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Share Patients with genetic lysosomal storage diseases – particularly children – are living longer because of better treatments. But with promising advances and longer lives comes complications, the loss of eyesight as these rare diseases take their toll over time. Lysosomal storage diseases are inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies, according to the National Organization for Rare Disorders. With these diseases, patients are missing the mucopolysaccharidosis type 1 (MPS1) gene. Current therapies that allow patients to live longer don’t address corneal clouding caused by these diseases. However, one Carolina-affiliated startup, RainBio, is developing a novel gene therapy for MPSI corneal blindness, giving patients a chance not only for longer lives but better ones.

Matt hirschJoanne kurtzbergJude samulskiBrian gilgerJoseph muenzerEpigenos bioscienceFran martinSchool of medicineNational organization for rare disordersDrug administrationKickstart venture servicesCarolina office of technology commercializationOffice of technology commercializationNc state school of veterinary medicineNc state universityGene therapy center