GenSight Biologics Announces Initial Results from New Meta-Analyses on Visual Outcomes with LUMEVOQ Gene Therapy at NANOS 2024 - Seite 3

About Leber Hereditary Optic Neuropathy (LHON)

Leber Hereditary Optic Neuropathy (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, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1^st eye, with the 2^nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of subjects have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously.

About LUMEVOQ (GS010; lenadogene nolparvovec)

LUMEVOQ (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. “LUMEVOQ” was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018. LUMEVOQ (GS010; lenadogene nolparvovec) has not been registered in any country at this stage.

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¹ Newman et al: J Neuro-Ophthalmol 2020 Dec;40(4):547-557.
² “Clinically Relevant Recovery”, or CRR, denotes an improvement in Best-Corrected Visual Acuity (BCVA) that satisfies one of two conditions: (1) A 10-letter (≥0.2 LogMAR) improvement for an on-chart starting visual acuity. (2) Improvement from “off-chart” to “on-chart” (≤1.6 LogMAR).
³ Nadir = lowest observation of visual acuity between baseline and time point of interest.
⁴ Three meta-analyses pre-definedin a statistical analysis plan
⁵ Sources of data: For idebenone and natural history data, systematic review of the literature and available clinical/regulatory reports on ND4 LHON patients; for Lumevoq, all phase 3 studies (RESCUE/RESTORE, REVERSE/RESTORE and REFLECT).
⁶ Carelli et al: J Neuro-Ophthalmol 2017; 0: 1-11

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The Gensight Biologics Stock at the time of publication of the news with a raise of +0,40 % to 0,497EUR on Lang & Schwarz stock exchange (12. März 2024, 07:32 Uhr).