Life Biosciences reports advances in nonhuman primate studies on partial epigenetic reprogramming for restoring visual function

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Key Takeaways

ER-100 gene therapy uses partial epigenetic reprogramming with transcription factors Oct-4, Sox-2, and Klf-4.
Intravitreal injection of ER-100, combined with doxycycline, improved visual function in non-human primates with NAION-like injury.
ER-100 effectively targets retinal ganglion cells, reducing visual function loss and improving axon density.
Human clinical trials for ER-100 in optic neuropathies are scheduled for late 2025, aiming to restore visual function.

According to the company, its data replicate and expand upon the potential of ER-100 to improve retinal ganglion cell function and restore visual function in a nonhuman primate model of nonarteritic anterior ischemic optic neuropathy.

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Life Biosciences presented data at the American Association of Ophthalmology’s 2024 annual meeting in Chicago, focusing in results that replicate previously reported nonhuman primate (NHP) data and further expand our knowledge on dosing and timing of treatment.

ER-100, a novel gene therapy utilizing partial epigenetic reprogramming with the transcription factors Oct-4, Sox-2, and Klf-4 (OSK), was delivered via a single intravitreal injection in combination with daily systemic doxycycline to non-human primates (NHPs) with an induced NAION-like injury. Immunohistochemistry confirmed the expression of these transcription factors (Oct-4, Sox-2, and Klf-4) in distinct perifoveal cells in the ER-100-treated group compared to the vehicle-treated controls.

In addition, ER-100 significantly mitigated deficits in pattern electroretinogram responses and axon density in both prevention and rescue treatment models. These findings suggest that ER-100 effectively targets retinal ganglion cells, reducing visual function loss in this NHP model of NAION. Life Bio is on schedule to launch the first human clinical trial of ER-100 for optic neuropathies in the latter half of 2025.

“Partial epigenetic reprogramming has the potential to restore visual function for those with optic neuropathies such as NAION and glaucoma,” Sharon Rosenzweig-Lipson, PhD, chief scientific officer of Life Bio, said in a news release.

Rosenzweig-Lipson added the data shared replicate and build upon the company’s understanding of the dosing of ER-100 and the timing of the treatment window relative to the onset of NAION-related damage.

“We look forward to advancing this program toward human clinical trials next year for patients affected by these age-related optic neuropathies,” she concluded.