Apply to trial NCT06996756

A few quick questions so the study team can decide if you might be a fit.

RecruitingPhase 1Biological intervention

Gene Therapy for Alpha 1- Antitrypsin Deficiency

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.

How this works

  1. Answer a few questions

    About 5 to 10 minutes. Skip-friendly where possible.

  2. We connect you with the study team

    When a site picks up your application, we introduce you to their team directly — with you on the thread.

  3. The team reaches out to schedule screening

    Usually within a few business days, via the contact you give.

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