GenSight Biologics Reveals Positive 5-Year Outcomes For Patients Treated Unilaterally With LUMEVOQ

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(RTTNews) - GenSight Biologics (GSGTF), a biopharma company focused on gene therapies for retinal neurodegenerative diseases and central nervous system disorders, announced Wednesday positive outcomes data from five years' follow-up of patients treated unilaterally with LUMEVOQ, the company's investigational gene therapy for Leber Hereditary Optic Neuropathy or LHON due to a mutated ND4 mitochondrial gene.

LHON is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness. It mainly affects adolescents and young adults.

The paper, published online by journal JAMA Ophthalmology in December 2024, found that patients "demonstrated a sustained bilateral improvement in BCVA [Best-Corrected Visual Acuity] and a good safety profile over 5 years after treatment".

The company noted that the patients had all participated in the Phase III trials RESCUE and REVERSE and accepted enrolling into the long-term study RESTORE at the end of the RESCUE and REVERSE studies.

As per the publication, the "persistent benefit" continues the durable effect observed at earlier time points and represents a significant addition to the body of evidence on the benefit-risk ratio of LUMEVOQ gene therapy in ND4 LHON patients.

Patrick Yu-Wai-Man, the lead author and Principal Investigator in the RESCUE, REVERSE and RESTORE studies, said, "The findings on the persistence of visual improvement show the promise of using LUMEVOQ gene therapy to treat patients with LHON due to the MT-ND4 gene variant. The results are especially encouraging given the poor visual prognosis of MT-ND4 LHON, which is the most frequent and severe clinical form of this rare mitochondrial genetic disease."

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