Gene therapy injection aims to halt blinding eye disease
NCT ID NCT07705568
First seen Jul 15, 2026 · Last updated Jul 17, 2026 · Updated 2 times
Summary
This trial tests a gene therapy called PST-611 in people aged 65 and older with geographic atrophy, an advanced form of age-related macular degeneration that causes blind spots. PST-611 is a DNA plasmid injected into the eye muscle that instructs cells to produce human transferrin, a protein that may protect the retina. Participants receive one injection every 20 weeks for three doses and are followed for a year to check safety and any changes in vision or retinal structure.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- a gene therapy called PST-611, a DNA plasmid that instructs cells to produce human transferrin, injected into the eye muscle
- What this could lead to
- If successful, PST-611 could slow or stop the progression of geographic atrophy, a leading cause of blindness in older adults, for which there are limited treatments.
- What could go wrong
- This is a small, early-phase trial focused on safety, so it is not designed to prove effectiveness. The treatment may not slow vision loss and could cause side effects like eye inflammation or increased pressure.
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 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 24 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Sep 2026
An estimate. Start dates often move.
- Expected to finish
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Jun 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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65 years and older
- 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: * Subjects must give written informed consent, be able to make the required trial visits and follow instructions. * Female and male subjects must be 65 years of age or older. * In the study eye (SE): cRORA must be present on OCT and attributed to AMD, as evaluated by the Investigator. * Documented recent history (i.e., over 3 to 12 months prior to first PST-611 administration) of GA lesion area progression, with a yearly growth projection of ≥ 1.6 mm2, in the SE * GA lesion area prior to the first PST-611 administration must be between 1.25 mm2 and 16 mm2, as assessed by OCT, in SE. * BCVA must be \< or = 75 ETDRS letters (Snellen \< or = 20/32) in the SE. * Fixation, either central or eccentric, of both eyes, must be compatible with the performance of the ocular imaging and functional assessments included in the trial, as evaluated by the Investigator. * If both eyes are eligible, the SE will be selected by the Investigator based on the totality of the clinical evaluation. Exclusion Criteria: * Both eyes (OU): any active intraocular or periocular infection or inflammation (eg, infectious blepharitis, infectious conjunctivitis, keratitis, scleritis, endophthalmitis), or history of intraocular or periocular infection or inflammation in the 12 weeks (84 days) prior to Dose 1. * SE: any intraocular surgery (including cataract surgery) or intravitreal (IVT) or periocular corticosteroid injection in the 12 weeks (84 days) prior to Dose 1. * SE: the documented need for more than 2 anti-VEGF IVT treatments per year as well as any anti-VEGF IVT treatment within 3 months prior to Dose 1, and lesion must be clinically inactive. * SE: media opacity that interferes with fundus imaging or is likely to require surgery during the trial period. * SE: subject with history of glaucoma filtering surgery (e.g., trabeculectomy or aqueous shunt implant) or who underwent eye surgery in the 12 weeks (84 days) prior to Dose 1. * SE: subject who has uncontrolled intraocular pressure of ≥ 25 mmHg in the SE at the Screening and Trial Baseline Visits. * SE: subject with intraocular hypotension (\<6 mmHg) in the SE that in the opinion of the Investigator would interfere with the PST-611 administrations or the evaluation of its safety or efficacy. * SE: subject with history of scleritis, scleral thinning, cicatrizing conjunctival diseases, severe ocular allergies, severe ocular surface disease, ocular scarring or intraocular hardware (e.g., retained implant device) that could interfere with the PST-611 administrations or the evaluation of its safety. * SE: any other concurrent ocular surface or intra-ocular condition, including retinal disease other than AMD, which, in the opinion of the Investigator, may pose a safety risk for the PST-611 administrations or interfere with the evaluation of its safety. * Subject has any condition, which in the opinion of the Investigator, could compromise the subject's safety or adherence to the trial protocol or follow-up. * Known/suspected hypersensitivity to any standard of care topical or local analgesics/anesthetics or other standard of care treatments used in the preparation of PST-611 administration procedure. * Treatment with investigational medicinal products in the 12 weeks (84 days) prior to Dose 1. * Subject previously exposed to any gene therapy product other than the non-viral gene therapy PST-611.
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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
3 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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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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CHU de Grenoble-Hôpital Michallon
Grenoble, France, 38043, France
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Hôpital Cochin
Paris, France, 75014, France
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Hôpital Lariboisière
Paris, France, 75010, France
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Other studies related to the condition(s) this trial covers.
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