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

Drug Profile

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

Alternative Names: BIVV-003; SB-BCLmR-HSPC; ST 400; ZFN-Bcl11a

Latest Information Update: 16 Aug 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 transference; Haemoglobin stimulants
  • 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

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

Most Recent Events

  • 08 Aug 2018 Sangamo Therapeutics initiates enrolment in the phase I/II Thales trial for Beta-thalassaemia in USA
  • 16 May 2018 BioVerativ and Sangamo plan a phase I/II trial for Sickle cell anaemia in USA in December 2018 (NCT03653247)
  • 16 May 2018 The US FDA approves IND application for BIVV 003 in Sickle cell anaemia
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