GenSight Biologics completes LUMEVOQ tech transfer to Catalent to enable global clinical expansion

GenSight Biologics S.A. (Euronext: SIGHT), the Paris-based clinical-stage gene therapy developer, has completed a critical milestone in the development of its lead candidate LUMEVOQ® (GS010; lenadogene nolparvovec), by successfully transferring the upstream manufacturing process to its new production partner Catalent, Inc. This strategic handover marks a foundational step in securing the supply chain for upcoming regulatory submissions and the planned global Phase III RECOVER trial targeting Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disorder causing irreversible vision loss.

The manufacturing shift to Catalent is expected to increase batch yield, enhance analytical robustness, and streamline the transition toward commercial scalability. These improvements come at a pivotal moment for GenSight Biologics as it prepares for broader regulatory engagements in the United States, Europe, and the United Kingdom. Institutional investors and biotech analysts view the partnership as a major de-risking event in the company’s trajectory, emphasizing the operational readiness required to meet stringent regulatory standards for advanced gene therapies.

How does the Catalent manufacturing transfer support GenSight Biologics’ Phase III trial and regulatory strategy for LUMEVOQ®?

The successful transfer of upstream production to Catalent is central to GenSight Biologics’ clinical and regulatory ambitions. The upstream phase encompasses the early stages of the gene therapy production process, and its completion enables the use of Catalent’s GMP-certified capabilities to generate clinical-grade materials suitable for human use. This development builds upon a prior manufacturing success in November 2024, when Catalent produced a drug product batch that passed all safety standards and is now designated for use in a named patient early access program (AAC) and a dose-ranging study in France.

The transfer is also significant because it ensures the continuity and scalability of supply as GenSight prepares to launch its Phase III RECOVER trial in 2026. Catalent will continue manufacturing future batches intended for this trial, as well as for use in eventual regulatory filings with the European Medicines Agency (EMA), the U.S. Food and Drug Administration (FDA), and the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA). These initiatives are aligned with GenSight Biologics’ broader clinical roadmap, which includes consultation with multiple regulators and the launch of global clinical activities designed to fulfill both FDA and EMA efficacy and safety requirements.

Why do analysts consider the Catalent partnership a de-risking milestone for GenSight Biologics?

Industry analysts and institutional investors have responded positively to GenSight Biologics’ operational execution, viewing the manufacturing transfer to Catalent as a significant milestone that reduces risks traditionally associated with gene therapy pipelines. Gene therapies require highly controlled production conditions and rigorous quality assurance protocols. By leveraging Catalent’s established infrastructure—which includes in-house analytical testing and previous success in commercializing gene therapies—GenSight Biologics gains access to a proven and scalable platform.

This partnership reassures stakeholders that the company has addressed prior production bottlenecks and now has the capability to consistently manufacture LUMEVOQ® to meet regulatory and commercial expectations. The collaboration also signals a turning point in GenSight’s readiness to transition from a clinical-stage developer to a commercially viable player in the gene therapy market.

What are the scientific and therapeutic foundations of LUMEVOQ®, and how does it aim to treat LHON?

LUMEVOQ® is an investigational gene therapy candidate designed to treat Leber Hereditary Optic Neuropathy (LHON), a rare and inherited mitochondrial disease. LHON typically presents in adolescents and young adults, leading to the rapid and irreversible loss of central vision. The disease affects both eyes in 97% of cases within a year of onset and is characterized by the degeneration of retinal ganglion cells, which play a key role in visual signal transmission.

GenSight Biologics has developed LUMEVOQ® using its proprietary Mitochondrial Targeting Sequence (MTS) platform, a technology developed in collaboration with the Institut de la Vision in Paris. This platform uses an adeno-associated virus (AAV) vector to deliver the gene of interest to retinal cells. Once inside the cell, the therapeutic gene expresses a functional protein designed to be directed into the mitochondria, where it compensates for the missing or defective protein responsible for the disease. The therapy is administered through a single intravitreal injection to each eye, with the goal of providing sustained visual recovery.

Though not yet approved in any country, LUMEVOQ® was recognized by the European Medicines Agency in 2018, which accepted the name for its active ingredient, lenadogene nolparvovec. The gene therapy has demonstrated promising outcomes in early-phase trials, and upcoming studies under the AAC and dose-ranging program are expected to generate additional safety and efficacy data for future marketing applications.

What future milestones does GenSight Biologics anticipate following this manufacturing advancement?

GenSight Biologics is now focused on the next phases of its development and regulatory agenda. The near-term priority is to execute the early access program and the dose-ranging study in France using the Catalent-manufactured batch released in late 2024. These efforts will support the generation of new clinical data and reinforce the treatment’s safety profile in preparation for broader filings.

In parallel, the company is preparing to initiate the RECOVER global Phase III trial, expected to begin in 2026. The trial design will adhere to the requirements of both the FDA and EMA and is aimed at producing confirmatory data for regulatory submissions. GenSight Biologics is also advancing preparations for a formal MHRA filing in the United Kingdom and has outlined a financing strategy to support the broader clinical and regulatory rollout of LUMEVOQ®.

With the upstream process now transitioned and validated at Catalent, GenSight Biologics will likely turn its attention to scaling downstream activities, should those responsibilities also be shifted to Catalent or another CDMO. The firm’s leadership, including CEO Laurence Rodriguez, views the Catalent partnership as instrumental in restoring confidence among regulators and ensuring that past manufacturing limitations no longer impede clinical or commercial progress.

How is institutional sentiment shaping around GenSight Biologics’ market positioning and gene therapy pipeline?

Institutional sentiment toward GenSight Biologics has been cautiously optimistic, particularly following recent updates that demonstrate operational and clinical momentum. Investors are watching closely for additional regulatory clarity, especially as the company engages in discussions with health authorities across major markets. The successful transition of manufacturing responsibilities to a globally recognized CDMO like Catalent is viewed as a critical risk mitigation step and enhances the biotech’s credibility within the competitive gene therapy space.

As a publicly traded company, GenSight Biologics has faced heightened pressure to deliver on clinical milestones and operational efficiency. The advances in manufacturing, combined with the potential initiation of the Phase III RECOVER trial, provide important validation points for shareholders and potential strategic partners. While the broader gene therapy market continues to evolve, with increasing regulatory scrutiny and demand for validated CMC processes, GenSight Biologics appears better positioned to navigate these challenges in 2025 and beyond.

What broader implications does this manufacturing success have for the gene therapy industry?

GenSight Biologics’ ability to successfully shift its upstream production to a commercial-grade CDMO reflects the maturing state of the gene therapy manufacturing ecosystem. As more gene therapies approach late-stage development, the importance of reliable, high-yield, and quality-controlled production processes becomes paramount. The partnership between a focused gene therapy innovator and a globally experienced manufacturer like Catalent illustrates a scalable model that may become more common as other developers seek to commercialize complex therapies for rare diseases.

From a regulatory and investor perspective, the GenSight-Catalent collaboration sends a positive signal that innovative biopharma firms can overcome legacy manufacturing hurdles through strategic outsourcing. It also demonstrates the critical role CDMOs play in supporting biotech pipelines from bench to bedside, ensuring that novel therapeutics are delivered with the precision and reliability required in high-stakes medical fields such as ophthalmology and mitochondrial disease treatment.

GenSight Biologics’ upstream manufacturing transfer of LUMEVOQ® to Catalent not only marks a critical operational milestone but also reinforces its broader readiness to enter the next phase of global clinical trials and regulatory engagements. As the biotech intensifies its focus on bringing a first-in-class therapy to patients with LHON, institutional interest is likely to grow alongside clinical evidence and manufacturing maturity. The success of this partnership may ultimately set a precedent for other gene therapy developers seeking a path to scalable, compliant, and commercially viable production.