RNA editing drug WVE-006 enters first human safety trial
NCT ID NCT06186492
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This Phase 1 trial tested WVE-006, an RNA editing drug, in 47 healthy volunteers to see if it is safe and how the body processes it. The study is the first step toward a possible treatment for Alpha-1 antitrypsin deficiency, a genetic condition that can cause lung and liver disease. Participants received either a single dose or multiple doses of the drug or a placebo.
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 lead to a treatment that corrects the genetic defect in Alpha-1 antitrypsin deficiency, potentially reducing lung and liver damage.
- What could go wrong
- This is an early Phase 1 trial in only 47 healthy volunteers, not patients. It only tests safety and dosing, not whether the drug works for the disease. Many drugs fail in later stages.
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
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
-
47 people
The number who actually took part.
- Started
-
Nov 2023
- Finished
-
Feb 2025
- 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 65 years
- Sex
-
Anyone
- Healthy volunteers
-
Accepted
You do not need to have the condition being studied to take part.
Show the full entry requirements Hide 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. * Genetic testing confirming PI\*MM. * 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. * Participant has a history of caffeine consumption exceeding 8 cups of coffee/day. * Use of prescription or non-prescription medications, including vitamin, dietary, and herbal supplements (including St John's Wort) within 7 days prior to the first dose of study treatment unless, in the opinion of the Investigator and Sponsor, the medication will not interfere with interpretation of study assessments. Contraception and hormone replacement therapy (HRT) are permitted. If needed, over-the-counter (OTC) medications such as paracetamol/acetaminophen may be used acutely. * Any recent or planned major surgery during the study. * 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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Simbec-Orion Clinical Pharmacology,
Merthyr Tydfil, Wales, CF48 4DR, United Kingdom
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can a smartphone breathing game help Alpha-1 patients breathe easier?
- Gene-Editing shot aims to fix rare lung and liver disease
- Hidden gene behind breathlessness? study tests link to rare lung condition
- New drug YOLT-202 tested in early trial for rare lung and liver disease