GenSight Biologics announced a pivotal regulatory agreement with France's medicines safety agency ANSM that establishes a clear pathway for opening early patient access to its gene therapy LUMEVOQ for treating Leber Hereditary Optic Neuropathy (LHON). The agreement enables expedited consideration of the French named early access program (AAC) upon approval of a focused dose-ranging clinical study.
Regulatory Milestone Achieved
Following extensive discussions, GenSight reached alignment with ANSM on opening the LUMEVOQ AAC program. The French regulatory agency agreed that authorization of a focused dose-ranging study could enable the early access program launch. The company has submitted a preliminary study design to ANSM and aims to finalize the dose-ranging study protocol in Q3 2025.
"In the interest of clearing the way for patients to be treated with LUMEVOQ, we are pleased to have achieved this important agreement with ANSM on a clear path forward for opening patient access to LUMEVOQ in France," said Laurence Rodriguez, Chief Executive Officer of GenSight Biologics. "We are thankful for the ANSM's ongoing engagement and openness to discussion, as we work towards the shared goal of providing access to LHON patients who urgently need a safe and effective treatment."
The AAC program is targeted to open in Q4 2025 at the latest. In parallel, GenSight will collaborate with ANSM to develop solutions for patients who may not be included in the dose-ranging study but could benefit from the early access program.
Strategic Timeline and Value Catalysts
GenSight has outlined multiple value-creating milestones for 2025-2026:
- Q3 2025: Clinical trial application for focused dose-ranging study
- Q4 2025: Completion of manufacturing technology transfer and AAC program opening
- Q2 2026: Completion of Phase III trial preparations
- Early H2 2026: Initiation of global Phase III clinical trial and pre-submission meeting with MHRA
Global Development Strategy
Beyond the French early access program, GenSight continues advancing its comprehensive marketing authorization strategy. The company is preparing for regulatory consultations in the US and EU, planning a global Phase III trial scheduled to begin in 2026 designed to meet FDA and EMA requirements.
The company is also completing transition to a new manufacturing partner to secure reliable clinical and commercial supply and advancing preparations for MHRA submission in the United Kingdom.
Financing and Operational Considerations
GenSight is implementing a financing strategy to bridge operations through the AAC program opening and prepare for the global Phase III clinical trial. "We are working on our financing strategy to manage the bridge between now and the opening of the AAC program and the initiation of the Phase III study," commented Jan Eryk Umiastowski, CFO of GenSight Biologics.
The company received €0.7 million of its Research Tax Credit in May, with the remaining €0.4 million expected in July 2025. Based on current operations and projections, GenSight's cash will support operations until mid-July 2025. The company acknowledges that current funds are insufficient to cover operational requirements for the next 12 months, with planned financing initiatives designed to extend the cash runway.
About LUMEVOQ and LHON
LUMEVOQ (GS010, lenadogene nolparvovec) is GenSight's lead gene therapy candidate in Phase III development for LHON, a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. The treatment leverages the company's Mitochondrial Targeting Sequence (MTS) technology platform and is designed to be administered as a single treatment to each eye by intravitreal injection to offer patients sustainable functional visual recovery.
As of June 12, 2025, GenSight Biologics had 131,466,495 outstanding ordinary shares.
