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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.
Neuros Medical, a Cleveland, Ohio based neurostimulation company, is focused on developing proprietary neurostimulation therapies for unmet needs to patients worldwide.
PharmaForm is a leading specialty contract development and manufacturing organization focusing on oral and transmucosal dosage forms. Founded in 1996, PharmaForm has built a reputation for delivering novel and innovative solutions to challenging problems
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).
Harmony Biosciences, LLC, is a private biopharmaceutical company headquartered in Plymouth Meeting, PA. The company was established in October 2017 with a vision to develop and commercialize novel treatment options for people living with rare diseases. With a focus on the central nervous system, including disorders of sleep and wakefulness, Harmony Biosciences is currently working to advance the understanding of narcolepsy and provide information and resources to people who live with, and healthcare professionals who treat, this chronic, debilitating neurologic disorder.