AMT 021

Drug Profile

AMT 021

Alternative Names: AAV5-AAT-PBGD; AAV5-PBGD gene therapy; AMT-020; AMT-021; rAAV2/5-PBGD

Latest Information Update: 16 Jul 2016

Price : $50

At a glance

  • Originator Digna Biotech; University of Navarra
  • Developer uniQure
  • Class Gene therapies
  • Mechanism of Action Hydroxymethylbilane synthase stimulants
  • Orphan Drug Status

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

    Yes - Acute intermittent porphyria
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • No development reported Acute intermittent porphyria

Most Recent Events

  • 16 Jul 2016 No recent reports of development identified for phase-I development in Acute-intermittent-porphyria in Spain (IV, Injection)
  • 16 Jul 2016 No recent reports of development identified for preclinical development in Acute-intermittent-porphyria in Netherlands (IV, Injection)
  • 01 Nov 2014 uniQure completes a phase I trial in Acute intermittent porphyria in Spain (NCT02082860)
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