Study Title Phenotype PI Location Criteria
RESCUE LHON m.11778 G>A Patrick Yu Wai Man Moorfields Eye Hospital Vision loss up to 6 months in duration
REVERSE LHON m.11778 G>A Patrick Yu Wai Man Moorfields Eye Hospital Vision loss from 6-12 months in duration





PI Contact:   

Funding Body:         GenSight Biologics

Dates of study:        February 2016 –

Ethical approval:      NRES Committee: London – West London & GTAC

REC number:          RESCUE (15/LO/1308) and REVERSE (15/LO/1309)


We would like to inform you about the RESCUE (NCT02652767) and REVERSE (NCT02652780) Phase III clinical trials of an experimental gene therapy for Leber Hereditary Optic Neuropathy (LHON) that may be of interest to you and to your patients. The experimental therapy is a recombinant adeno-associated viral vector 2 (rAAV2) carrying the wild-type ND4 gene (rAAV2/2-ND4) that has been developed for the treatment of vision loss in patients diagnosed with LHON due to the m.11778G>A ND4 mitochondrial mutation.

  • RESCUE will include patients with vision loss of up to 6 months in duration.
  • REVERSE will include patients with vision loss from 6 months up to one year in duration.
  • In both RESCUE and REVERSE, each patient will receive rAAV2/2-ND4 in one of their randomly selected eyes via an intravitreal injection. The fellow eye will receive a sham intravitreal injection.


Main inclusion criteria:

(1) Confirmed LHON with the 11778 mitochondrial DNA mutation

(2) Disease duration of less than 12 months (in both eyes)

(3) Age 18 years or older




The UK trial site will be Moorfields Eye Hospital in London. Travel and accommodation expenses in London will be fully covered for the patient and an accompanying person if required. We now have regulatory approval to start recruitment, so please do get in touch with us if you have any eligible patients.


Contact details for referral of patients to study: