GenSight Biologics has submitted an updated regulatory file for its LUMEVOQ® gene therapy to the French medicines safety agency (ANSM). This submission paves the way for the restart of the early access program in France. The company successfully manufactured LUMEVOQ®, blending two GMP drug substance batches to maximize available clinical vials and pass all necessary quality control tests.
LUMEVOQ® is designed to treat Leber Hereditary Optic Neuropathy (LHON) caused by a mutated ND4 mitochondrial gene. LHON is a rare genetic condition leading to acute and often irreversible vision loss. The resubmission signifies a shift from the manufacturing phase to the regulatory review process. The company and the LHON patient community anticipate the swift resumption of the early access program by the end of the year.
The updated file will facilitate the ANSM’s evaluation of individual compassionate use applications, now submittable by healthcare professionals under the early access program. GenSight Biologics anticipates a review period for these applications and will collaborate with the ANSM to streamline the assessment process. The company is preparing to supply the drug to the designated treatment center, the Quinze-Vingts hospital in Paris, by mid-December, working alongside the hospital’s administrative and medical staff to administer the first injections by the end of December.
GenSight Biologics is a clinical-stage biopharmaceutical company focused on developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. Its pipeline utilizes two primary technology platforms: Mitochondrial Targeting Sequence (MTS) and optogenetics. These technologies aim to preserve or restore vision in patients with blinding retinal diseases. The company’s lead product candidate, GS010 (also known as LUMEVOQ®), is undergoing Phase III trials for LHON, primarily affecting adolescents and young adults, causing irreversible blindness.
LHON is a rare, maternally inherited mitochondrial genetic disease. The disease is characterized by the degeneration of retinal ganglion cells, leading to rapid and irreversible vision loss, often resulting in legal blindness. Typically, patients experience painless, sudden central vision loss in one eye, followed by impairment in the other. LHON is a symmetrical condition with limited functional visual recovery. Within a year of vision loss onset, 97% of individuals experience bilateral involvement, and in 25% of cases, both eyes are affected simultaneously.
LUMEVOQ® utilizes a proprietary Mitochondrial Targeting Sequence (MTS) technology platform developed from research at the Institut de la Vision in Paris. This technology, combined with the gene of interest, enables precise targeting of mitochondrial defects using an AAV (Adeno-Associated Virus) vector. The gene of interest is introduced into the cell, where it expresses the functional protein. This protein is transported to the mitochondria via specific nucleotide sequences to restore the missing or deficient mitochondrial function. The European Medicines Agency (EMA) approved “LUMEVOQ” as the invented name for GS010 (lenadogene nolparvovec) in October 2018. Currently, LUMEVOQ® is not registered in any country.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
