GenSight was invited to ARVO in Seattle (Wash), US.

Scott Urestky, MD, Neuro-Ophthalmology, Director of Clinical Development at GenSight, gave a talk on “Allotopic Gene Therapy for ND4 Leber Hereditary Optic Neuropathy with rAAV2/2-ND4 (GS010): From Pre-Clinical Development to Phase III“.

The company presented also several posters on both its lead products as well as an oral presentation on its optogenetics product GS030

– GS010 aiming at treating LHON, a rare mitochondrial genetic disease

Intravitreal injection of rAAV2/2–ND4 (GS010): immune responses and ocular inflammation in non-human primates and patients with Leber Hereditary Optic Neuropathy (LHON). Session: Gene editing and therapies (Presentation time: Sun, May 01, 3:15pm – 5:00pm Abstract Number: 1177 – C0027)

– GS030 based on optogenetics to treat RP

Optogenetic engineering of retinal ganglion cells with AAV2.7m8-ChrimsonR-tdTomato in combination with an event-based camera photostimulation device. Session: Diseases and Protection (Room Tahoma 5, TCC. Presentation Time: Wed, May 04, 5:00pm – 5:15pm. Abstract Number: 5260)

Optogenetic visual restoration using ChrimsonR: Photoactivation below safety radiation limit in retinal ganglion cell populations from non-human primates. (Abstract Number: 598 – B0071)

Optogenetic visual restoration using ChrimsonR: Photocurrent analysis in primate retinal ganglion cells by using two-photon guided patch-clamp recording. (Abstract Number: 599 – B0072)

Optogenetic visual restoration using ChrimsonR: Validation in degenerative rodent models, rd1 and P23H. (Abstract Number: 600 – B0073)

Optogenetic visual restoration using ChrimsonR: Biophysical properties study in HEK cells using patch clamp recordings. (Abstract Number: 601 – B0074)

All poster will be presented at the session “Visual Disease Models and Restoration”, Sun, May 01, 1:30pm – 3:15pm