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RNA editing drug hopes to fix genetic flaw in rare lung and liver disease

NCT ID NCT06405633

Disease control Sponsor: Wave Life Sciences USA, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.
Why investors are watching

Wave Life Sciences is testing WVE-006 in 24 people with alpha-1 antitrypsin deficiency, a genetic condition that can damage the lungs and liver. This early-stage study checks whether the drug is safe and how it behaves in the body, and a clear result matters because the company is small and this is one of its main experimental programs.

If it works: If the drug proves safe and shows signs of working, Wave could move it into larger studies and build confidence in its pipeline. That could strengthen the company's position with partners or investors.

If it fails: Early-stage trials often fail on safety or effectiveness, and this one has only 24 participants, so results may be unclear. A poor outcome or delay could hurt the company's prospects, since it has few other late-stage assets.

AI-written from the trial record. Speculative, and not investment advice.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 24 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2024

Expected to finish

Sep 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 70 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Healthy as determined by the Investigator, based on a medical evaluation, or has mild to moderate AATD-induced lung disease (FEV1 ≥50%) and/or stable mild AATD-induced liver disease (≤F2 (≤10 kPa) on FibroScan. * Genetic testing confirming Pi\*ZZ. * Participant has been a non-smoker for at least 1 year prior to screening. Exclusion Criteria: * • Participant has a history of multiple drug allergies or of allergic reaction to an oligonucleotide or to N-acetylgalactosamine (GalNAc). * Participant has a history of intolerance or any medical condition that might interfere with subcutaneous injections. * Any ongoing or recent infections. * Any recent or planned vaccinations during the study. * Participant has a history of regular alcohol consumption exceeding 14 standard drinks/week. * Unwilling to abstain from alcohol for 48 hours prior to dosing at each of the dosing visits. * Any recent or planned major surgery during the study. * Participant has any medical condition or social circumstance that, in the opinion of the Investigator, would make the participant unsuitable for participation in the study or for dosing on Day 1, or could interfere with the assessments of safety, pharmacodynamics, or pharmacokinetics, or completion of the study. * Participant currently on AAT augmentation therapy, planned to be on augmentation therapy anytime during the study, or has been on augmentation therapy within 30 days prior to Screening Visit. * Donation of blood or blood products in excess of 500 mL within 12 weeks prior to Screening Visit and/or unwilling to refrain from blood donation for the duration of the study. * Participant has received an investigational agent within 3 months of the Screening Visit.

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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

Locations

  • Cambridge University - Addenbrooke's Hospital

    Cambridge, CB2 0QQ, United Kingdom

  • Dalhousie University - Queen Elizabeth II Health Sciences Centre

    Halifax, Nova Scotia, B3H 1V7, Canada

  • Inspiration Research Limited

    Toronto, Ontario, M5T 3A9, Canada

  • Queen Elizabeth Hospital, University Hospital Birmingham

    Birmingham, B15 2GW, United Kingdom

  • Royal Free London NHS Foundation Trust

    London, SW8 3RN, United Kingdom

  • St. Vincent's Hospital, Melbourne

    Fitzroy, Victoria, 3065, Australia

  • Turku University Hospital

    Turku, 20520, Finland

  • Universitaetsklinikum Aachen, AoeR

    Aachen, 52074, Germany

  • Waikato Hospital

    Hamilton, 3204, New Zealand

More trials for these conditions

Other studies related to the condition(s) this trial covers.