Gene editing breakthrough targets rare lung and liver disease
NCT ID NCT06389877
First seen Mar 27, 2026 · Last updated May 25, 2026 · Updated 13 times
Summary
This study tests a new gene editing medicine called BEAM-302 in 106 adults with Alpha-1 antitrypsin deficiency, a genetic condition that can cause lung and liver damage. The goal is to see if the treatment is safe and can raise levels of a protective protein in the blood. Participants will receive one dose and be monitored for side effects and protein levels over time.
Disclaimer
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This is a summary of
the original study
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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 Center
RECRUITINGBirmingham, Alabama, 35233, United States
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Clinical Study Center
RECRUITINGBoston, Massachusetts, 02215, United States
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Clinical Study Center
RECRUITINGCharleston, South Carolina, 29425, United States
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Clinical Study Center
RECRUITINGAdelaide, Australia
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Clinical Study Center
RECRUITINGFitzroy, Australia
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Clinical Study Center
RECRUITINGDublin, Ireland
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Clinical Study Center
RECRUITINGLeiden, Netherlands
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Clinical Study Center
RECRUITINGAuckland, New Zealand
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Clinical Study Center
RECRUITINGHamilton, New Zealand
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Clinical Study Center
RECRUITINGLondon, United Kingdom
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Clinical Study Center
RECRUITINGSouthampton, United Kingdom
Conditions
Explore the condition pages connected to this study.