Gene therapy breakthrough aims to control rare metabolic disease

NCT ID NCT06735755

Recruiting now Disease control Sponsor: Beam Therapeutics Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a new gene therapy called BEAM-301 in 36 adults with glycogen storage disease type Ia (GSDIa) who have a specific gene change. The goal is to see if a single dose can safely improve blood sugar control and reduce the need for cornstarch supplements. Participants will be monitored for side effects and changes in their condition over time.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

glycogen storage disease due to glucose-6-phosphatase deficiency type IA

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

GLYCOGEN STORAGE DISEASE TYPE IA

Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Clinical Study Site
RECRUITING

Orange, California, 92868, United States
Clinical Study Site
RECRUITING

Farmington, Connecticut, 06030, United States
Clinical Study Site
RECRUITING

Houston, Texas, 77030, United States