Research programme: gene regulation therapeutics - Sangamo Therapeutics/Shire

Drug Profile

Research programme: gene regulation therapeutics - Sangamo Therapeutics/Shire

Alternative Names: hFVIII; SB-FVIII; ZFN genome editing therapeutics - Sangamo/Shire; ZFP therapeutics - Sangamo/Shire

Latest Information Update: 11 Jan 2017

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At a glance

  • Originator Sangamo BioSciences; Shire
  • Developer Sangamo Therapeutics
  • Class Gene therapies; Transcription factors; Zinc finger DNA binding proteins
  • Mechanism of Action Blood coagulation factor modulators; DNA binding protein modulators; RNA interference
  • 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

  • Preclinical Haemophilia A; Huntington's disease

Most Recent Events

  • 06 Jan 2017 Sangamo Biosciences is now called Sangamo Therapeutics
  • 07 Dec 2016 Preclinical development for Huntington's disease and Haemophilia A is ongoing in USA
  • 16 Jul 2016 No recent reports of development identified for preclinical development in Haemophilia-A in USA (Parenteral)
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