Long title
Efficacy and safety of bilateral intravitreal injection of GS010: a randomized, double-masked, placebo-controlled trial in subjects affected with G11778A ND4 leber hereditary optic neuropathy for up to one year.
Date receipt dossier
EudraCT number
2017-002187-40
Company / Sponsor
GenSight Biologics
Treated organism
Humans
Indication category
Genetic disorders
Disease
Leber hereditary optic neuropathy
Therapeutic approach
Gene therapy
Genetic modification
human wild-type MT-NDA (mitochondrial NADH Dehydrogenase 4)
Method of transfer of nucleic acid of interest
AAV2
Administered biological material
Recombinant AAV2 expressing MT-NDA
Route of administration
Intravitreal
Locations in Belgium
Universitair Ziekenhuis Gent
Type of procedure
Contained use only
Current status
Authorized
Pharmaceutical study code
GS010