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Stem cell therapeutics - ExCellThera

Drug Profile

Stem cell therapeutics - ExCellThera

Alternative Names: ECT 001; ECT 002; ECT 003; ECT 005; ECT-001 cell therapy; ECT-001-CB; ECT-100; UM 171; UM171 + UM092; UM171 + UM390; UM171-Expanded CB; UM171-Expanded Cord Blood Transplantation

Latest Information Update: 15 Jan 2024

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At a glance

  • Originator ExCellThera
  • Developer California Institute for Regenerative Medicine; ExCellThera; Fred Hutchinson Cancer Research Center; Memorial Sloan-Kettering Cancer Center
  • Class Antineoplastics; Gene therapies; Haematopoietic stem cells therapies; Immunotherapies
  • Mechanism of Action CD34 antigen stimulants; Stem cell stimulants; Ubiquitin protein ligase modulators
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Graft-versus-host disease
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Preregistration Haematological malignancies
  • Phase II Acute myeloid leukaemia; Lymphoid leukaemia; Myelodysplastic syndromes
  • Phase I/II Multiple myeloma
  • Phase I Sickle cell anaemia
  • No development reported Immunological disorders

Most Recent Events

  • 02 Jan 2024 Preregistration for Haematological malignancies in European Union (Parenteral)
  • 02 Jan 2024 ExCellThera completes a phase II trial in Myelodysplastic syndrome in USA and Netherlands (parenteral) (NCT04103879; EudraCT-2022-002458-26)
  • 12 Dec 2023 ExCellThera plans a phase III trial for Acute leukemia and Myelodysplasias in 2024
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