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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.
Clinical trials are designed around endpoints, but its the beginning that sets them up for success—including selecting the best RTSM partner for continuous and flexible support throughout the duration of a trial. With decades of experience, Endpoint Clinical is obsessed with proactive problem-solving, ensuring that issues are anticipated and resolved before they escalate, and any changes are easily incorporated. From day one, we become an extension of your team, seamlessly integrating into your trial operations. We minimize friction, reduce risk, and enable your clinical teams to focus where it matters most—on trial success. Endpoint is headquartered in Raleigh, NC, with offices across the US, EU, and Asia.
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Welcome to a different kind of CRO. Bringing your product to market takes something different. At Rho, we`re experienced, creative problem-solvers who provide outstanding clinical research fueled by our unique team approach. Our dedication to collaboration makes your clinical trials and programs run smarter and more efficiently.
Discovering new medicines by combining ultra throughput biochemistry and machine learning.