Research programme: zinc finger DNA-binding protein nucleases - Sangamo Therapeutics

Drug Profile

Research programme: zinc finger DNA-binding protein nucleases - Sangamo Therapeutics

Alternative Names: SB-BCLmR-HSPC; SB-LSD3; SB-LSD4; ZFN Bcl11a; ZFN therapeutics - Sangamo; ZFP nucleases - Sangamo; ZFP Therapeutic® haemoglobinopathy programme - Sangamo/Bioverativ

Latest Information Update: 14 Mar 2017

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At a glance

  • Originator Sangamo BioSciences
  • Developer Bioverativ; Sangamo Therapeutics; University of Massachusetts Medical School
  • Class Antineoplastics; Cell therapies; Gene therapies; Stem cell therapies; Zinc finger DNA binding proteins
  • Mechanism of Action Alpha 1 antitrypsin modulators; CCR5 receptor modulators; Cell replacements; Gene modulators; Gene silencing; Genetic transcription inhibitors; Haemoglobin modulators; Interleukin 2 receptor modulators; T-cell receptor gene modulators
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Mucopolysaccharidosis I
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Alpha 1-antitrypsin deficiency; Beta-thalassaemia; Cancer; Immunodeficiency disorders; Mucopolysaccharidosis I; Mucopolysaccharidosis II; Sickle cell anaemia
  • Research Down syndrome; HIV infections

Most Recent Events

  • 01 Feb 2017 Biogen has separated its Haemophilia Business under a new company Bioverativ
  • 06 Jan 2017 Sangamo Biosciences is now called Sangamo Therapeutics
  • 20 Jun 2016 The US FDA approves IND application for SB 913 in Mucopolysaccharidosis II
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