Drug Profile


Alternative Names: Factor IX gene therapy - REGENXBIO; FIX gene therapy - REGENXBIO; scAAV 2/8-LP1-hFIXco

Latest Information Update: 11 Apr 2016

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At a glance

  • Originator ReGenX Biosciences
  • Developer St. Jude Childrens Research Hospital; University College London
  • Class Antihaemorrhagics; Blood coagulation factors; Coagulants; 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

  • Discontinued Haemophilia B

Most Recent Events

  • 14 Dec 2011 Phase-I/II clinical trials in Haemophilia B in United Kingdom (IV)
  • 14 Dec 2011 Phase-I/II clinical trials in Haemophilia B in USA (IV)
  • 20 Apr 2011 Preclinical trials in Haemophilia B in USA (IV)
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