A new gene therapy for Glycogen Storage Disease 1b
Official title: ND Enabling Studies for CMT-101, a Novel Gene Therapy for Glycogen Storage Disease 1b
Funded at Cometa Therapeutics INC · NIH-11067362 · Data updated 2026-11-12
This study is testing a new gene therapy to help people with Glycogen Storage Disease 1b (GSD-Ib) by using a special delivery method to improve their blood sugar control and overall health.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cometa Therapeutics INC NIH-funded |
| Lab location | 1 site (Towson, United States) |
| Project start | June 1, 2025 |
| Project end | May 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $299,648 |
| NIH project number | 1R42DK141304-01A1 |
| Project ID | NIH-11067362 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Glycogen Storage Disease 1b.
✕Less likely to help
Patients with other types of glycogen storage diseases or those without a confirmed diagnosis of GSD-Ib may not benefit from this research.
What this research studies
This research focuses on developing a novel gene therapy using an AAV vector to treat Glycogen Storage Disease 1b (GSD-Ib), a rare metabolic disorder caused by mutations in the glucose-6-phosphate transporter gene. Patients with GSD-Ib struggle to maintain normal blood sugar levels, leading to severe hypoglycemia and other serious health issues. The therapy aims to enhance gene expression and restore metabolic functions by utilizing a human G6PT gene promoter. Preliminary studies in animal models have shown promising results, indicating potential for improved patient outcomes.
Why it matters
Potential benefit: If successful, this research could significantly improve blood sugar regulation and overall health for patients with GSD-Ib.
How similar studies have gone: Other research in gene therapy for metabolic disorders has shown success, suggesting a promising outlook for this approach.
Where this research is happening
-
Cometa Therapeutics INC
Towson, United States Active
Researchers
- Principal investigator: Hawkins, Al — Cometa Therapeutics INC
- Study coordinator: Hawkins, Al
Related research
Other NIH-funded projects on the same topics.
-
Gene therapy approach to GSD-1b with a novel G6PT1 promoter-driven AAV vector
A new gene therapy approach aims to restore normal blood sugar in people with Glycogen Storage Disease type 1b by delivering the G6PT1 gene using an AAV vector.
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.