ARVO, May 3-7, 2015, Denver, Colorado

GenSight attended ARVO, Denver (Colorado) and presented 3 posters:

• Preliminary safety and tolerability results of intravitreal administration of GS010, a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing human wild type mitochondrial NADH dehydrogenase 4 (ND4) gene in patients with LeberHereditary Optic Neuropathy (LHON) due to G11778A mitochondrial DNA mutation.
• Leber’s Hereditary Optic Neuropathy (LHON): a qualitative study on the experiences and hopes of patients and caregivers.
• Relevance of NHP safety studies for ocular delivery of AAV-based gene therapy in humans: Example of GS010 (rAAV2/2-ND4) local tolerability, shedding and immunogenicity for the treatment of Leber Hereditary Optic Neuropathy (LHON)), after intravitreal (IVT) injection in non-human primates (NHP) and in LHON patients.