Gene therapy trial aims to halt vision loss in dry AMD
NCT ID NCT07158775
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new gene therapy called BS01 for people with geographic atrophy, a severe form of dry age-related macular degeneration that causes blind spots. The treatment uses a harmless virus to deliver a light-sensitive protein to the eye, aiming to restore or preserve vision. The trial involves 40 adults aged 50-85 and will first check safety, then measure if it improves eyesight compared to a sham procedure.
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 40 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jan 2026
- Expected to finish
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Dec 2031
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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50 to 85 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: General Inclusion Criteria 1. Signed informed consent obtained before screening. 2. Men or women between 50 and 85 years of age inclusive at the time of signing the informed consent. Ophthalmic Inclusion Criteria 1. Geographic atrophy with some macula foveal involvement secondary to dry AMD. 2. Total GA area ≥ 5 and ≤ 17.5 mm2 (2 and 7 disk areas respectively), based on Heidelberg Region Finder or equivalent automated software. 3. If GA is multifocal, at least one focal lesion should measure ≥ 1.25 mm2 (0.5 disk area) to ensure measurable focal effects for efficacy evaluation. 4. Composite lesion encompassing confluent GA regions with total area meeting thresholds given in 2 above. 5. GA in part within 1200 microns from the foveal center. 6. The atrophic lesion must be able to be photographed in its entirety. 7. BCVA between 20/50 to 20/400, inclusive, using letter score per EDTRS chart. 8. Clear ocular media and adequate pupillary dilatation in both eyes to allow for all imaging procedures, including good quality stereoscopic fundus photography and fundus autofluorescence (FAF). 9. Central fixation. Exclusion Criteria: General Exclusion Criteria 1. Previous therapeutic radiation in the region of the SE. 2. Previous treatment with any ocular or systemic gene transfer product. 3. Any treatment with an investigational agent in the past 60 days for any condition. 4. Women who are pregnant or nursing. 5. Known hypersensitivity to topical ocular anesthetics or diagnostic drops to be used during the study. 6. Any other medical condition, which, in the Investigator's judgment, will interfere with the patient's ability to comply with the protocol, compromises patient safety, or interferes with the interpretation of the study results. Ophthalmic Exclusion Criteria 1. \> 30% difference of BCVA using EDTRS in two baseline visual acuity assessments with at least 14 days apart during screening. 2. Any intraocular surgery or thermal laser within 3 months of study entry. Any prior thermal laser in the macular region, regardless of indication. 3. Any ocular or periocular infection (including blepharitis), or ocular surface inflammation in the past 12 weeks. 4. History of any of the following procedures: Posterior vitrectomy, filtering surgery (e.g., trabeculectomy), glaucoma drainage device, corneal transplant, or retinal detachment. 5. Any sign of diabetic retinopathy in either eye. 6. Intraocular pressure (IOP) \> 25 mmHg in either eye. 7. Completely atrophic centrally located lesions based on ellipsoid zone (EZ) loss in spectraldomain optical coherence tomography (SD-OCT).
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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
1 site. 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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NJ Retina
RECRUITINGEdison, New Jersey, 08820, United States
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