Leber Hereditary Optic Neuropathy (LHON) caused by mutation of the ND4 gene - Healing Genes

Leber Hereditary Optic Neuropathy (LHON) caused by mutation of the ND4 gene

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Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year (REFLECT)

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

Phase 3


Doctors in CA, CO, GA, MA, NY, PA, and TN are recruiting participants with a specific type of Leber’s Hereditary Optic Neuropathy (LHON) to help assess the effectiveness of an investigational gene therapy to improve retina structure and function. The mutation targeted by this treatment is the G11784ND4 mitochondrial mutation, where vision has been lost up to one year.

Participants will be assigned to receive either the investigational drug in both eyes or to receive a placebo injection is one eye and the actual therapy in the second eye. This drug replaces the dysfunctional gene that causes LHON through the mechanism of a harmless virus that inserts the correct gene into the patient’s cells. Patients will be followed up to 2 years post treatment.


  • Be 15 years of age or older
  • Have vision loss of a certain duration of time due to ND4 LHON, in at least one eye
  • Not have alcoholism, alcohol dependence, or alcohol or drug abuse


  1. Prescreening to confirm eligibility of the patient to participate
  2. A single intravitreal injection in each eye
  3. 4 weeks post-treatment, a blood assay will be required
  4. Further follow up will continue to the 2 years post treatment, including lab testing, electrocardiogram, physical evaluation, patient surveys, and particularly vision assessments.


Primary contact: Barrett Katz, MD | [email protected] | 646 831 3799

The following locations are participating in the trial:

Doheny Eye Center UCLA Pasadena | Pasadena, CA

University of Colorado Health Eye Center | Aurora, CO

Emory Healthcare – The Emory Clinic | Atlanta, GA

Massachusetts Eye and Ear Infirmary | Boston, MA

Icahn School of Medicine at Mount Sinai | New York, NY

Wills Eye Institute – Ocular Oncology Service | Philadelphia, PA

Vanderbilt Eye Institute | Nashville, TN


GenSight Biologics

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