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Research programme: gene editing therapeutics - Intellia Therapeutics/Regeneron Pharmaceuticals

Drug Profile

Research programme: gene editing therapeutics - Intellia Therapeutics/Regeneron Pharmaceuticals

Alternative Names: CRISPR/Cas9 gene editing therapies - Intellia Therapeutics/Regeneron Pharmaceuticals

Latest Information Update: 28 Feb 2024

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At a glance

  • Originator Intellia Therapeutics
  • Developer Intellia Therapeutics; Regeneron Pharmaceuticals
  • Class Antihaemorrhagics; Blood coagulation factors; Coagulants; Gene therapies
  • Mechanism of Action Factor VIII modulators; Gene modulators; 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 Haemophilia B
  • Research Haemophilia A; Musculoskeletal disorders; Neurological disorders
  • No development reported Transthyretin-related hereditary amyloidosis

Most Recent Events

  • 22 Feb 2024 Intellia Therapeutics and Regeneron plan a first-in-human phase I trial for Haemophilia B in mid-2024
  • 22 Feb 2024 US FDA approves IND application for gene editing therapeutics in Haemophilia B
  • 02 Nov 2023 Regeneron and Intellia Therapeutics expands existing collaboration to develop products for the treatment of Neurological and Muscular disorders
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