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Alpha-1 Antitrypsin Deficiency (AATD)

2
Pipeline Programs
5
Companies
5
Clinical Trials
1 recruiting
0
Approved Products

Pipeline by Development Stage

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

Drug Modality Breakdown

Small Molecule
1100%
+ 4 programs with unclassified modality

Competitive Landscape

5 companies ranked by most advanced pipeline stage

CRISPR Therapeutics
1 program
1
CTX460Phase 1/21 trial
Active Trials
NCT07850076Not Yet Recruiting68Est. Jun 2030
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
NCT07227207Recruiting72Est. 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
2030
Mereo BioPharmaAlvelestat
CRISPR TherapeuticsCTX460
Tessera TherapeuticsTSRA-196
WaVe Life SciencesWVE-006
ChiesiPulmonary Rehabilitation

Clinical Trials (5)

Total enrollment: 300 patients across 5 trials

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

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

A Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of CTX460 in Adults With Emphysema Associated With Severe Alpha-1 Antitrypsin Deficiency (AATD)

Start: Oct 2026Est. completion: Jun 203068 patients
Phase 1/2Not Yet Recruiting

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

Start: Apr 2026Est. completion: Mar 202972 patients
Phase 1/2Recruiting

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

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

1 actively recruiting trials targeting 300 patients
5 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.