Can a single Gene-Editing injection stop a blinding corneal disease?
NCT ID NCT07750678
First seen Aug 06, 2026 · Last updated Sep 11, 2026 · Updated 3 times
Summary
This trial tests a single injection of GEB-101, a gene-editing treatment, directly into the cornea of people with TGFBI-related corneal dystrophy, a hereditary condition that causes progressive vision loss. The study aims to see if the treatment is safe and whether it can slow or stop the disease by correcting the underlying genetic mutation. Participants are adults aged 18-70 with a confirmed TGFBI gene mutation and symptoms of corneal dystrophy. The trial will monitor for side effects, changes in vision, and how long any benefit lasts.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- GEB-101, a gene-editing injection given directly into the cornea
- What this could lead to
- If successful, this could lead to a one-time treatment that corrects the genetic cause of TGFBI-related corneal dystrophy, potentially preventing vision loss and the need for repeat surgeries.
- What could go wrong
- This is an early-phase trial with a small number of participants, so safety and effectiveness are not yet proven. Risks include possible eye inflammation, infection, or the treatment not working as intended.
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
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 30 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Oct 2026
An estimate. Start dates often move.
- Expected to finish
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Dec 2028
An estimate. End dates often move.
- Lead sponsor
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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
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18 to 70 years
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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: * Males or females, age 18-70 years (inclusive). * TGFBI gene mutation confirmed. * Patients with corneal dystrophy who are symptomatic. * Willing and able to give signed informed consent and follow study instructions. Exclusion Criteria: * In either eye: Have any active infection, glaucoma, corneal disease, or other ocular conditions that may affect the assessment of efficacy or safety. * In the study eye: Have a history of specific intraocular or corneal surgery, or the presence of active intraocular inflammation or vitreous hemorrhage. * Patient with other corneal disorders that may hinder therapeutic surgery or interfere with the interpretation of study results. * Prior gene therapy administration in either eye. * Active infection occurring or suspected within 4 weeks prior to the investigational product, requiring systemic anti-infective therapy. * Subjects with a history of major cardiovascular events within 6 months, the presence of any clinically significant condition, a diagnosis of malignancy within 5 years (with specified exceptions), or active infection with HIV, syphilis, hepatitis C, or hepatitis B. * Female subjects who are pregnant, breastfeeding, or planning to become pregnant during the study period. * Disease, condition, or disorder that in the judgement of Investigator could confound study assessments or limit compliance to study protocol.
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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.
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The places running it
2 sites in 2 countries. The list below names each one and where it is.
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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.
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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
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Fudan University Eye & ENT Hospital
Shanghai, Shanghai Municipality, 200031, China
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University of California, Los Angeles (UCLA)
Los Angeles, California, 90095, United States
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