Our mission is to turn scientific advances in gene therapy into novel treatments for patients with severe degenerative diseases of the eye and the central nervous system.
Our initial focus is on severe retinal diseases, with the goal of preserving or restoring vision in patients who suffer from sight-threatening ophthalmic diseases. We have been using an integrated development platform based on the combination of a gene therapy-based approach with technologies such as Mitochondrial Target Sequence (MTS) and optogenetics. We are proud of the two promising product candidates that, to date, have advanced to the stage of being tested in clinical trials. And we are investigating others.
Our goal: to transform the lives of patients suffering from severe degenerative diseases of the eye and central nervous system
Our gene therapy-based approach appeals to us not just because we are excited about bringing advanced, innovative science to bear on diseases with a severe impact on patients’ quality of life. Our approach also energizes us because it holds out the promise of a long-lasting cure administered in a manner that is simple and convenient for patients and their physicians. Thus, our two leading product candidates are currently designed to be administered in a single treatment to each eye by intravitreal, or IVT, injection.
We have broader ambitions. We believe that our technology platforms have wide applicability, not just within ophthalmology but outside as well, potentially to other disorders of the central nervous system. Even as we push forward on getting our lead products to market, our research teams are exploring gene therapy-based therapy options for other neurodegenerative conditions for which no satisfactory solutions have been found.