AMT 060

Drug Profile

AMT 060

Alternative Names: AAV5-hFIX; AMT-060

Latest Information Update: 05 Sep 2017

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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

  • 28 Aug 2017 Protein Sciences Corporation has been acquired by Sanofi
  • 31 Jul 2017 uniQure reacquires co-development and commercialisation rights from Chiesi Farmaceutici SpA for Haemophilia B in Europe and other select territories, and terminates their co-development and license agreement
  • 10 Jul 2017 Updated efficacy data from a phase I/II trial in Haemophilia B released by uniQure
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