[Clinical endpoints and translational challenges]

Optogenetic vision-restorative approaches have entered the clinical stage of development.

This article provides an overview of the current status of translation. It discusses specific aspects relating to the identification of a suitable study group and the selection of endpoints. It also focuses on patients' expectations on this novel therapy.

A narrative review article is presented.

Clinicaltrials.gov lists eight different treatment studies pursuing the approach of optogenetic restoration of vision. The results from three of these have been published in peer-reviewed journals. Now that proof of concept has been successfully demonstrated in patients, future approaches must focus on restoring more complex visual perception in order to offer maximum added value. When identifying suitable patient groups, the status of retinal remodelling must be considered, as must the fact that patients with no remaining native visual impressions already benefit from a lower functional gain. Study endpoints should be chosen so that they specifically probe the function of the optogenetic actuator and meet the requirements of the regulatory authorities for treatment approval.

Optogenetics has reached the clinical development phase. Following proof of concept, the focus is now on further developing the approach so that it will also enable complex vision.