A new gene therapy for Glycogen Storage Disease 1b
ND Enabling Studies for CMT-101, a Novel Gene Therapy for Glycogen Storage Disease 1b
['FUNDING_OTHER'] · COMETA THERAPEUTICS INC · NIH-11067362
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
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | COMETA THERAPEUTICS INC (nih funded) |
| Locations | 1 site (TOWSON, UNITED STATES) |
| Trial ID | NIH-11067362 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Glycogen Storage Disease 1b.
Not a fit: Patients with other types of glycogen storage diseases or those without a confirmed diagnosis of GSD-Ib may not benefit from this research.
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 performed: Other research in gene therapy for metabolic disorders has shown success, suggesting a promising outlook for this approach.
Where this research is happening
TOWSON, UNITED STATES
- 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 below.