ASGCT, Washington, DC, US

GenSight gave several oral presentations at ASGCT, Washington DC (US), on its lead products for the treatment of retinal degenerative diseases that lead to blindness. 

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

Phase I Gene Therapy Preliminary Clinical Results for treatment of ND4 Leber Hereditary Optic Neuropathy with rAAV2-2-ND4. Session: Gene Therapy for Neurosensory Diseases (Room: Washington 1-2. Presentation Time: Thursday May 5, 4:30pm – 4:45pm. Abstract number: 267)

Relationship between immune responses and ocular inflammation: what is learnt from intravitreal injection of rAAV2/2–ND4 (GS010) in non-human primates and Leber Hereditary Optic Neuropathy (LHON) patients. Session: Immunological Aspects of Gene Therapy I: AAV Vectors  (Room Washington 5-6, Presentation time: Wednesday May 4, 2016 5:00pm – 5:15pm, Abstract number: 83)

– GS030 based on optogenetics to treat RP

Optogenetic engineering of retinal ganglion cells with AAV2.7m8-ChrimsonR-tdTomato is well tolerated and induces functional responses to light in non-human primates. Session: Gene Therapy for Neurosensory Diseases (Room: Washington 1-2. Presentation Time: Thursday May 5, 4:45pm – 5:00pm. Abstract number: 268)