Advanced therapies for retinitis pigmentosa

Gil Carrasquinho, MD, MBA, Santen Pharmaceutical Co. Ltd., provides an overview of the current research landscape in retinitis pigmentosa (RP), a degenerative retinal disease that leads to a loss of vision over time. While current treatment options for RP are limited, the gene therapy voretigene neparvovec was recently approved, however, this can only be used to treat a small subset of patients with RP. A number of cell and gene therapies are currently under investigation, however the high level of genotypic and phenotypic heterogeneity seen in patients with RP makes the design of clinical trials and therefore the development and approval of new therapies challenging. This interview took place at the International Society for Stem Cell Research (ISSCR) 2021 Annual Meeting.