Gene-Editing shot aims to fix lung and liver damage in rare disease
NCT ID NCT06389877
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.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for ALPHA 1-ANTITRYPSIN DEFICIENCY are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Clinical Study Center
RECRUITINGBirmingham, Alabama, 35233, United States
-
Clinical Study Center
RECRUITINGBoston, Massachusetts, 02215, United States
-
Clinical Study Center
RECRUITINGCharleston, South Carolina, 29425, United States
-
Clinical Study Center
RECRUITINGAdelaide, Australia
-
Clinical Study Center
RECRUITINGFitzroy, Australia
-
Clinical Study Center
RECRUITINGDublin, Ireland
-
Clinical Study Center
RECRUITINGLeiden, Netherlands
-
Clinical Study Center
RECRUITINGAuckland, New Zealand
-
Clinical Study Center
RECRUITINGHamilton, New Zealand
-
Clinical Study Center
RECRUITINGLondon, United Kingdom
-
Clinical Study Center
RECRUITINGSouthampton, United Kingdom