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Gene Therapy for Alpha 1- Antitrypsin Deficiency
Sponsor: Weill Medical College of Cornell University
Summary
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.
Key Details
Gender
All
Age Range
18 Years - 70 Years
Study Type
INTERVENTIONAL
Enrollment
16
Start Date
2025-02-26
Completion Date
2032-08-01
Last Updated
2026-03-13
Healthy Volunteers
No
Conditions
Interventions
AAV8hAAT(AVL)
AAV8hAAT(AVL) gene transfer vector
Locations (1)
WCMC Department of Genetic Medicine
New York, New York, United States