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Tremtelectogene empogeditemcel - Vor Biopharma

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Drug Profile

Tremtelectogene empogeditemcel - Vor Biopharma

Alternative Names: trem-cel; VOR-33

Latest Information Update: 20 Aug 2025

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At a glance

  • Originator Columbia University
  • Developer Vor Biopharma
  • Class Antineoplastics; Gene therapies; Haematopoietic stem cells therapies; Immunotherapies; Stem cell therapies
  • Mechanism of Action Cell replacements
  • Orphan Drug Status

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

    Yes - Acute myeloid leukaemia
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Acute myeloid leukaemia; Myelodysplastic syndromes
  • Preclinical Myeloproliferative disorders

Most Recent Events

  • 28 May 2025 Vor Biopharma terminates a first-in-human phase I/IIa trial in Acute myeloid leukaemia and Myelodysplastic syndromes (Combination therapy, Second-line therapy or greater) in USA, Canada (Parenteral, Infusion), due to lack of funding (NCT04849910)
  • 30 Apr 2025 Vor receives a supportive feedback from the US FDA regarding a registrational phase III clinical trial design in Acute myeloid leukemia
  • 30 Apr 2025 Efficacy data from a phase I/II trial in Acute myeloid leukaemia and Myelodysplastic syndrome released by PureTech

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