Gene therapy trial aims to fix protein deficiency behind lung and liver disease

NCT ID NCT06389877

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

Summary

This early-stage study is testing a new gene therapy called BEAM-302 in adults with Alpha-1 antitrypsin deficiency, a genetic condition that can damage the lungs and liver. The main goals are to find a safe dose and see if the treatment can increase levels of a missing protective protein in the blood. The study will enroll about 106 participants who have either lung disease, liver disease, or both related to this condition.

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Contacts and locations

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Study contacts

Contact

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

Locations

Clinical Study Center
RECRUITING

Birmingham, Alabama, 35233, United States
Clinical Study Center
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Boston, Massachusetts, 02215, United States
Clinical Study Center
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Charleston, South Carolina, 29425, United States
Clinical Study Center
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Adelaide, Australia
Clinical Study Center
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Fitzroy, Australia
Clinical Study Center
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Dublin, Ireland
Clinical Study Center
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Leiden, Netherlands
Clinical Study Center
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Auckland, New Zealand
Clinical Study Center
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Hamilton, New Zealand
Clinical Study Center
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London, United Kingdom
Clinical Study Center
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Southampton, United Kingdom

Conditions

Explore the condition pages connected to this study.

ALPHA 1-ANTITRYPSIN DEFICIENCY