Administration of ER-100 was well-tolerated through Day 56 in three participants with glaucoma, supporting dose escalation in the ongoing trial

BOSTON, Oct. 08, 2026 (GLOBE NEWSWIRE) -- Life Biosciences (“Life Bio”), a biotechnology company pioneering cellular rejuvenation medicines to reverse diseases of aging, today announced interim data from its Phase 1 clinical trial of ER-100 in three participants with open angle glaucoma (OAG). The data demonstrated that a single intravitreal administration along with daily oral doxycycline was well-tolerated through Day 56 in all three participants and showed preliminary signals of improvement in visual field testing in two participants.

“These initial results provide early information regarding the tolerability of ER-100 and epigenetic restoration in people with open angle glaucoma. We are encouraged by the Data Safety Monitoring Board’s recommendation to escalate to Dose Level 2, a decision that allows us to keep moving this trial forward on behalf of people living with optic neuropathies like glaucoma, a disease where vision loss is often considered irreversible,” said Sharon Rosenzweig-Lipson, Ph.D., Chief Scientific Officer of Life Bio. “While this Phase 1 study is designed to evaluate safety and tolerability, the preliminary observations of visual field improvements in two participants are encouraging as we continue to advance the ER-100 program. This milestone marks an important step in Life Bio’s progress translating epigenetic restoration into a new class of medicines for age-related diseases.”

Presentation highlights:

Safety and Tolerability Observations

  • A single intravitreal administration of Dose Level 1 ER-100 (2x1011 vg) along with daily oral doxycycline to induce OSK expression was well-tolerated in 3 participants with open angle glaucoma (OAG) through Day 56
  • There were no reported dose-limiting toxicities, adverse events of special interest or serious adverse events
  • All reported adverse events were related to procedure, steroid, or pre-existing conditions and were not directly associated with ER-100
  • Data Safety Monitoring Board recommendation was to escalate to Dose Level 2 (6x1011 vg) in OAG participants, with the trial currently ongoing


Visual Field Observations

  • Improvements in Humphrey Visual Fields measurements, a standard eye exam that measures a participant’s ability to detect light across many points in their field of vision, scored in decibels (dB), were observed in 2 of 3 OAG participants at Day 56
  •  In each of these 2 participants, ≥ 28% of points in the visual fields showed a >4 dB improvement

This first-in-human, Phase 1, open-label clinical trial (NCT07290244) is evaluating the safety and tolerability of ER-100 in adults with optic neuropathies. ER-100 uses controlled expression of three transcription factors, OSK (OCT4, SOX2, KLF4), delivered via a single intravitreal injection, with OSK expression induced by systemic doxycycline administered for 8 weeks (56 days). An oral prednisolone taper is administered to minimize risk of inflammation due to AAV-2 immune response. Multiple study visits assessing tolerability and visual endpoints were included in this first 56 day evaluation.

About Optic Neuropathies
Optic neuropathies are a group of disorders characterized by damage to retinal ganglion cells (RGCs), the primary neurons connecting the eye to the brain. Because RGCs do not naturally regenerate, damage results in permanent vision impairment. One such optic neuropathy, open-angle glaucoma (OAG) is a chronic neurodegenerative disease and a leading cause of blindness in older adults. While often associated with elevated intraocular pressure, disease progression frequently continues despite treatment, and some patients suffer from OAG despite normal intraocular pressure. Non-arteritic anterior ischemic optic neuropathy (NAION) is the most common acute optic neuropathy in adults over fifty. It involves sudden, painless vision loss due to insufficient blood flow, for which there are currently no approved treatments.

About ER-100
ER-100 is an investigational therapy in clinical development for the treatment of optic neuropathies including OAG and NAION. ER-100 is designed to restore function in retinal ganglion cells using Life Biosciences’ Epigenetic Restoration platform, which utilizes controlled expression of three transcription factors, OCT4, SOX2 and KLF4 (OSK), to reset cellular gene expression patterns and restore cells to a more youthful and functional state. ER-100 is currently being evaluated in a Phase 1 clinical trial. More information can be found at clinicaltrials.gov (NCT07290244): https://clinicaltrials.gov/study/NCT07290244

About Life Biosciences
Life Biosciences is a biotechnology company pioneering cellular rejuvenation using epigenetic restoration to reverse diseases of aging. The company’s proprietary Epigenetic Restoration platform utilizes three transcription factors, OCT4, SOX2, and KLF4 (OSK), to restore older and damaged cells to a younger and healthier state. This innovative approach targets a root cause of aging at the epigenetic level, and has the potential to address a wide range of serious age-related diseases. Life Bio’s lead program, ER-100, is in development for optic neuropathies, including open-angle glaucoma (OAG) and non-arteritic anterior ischemic optic neuropathy (NAION), with a Phase 1 clinical trial initiated in the first quarter of 2026. Beyond ER-100, the company is strategically broadening its therapeutic pipeline to address additional age-related diseases, underscoring the platform’s versatility and transformative potential. For more information, visit www.lifebiosciences.com or follow Life Bio on Twitter (X) and LinkedIn.

Media Contact
Madelin Hawtin
LifeSci Communications
lifebio@lifescicomms.com