Life Biosciences Presents New Data on Epigenetic Reprogramming for Optic Neuropathies at AAO 2024
Life Biosciences, a biotech company advancing therapies to reverse aging-related diseases, shared new findings at the American Academy of Ophthalmology (AAO) Annual Meeting, held from October 18-21, 2024, in Chicago. The company presented updates on its gene therapy platform, which aims to restore vision in optic neuropathies like nonarteritic anterior ischemic optic neuropathy (NAION) and glaucoma by rejuvenating retinal cells.
Key Findings:
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ER-100 Gene Therapy: ER-100 uses partial epigenetic reprogramming to "reset" the biological age of retinal cells by expressing three key transcription factors: OCT4, SOX2, and KLF4 (OSK). These factors help reprogram the epigenome, the regulatory system that controls gene expression, bringing older cells closer to a more youthful state. ER-100 is administered via an intravitreal injection combined with daily oral doxycycline, which activates the therapy.
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Effectiveness in Nonhuman Primate Models: In a nonhuman primate (NHP) model mimicking NAION-related damage, ER-100 successfully improved retinal ganglion cell function and axon survival.
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Targeted and Safe: Immunohistochemistry, a method to visualize specific cells in tissue, confirmed that the therapy specifically affected cells in the retina while sparing other tissues. This precision is critical to ensuring the therapy only targets the affected areas. Additionally, no adverse safety findings were reported after systemic delivery in preclinical studies. Systemic delivery means the therapy affects the entire body, and the absence of negative effects suggests its safety for future use.
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Prevention and Rescue: The therapy showed benefits in both prevention and rescue (administered after damage) settings.
Mechanism of action of the Partial Epigenetic Reprogramming (Source: Life Biosciences)
Dr. Sharon Rosenzweig-Lipson, Chief Scientific Officer at Life Bio:
"Partial epigenetic reprogramming has the potential to restore visual function for those with optic neuropathies such as NAION and glaucoma. The data shared today build upon our understanding of ER-100’s effectiveness in targeting retinal ganglion cells at different stages of damage."
Looking ahead, Life Biosciences plans to begin human clinical trials for ER-100 in 2025, further exploring its potential to treat optic neuropathies. The company’s broader platform, which also includes chaperone-mediated autophagy (CMA)—a process that clears unwanted proteins from cells—targets aging-related diseases such as neurodegeneration and retinal degeneration. Both platforms represent Life Bio's commitment to addressing unmet needs in aging-related diseases.
