RNA editing drug hopes to fix genetic flaw in rare lung and liver disease
NCT ID NCT06405633
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tests an experimental drug called WVE-006 in 24 people with a severe form of alpha-1 antitrypsin deficiency (Pi*ZZ). The drug is designed to edit RNA to help the body produce a working version of the protective protein. The main goal is to check safety, but researchers will also measure whether the drug raises 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
WVE-006 (RNA editing oligonucleotide)
What this could lead to
If successful, this could point toward a treatment that corrects the genetic defect in alpha-1 antitrypsin deficiency, potentially reducing lung and liver damage.
What could go wrong
This is a very early, small study (24 people) focused on safety. It may not show enough benefit or could have side effects. Even if safe, much larger trials are needed to prove it works.
Disclaimer
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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.
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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
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Locations
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Cambridge University - Addenbrooke's Hospital
Cambridge, CB2 0QQ, United Kingdom
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Dalhousie University - Queen Elizabeth II Health Sciences Centre
Halifax, Nova Scotia, B3H 1V7, Canada
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Inspiration Research Limited
Toronto, Ontario, M5T 3A9, Canada
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Queen Elizabeth Hospital, University Hospital Birmingham
Birmingham, B15 2GW, United Kingdom
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Royal Free London NHS Foundation Trust
London, SW8 3RN, United Kingdom
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St. Vincent's Hospital, Melbourne
Fitzroy, Victoria, 3065, Australia
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Turku University Hospital
Turku, 20520, Finland
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Universitaetsklinikum Aachen, AoeR
Aachen, 52074, Germany
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Waikato Hospital
Hamilton, 3204, New Zealand