Research programme: gene editing therapeutics - Intellia Therapeutics/Novartis

Drug Profile

Research programme: gene editing therapeutics - Intellia Therapeutics/Novartis

Alternative Names: CRISPR/Cas9-based CAR-T cell therapies - Intellia Therapeutics/Novartis; CRISPR/Cas9-based haematopoietic stem cell therapies - Intellia Therapeutics/Novartis

Latest Information Update: 14 Dec 2017

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At a glance

  • Originator Intellia Therapeutics
  • Class CAR-T cell therapies; Gene therapies; Stem cell 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

  • Research Sickle cell anaemia

Most Recent Events

  • 11 Dec 2017 Pharmacodynamics data from a preclinical studies in Sickle cell disease presented at the 59th Annual Meeting and Exposition of the American Society of Haematology (ASH-Hem 2017)
  • 25 Jun 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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