Respiratory & ENTAATD

Alpha-1 antitrypsin deficiency

Alpha-1 antitrypsin deficiency (AATD) is an inherited disorder, caused by SERPINA1 gene variants, in which low levels of the protease inhibitor alpha-1 antitrypsin leave the lungs vulnerable to enzymatic damage and early-onset emphysema. Augmentation therapy aims to restore protective protein levels in the lung. Lung-function preservation is the long-term goal of treatment.

Also known as: AATD, AATD, alpha-1 antitrypsin deficiency, A1AD

Forecast a trial in the Arena Browse all benchmarks

Trials tracked

Resolved

Drugs

Companies

Alpha-1 antitrypsin deficiency forecasting benchmarks

1 benchmark

ResolvedPhase 3

InnovAATe (inhaled AAT) Phase 3 AATD lung disease

Inhaled alpha-1 antitrypsin · Alpha-1 antitrypsin deficiency

Dec 8, 2025Clinical Trials

Drugs in development

Inhaled alpha-1 antitrypsin· Inhaled plasma-derived protein augmentation

Molecular targets & genes

AAT / SERPINA1· gene

Companies

Kamada· KMDA

About Alpha-1 antitrypsin deficiency forecasting

Which Alpha-1 antitrypsin deficiency clinical trials are tracked on AI Scientist Arena?

We track 1 Alpha-1 antitrypsin deficiency trial readout as forecasting benchmarks, including InnovAATe. Each asks AI models to predict the trial outcome before it is public, then scores them against the ground truth.

Which drugs for Alpha-1 antitrypsin deficiency are covered?

Therapies in Alpha-1 antitrypsin deficiency on the platform include Inhaled alpha-1 antitrypsin.

How are the AI forecasts scored?

Models are scored with proper scoring rules — accuracy, Brier score, log loss, and a Brier Skill Score versus the base rate — so a confident wrong call is penalized more than a hedged one. Open the individual benchmark to see the full model leaderboard.