GenSight Biologics Secures French Regulatory Approvals for GS010/LUMEVOQ Gene Therapy in Rare Blindness Disease
Key Insights
GenSight Biologics received French regulatory approval for the REVISE dose-ranging study investigating GS010/LUMEVOQ gene therapy for ND4-LHON (search), a rare mitochondrial disease causing irreversible vision loss (search).
The French medicines agency ANSM (search) granted compassionate use authorization (AAC) for GS010/LUMEVOQ, enabling named patient early access for eligible patients with serious unmet medical needs.
The Phase II REVISE study will enroll 14 patients and investigate two clinical doses, with the primary endpoint measuring visual acuity changes 1.5 years post-treatment versus baseline.
GenSight Biologics has achieved significant regulatory milestones in France for its investigational gene therapy GS010/LUMEVOQ (lenadogene nolparvovec), designed to treat vision loss (search) caused by Leber Hereditary Optic Neuropathy (search) (LHON (search)) with ND4 mitochondrial mutations. The French medicines safety agency ANSM (search) has both authorized a dose-ranging clinical study and granted compassionate use authorization for the therapy.
Regulatory Approvals Support Clinical Development
The ANSM (search) authorized the REVISE dose-ranging study after assessing the GS010/LUMEVOQ quality dossier and clinical aspects. The study also received approval from the Ethics Committee. This regulatory approval supports the ongoing review of GenSight Biologics' application for the French named patient early access program (AAC).
REVISE is designed as an open-label, single center, Phase II study targeting enrollment of 14 patients. All patients must have confirmed mutations in the ND4 gene (search) and must have experienced 6 months to 1.5 years of vision loss (search) at the date of treatment. The study will investigate two clinical doses, with patients distributed equally between the two dose levels.
The primary endpoint measures efficacy through changes in Best Corrected Visual Acuity (BCVA) at 1.5 years post-treatment versus baseline. The study is expected to begin in January 2026.
Compassionate Use Authorization Granted
In a separate regulatory decision, ANSM (search) granted compassionate use authorization (Autorisation d'Accès Compassionnel, or AAC) for GS010/LUMEVOQ. The AAC Program in France enables patients suffering from serious, rare or disabling diseases to benefit from treatments that do not have marketing authorization when there is an unmet medical need and no appropriate therapy available.
To be eligible for an AAC program, the candidate treatment must present a favorable benefit-risk ratio. Healthcare professionals may initiate requests for AAC by submitting named patient requests to the ANSM (search), which evaluates and authorizes the access requests. Patients for whom applications for treatment with GS010 are submitted must meet specific eligibility criteria, including requirements related to the length of time since the onset of their vision loss (search).
Strategic Development Priorities
While the AAC application was under review, GenSight Biologics continued advancing according to target timelines with the final stages of technology transfer to its new manufacturing partner and preparations to finalize the protocol for the Phase III study. The company is pursuing opportunities to out-license GS010 in markets outside the USA and Europe, while exploring paid Early Access Programs worldwide.
Targeting Rare Mitochondrial Disease
LHON (search) is a rare, maternally inherited mitochondrial genetic disease characterized by the degeneration of retinal ganglion cells (search), which results in precipitous and usually irreversible vision loss (search) and typically leads to legal blindness (search). The ND4 mitochondrial mutation (search) is the most common of the mutations that cause LHON and is associated with the worst prognosis among the leading mutations.
GS010/LUMEVOQ targets LHON (search) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision (search) in Paris. When associated with the gene of interest, the platform allows specific addressing of defects inside the mitochondria (search) using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which is then shuttled to the mitochondria through specific nucleotidic sequences to restore the missing or deficient mitochondrial function.
The gene therapy is designed to be administered as a single intravitreal injection per eye. GS010/LUMEVOQ is currently in Phase III clinical development and has not been granted marketing authorization in any country.