Gene therapy approach to GSD-1b with a novel G6PT1 promoter-driven AAV vector
Official title: ND Enabling Studies for CMT-101, a Novel Gene Therapy for Glycogen Storage Disease 1b
Funded at Cometa Therapeutics INC · NIH-11467059 · Data updated 2026-11-12
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.
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 | August 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $845,383 |
| NIH project number | 4R42DK141304-02 |
| Project ID | NIH-11467059 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with Glycogen Storage Disease type 1b who might participate in future translational studies or have samples available for research; at this stage, it is preclinical.
✕Less likely to help
Patients with other metabolic diseases or those not represented by the G6PT1 gene therapy approach may not benefit from this project.
What this research studies
Cornela Therapeutics is working on an AAV-based gene therapy for GSD-1b, a rare metabolic disorder that causes dangerous lows in blood sugar. The project focuses on delivering the G6PT1 gene with a promoter that matches human liver biology to boost gene expression and improve metabolic stability. The study involves preclinical testing in mouse models to measure outcomes like blood glucose, neutrophil counts, and organ health, with the goal of informing future human trials. If successful, this work could pave the way for a treatment that reduces hypoglycemia risk and infections, improving quality of life for patients and families.
Why it matters
Potential benefit: If successful, this therapy could stabilize blood sugar and reduce complication risks for people with GSD-1b.
How similar studies have gone: Previous AAV-G6PT1 approaches showed partial rescue in mouse models; this project aims to achieve fuller restoration of survival and metabolic parameters.
Where this research is happening
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Cometa Therapeutics INC
Towson, United States Active
Researchers
- Principal investigator: Hawkins, Al — Cometa Therapeutics INC
- Study coordinator: Hawkins, Al
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.