January 15, 2025 - 🧬 [nGram] Today’s News Scoop: GenSight's 5-Year LUMEVOQ® Results, Annovis Patent, Mitophagy in Neurodegeneration


  1. GenSight Biologics publishes 5-year outcomes for LUMEVOQ gene therapy
    • GenSight Biologics announced 5-year follow-up results for LUMEVOQ gene therapy in treating Leber Hereditary Optic Neuropathy (LHON).
    • The study showed sustained bilateral improvement in Best-Corrected Visual Acuity (BCVA) and a good safety profile.
    • 66.1% of participants achieved clinically meaningful improvement in at least one eye, with 80.6% having on-chart vision.
    • The therapy was well-tolerated with no severe ocular events, and quality of life scores improved significantly.
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  2. Annovis granted U.S. patent for treatment and prevention of acute brain or nerve injuries
    • Annovis Bio has been granted a U.S. patent for the use of buntanetap in treating acute brain or nerve injuries.
    • The patent covers methods to reduce neurotoxicity and mitigate neurodegenerative processes in conditions like stroke and traumatic brain injury.
    • This U.S. patent complements existing patents in the EU, Japan, and worldwide, securing global protection.
    • The patent was issued by the U.S. Patent and Trademark Office on January 2, 2025.
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  3. Mission Therapeutics highlights mitophagy's role in tackling neurodegenerative diseases
    • Mission Therapeutics published a review in Nature Reviews Drug Discovery on enhancing mitophagy to address neurodegenerative diseases.
    • The review discusses mitochondrial dysfunction as a hallmark of diseases like Parkinson's, Alzheimer's, ALS, and Huntington's.
    • Two molecular approaches, USP30 inhibition and PINK1 activation, are being tested in early-stage clinical trials.
    • Three selective mitophagy enhancers have entered clinical trials in the past year, aiming to modify disease progression.
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