Evaluating ER-100 for Safety in People With Glaucoma or Non-Arteritic Anterior Ischemic Optic Neuropathy (Optic Nerve Conditions)

Conditions

• NAION( Non-arteritic Anterior Ischemic Optic Neuropathy)
• Open Angle Glaucoma (OAG)

Eligibility Criteria

Sex

ALL

Age

40 Years - 85 Years

Healthy Volunteers

Not accepted

Interventions

• ER-100 epigenetic therapy — GENETIC
  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.

Study Details

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

Key Dates

Start date

Mar 2, 2026

Status verified

Mar 2026

Primary completion

May 31, 2027

Completion

Mar 31, 2032

Study Design

Enrollment

18 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

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.

Primary Outcome Measure

Incidence of Treatment-Emergent Adverse Events (TEAEs) During and Post-Doxycycline Activation Period [ Time Frame: Baseline to Day 56, Day 112 ]

Central Contacts

• Life Biosciences
  (857) 400-9245
  [email protected]

Locations (4)

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