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: 06 Nov 2017

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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

  • 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
  • 19 Jun 2017 China’s State Intellectual Property Office ("SIPO") announced its intention to grant a patent for CRISPR/Cas9 genome editing technology
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