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Research programme: gene regulation therapeutics - Sangamo Therapeutics/Takeda

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Drug Profile

Research programme: gene regulation therapeutics - Sangamo Therapeutics/Takeda

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

Latest Information Update: 24 Nov 2023

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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.

    No
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Huntington's disease
  • Discontinued Haemophilia A

Most Recent Events

  • 24 Nov 2023 Preclinical trials in Huntington's disease in USA (Parenteral) (Sangamo Therapeutics pipeline; November 2023)
  • 28 Feb 2023 gene regulation therapeutics is still in early research in Huntington's disease in USA
  • 23 Feb 2023 Discontinued - Preclinical for Haemophilia A in USA (Parenteral) (Sangamo Therapeutics pipeline, February 2023)

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