Therapeutic landscape for inherited ocular diseases: current and emerging therapies

Inherited ocular diseases comprise a heterogeneous group of rare and complex diseases, including inherited retinal diseases ( IRDs) and inherited optic neuropathies. Recent success in adeno-associated virus-based gene therapy, voretigene neparvovec (Luxturna (R)) for RPE65-related IRDs, has heralded rapid evolution in gene therapy platform technologies and strategies, from gene augmentation to RNA editing, as well as gene agnostic approaches such as optogenetics. This review discusses the fundamentals underlying the mode of inheritance, natural history studies and clinical trial outcomes, as well as current and emerging therapies covering gene therapy strategies, cell-based therapies and bionic vision.

Automated summary

Inherited ocular diseases, such as inherited retinal diseases and inherited optic neuropathies, are a diverse and complex group of rare diseases. Recent advancements in gene therapy, including adeno-associated virus-based gene therapy, have revolutionized the field and opened up new possibilities for treating these diseases. This review explores the mode of inheritance, natural history studies, clinical trial outcomes, and current and emerging therapies, including gene therapy strategies, cell-based therapies, and bionic vision.