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Benitec Biopharma Inc is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines. Its technology has the potential to provide a one-shot cure for a wide range of diseases that are addressed by strict ongoing treatment regimens or that have no effective treatment or only palliative care options. The Company is developing a silence and replace-based therapeutic (BB-301) for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD), a chronic, life threatening genetic disorder.
Taysha Gene Therapies Inc is a patient-centric gene therapy company to eradicate monogenic CNS disease. It is focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the CNS in both rare and large patient populations. The company develops and commercializes transformative gene therapy treatments. Its product pipeline includes, TSHA-102 which is a is a self-complementary intrathecally delivered AAV9 gene transfer therapy in clinical evaluation for Rett syndrome.