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Intellia Therapeutics Inc. ist ein US-amerikanisches Pharmaunternehmen mit Sitz in Cambridge (Massachusetts), Vereinigte Staaten. Der Hauptzweck des Unternehmens ist die Bearbeitung von Genomen. (de) Intellia Therapeutics is a clinical-stage biotechnology company focused on developing novel, potentially curative therapeutics leveraging CRISPR-based technologies. To fully realize the transformative potential of CRISPR-based technologies, Intellia is pursuing two primary approaches. The company’s in vivo programs use intravenously administered CRISPR as the therapy, in which the company's proprietary delivery technology enables highly precise editing of disease-causing genes directly within specific target tissues. Intellia’s ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases. The CRISPR gene editing system was originally invented by Jennifer Doudna, one of Intellia's scientific founders, (with colleagues at University of California, Berkeley) and Virginijus Šikšnys (with colleagues at Vilnius University). The company has entered into a number of different research and development collaborations with leading and emerging biotechnology companies including Novartis, Regeneron, Avencell, SparingVision, Kyverna, and ONK Therapeutics. Intellia has two in vivo programs in ongoing clinical trials. NTLA-2001 is an investigational CRISPR therapy candidate for the treatment for ATTR amyloidosis currently in Phase 1 studies. NTLA-2002 is an investigational CRISPR therapy candidate for the treatment of hereditary angioedema (HAE) currently in Phase 1 / 2 studies. Intellia’s proprietary non-viral gene knock out platform deploys lipid nanoparticles to deliver to the liver a two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which carries out the precision editing.Intellia also has a number of research programs for in vivo and ex vivo therapeutic candidates with potential applications in diseases including cancer, alpha-1 antitrypsin deficiency, and hemophilia. The company is also working on a variety of additional gene editing technologies including base editing and DNA writing. (en) |
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Intellia Therapeutics Inc. ist ein US-amerikanisches Pharmaunternehmen mit Sitz in Cambridge (Massachusetts), Vereinigte Staaten. Der Hauptzweck des Unternehmens ist die Bearbeitung von Genomen. (de) Intellia Therapeutics is a clinical-stage biotechnology company focused on developing novel, potentially curative therapeutics leveraging CRISPR-based technologies. To fully realize the transformative potential of CRISPR-based technologies, Intellia is pursuing two primary approaches. The company’s in vivo programs use intravenously administered CRISPR as the therapy, in which the company's proprietary delivery technology enables highly precise editing of disease-causing genes directly within specific target tissues. Intellia’s ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases. (en) |
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