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Clinical trials

The initial focus of GenSight is on developing therapies for severe retinal diseases, with the goal of preserving or restoring vision in patients suffering from sight-threatening ophthalmic diseases. Using our gene-therapy based approach, our product candidates are designed to be administered in a single treatment to each eye by intravitreal or subretinal injection in order to provide patients with a long-lasting functional cure.

Our pipeline currently comprises two lead product candidates for the treatment of sight-threatening retinal degenerative diseases, together with products in preclinical development targeting ophthalmic and neurodegenerative diseases. 

Visit www.clinicaltrials.gov and search for “GenSight Biologics” to learn more about our ongoing clinical trials listed below.

For GS010, a Phase 3 gene therapy product candidate aiming at treating LHON

  • Phase I/II : Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients 
  • RESCUE : Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation
  • REVERSE : Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation
  • REFLECT : Efficacy and Safety Study of Bilateral Intravitreal Injection of GS010 for the Treatment of Vision Loss up to 1 Year From Onset in LHON Due to the ND4 Mutation 
  • REALITY : LHON Registry Study to Understand the Evolution of Visual Functional and Structural Changes and Other Associated Symptoms in Patients with LHON
  • RESCUE and REVERSE Long-term Follow-up (RESCUE/REVERSE)

For GS030, our second gene therapy product candidate based on Optogenetics to treat Retinitis Pigmentosa


    Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa


For more information on clinical trials, please contact us:



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