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AMT 060

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Drug Profile

AMT 060

Alternative Names: AAV5-FIX; AAV5-hFIX; AMT-060; CSL-220

Latest Information Update: 18 Feb 2025

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At a glance

  • Originator Amsterdam Molecular Therapeutics
  • Developer St. Jude Childrens Research Hospital; uniQure; University College London
  • Class Antihaemorrhagics; Gene therapies
  • Mechanism of Action Factor IX replacements; 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

  • 07 Dec 2024 Efficacy and adverse events data from a phase I/II trial in Haemophilia B presented at the 66th American Society of Hematology Annual Meeting and Exposition (ASH-Hem-2024)
  • 05 Dec 2020 Adverse events and safety data from a phase I/II trial in Hemophilia B presented at the 62nd Annual Meeting and Exposition of the American Society of Hematology(ASH-Hem-2020)
  • 13 Nov 2020 uniQure initiates a phase I/II extension trial in Haemophilia B in Denmark (IV) (EudraCT2020-000739-28)

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