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Spruce is a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need. Our wholly-owned product candidate, tildacerfont, is a CRF1 receptor antagonist currently in late-stage trials in adult patients with classic congenital adrenal hyperplasia (CAH), with a planned Phase 2 trial in pediatric classic CAH. We aim to advance the treatment paradigm for classic CAH with a well-tolerated, non-steroidal approach designed to offer markedly improved disease control and reduced steroid burden for patients. Tildacerfont may also benefit patients with other disorders characterized by elevated levels of or hyperresponsiveness to adrenocorticotropic hormone (ACTH), a hormone involved in the production of cortisol, including a rare form of polycystic ovary syndrome (PCOS).
GenSight Biologics is is a clinical-stage biotechnology company discovering and developing novel therapies for neurodegenerative retinal diseases and diseases of the central nervous system. GenSight Biologics` pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics for retinitis pigmentosa, to help preserve or restore vision in patients suffering from severe degenerative retinal diseases. GenSight Biologics` lead product candidate, GS010, is in Phase III trials in Leber`s Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible low vision and legal blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics` product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.
Athersys is a Cleveland, OH-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Alexion is a global biopharmaceutical company focused on developing and delivering life-transforming therapies for patients with devastating and rare diseases. Our shared purpose to serve patients in need drives us to find answers, change the world, and create a legacy. Alexion`s three highly innovative therapies treat patients with four severe and ultra-rare diseases, and we are committed to developing additional therapies that have the potential to transform patients` lives. Today, Alexion is advancing the most robust rare disease pipeline in the biotech industry, which, in addition to our complement and metabolic clinical programs, includes more than 30 diverse preclinical programs across a range of therapeutic modalities. Alexion has more than 3,000 employees serving patients in 50 countries. Our global headquarters and research operations are based in New Haven, Connecticut and our EMEA headquarters are in Zürich, Switzerland. We also have global supply chain and operations headquarters in Ireland, manufacturing facilities in the United States, and local and regional operations in countries around the world. Alexion was ranked number 5 by Science magazine in its Top Employer Survey and has also been ranked as one of the top companies on the Forbes "World`s Most Innovative Companies" list every year since 2012.
Concerro is a San Diego, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.