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Preclinical and Phase I/II Trials of AAV-AAT Vectors: Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Alpha 1-Antitrypsin (rAAV1-CB-hAAT) Gene Vector to AAT-Deficient Adults

Trial Profile

Preclinical and Phase I/II Trials of AAV-AAT Vectors: Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Alpha 1-Antitrypsin (rAAV1-CB-hAAT) Gene Vector to AAT-Deficient Adults

Status: Completed
Phase of Trial: Phase I

Latest Information Update: 20 Dec 2016

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At a glance

  • Drugs AGTC 0106 (Primary)
  • Indications Alpha 1-antitrypsin deficiency
  • Focus Adverse reactions; Pharmacogenomic; Pharmacokinetics
  • Most Recent Events

    • 31 Aug 2018 Biomarkers information updated
    • 05 May 2016 Results published in the media release from American Society of Gene Therapy.
    • 05 May 2016 According to a media release from American Society of Gene Therapy, results from this trial were presented in a podium session at the American Society of Gene and Cell Therapy (ASGCT) 19th Annual Meeting.
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