ADVM 043

Drug Profile

ADVM 043

Alternative Names: AAVrh 10halpha1AT; AAVrh-10hA1AT; AAVrh.10hA1AT; AAVrh.10halpha1AT; ADVM-043- Adverum; ANN-001

Latest Information Update: 09 Jan 2018

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At a glance

  • Originator Weill Cornell Medical College
  • Developer Adverum Biotechnologies
  • 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.

  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Alpha 1-antitrypsin deficiency

Most Recent Events

  • 05 Dec 2017 Annapurna Therapeutics entered into a series of licensing agreements with Weill Cornell Medicine for the advancement of ADVM 043
  • 28 Nov 2017 Phase-I/II clinical trials in Alpha 1-antitrypsin deficiency in USA (Intrapleural) (NCT02168686)
  • 28 Nov 2017 Phase-I/II clinical trials in Alpha 1-antitrypsin deficiency in USA (IV) (NCT02168686)
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