RNA editing drug WVE-006 enters first human safety trial

NCT ID NCT06186492

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

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for ALPHA-1 ANTITRYPSIN DEFICIENCY (AATD) are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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 (AATD)

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.