Leber’s Hereditary Optic Neuropathy Clinical Trial

If anyone reading this has Leber’s Hereditary Optic Neuropathy
and is interested in a clinical trial please get in touch!

Gensight Biologics is working on a phase III clinical trial named RESCUE that is actively seeking patients suffering from Leber’s Hereditary Optic Neuropathy (LHON) in the United States and Canada The purpose of the RESCUE study is to evaluate an investigational gene therapy, GS010, as a potential treatment for the most common variant (ND4/G11778A gene mutation) of LHON. Three clinical sites have been selected to treat the patients, they are located in Los Angeles, Philadelphia and Atlanta. Travel costs and other appropriate expenses for taking part in the trial, including those for one accompanying companion, will be supported, in accordance with GenSight Biologics.


Patients may qualify if they meet these main selection/inclusion criteria listed below

  • Age 15 years or older.
  • Onset of vision loss based on medically documented history or subject testimony, in at least one eye for ≤180 days in duration
  • Each eye of the subject maintaining visual ability to allow at least for counting of the examiner’s fingers at any distance.
  • Documented results or pedigree evidence of genotyping showing the presence of the G11778A mutation in the ND4 gene and the absence of the other primary LHON-associated mutations (ND1 or ND6) in the subject’s mitochondrial DNA.