Breakthrough programs advancing quickly to the clinic
Our proprietary technology platforms target genetic diseases for which there are currently no therapeutic options. Beginning with advanced gene replacement programs licensed from our partners, we are expanding into additional disease areas and accelerating our pipeline to develop multiple drug candidates.
Our most advanced programs aim to treat inherited retinal dystrophies (IRDs), which cause profound vision loss often early in life. The underlying genetic causes of these monogenic, recessively inherited IRDs provide well-defined therapeutic targets.
We are focused on treating diseases which cause serious complications, and if left untreated, can be fatal. Diseases of interest are those caused by mutations, which cannot be treated by standard gene replacement as well as diseases where re-dosing can extend or augment clinical benefit
We are using our platform technologies to address genetic neurological disorders that lead to severe impairments that affect nearly every aspect of a person’s life. Diseases of interest are those with few or no available therapies, and for which current gene therapy approaches are inadequate.
Like inherited retinal dystrophies, genetic disorders of hearing are debilitating conditions producing permanent disability and deafness. Our advanced technologies allow us to address genetic causes of hearing loss where current gene therapy approaches fall short.