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Adverum Biotechnologies Inc is a clinical-stage company that aims to establish gene therapy as a new standard of care for prevalent ocular diseases. It develops gene therapy product candidates intended to provide durable efficacy by inducing sustained expression of a therapeutic protein. Its product candidate, ixoberogene soroparvovec (Ixo-vec), is a single, in-office intravitreal (IVT) injection gene therapy product designed to deliver long-term durable therapeutic levels of aflibercept associated with a robust, sustained treatment response, reducing the treatment burden and fluctuations in macular fluid associated with bolus anti-vascular endothelial growth factor (VEGF) IVT injections.
Precision BioSciences Inc is a genome editing company dedicated to improving life through its genome editing platform, ARCUS. It leverages ARCUS in the development of its product candidates, which are designed to treat human diseases and provide food and agricultural solutions. The ARCUS is in its relatively small size which potentially allows delivery to a wider range of cells and tissues using viral and non-viral gene delivery methods.