Mark Moster: RESCUE, REVERSE and RESTORE clinical trials: Investigating gene therapy for Leber’s hereditary optic neuropathy

Leber’s hereditary optic neuropathy (LHON) is characterized by bilateral, painless subacute loss of central vision during young adult life. We were delighted to speak with Prof. Mark Moster (Wills Eye Hospital, Philadelphia, PA, USA) to discuss the findings in the RESCUE, REVERSE and RESTORE clinical trials. 

Questions:

1. Could you give us a brief overview of Leber’s hereditary optic neuropathy (LHON) and its prognosis? (00:23)
2. Please could you describe the gene therapy you investigated in the RESCUE, REVERSE and RESTORE clinical trials? (01:21)
3. What were the efficacy and safety findings from the RESCUE and REVERSE clinical trials? (02:13)
4. What were the findings of the RESTORE long term follow up study? (03:57)
5. What is the current status of the European Medicines Agency (EMA) regulatory review? (04:45)

Disclosures: Mark Moster is a consultant for Gensight Biologics, and discloses grant/research support from Gensight Biologics.

Support: Interview and filming supported by Touch Medical Media Ltd. Interview conducted by Shanice Allen.