GENSIGHT BIOLOGICS

NICE has recommended idebenone (Raxone) as the first NHS treatment for Leber's hereditary optic neuropathy (LHON) in England, benefiting an estimated 250 eligible patients aged 12 and over.

GenSight Biologics has successfully transferred the upstream manufacturing process for LUMEVOQ, its gene therapy for Leber Hereditary Optic Neuropathy (LHON), to manufacturing partner Catalent Inc.

GenSight Biologics reached agreement with French regulatory agency ANSM to expedite opening of early access program for LUMEVOQ gene therapy following approval of a dose-ranging study.

• GenSight Biologics' LUMEVOQ demonstrates sustained visual acuity improvement in Leber Hereditary Optic Neuropathy (LHON) patients five years post-treatment. • Bilateral injections of LUMEVOQ resulted in a higher rate of clinically relevant visual recovery compared to unilateral treatment in the REFLECT Phase III trial. • The gene therapy maintained a favorable safety profile over the five-year study period, with no serious ocular adverse events reported. • RESTORE study showed that Lumevoq-treated eyes achieved a mean improvement against nadir of +22 letters equivalent and sham-treated eyes demonstrated a mean improvement of +20 letters equivalent.

• A 5-year follow-up study (RESTORE) showed that lenadogene nolparvovec gene therapy provides sustained visual acuity improvement in both eyes of LHON patients with the MT-ND4 mutation. • The study demonstrated a mean change in BCVA of -0.4 logMAR (more than +4 lines) in treated eyes, similar to sham-treated eyes, indicating a potential bilateral therapeutic effect. • Clinically relevant recovery, defined as a BCVA improvement of at least -0.3 logMAR (+3 lines), was observed in 66.1% of participants, with improvements in quality of life. • The gene therapy demonstrated a favorable long-term safety profile, with manageable intraocular inflammation and no permanent complications reported.

• Leading biotech companies and academics gather at the 2nd Mitochondria-Targeted Drug Development Summit to tackle unmet medical needs in mitochondrial dysfunction disorders. • The summit will explore innovative approaches including gene therapy, mitochondrial transfer, and precision medicine for treating conditions ranging from muscle dystrophy to neurodegenerative diseases. • Industry leaders from Mitobridge/Astellas, Minovia Therapeutics, and other prominent companies will present advances in mitochondrial quality control and oxidative phosphorylation research.

• Industry leaders from Stealth BioTherapeutics, Cohbar, and GenSight Biologics will gather at the exclusive Mitochondria-Targeted Drug Development Digital Summit to share breakthrough research developments. • The summit will showcase innovative approaches including Mitokinin's PINK1 activation for Parkinson's disease and Larimar Therapeutics' CTI-1601 protein replacement therapy for Friedreich's Ataxia. • Over 80 key industry experts will participate in 20+ hours of content and workshops, focusing on advancing therapeutics for mitochondrial and age-related diseases.