RNA editing drug WVE-006 enters first human safety trial

NCT ID NCT06186492

First seen Mar 23, 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.

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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.

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Contacts and locations

Locations

  • Simbec-Orion Clinical Pharmacology,

    Merthyr Tydfil, Wales, CF48 4DR, United Kingdom

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.

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.