Weill Medical College of Cornell University
This is a study of gene therapy to treat alpha 1-antitrypsin (AAT) deficiency. This study aims to treat AAT deficiency with a single administration of AAV8hAAT(AVL), a gene therapy that codes for an oxidation resistant form of the AAT protein, which if safe and if efficacious, will protect the lung on a persistent basis. We hope to learn the safety/toxicity and initial evidence of efficacy of intravenous delivery of this gene therapy to alpha 1-antitrypsin deficient individuals.
Inclusion Criteria: * AAT genotype ZZ, or Z null heterozygotes, and if on augmentation therapy, pre-therapy AAT serum levels \<11 μM * Evidence of mild to moderate disease by the following lung function parameters on PFT and/or chest high resolution computational tomography (HRCT) * Emphysema as assessed by HRCT and/or * Lung function parameters consistent with mild to moderate loss of lung function, in either category 1 or category 2 below: 1. Mild lung dysfunction: FEV1/FVC \< 0.70 and FEV1 ≥ 80% predicted 2. Moderate lung dysfunction: FEV1/FVC \< 0.70 and FEV1 40 - \< 80% pre…
AAV8hAAT(AVL) gene transfer vector
Arctuva estimate
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