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Alpha-1 Antitrypsin Deficiency (AATD)

1
Pipeline Programs
4
Companies
4
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
1100%
+ 3 programs with unclassified modality

Competitive Landscape

4 companies ranked by most advanced pipeline stage

WaVe Life Sciences
WaVe Life SciencesJapan - Kagoshima
1 program
1
WVE-006Phase 11 trial
Active Trials
NCT06186492Completed47Est. Feb 2025
Chiesi
ChiesiBrazil - Santana de Parnaíba
1 program
Pulmonary RehabilitationN/A1 trial
Active Trials
NCT07242079Completed50Est. Jun 2025
Tessera Therapeutics
Tessera TherapeuticsMA - Somerville
1 program
TSRA-196PHASE_1_21 trial
Active Trials
NCT07227207Not Yet Recruiting72Est. Mar 2029
Mereo BioPharma
Mereo BioPharmaUK - London
1 program
AlvelestatPHASE_2Small Molecule1 trial
Active Trials
NCT03679598Completed63Est. Dec 2023

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
Mereo BioPharmaAlvelestat
Tessera TherapeuticsTSRA-196
WaVe Life SciencesWVE-006
ChiesiPulmonary Rehabilitation

Clinical Trials (4)

Total enrollment: 232 patients across 4 trials

Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency

Start: Apr 2019Est. completion: Dec 202363 patients
Phase 2Completed

A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)

Start: Dec 2025Est. completion: Mar 202972 patients
Phase 1/2Not Yet Recruiting

A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1)

Start: Nov 2023Est. completion: Feb 202547 patients
Phase 1Completed
NCT07242079ChiesiPulmonary Rehabilitation

Effect Of Pulmonary Rehabilitation in Patients With Alpha-1 Antitrypsin Deficiency

Start: Nov 2024Est. completion: Jun 202550 patients
N/ACompleted

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

4 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.