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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.
Amicus Therapeutics Inc is a biotechnology company focused on discovering, developing and delivering medicines for people living with rare metabolic diseases. The company is engaged in developing a pipeline of medicines for rare metabolic diseases, including a rare disease gene therapy portfolio. It has a portfolio of product opportunities, including oral precision medicine for people living with Fabry disease who have amenable genetic variants; a clinical-stage, treatment paradigm for Pompe disease, and a rare disease gene therapy portfolio. The company has one segment focused on the discovery, development, and commercialization of advanced therapies to treat a range of devastating rare and orphan diseases.