Basic Information
RainBio is the only pioneer in ocular gene therapy focused on corneal blindness. Our adeno-associated virus (AAV) vector gene therapy directly corrects corneal cell function to maintain or restore vision in patients at risk of blindness due to various diseases. Our groundbreaking first-in-class lead program targeting corneal clouding in Type 1 Mucopolysaccharidosis (MPS1) patients, who have lost vision due to this rare genetic disorder, is opening new frontiers in ocular gene therapy.