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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.
Sobi is an international specialty healthcare company dedicated to developing treatments and extensive patient support networks for people with rare and difficult to treat diseases. These patients are often misunderstood, misdiagnosed and have limited medical options otherwise. This commitment has led to our world-class capabilities in protein biochemistry and biologics manufacturing, recognized by our leading industry partners. In North America, we have established our home bases in Waltham, MA and Toronto, Canada. Our employees not only have deep expertise in rare diseases, but are also dedicated to listening and learning from patients, payers and HCPs to ensure we’re bringing value to the region in the smartest, most impactful ways.
AmCyte Inc. is a Santa Monica, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Enzyvant is also advancing the development of RVT-802, an investigational tissue-based biologic therapy for the potential treatment of primary immune deficiency associated with complete DiGeorge Syndrome. RVT-802 has been granted orphan drug designation, Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, and pediatric rare disease designation by the U.S. Food and Drug Administration. Enzyvant anticipates a potential BLA filing for RVT-802 in the first half of 2018. Enzyvant plans to develop treatments for additional rare diseases with high unmet need.
Roswell Biotechnologies is a molecular electronics company transforming DNA sequencing with their latest platform, designed to deliver the disruptive performance required for future biomedical and industrial applications.