Can a One-Time gene edit stop glaucoma in its tracks?

NCT ID NCT06465537

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused This study
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 20, 2026 · Last updated Aug 21, 2026 · Updated 1 time

Summary

This trial tests a new gene-editing therapy called BD113vVLP for people with primary open-angle glaucoma caused by a specific genetic mutation (MYOC). The therapy is injected into the eye and aims to disable the faulty gene that leads to high eye pressure. The study will evaluate safety and tolerability, and also look at whether it can lower eye pressure to a healthier level. It involves a small group of adults aged 18 to 65 with this type of glaucoma.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
BD113vVLP, a virus-like particle carrying CRISPR/Cas9 gene editing components, injected into the eye to target the mutated MYOC gene.
What this could lead to
If successful, this could lead to a one-time gene therapy that lowers eye pressure and potentially halts glaucoma progression, reducing the need for lifelong treatment.
What could go wrong
This is an early, small trial (9 participants) focused on safety and tolerability. Gene editing carries risks like inflammation or infection, and the effect may not be durable or effective in all patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

About 9 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2024

Expected to finish

Jul 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Signed ICF; 2. Aged 18 to 65 years old; 3. Primary open Angle glaucoma (POAG) with elevated intraocular pressure (IOP) was diagnosed with ≥1 year medical history record ; 4. Good function level of organs; 5. Good compliance and willing to comply with the visit schedule, laboratory tests and other specified test etc. per protocol; 6. Agreeing to accept a long-term safety follow-up after 1 year of study. Special Inclusion Criteria for Group 1: * Target intervenning eye is no visual acuity; * The intraocular pressure (IOP) was ≤35 mmHg and \> 21 mmHg after receiving a combination therapy of 2 or more drugs lowering IOP. Special Inclusion Criteria for Group 2: * MYOC gene mutation was detected in peripheral blood nucleated cells ; * The intraocular pressure (IOP) was ≤30 mmHg and \> 21 mmHg after receiving a combination therapy of 2 or more drugs lowering IOP; * Both eyes have a Shaffer Angle mirror rating greater than 3. Exclusion Criteria: 1. Secondary glaucoma; 2. Any active or recurrent intraocular infection or inflammation, including but not limited to uveitis; 3. The target intervenning eye has severe xerophthalmia or clinically significant active corneal disease; 4. Any condition no accepting the measure of IOP; 5. Any positive of human immunodeficiency virus type 1/2 (HIV-1/HIV-2) antibody, treponema pallidum (TP) specific antibody, human T-lymphotropic virus type 1 or 2 (HTLV-1/HTLV-2) antibody, or vesicular stomatitis virus G (VSV-G) antibody; 6. Any of hepatitis B virus (HBV) HbsAg or HBV-DNA, hepatitis C virus (HCV) HCAb, or epstein-barr virus (EBV), or cytomegalovirus (CMV) nucleic acid test is positive; 7. Severe active bacterial, viral, fungal, malaria or parasitic systemic infection; 8. Any past or present malignancy, myeloproliferative or immunodeficient disease; 9. History of major organ diseases or abnormalities in laboratory tests, including: 1. Liver cirrhosis, liver fibrosis or active hepatitis, and/or abnormal liver function tests (serum total bilirubin (TBIL) ≥1.5 x upper limit of normal (ULN); Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≥2.5×ULN; Alkaline phosphatase ≥2.5 × ULN); 2. Cardiovascular and cerebrovascular diseases, including uncontrolled hypertension, myocardial infarction, myocarditis, arrhythmia, stroke, etc.; 3. Kidney disease, or creatinine ≥ 1.5ULN and creatinine clearance \< 30% normal level (using the Cockcroft-Gault equation); 4. Endocrine disorders, such as insulin-dependent diabetes mellitus, hyperthyroidism or hypothyroidism; 5. Severe pulmonary hypertension, chronic obstructive pulmonary disease, interstitial pneumonia; 10. Any severe psychiatric disorders; 11. Participating in another clinical study of a drug or device, or administrated the investigational drug within 42 days prior to the screening visit; 12. Pregnant or lactating women; 13. Refusing to accept any contraception measures; 14. Allergic to clinical investigational drugs or their excipients; 15. Other conditions assessed by the investigator as unsuitable for participation in this study. Special Exclusion Criteria for Group 2: * Retinal diseases: complicated with unexplained quadrant blindness, neovascularization age-related macular degeneration, retinal branch vein obstruction, central retinal vein obstruction, cystoid macular edema, macular hiatal hole and central serous retinopathy; * A history of anterior chamber angle stenosis, congenital glaucoma, or angle closure, clinically significant anterior peripheral adhesion, or extensive cicatricial adhesion caused by surgery/laser therapy in the anterior chamber angle; * The central corneal thickness is less than 480 μm or more than 620 μm.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Beijing Tongren Hospital, Capital Medical University

    Beijing, Beijing Municipality, 100730, China

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