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ST 400

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Drug Profile

ST 400

Alternative Names: Autologous CD34 + hematopoietic stem cells; Autologous haematopoietic stem/progenitor cell therapy - Bioverativ/Sangamo Therapeutics; BIVV-003; SAR-445136; SB-BCLmR-HSPC; ST-400; ZFN-Bcl11a

Latest Information Update: 17 Jan 2023

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At a glance

  • Originator Sangamo Therapeutics
  • Developer Bioverativ; Sangamo Therapeutics
  • Class Gene therapies; Haematopoietic stem cells 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.

    Yes - Sickle cell anaemia; Beta-thalassaemia
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

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

Most Recent Events

  • 10 Dec 2022 Safety and Efficacy data from a phase I/II PRECIZN-1 trial in Sickle cell anaemia presented at the 64th American Society of Hematology Annual Meeting (ASH-2022)
  • 17 Nov 2022 Sangamo Therapeutics and Sanofi completes a phase-I/II trial in Beta-thalassaemia in USA (Parenteral) (NCT03432364)
  • 03 Nov 2022 ST 400 receives Regenerative Medicine Advanced Therapy (RMAT) status for Sickle cell anaemia in USA

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