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OTQ 923

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Drug Profile

OTQ 923

Alternative Names: CRISPR/Cas9-based engineered cell therapy- Novartis/ Intellia Therapeutics; OTQ-923

Latest Information Update: 21 Feb 2025

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At a glance

  • Originator Intellia Therapeutics
  • Developer Novartis
  • Class Antianaemics; Gene therapies; Haematopoietic stem cells therapies; Stem cell therapies
  • Mechanism of Action Cell replacements; Fetal haemoglobin expression stimulants
  • 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

  • Discontinued Sickle cell anaemia

Most Recent Events

  • 11 Feb 2025 Novartis terminates a phase I/II trial in Sickle cell anaemia (In children, In adolescents, In adults) in USA (IV), due to the decision of early termination was made due to business reasons and was not a consequence of any safety concern (NCT04443907)
  • 23 Feb 2023 Discontinued - Phase-I/II for Sickle cell anaemia (In adolescents, In children, In adults) in Italy (IV)
  • 10 Dec 2022 Safety and efficacy data from a phase I/II trial in Sickle cell disease presented at the American Society of Hematology Annual Meeting and Exposition (ASH-Haem-2022)

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