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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: 15 Apr 2025

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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

  • No development reported Anaemia; Beta-thalassaemia

Most Recent Events

  • 30 Mar 2025 EdiGene terminates Phase-I clinical trials in Beta-thalassaemia (In children, In adolescents, In adults) in China (IV) (NCT04925206)
  • 28 Mar 2025 No recent reports of development identified for research development in Anaemia in China (IV)
  • 12 Mar 2025 EdiGene terminates a Phase-I clinical trials in Beta-thalassaemia (In children, In adolescents, In adults) in China (IV) due to change in development plan (NCT04390971)

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