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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.
Acadia Pharmaceuticals Inc is a biopharmaceutical company focused on the development and commercialization of medicines that address unmet medical needs in central nervous system (CNS) disorders and rare diseases. It has two core franchises in neuroscience and neuro-rare diseases. The neuroscience franchise is anchored by its commercial product NUPLAZID (pimavanserin), which is used for the treatment of hallucinations and delusions associated with Parkinson's Disease Psychosis (PDP). The neuro-rare disease franchise is anchored by the commercial product DAYBUE, which is used for the treatment of Rett syndrome. In addition, the company has various product candidates under development in different stages, including ACP-204, ACP-101, ACP-211, ACP-711, ACP-2591, etc.