Recruiting
Phase 1

ER-100

Sponsor:

Life Biosciences Inc.

Code:

NCT07290244

Conditions

Open Angle Glaucoma (OAG)

NAION( Non-arteritic Anterior Ischemic Optic Neuropathy)

Eligibility Criteria

Sex: All

Age: 40 - 70+

Healthy Volunteers: Not accepted

Interventions

ER-100 epigenetic therapy

Study Details

Brief summary:

The goal of this clinical trial is to evaluate the safety and tolerability of a single dose of ER-100 in adults with optic nerve conditions, specifically Open Angle Glaucoma (OAG) and Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION). The main questions it aims to answer are:

  • Is ER-100 safe when given as a single dose to people with OAG or NAION
  • What side effects may occur, if any, after taking ER-100?

Participants will:

  • Receive a single dose of ER-100
  • Undergo safety assessments including detailed eye examination and laboratory tests
  • Provide body fluid samples (tears, saliva, feces, urine) to help researchers understand how the drug is processed and cleared from the body
  • Complete questionnaires about their quality of life
  • Be followed for up to 5 years to monitor long-term health and vision outcomes

Conditions

Open Angle Glaucoma (OAG)

NAION( Non-arteritic Anterior Ischemic Optic Neuropathy)

Study ID

NCT07290244

Start date

Mar 2, 2026

Status verified date

Mar, 2026

Completion date

Mar, 2032

Anticipated

Primary completion date

May, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 40 - 70+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Have clear eye structures and be able to have your pupils safely dilated so the doctor can examine the back of your eye.
  • Able to understand the study and sign a consent form.
  • Be between 40 and 85 years old.
  • Willing and able to follow the study schedule, including all visits and tests, and speak a language for which the study materials are available.
  • If a participant can become pregnant, must agree to use a condom and one highly effective form of birth control during sex for at least 4 months after receiving the study drug (ER-100).

For participants with open-angle glaucoma (OAG):

  • Diagnosis of open-angle glaucoma in the study eye.
  • Eye pressure must be less than 30 mmHg, measured with a standard test.
  • Visual field test must show moderate to advanced vision loss (MD score between -6 and -20 dB).
  • Not expected to need glaucoma surgery in the study eye within 2 months after receiving ER-100.
  • Have reasonably good vision in the study eye (at least 20/80 on a standard eye chart).

For participants with NAION (non-arteritic anterior ischemic optic neuropathy):

  • Had a sudden, painless loss of vision in one eye within 14 days before receiving ER-100, confirmed by a specialist. Having had NAION in the other eye is okay.
  • The affected eye must show swelling of the optic nerve.
  • Visual field test must show vision loss consistent with optic nerve damage (MD worse than -3.0 dB).
  • If only one eye is affected, there must be a difference in pupil response between the two eyes.
  • Have vision in the affected eye between 20/40 and 2/500 on a standard eye chart.

Exclusion Criteria:

  • History of optic neuritis (inflammation of the optic nerve) or repeated episodes of eye inflammation (uveitis) not caused by injury or surgery.
  • Allergic reactions to tetracycline antibiotics or steroid medications.
  • Moderate to severe cataracts, macular problems, or corneal issues that could interfere with eye testing.
  • Unable to keep your eyes focused on a target during testing.
  • Had cataract surgery or other eye surgery (including laser procedures) within 3 months before receiving the study drug.
  • Had cancer (except for basal cell skin cancer) within the past 5 years.
  • Have Type 1 diabetes, or poorly controlled Type 2 diabetes (A1c greater than 7 despite treatment).
  • Have memory or thinking problems that prevent you from understanding the study or completing the required tests.
  • Pregnant or breastfeeding.
  • Have a weakened immune system, including a history of organ transplant, or test positive for HIV, hepatitis B or C, or tuberculosis.
  • Have any other condition that, in the opinion of the study doctor, could increase your risk from the study drug or procedures, affect the study results, or make it hard for you to complete the study.
  • Have macular disease, advanced diabetic eye disease, or other eye conditions that limit vision in the study eye.
  • Eye pressure at screening is 30 mmHg or higher.
  • Taking certain medications (warfarin, dilantin, carbamazepine, or barbiturates) within 14 days before starting the study or during the first 8 weeks.
  • Have any other eye or vision problem that, in the opinion of the study doctor, could affect safety or interfere with vision testing.
  • Have previously received any gene therapy using adeno-associated virus (AAV).

Additional Exclusion Criteria for Participants with Open-Angle Glaucoma (OAG):

\- Diagnosed with glaucoma before age 40.

Additional Exclusion Criteria for Participants with NAION:

  • Show signs of giant cell arteritis (a type of blood vessel inflammation), based on abnormal blood tests.
  • Had NAION start in both eyes at the same time.

Study Design

Enrollment

18 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: OAG - Low Dose ER-100 (2 x 10^11 vg/eye)

Participants with Open Angle Glaucoma will receive a low dose of ER-100 administered to one eye. ER-100 is delivered via a modified adeno-associated virus (AAV) vector and activated by systemic doxycycline taken for 8 weeks (56 days). This dose level begins with a sentinel participant followed by additional participants after DSMB review.

experimental: OAG - High Dose ER-100 (6 x 10^11 vg/eye)

Participants with Open Angle Glaucoma will receive a higher dose of ER-100 administered to one eye. ER-100 is delivered via a modified AAV vector and activated by systemic doxycycline for 8 weeks. This dose level also begins with a sentinel participant and proceeds following DSMB review.

experimental: NAION - Selected Dose ER-100

Participants with Non-Arteritic Anterior Ischemic Optic Neuropathy will receive ER-100 at a dose selected based on safety and tolerability data from the OAG cohort. ER-100 is administered to one eye and activated by systemic doxycycline for 8 weeks. Initial enrollment is limited to three participants, with potential expansion to six following DSMB review.

Interventions

ER-100 epigenetic therapy

ER-100 is an investigational AAV-based epigenetic therapy administered via intravitreal injection to one eye. It uses a modified adeno-associated virus (AAV) vector to deliver instructions for producing three transcription factors-OCT4, SOX2, and KLF4 (collectively referred to as OSK)-intended to reverse age-related epigenetic changes in retinal cells. Systemic doxycycline is administered for 8 weeks (56 days) to activate OSK expression. ER-100 does not alter the participant's existing genes, and the AAV vector has been engineered to eliminate its ability to cause infectious disease.

Primary outcome measure

  • Incidence of Treatment-Emergent Adverse Events (TEAEs) During and Post-Doxycycline Activation Period [ Time Frame: Baseline to Day 56, Day 112 ]
  • Incidence of Dose-Limiting Toxicities During and Post-Doxycycline Activation Period [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change in Safety Laboratory Tests (Liver Function Tests - LFTs) During and Post-Doxycycline Activation Period [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change in Safety Laboratory Tests (Blood Protein Levels) During and Post-Doxycycline Activation Period [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change in Safety Laboratory Tests (Calcium, Glucose, Bilirubin, Creatinine, Blood Urea Nitrogen) During and Post-Doxycycline Activation Period [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change in Safety Laboratory Tests (Electrolytes: Sodium, Potassium, Chloride) During and Post-Doxycycline Activation Period [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change in Safety Laboratory Tests (Electrolytes: Bicarbonate and Magnesium) During and Post-Doxycycline Activation Period [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change in Safety Laboratory Tests (Hemoglobin) During and Post-Doxycycline Activation Period [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change in Safety Laboratory Tests (Hematocrit) During and Post-Doxycycline Activation Period [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change in Safety Laboratory Tests (White Blood Cell and Platelet Counts) During and Post-Doxycycline Activation Period [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change in Safety Laboratory Tests (Red Blood Cell Count) During and Post-Doxycycline Activation Period [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change in Safety Laboratory Tests (Erythrocyte Sedimentation Rate) During and Post-Doxycycline Activation Period [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change in Safety Laboratory Tests (C-Reactive Protein) During and Post-Doxycycline Activation Period [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change in Safety Laboratory Tests (Urine Test Results) During and Post-Doxycycline Activation Period [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change from Baseline in the Intraocular Pressure (IOP) in the Treated Eye During and Post-Doxycycline Activation Period - Safety [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change from Baseline in the Best Corrected Visual Acuity (BCVA) Letter Score in the Treated Eye During and Post-Doxycycline Activation Period - Safety [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change from Baseline in Humphrey Visual Field (HVF) Test Results During and Post-Doxycycline Activation Period - Safety [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change from Baseline in Pattern Electroretinogram (pERG) During and Post-Doxycycline Activation Period - Safety [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change from Baseline in Quantitative Contrast Sensitivity Function (qCSF) During and Post-Doxycycline Activation Period - Safety [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change from Baseline in Optical Coherence Tomography (OCT): Ganglion Cell Layer (GCL) Thickness During and Post-Doxycycline Activation Period - Safety [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change from Baseline in Optical Coherence Tomography (OCT): Retinal Nerve Fiber Layer (RNFL) Thickness During and Post-Doxycycline Activation Period - Safety [ Time Frame: Baseline to Day 56, Day 112 ]
  • Change from Baseline in Slit Lamp Exam Results During and Post-Doxycycline Activation Period - Safety [ Time Frame: Baseline to Day 56, Day 112 ]

Central Contacts and Locations

Central contacts

Life Biosciences

(857) 400-9245ER100trial@lifebiosciences.com

Locations

Global Research Management, Inc.

Recruiting

Glendale, California, United States, 91204

Contacts

Logi El-Harazi

818-246-2560logi@grmclinical.com

Sevada Minassians

818-246-2560Sevada@grmclinical.com

Mass Eye and Ear

Recruiting

Boston, Massachusetts, United States, 02114

Contacts

Joseph Rizzo, MD

617-573-6060centerforclinicalresearchoperations@meei.harvard.edu

Principal Investigator:

Joseph Rizzo, MD

Columbia University Irving Medical Center/Edward S. Harkness Eye Institute

Recruiting

New York, New York, United States, 10032

Contacts

Ioannis Michalopoulos

212-305-0011im2482@cumc.columbia.edu

Principal Investigator:

Aakriti Shukla, MD, MSc

Charleston Neuroscience Institute

Recruiting

Charleston, South Carolina, United States, 29414

Contacts

Alise Chatelain

843-998-7659a.chatelain@retinasouthcarolina.com

Terry Blewer

843-998-7659t.blewer@retinasouthcarolina.com

More Information

Sponsor

Life Biosciences Inc.

Last update posted

May 19, 2026

Last verified

Mar, 2026

Keywords

  • Optic Neuropathy
  • Retinal Diseases
  • Aging/Cellular Aging
  • Open-Angle Glaucoma
  • Non-Arteritic Anterior Ischemic Optic Neuropathy
  • Partial Epigenetic Reprogramming
  • Epigenetic Therapy

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-24. This information was provided to ClinicalTrials.gov by Life Biosciences Inc. on 2026-05-19. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.