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Research programme: gene editing therapeutics - Intellia Therapeutics

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Drug Profile

Research programme: gene editing therapeutics - Intellia Therapeutics

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

Latest Information Update: 20 Nov 2023

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At a glance

  • Originator Intellia Therapeutics
  • Class Gene therapies
  • Mechanism of Action Gene modulators
  • 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 Alpha 1-antitrypsin deficiency; Sickle cell anaemia
  • No development reported Hepatitis B; Inborn error metabolic disorders; Primary hyperoxaluria

Most Recent Events

  • 09 Nov 2023 Preclinical development is ongoing for Alpha-1-antitrypsin-deficiency in USA (Parenteral)
  • 28 Apr 2023 No recent reports of development identified for preclinical development in Primary-hyperoxaluria in USA (Parenteral)
  • 28 Feb 2022 The US Patent and Trademark Office issues decision in favor of Broad Institute for interference claim for patent related to CRISPR-Cas9 genome editing technology in USA

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