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Gain Therapeutics Inc is a biotechnology company developing novel small molecule therapeutics to treat diseases across several therapeutic areas, including, lysosomal storage disorders, central nervous system disorders, metabolic disorders, and oncology. The company's clinical-stage product candidate, GT-02287, is being developed for the treatment of GBA1 Parkinson's disease. The company uses its computational target and drug discovery platform, Magellan, to discover novel allosteric binding sites on proteins implicated in disease and to identify proprietary small molecules that bind these sites to modulate protein function and treat the underlying cause of the disease. Geographically, the company operates in Switzerland, Spain, the United States, and Australia.
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.