Research programme: gene editing therapeutics - Intellia Therapeutics

Drug Profile

Research programme: gene editing therapeutics - Intellia Therapeutics

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

Latest Information Update: 07 May 2018

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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.

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Alpha 1-antitrypsin deficiency; Hepatitis B; Inborn error metabolic disorders; Primary hyperoxaluria

Most Recent Events

  • 01 May 2018 Pharmacodynamics data from preclinical studies in Alpha-1 antitrypsin deficiency and Primary hyperoxaluria released by Intellia Therapeutics
  • 31 Oct 2017 Updated pharmacodynamics data from preclinical studies in Inborn error metabolic disorders released by 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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