

Developer of a gene therapy designed to maintain and restore sight in patients with inherited retinal dystrophies. The company's gene therapy is potentially a sight restoring therapy for a rare degenerative disease causing blindness known as Choroideremia, enabling patients to get treated for several rare conditions caused by mutations in a single gene. Among its major competitors, NightstaRx is ranked in 4th place for NPS while Ultragenyx Pharmaceutical is 1st, and Illumina is 2nd.