Life Biosciences Presents New Data on Novel Gene Therapy at AAO
Life Biosciences announced new data at the 2024 American Association of Ophthalmology (AAO) annual meeting on its gene therapy for optic neuropathies. According to Life Biosciences, the data replicates earlier findings in nonhuman primates (NHPs) and provides insights into the dosing and timing of the therapy.
The gene therapy, ER-100, employs partial epigenetic reprogramming and features three transcription factors—Oct-4, Sox-2, and Klf-4 (OSK)—administered via a single intravitreal injection. This was paired with daily systemic doxycycline in NHPs with a NAION-like (non-arteritic anterior ischemic optic neuropathy) injury. The study confirmed that ER-100 successfully expresses these factors in specific retinal cells and demonstrated significant protection against visual function loss.
Immunohistochemistry revealed the expression of each transcription factor in discrete cells within the perifoveal regions in ER-100-treated NHPs, compared to controls receiving the vehicle. Furthermore, ER-100 markedly reduced damage to retinal ganglion cells, showing improvements in both pattern electroretinogram measures and axon density. This was seen in both preventive and rescue treatment protocols, highlighting ER-100’s potential to halt or even reverse vision loss when administered after injury.
“Partial epigenetic reprogramming has the potential to restore visual function for those with optic neuropathies such as NAION and glaucoma," Dr. Sharon Rosenzweig-Lipson, Chief Scientific Officer of Life Biosciences, said in a company news release. "The data shared today replicate and build upon our 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.”
With these results, Life Bio said it remains on track to launch the first human clinical trials of ER-100 for optic neuropathies in the second half of 2025.