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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.
Rocket Pharmaceuticals Inc is a late-stage biopharmaceutical company. It is focused on the development of gene therapies for rare and devastating pediatric diseases. The pipeline products of the company include RP-A501 for Danaon Disease, RP-L102 for Fanconi Anemia, RP-L201 for Leukocyte Adhesion, RP-L301 for Pyruvate Kinase Deficiency, and RP-L401 for Infantile Malignant Osteopetrosis. The Company has one reportable segment related to R&D and commercial readiness of its gene therapies.