Israel Grants Regulatory Authorization for Early Access Use of GS010/LUMEVOQ in ND4-LHON Patients
GenSight Biologics, a biopharmaceutical company dedicated to the development and commercialization of innovative gene therapies for retinal neurodegenerative diseases and disorders of the central nervous system, has announced that Israel’s Ministry of Health has authorized early access treatment for individual patients with its investigational gene therapy GS010/LUMEVOQ®. The authorization represents an important milestone in expanding access to the therapy for patients suffering from Leber Hereditary Optic Neuropathy linked to the ND4 mitochondrial mutation (ND4-LHON), a rare and severely debilitating genetic eye disease.
GenSight Biologics is listed on Euronext under the ticker SIGHT (ISIN: FR0013183985) and is eligible for the PEA-PME investment scheme. The company’s announcement underscores its continued commitment to addressing unmet medical needs through regulatory collaboration, compassionate use programs, and strategic global partnerships.
Regulatory Approval Enables Early Access for Individual Patients in Israel
The authorization was granted by Israel’s Ministry of Health Pharmaceutical Division and permits the use of GS010/LUMEVOQ® for early access treatment on a named-patient basis. Under this framework, eligible patients may receive the investigational therapy outside of a formal clinical trial setting, provided specific regulatory and ethical requirements are met.
Early access treatment in Israel is reserved for patients with life-threatening or seriously debilitating conditions for which no satisfactory, approved treatment options exist either domestically or internationally. Additionally, patients must be unable to participate in a suitable clinical trial. ND4-LHON meets these criteria due to its rapid onset, severe vision loss, and lack of approved curative therapies.
The process for early access treatment begins with a treating physician, who must determine that the investigational therapy is medically justified for the patient. The request is then reviewed by the hospital’s ethics committee, commonly referred to as the Helsinki Committee, and subsequently evaluated by the Ministry of Health. Approval is contingent upon a thorough assessment of scientific rationale, non-clinical and clinical data, and published professional literature supporting the therapy’s potential benefit-risk profile.
GenSight Biologics confirmed that bilateral intravitreal injection of GS010/LUMEVOQ® for eligible patients is expected to be performed in the first quarter of 2026, subject to final approvals and logistical readiness.
Addressing an Urgent Unmet Need in ND4-LHON
Leber Hereditary Optic Neuropathy is a rare mitochondrial genetic disorder that typically affects young adults, often leading to rapid and profound vision loss in both eyes. The ND4 mutation is one of the most common genetic causes of LHON. Patients frequently experience sudden central vision loss that can progress within weeks or months, resulting in long-term disability and significantly reduced quality of life.
GS010/LUMEVOQ® is designed to address the underlying genetic defect responsible for ND4-LHON. By delivering a functional copy of the ND4 gene directly to retinal ganglion cells, the therapy aims to restore mitochondrial function and preserve or improve visual outcomes. Although GS010/LUMEVOQ® has not yet received full market authorization, it has been evaluated in multiple clinical studies, generating a body of data that has supported regulatory discussions and early access initiatives in several countries.
Collaboration with Local Partner SK-Pharma
To facilitate patient access in Israel, GenSight Biologics is working closely with SK-Pharma, its strategic partner in the country. The collaboration focuses on ensuring that eligible patients with urgent unmet medical needs can navigate the early access process efficiently while complying with all regulatory and ethical requirements.
Through this partnership, GenSight aims to support physicians, hospitals, and regulatory bodies by providing necessary clinical documentation, scientific evidence, and logistical coordination for the delivery and administration of GS010/LUMEVOQ®.
Executive Commentary Highlights Global Early Access Strategy
Commenting on the authorization, Laurence Rodriguez, Chief Executive Officer of GenSight Biologics, expressed satisfaction with the regulatory outcome and emphasized the company’s patient-focused mission.
“We are very pleased that we are able to provide limited quantities of GS010/LUMEVOQ® for early access treatment worldwide, following authorization by relevant competent authorities,” said Ms. Rodriguez. “We are particularly gratified that the clinical and scientific evidence we were able to submit supported the compassionate treatment application in Israel.”
She further noted that the ability to offer early access reflects both the maturity of the GS010 clinical data package and the company’s ongoing dialogue with regulatory agencies across multiple jurisdictions.
Balancing Early Access with Clinical Development Priorities
While expanding early access opportunities internationally, GenSight Biologics emphasized that it remains focused on ensuring sufficient supply of GS010/LUMEVOQ® for its ongoing and planned clinical programs. At present, the company is prioritizing supply for the REVISE dose-ranging study as well as the named patient Early Access Program (Autorisation d’Accès Compassionnel, AAC) recently authorized in France.
“These programs represent essential steps toward our ultimate goal of maximizing patient access to GS010/LUMEVOQ® through full market authorizations in key markets around the world,” Ms. Rodriguez explained.
The company noted that early access initiatives are carefully managed to avoid disruption to clinical trials, which remain critical for generating the robust evidence required for regulatory approval.
Manufacturing Transition and Supply Expansion
As part of its broader operational strategy, GenSight Biologics is in the final stages of completing a technology transfer to its new manufacturing partner, Catalent. This transition is expected to be finalized by the end of 2025 and is intended to strengthen the company’s long-term manufacturing capabilities.
Once complete, the partnership with Catalent will enable the production of new GS010/LUMEVOQ® batches beginning in 2026. These additional manufacturing capacities are expected to fully address projected clinical and early access needs, supporting both ongoing studies and potential expansion of compassionate use programs globally.
Preparing for Pivotal Phase III Development
In parallel with manufacturing scale-up, GenSight Biologics is preparing to reengage with global regulatory agencies to advance the next phase of clinical development for GS010/LUMEVOQ®. The company plans to pave the way for the launch of the pivotal Phase III RECOVER study in the second half of 2026.
The RECOVER study is designed to confirm the therapy’s efficacy and safety in a larger patient population and represents a key step toward potential regulatory submissions. GenSight also indicated that it is preparing for a future submission in the United Kingdom, reflecting its intention to pursue market authorization in multiple regions.
Strategic Business Development Initiatives
Beyond Europe and the United States, GenSight Biologics continues to explore opportunities to out-license GS010/LUMEVOQ® in additional markets. These discussions aim to leverage regional expertise and infrastructure to accelerate patient access while optimizing the commercial potential of the therapy.
At the same time, the company is evaluating the feasibility of paid Early Access Programs in select countries, where regulatory frameworks permit such arrangements. These programs could provide earlier treatment options for patients while contributing to the sustainability of GenSight’s development efforts.
Financial Calendar and Upcoming Milestones
GenSight Biologics also provided an overview of its upcoming financial and corporate milestones for 2026. These include regular cash position updates, shareholder meetings, and financial statement releases that will inform investors and stakeholders of the company’s progress.
Key dates include the renewal of financial authorizations for potential fundraising, publication of full-year and half-year financial statements, and scheduled annual and extraordinary general meetings.
About Leber Hereditary Optic Neuropathy (LHON)
LHON is a rare, maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells, which results in precipitous and usually irreversible vision loss and typically leads to legal blindness. The ND4 mitochondrial mutation is the most common of the mutations that cause LHON and is associated with the worst prognosis among the leading mutations.
About GenSight Biologics
GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics’ pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics’ lead product candidate, GS010 (lenadogene nolparvovec) is being investigated as a treatment for Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. GS010 is currently in clinical development, has not to date been granted marketing authorization in France or any other jurisdiction, and is not available commercially. Using its gene therapy-based approach, GenSight Biologics’ product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.
