Gene therapy breakthrough for rare metabolic disease
NCT ID NCT06735755
First seen Jan 05, 2026 · Last updated May 11, 2026 · Updated 17 times
Summary
This study tests a new gene therapy called BEAM-301 for adults with a specific form of glycogen storage disease type Ia (GSDIa), a rare genetic disorder that causes dangerously low blood sugar. The treatment aims to fix a faulty gene to help the body better control blood sugar levels. About 36 participants will receive a single dose to see if it is safe and effective.
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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.
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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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Clinical Study Site
RECRUITINGOrange, California, 92868, United States
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Clinical Study Site
RECRUITINGFarmington, Connecticut, 06030, United States
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Clinical Study Site
RECRUITINGHouston, Texas, 77030, United States
Conditions
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