, Daniel
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.