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Intellia Therapeutics is a gene editing company focused on the development of Crispr/Cas9-based therapeutics. Crispr/Cas9 stands for Clustered Regularly Interspaced Short Palindromic Repeats (Crispr)/Crispr-associated protein 9 (Cas9), which is a revolutionary technology for precisely altering specific sequences of genomic DNA. Intellia is focused on using this technology to treat genetically defined diseases. It's evaluating multiple gene editing approaches using in vivo and ex vivo therapies to address diseases with high unmet medical needs, including ATTR amyloidosis, hereditary angioedema, sickle cell disease, and immuno-oncology. Intellia has formed collaborations with several companies to advance its pipeline, including narrow-moat Regeneron and wide-moat Novartis.
Anavex Life Sciences Corp is a clinical-stage biopharmaceutical company engaged in the development of differentiated therapeutics by applying precision medicine to central nervous system diseases with high unmet need. It analyzes genomic data from clinical studies to identify biomarkers, which are used to select patients that will receive the therapeutic benefit for the treatment of neurodegenerative and neurodevelopmental diseases. Its necessary compound ANAVEX 2-73 is being developed to treat Alzheimer's disease, Parkinson's disease, and potentially other central nervous system diseases, including rare diseases, such as Rett syndrome, a rare severe neurological monogenic disorder. The company's operating segment is into developing novel therapies for the management of CNS diseases.