RBIO-1

**RBIO-1** is an investigational adeno-associated virus gene therapy being developed by RainBio for corneal opacity and blindness associated with mucopolysaccharidosis I. The program is designed to deliver an optimized **alpha-L-iduronidase** gene cassette to the cornea in order to restore local IDUA enzyme activity, reduce glycosaminoglycan accumulation, and thereby prevent or reverse MPS I corneal clouding. Publicly available descriptions indicate administration by **corneal intrastromal injection** and position RBIO-1 as a potentially first-in-class anterior ocular AAV therapy; preclinical reports describe restoration of IDUA activity in patient cells and human corneas, as well as prevention and reversal of corneal opacity in a canine MPS I model.

Other names

optimized IDUA cDNA genetic cassetteAAV-IDUA gene therapy