GS010 is an AAV2 gene therapy vector that encodes the human wild-type ND4 protein.
The ND4 gene is normally located in the mitochondria where ND4 proteins are synthesized. GS010 allows allotopic expression of the mitochondrial gene ND4 in the nucleus thanks to a proprietary Mitochondrial Targeting Sequence that shuttles the messenger RNA from the nucleus directly to the outer membrane of the mitochondria. There, the ND4 proteins are synthesized and incorporated into the mitochondria. Wild-type ND4 proteins then integrate into Complex I of the respiratory chain and rescue the deficiency.
Trials with enrollment already closed
A Phase I/II trial, completed in France subjects with long-standing vision loss from LHON with the ND4 gene mutation.
Two Phase 3 trials (RESCUE and REVERSE), to evaluate the efficacy of GS010 in LHON patients with a recent onset of disease (i.e., less than one year), were completed in 2019 in Europe and the US. Results of follow-up visits up to Week 96 in both trials were reported in 2019. Patients in both trials were invited to participate in a long-term follow-up study, which will monitor safety and efficacy up to 5 years after injection.
A third Phase 3 trial, is the ongoing REFLECT study, which evaluates the efficacy and safety of bilateral intravitreal injection of GS010 for the treatment of vision loss up to 1 year.
Finally, the company also completed a retrospective registry study REALITY to study the evolution of visual function among untreated LHON patients. This study completed patient recruitment in 2019.
Studies being initiated
There are currently no trials involving GS010 that are enrolling patients.
Expanded access / compassionate use
For any request regarding Early Access Program please use the contact form: Contact Us – GenSight Biologics (gensight-biologics.com)