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Crispr 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. The company is focused on using this technology to treat genetically defined diseases. Crispr's first approved drug is Casgevy, which was developed in collaboration with Vertex Pharmaceuticals and targets sickle-cell disease and transfusion-dependent beta-thalassemia, which have high unmet medical needs. The company is advancing a variety of gene editing programs in immuno-oncology, cardiovascular, and a stem cell-derived therapy to treat Type 1 diabetes.
Axsome Therapeutics Inc is a clinical-stage biopharmaceutical company. It is engaged in developing novel therapies for the management of the central nervous system, or CNS, disorders for which there are limited treatment options. Its pipeline products includes AXS-05, AXS-07, AXS-12, and AXS-14. It manages its business as one operating segment and reporting unit, which is the business of developing and delivering novel therapies for the management of CNS disorders.