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ET 01

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Drug Profile

ET 01

Alternative Names: BCL11A enhancer modified autologous haematopoietic stem cells; ET-01 - EdiGene

Latest Information Update: 28 Sep 2024

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At a glance

  • Originator EdiGene Inc
  • Class Gene therapies; Haematopoietic stem cells therapies; Stem cell therapies
  • Mechanism of Action Cell replacements; Fetal haemoglobin expression stimulants; Gene modulators
  • 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

  • Research Anaemia
  • No development reported Beta-thalassaemia

Most Recent Events

  • 28 Sep 2024 No recent reports of development identified for phase-I development in Beta-thalassaemia(In adolescents, In children, In adults) in China (IV, Infusion)
  • 10 Dec 2022 Efficacy and adverse events data from a phase I trial in Beta-thalassemia presented at the 64th American Society of Hematology Annual Meeting and Exposition
  • 10 Nov 2022 Efficacy and safety data from phase I trial in Beta-thalassaemia released by EdiGene

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