GenSight Biologics Announces LUMEVOQ® Scientific Updates at AAO 2024
GenSight Biologics (Euronext: SIGHT), a biopharmaceutical company specializing in innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, has announced new scientific data on its gene therapy, LUMEVOQ®, to be presented at the 2024 American Academy of Ophthalmology (AAO) annual meeting in Chicago, Illinois, from October 18-21, 2024.
Key Opinion Leaders in Leber Hereditary Optic Neuropathy (LHON) will discuss the following topics:
- Comparison of LHON natural history with idebenone and LUMEVOQ®
- Real-world experiences with LUMEVOQ®
- Evidence of the contralateral effect
- Long-term outcomes from bilateral injection of LUMEVOQ®
Presentations and Posters:
- Poster: “Meta-analysis of Treatment Outcomes for Patients with m.11778G>A MT-ND4 Leber Hereditary Optic Neuropathy” (Poster PO004)
- Presenter: Nancy J. Newman, MD, Emory University School of Medicine
- Time: Saturday, October 19, 2024, 9:15 – 10:15 AM CDT
- Location: Hall A, Poster Theater
- Presentation: “Efficacy and Safety of Lenadogene Nolparvovec Gene Therapy for LHON in Real-Life Settings” (PA023)
- Presenter: Valerie Biousse, MD, Emory University School of Medicine
- Session: OP04 Neuro-Ophthalmology Original Papers
- Time: Saturday, October 19, 2024, 2:48 – 3:00 PM CDT
- Location: S405
- Poster: “Post-Mortem Analyses with Histopathological and Molecular Assessments Following AAV2 Gene Therapy in LHON” (Poster PO093)
- Presenter: Alfredo A. Sadun, MD, PhD, Doheny Eye Centers-UCLA
- Time: Sunday, October 20, 2024, 9:15 – 9:45 AM CDT
- Location: Station 2, Poster Discussion Lobby
- Presentation: “Four-Year Results of Bilateral Injection of Lenadogene Nolparvovec Gene Therapy for LHON” (PA047)
- Presenter: Patrick Yu-Wai-Man, FRCOphth, MBBS, PhD, University of Cambridge
- Session: OP08 Retina, Vitreous Original Papers
- Time: Sunday, October 20, 2024, 2:48 – 2:55 PM CDT
- Location: S405
About GenSight Biologics
GenSight Biologics S.A. is a clinical-stage biopharmaceutical company dedicated to developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. Its pipeline utilizes two core technology platforms: the Mitochondrial Targeting Sequence (MTS) and optogenetics. The lead product candidate, GS010, is in Phase III trials for LHON, a rare mitochondrial disease causing irreversible blindness in young people. This gene therapy is designed for intravitreal injection, offering potential long-term visual recovery.
About Leber Hereditary Optic Neuropathy (LHON)
LHON is a rare, maternally inherited mitochondrial disease characterized by the degeneration of retinal ganglion cells, leading to severe, irreversible vision loss, predominantly affecting adolescents and young adults. It typically presents as a painless, sudden loss of central vision, often affecting both eyes within a year.
About LUMEVOQ® (GS010; lenadogene nolparvovec)
LUMEVOQ® (GS010; lenadogene nolparvovec) targets LHON using a proprietary mitochondrial targeting sequence technology. It utilizes an AAV vector to deliver the gene of interest directly into cells, where it produces the necessary functional protein to restore mitochondrial function. The European Medicines Agency accepted “LUMEVOQ” as the name for GS010 in October 2018, but it has not yet been registered in any country.
