Autologous haematopoietic stem/progenitor cell therapy - Sangamo Therapeutics/Bioverativ

Drug Profile

Autologous haematopoietic stem/progenitor cell therapy - Sangamo Therapeutics/Bioverativ

Alternative Names: SB BCLmR HSPC; ST 400; ZFN-Bcl11a

Latest Information Update: 10 Apr 2018

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At a glance

  • Originator Sangamo Therapeutics
  • Developer Bioverativ; Sangamo Therapeutics
  • Class Gene therapies; Stem cell therapies
  • Mechanism of Action Cell replacements; Gene silencing; Haemoglobin stimulants
  • 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

  • Phase I/II Beta-thalassaemia
  • Preclinical Sickle cell anaemia

Most Recent Events

  • 10 Apr 2018 Preclinical development for Sickle cell anaemia is ongoing in USA (Sangamo Therapeutics pipeline, April 2018)
  • 08 Mar 2018 Bioverativ has been acquired by Sanofi
  • 01 Mar 2018 Phase-I/II clinical trials in Beta-thalassaemia in USA (Parenteral) (NCT03432364)
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