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AGTC 0106

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Drug Profile

AGTC 0106

Alternative Names: AGTC-0106; Alpha-1 antitrypsin deficiency gene therapy - Beacon Therapeutics; rAAV1-CB-hAAT; Recombinant AAV-AAT vector gene therapy - Beacon Therapeutics

Latest Information Update: 29 Jun 2023

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At a glance

  • Originator Applied Genetic Technologies Corporation
  • Developer Beacon Therapeutics; National Heart, Lung and Blood Institute
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Alpha 1-antitrypsin deficiency
  • New Molecular Entity No

Highest Development Phases

  • Phase II Alpha 1-antitrypsin deficiency

Most Recent Events

  • 07 Feb 2019 Phase II development is ongoing for Alpha 1-antitrypsin deficiency in Ireland and USA
  • 05 May 2016 Pharmacodynamics data from a phase I trial in Alpha-1-antitrypsin deficiency released by Applied Genetic Technologies Corporation
  • 20 Jan 2016 Pharmacodynamics data from a phase II trial in Alpha-1-antitrypsin deficiency released by Applied Genetic Technologies Corporation

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