3. Gene therapy for inherited retinal diseases

Gene therapy for inherited retinal diseases is expected to receive a number of FDA approvals in 2018, if not late 2017. Currently, there are no known treatments for the rare diseases, but the growing research of specific genetic etiology may soon restore vision loss in patients.

Recombinant AAV2 vector encoding a functional copy of the RPE65 gene is expected to receive FDA approval in the next year to treat retinal diseases that are caused by biallelic RPE65 mutations.

According to Dr. Aleksandra Rachitskaya of the Cleveland Clinic’s Cole Eye Institute, the gene therapy won’t cure blindness completely, but will instead preserve the vision that is already there. However, there are currently retinal implants out there that help cure blindness and researchers are using stem cell therapies to combat retinal diseases. She also said that a new methodology called optogenetics, which involved modifying cells to function as other cells, could bring new hope to treating the diseases.

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