Gene therapy manufacturing and testing for a rare brain disease (MPS IIIC) to help affected children
Official title: Manufacturing and Analytical Development for JLK-247 Gene Therapy
Funded at Phoenix Nest, INC. · NIH-11315125 · Data updated 2026-11-12
A NIH-funded project aims to develop and refine the manufacturing and analytical processes for a gene therapy intended to help children with MPS IIIC, a rare and progressive neurological disease.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Phoenix Nest, INC. NIH-funded |
| Lab location | 1 site (Brooklyn, United States) |
| Project start | July 14, 2026 |
| Project end | June 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $1,492,329 |
| NIH project number | 1R44NS147802-01 |
| Project ID | NIH-11315125 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children with MPS IIIC or related lysosomal storage diseases who may be eligible for future trials or related studies.
✕Less likely to help
This project focuses on the manufacturing process and may not directly involve patients in the current phase.
What this research studies
This project focuses on creating reliable production and quality control steps for a gene therapy targeting MPS IIIC, a serious childhood disorder. Researchers will develop scalable manufacturing methods and analytical assays to ensure the therapy is safe and effective for patients. The work is designed to prepare for future clinical testing and bring a potential treatment closer to children who currently have few options.
Why it matters
Potential benefit: If successful, the therapy could offer long-term treatment that slows or halts neurological decline in children with MPS IIIC.
How similar studies have gone: Gene therapy approaches for similar lysosomal storage diseases have shown promise in early stages, but this disease-specific therapy is still in development.
Where this research is happening
-
Phoenix Nest, INC.
Brooklyn, United States Active
Researchers
- Principal investigator: Singamsetty, Srikanth — Phoenix Nest, INC.
- Study coordinator: Singamsetty, Srikanth
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.