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Research programme: in-vivo CRISPR editing gene therapies - Orna Therapeutics

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Drug Profile

Research programme: in-vivo CRISPR editing gene therapies - Orna Therapeutics

Latest Information Update: 24 Oct 2024

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At a glance

  • Originator Orna Therapeutics
  • Class Antianaemics; Gene therapies; RNA
  • Mechanism of Action Gene transference
  • 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

  • Preclinical Beta-thalassaemia; Sickle cell anaemia

Most Recent Events

  • 22 Oct 2024 Preclinical trials in Beta-thalassaemia in USA (Parenteral) before October 2024
  • 22 Oct 2024 Preclinical trials in Sickle cell anaemia in USA (Parenteral) before October 2024
  • 22 Oct 2024 Pharmacodynamics data from preclinical studies in Sickle cell anaemia and Beta thalassaemia released by Orna Therapeutics

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