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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.
Soleno Therapeutics Inc is a biopharmaceutical company developing novel therapeutics for the treatment of rare diseases. Its product candidate DCCR (Diazoxide Choline) Extended-Release tablets, is a proprietary extended-release formulation of a crystalline salt of diazoxide. DCCR has demonstrated the ability to activate the KATP channel in the brain, pancreas, and fat tissue.