Life Biosciences recently announced that the first participant has been dosed in its phase 1 clinical trial of ER-100, a novel therapy intended to treat optic neuropathies including Open-Angle Glaucoma (OAG) and Non-arteritic Anterior Ischemic Optic Neuropathy (NAION).

The phase 1 trial will evaluate the safety and tolerability of ER-100, with additional endpoints assessing visual function. ER-100 is the first clinical candidate from Life Bio’s Epigenetic Restoration platform, which uses controlled expression of 3 transcription factors, OCT4, SOX2 and KLF4 (OSK) to restore cellular function by resetting the epigenetic code to more youthful patterns of gene expression.

David Sinclair, PhD., Co-Founder, Life Biosciences and Professor—Genetics, Harvard Medical School, said, “This is an important moment for Life Bio and for the field of aging biology. Our research has suggested that aging is driven in large part by the loss of epigenetic information, not irreversible damage. This clinical study represents the first opportunity to test whether restoring that information can ameliorate human disease.”

Optic neuropathies represent a large unmet medical need. Current treatments primarily address risk factors, such as intraocular pressure in glaucoma, but do not directly target the damage to retinal ganglion cells. As a consequence, the disease often leads to irreversible vision loss despite treatment. Vision loss not only directly impacts patients’ lives, but also increases the risk of loss of independence, damaging falls, and depression and dementia due to social isolation, underscoring the need for disease-modifying therapies.

Sharon Rosenzweig-Lipson, Ph.D., Chief Scientific Officer (CSO), Life Biosciences, said, “This milestone reflects years of rigorous scientific development and translational research. Our preclinical studies have demonstrated that controlled OSK expression can reset epigenetic patterns associated with healthy cellular function, improve tissue performance, and restore visual function in animal models. Advancing ER-100 into the clinic is an important step toward translating epigenetic restoration into a new class of medicines for age-related diseases.”

Beyond ER-100, Life Bio is developing applications of its proprietary Epigenetic Restoration platform for multiple indications in a variety of organs, reflecting the broad therapeutic potential of this platform.