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WVE-006

Phase 1

Alpha-1 Antitrypsin Deficiency | Small molecule | Rare Disease |Wave Life Sciences Ltd.|Last Updated: Apr 14, 2026

Target and mechanism

Molecular targetAAT
Target classProtein
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment24

FDA Designations

ACCELERATED_APPROVAL

Clinical trial landscape

WVE-006 · 2 trials · 2 indications

Phase 1 2
NCT06405633A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATDAlpha-1 Antitrypsin Deficiency
ACTIVE NOT_RECRUITING24 Analytics
NCT06186492A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1)Alpha-1 Antitrypsin Deficiency (AATD)
COMPLETED47 Analytics
PHASE1ACTIVE NOT_RECRUITING
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD
Alpha-1 Antitrypsin DeficiencyUnlock trial analytics
PHASE1COMPLETED
A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1)
Alpha-1 Antitrypsin Deficiency (AATD)Unlock trial analytics

Study Endpoints

Primary Endpoints

The proportion of participants with adverse events
Up to 36 Weeks

Secondary Endpoints

Single Ascending Dose - Change from baseline in levels of serum M-AAT protein.
Up to 12 Weeks
Multiple Ascending Dose - Change from baseline in levels of serum M-AAT protein
Up to 24 Weeks
Single Ascending Dose - Area under the plasma concentration time curve for WVE-006 from time of dosing to the last measurable concentration (AUClast)
Up to 12 Weeks
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
WVE-006 (Dose 1)EXPERIMENTAL -
WVE-006 (Dose 2)EXPERIMENTAL -
WVE-006 (Dose 3)EXPERIMENTAL -
Single Ascending Dose (SAD): WVE-006 30 milligram (mg) or placeboEXPERIMENTAL -
SAD: WVE-006 100 mg or placeboEXPERIMENTAL -
SAD WVE-006 200 mg or placeboEXPERIMENTAL -
SAD: WVE-006 400 mg or placeboEXPERIMENTAL -
SAD: WVE-006 600 mg or placeboEXPERIMENTAL -
Multiple Dose: WVE-006 600 mg Every 2 weeks (Q2W) or placeboEXPERIMENTAL -

Interventions

NameTypeDescription
WVE-006DRUGRNA editing oligonucleotide
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Eligibility Criteria

Age Range18 Years to 70 Years
SexALL
Healthy VolunteersNo
Study Sites9

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-...

Countries:AustraliaCanadaFinlandGermanyNew ZealandUnited Kingdom
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Frequently asked questions about WVE-006

What is WVE-006 used for?

WVE-006 is an investigational small molecule being developed for Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic condition. It is designed to target the AAT protein. The drug is currently in Phase 1 clinical development and has not been approved by the FDA.

What does WVE-006 target?

WVE-006 targets the AAT protein, which is associated with Alpha-1 Antitrypsin Deficiency. By targeting this protein, the drug aims to address the underlying cause of the condition. It is currently being studied in early-stage clinical trials.

Who is developing WVE-006?

WVE-006 is being developed by Wave Life Sciences Ltd., a biopharmaceutical company listed on the stock exchange under the ticker WVE. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational drug for Alpha-1 Antitrypsin Deficiency.

What phase is WVE-006 in?

WVE-006 is in Phase 1 clinical development. It is an investigational drug, meaning it has not yet received FDA approval. The drug is being studied in healthy volunteers and in patients with Alpha-1 Antitrypsin Deficiency to assess its safety, tolerability, and pharmacokinetics.

What clinical trials is WVE-006 in?

WVE-006 is being studied in two clinical trials. The first, NCT06186492, is a completed Phase 1 study in healthy participants with wild-type AAT expression. The second, NCT06405633, is an active Phase 1b/2a study in participants with Pi*ZZ AATD, a specific genotype of the condition.

Is WVE-006 the same as RestorAATion-1?

WVE-006 is the drug being evaluated in the RestorAATion-1 trial, which is a Phase 1 study with the clinical trial identifier NCT06186492. This trial is designed to evaluate the safety, tolerability, and pharmacokinetics of WVE-006 in healthy participants with wild-type AAT expression.