AMT 060

Drug Profile

AMT 060

Alternative Names: AAV5-FIX; AAV5-hFIX; AMT-060

Latest Information Update: 28 May 2018

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At a glance

  • Originator Amsterdam Molecular Therapeutics
  • Developer St. Jude Childrens Research Hospital; uniQure; University College London
  • Class Gene therapies
  • Mechanism of Action Factor IX stimulants; Gene transference
  • Orphan Drug Status

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

    Yes - Haemophilia B
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Haemophilia B

Most Recent Events

  • 21 May 2018 Updated efficacy data from a phase I/II trial in Haemophilia B released by uniQure
  • 07 Feb 2018 AMT 060 is still in phase I/II trials for Haemophilia B in Denmark, Germany and Netherlands (NCT02396342)
  • 11 Dec 2017 Updated adverse events and efficacy data from a phase I/II trial in presented at the 59th Annual Meeting and Exposition of the American Society of Haematology (ASH-Hem-2017)
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