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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.
Applied Therapeutics Inc is a clinical-stage biopharmaceutical company, developing a pipeline of novel product candidates against validated molecular targets in indications of high unmet medical need. The company's drug compounds pipeline includes AT-007 which is a novel central nervous system, penetrant ARI that it is developing for the treatment of rare metabolic diseases, including Galactosemia and Sorbitol Dehydrogenase (SORD) Deficiency, AT-001 for the treatment of diabetic cardiomyopathy, and AT-003 for the treatment of diabetic retinopathy.