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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.
At Spark Therapeutics, we don`t follow footsteps. We create the path. We were born of innovation, springing from the curiosity, imagination and dedication of remarkable scientists and healthcare visionaries. Our mission is seemingly impossible to others, but not to us: Challenge the inevitability of genetic disease by discovering, developing and delivering treatments in ways unimaginable – until now. Since our founding, we have been committed to bringing a wide range of expertise to build a fully integrated gene therapy company focused on inherited retinal diseases (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver, such as hemophilia. We are seeking talented individuals with diverse experiences, abilities and interests who have the curiosity, courage and drive to reimagine a new health care paradigm. Join us on a journey through uncharted territory – seeking to bring gene therapy for genetic diseases to people worldwide. The resilience of the people we serve is our inspiration to break barriers, as we strive to turn genes into medicine for those with inherited diseases. Spark is a member of the Roche Group.
InCarda Therapeutics is a clinical stage drug delivery company pioneering a novel approach of treating cardiovascular diseases and conditions by the inhalation route.
Seagen Inc. is an American biotechnology company focused on developing and commercializing innovative, empowered monoclonal antibody-based therapies for the treatment of cancer.
Prosetta Biosciences, Inc. is an 11-year old biotechnology company developing novel small-molecule antiviral therapeutics based on over 25 years of basic research on the Cell-Free Protein Synthesizing Systems (CFPSS) by scientists at the University of California, San Francisco and the University of Washington, Seattle. The incorporation of the CFPSS in our drug discovery platform, has lead to the identification of small molecules which disrupt or alter the key protein-protein interactions involved with the assembly of viral capsids (the protein shell that protects the DNA or RNA genome of a virus). Prosetta has successfully produced highly potent compounds which display activity against multiple strains of many viral families including lead series of molecules that are effective antiviral agents against Influenza, HIV and HCV.