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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.
Ultragenyx Pharmaceutical Inc is a USA-based biopharmaceutical company. It identifies, acquires, develops, and commercializes novel products for the treatment of serious rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. The company's medicine portfolio includes Crysvita, Dojolvi and Mepsevii. Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older. Mepsevii is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII.