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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- AI model could predict who needs a lung transplant for rare emphysema
- Could a simple shot replace IV drips for Alpha-1 patients?
- Hidden liver harm: study tracks silent damage in genetic disorder
- Promising liver drug trial halted early: what it means for patients
- Gene-Editing shot aims to fix lung and liver damage in rare disease
- New drug AIR-001 enters first human tests for rare lung condition