Gene-Editing shot aims to fix lung and liver damage in rare disease

NCT ID NCT06389877

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

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This study tests a new gene-editing treatment called BEAM-302 for adults with Alpha-1 antitrypsin deficiency, a genetic condition that can cause lung and liver disease. The therapy is given as an infusion and aims to correct the faulty gene. The trial will enroll 106 people to check safety and whether it raises protective protein levels in the blood.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: BEAM-302 (a gene-editing therapy delivered via lipid nanoparticles)
What this could lead to: If it works, this could provide a one-time treatment that corrects the genetic defect, potentially reducing lung and liver damage in people with Alpha-1 antitrypsin deficiency.
What could go wrong: This is an early phase 1/2 trial with only 106 participants, so safety and effectiveness are not yet proven. Gene-editing therapies can have off-target effects or immune reactions, and long-term outcomes are unknown.

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Conditions

The condition(s) this trial relates to.

alpha 1-antitrypsin deficiency

As listed by the trial registrant

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

ALPHA 1-ANTITRYPSIN DEFICIENCY

Contacts and locations

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Study contacts

Contact

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
RECRUITING

Southampton, United Kingdom

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