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Main focus: Gene editing for the treatment of genetic diseases
Company stage: Clinical
Diseases: Transthyretin (ATTR) amyloidosis, hereditary angioedema, haemophilia A and B, sickle cell disease
acute myeloid leukaemia, solid tumours
Genome editing technology: CRISPR-Cas9, Base editing
Funding stage: Public (NASDAQ:NTLA)
Location: Cambridge, MA, USA
Intellia Therapeutics is a clinical-stage, gene-editing company applying its proprietary CRISPR-Cas9 platform to develop novel gene-editing and cellular therapies. The company was co-founded by Nobel Prize winner Jennifer Doudna. The company persues both in vivo and ex vivo therapeutic approahes and was the first to demonstrate safety and efficacy using an in vivo-administered CRISPR-Cas9-based therapy. These data were demonstrated for a programme developed for the treatment of a fatal cardiovascular disease called Transthyretin (ATTR) amyloidosis. The company is also engaged in multiple large collaborations with companies such as Regeneron and Novartis.