Gene Therapy Against Blindness
We are a clinical-stage biotechnology company discovering and developing novel therapies for mitochondrial and neurodegenerative diseases of the eye and central nervous system. To address these therapeutic areas, we leverage our integrated development platform by combining a gene therapy-based approach with our core technology platforms of mitochondrial targeting sequence, or MTS, and optogenetics.
Our management and scientific teams have extensive experience in gene therapy and drug development, particularly in the field of ophthalmology, and have served in leadership roles at several innovative ophthalmology companies.
Our initial focus has been on developing therapies for severe retinal diseases, with the goal of preserving or restoring vision in patients suffering from sight-threatening ophthalmic diseases.