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News from the Foundation
Cell-Based Treatment Preserves Vision in Usher Syndrome Model
A research team funded by the Foundation Fighting Blindness has used cell transplantation to restore vision in a mouse model of Usher syndrome type 2A (USH2A), a leading cause of combined deafness and blindness in humans. Never before has a cell-based treatment been used to save vision in an Usher syndrome study, in large part because no other Usher syndrome animal models have exhibited vision loss or retinal degeneration. The advancement is a critical step forward in developing a vision treatment for humans with the condition.

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Oxford BioMedica Receives Favorable Designation for Usher Syndrome Gene Therapy
Oxford BioMedica, a U.K. partner of the Foundation Fighting Blindness, has received orphan drug designation from the European Medicines Agency (EMEA) for their emerging Usher syndrome gene therapy known as UshStat. The orphan designation provides Oxford BioMedica with benefits and incentives including 10 years of marketing exclusivity, reduced regulatory fees, and clinical development guidance for UshStat.

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Researchers Target Innovative Stem Cell Treatment for AMD
An international research team has rescued vision in rats through transplantation of induced pluripotent stem cells or iPS — stem cells that are derived from mature cells or tissue that reside almost anywhere in the body. The team is working toward using this innovative approach to treat age-related macular degeneration (AMD), and potentially other retinal degenerative diseases, in humans. 

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Promising Dry AMD Treatment Moves into Phase II Clinical Trial
An oral medication for the dry form of age-related macular degeneration (AMD) is moving into a Phase II clinical trial after showing positive results for safety and effectiveness in prior preclinical studies and a Phase I human trial. Developed by Acucela, the drug works by slowing the buildup of toxic waste products that lead to retinal degeneration and vision loss in dry AMD.
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