GenSight Biologics has achieved a critical manufacturing milestone by successfully transferring the upstream phase of its LUMEVOQ gene therapy production process to contract development and manufacturing organization Catalent Inc. The transfer supports the company's strategy to secure reliable supply for clinical trials and regulatory submissions targeting Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease causing irreversible blindness.
Manufacturing Partnership Delivers Key Capabilities
Catalent Inc. stands as the only contract development and manufacturing organization with a successfully commercialized gene therapy produced in its facility. The partnership provides GenSight with both production and in-house testing capabilities, while the GMP capacity at Catalent's facility offers greater manufacturing flexibility for the gene therapy.
"This outstanding result is a significant milestone in GenSight's strategy for securing the supply of LUMEVOQ for clinical use and for supporting the planned regulatory submissions," commented Scott Jeffers, Chief Technical Officer of GenSight Biologics. "Our new partnership is proving to be highly effective, not just in completing the tech transfer process successfully, but also in improving the yield and upgrading the analytical methods used to reinforce control over the safety and quality of each batch."
Clinical Supply Chain Secured
Catalent successfully manufactured a drug product batch that received safety clearance for human use in November 2024. This batch will serve as the product supply source for France's named patient early access program (AAC) and dose-ranging study. Following completion of the technology transfer, Catalent will manufacture drug supply for the planned global Phase III trial RECOVER and regulatory submissions.
The French agency ANSM has agreed to consider opening the AAC program expeditiously after approval of the dose-ranging study, providing a pathway for patient access while the company advances its global marketing authorization strategy.
Strategic Positioning for Global Development
GenSight is implementing a comprehensive strategy that includes preparation for regulatory consultations in the US and EU, planning for the global Phase III trial scheduled to begin in 2026 designed to meet FDA and EMA requirements, and advancing preparations for MHRA submission in the United Kingdom.
"Our partnership with a manufacturing powerhouse like Catalent is a critical enabler of our global strategy," explained Laurence Rodriguez, CEO of GenSight Biologics. "The outputs from their work with our team will allow us to reassure regulators that we have surmounted the challenges we faced in the past and, ultimately, to fulfill our mission to provide LHON patients a safe and effective treatment for their urgent unmet need."
About LHON and LUMEVOQ
Leber Hereditary Optic Neuropathy is a rare maternally inherited mitochondrial genetic disease characterized by degeneration of retinal ganglion cells, resulting in brutal and irreversible vision loss that can lead to legal blindness. The condition mainly affects adolescents and young adults, with 97% of subjects experiencing bilateral involvement within one year of onset, and 25% of cases involving simultaneous vision loss in both eyes.
LUMEVOQ (GS010; lenadogene nolparvovec) leverages GenSight's proprietary mitochondrial targeting sequence technology platform to address defects inside mitochondria using an AAV vector. The therapy transfers genes into cells to produce functional proteins that restore missing or deficient mitochondrial function through intravitreal injection to each eye.
