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: 22 Mar 2018

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At a glance

  • Originator Intellia Therapeutics
  • Developer Intellia Therapeutics; Regeneron Pharmaceuticals
  • 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.

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Amyloidosis

Most Recent Events

  • 01 May 2018 Intellia Therapeutics announces intention to submit an IND application for Transthyretin amyloidosis by the end of 2019
  • 14 Mar 2018 Pharmacodynamics results from preclinical studies in Amyloidosis released Intellia Therapeutics
  • 25 Jul 2017 University of California, University of Vienna and Charpentier submit appellate brief seeking reversal of decision by PTAB, which dismissed interference proceedings against Broad Institute, MIT and Harvard University for a patent related to CRISPR-Cas9 genome editing technology, in USA
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