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Anavex Life Sciences Corp is a clinical-stage biopharmaceutical company engaged in the development of differentiated therapeutics by applying precision medicine to central nervous system diseases with high unmet need. It analyzes genomic data from clinical studies to identify biomarkers, which are further used in the analysis of clinical trials. The company's focus is on developing treatments for Alzheimer's disease, Parkinson's disease, schizophrenia, neurodevelopmental, neurodegenerative, and rare diseases, including Rett syndrome, and other central nervous system (CNS) disorders. Its various therapeutic candidates in different stages of development include Anavex 2-73 (Blacamesine), Anavex 3-71 (AF710B), Anavex 1-41, and Anavex 1066.
Neurogene Inc is a clinical-stage biotechnology company committed to overcoming the limitations of neurological diseases into treatable conditions, by harnessing its proprietary transgene regulation technology, EXACTTM (Expression Attenuation via Construct Tuning). It is building a differentiated product portfolio of genetic medicines for rare neurological diseases with high unmet needs. The drug candidates in the company's product pipeline include NGN-401, which is in development for the treatment of Rett syndrome, using its EXACT platform, and NGN-101, a non-EXACT, conventional gene therapy development candidate for the treatment of CLN5 Batten disease, designed to deliver the CLN5 gene and packaged in an AAV9 capsid.