Intravitreal BS01 for geographic atrophy from dry AMD

A Phase 1/2 Study to Evaluate the Safety and Efficacy of Intravitreal Administration of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP (AAV2-CAGChronosFP) in Patients With Geographic Atrophy Secondary to Dry AMD

This trial will test whether injections of BS01 into the eye can slow or reduce geographic atrophy in adults 50–85 with GA from dry age-related macular degeneration.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 50 Years to 85 Years
• Sex: All
• Sponsor: Bionic Sight LLC (Industry-sponsored)
• Drugs / interventions: radiation
• Locations: 1 site (Edison, New Jersey)
• Trial ID: NCT07158775 on ClinicalTrials.gov

What this trial studies

This is a seamless Phase 1/2, multi-center, dose-escalation and expansion trial of intravitreal BS01 in patients with geographic atrophy secondary to dry AMD. Part 1 is an open-label dose-escalation cohort enrolling up to 10 patients to define safety and an appropriate dose, and Part 2 is a dose-expansion cohort of up to 30 patients that includes a selected BS01 dose and an untreated (sham injection) control group for controlled comparison of safety and efficacy. Key ophthalmic entry criteria include GA with some foveal involvement, total lesion area between 5 and 17.5 mm2, at least one focal lesion ≥1.25 mm2 if multifocal, and BCVA between 20/50 and 20/400. Participants will receive intravitreal injections or sham procedures with scheduled imaging and visual acuity follow-up to monitor lesion size, function, and adverse events.

Who should consider this trial

Good fit: Ideal candidates are adults 50–85 with geographic atrophy from dry AMD that involves the fovea, total GA area 5–17.5 mm2 (with at least one lesion ≥1.25 mm2 if multifocal), and BCVA between 20/50 and 20/400.

Not a fit: Patients with very small or very large GA outside the specified area range, predominant non-dry AMD causes, active neovascular (wet) AMD, or vision outside the enrollment range are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, BS01 injections could slow the growth of GA lesions and help preserve central vision for patients with dry AMD.

How similar studies have performed: Other intravitreal approaches for GA, including complement inhibitors, have shown modest slowing of lesion growth, but BS01 itself is a novel agent that is being tested in humans through this phased escalation and expansion design.

Eligibility criteria

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).

Where this trial is running

Edison, New Jersey

• NJ Retina — Edison, New Jersey, United States (Recruiting)

Study contacts

• Study coordinator: Sheila Nirenberg, PhD+

Conditions Geographic Atrophy Secondary to Dry Age-related Macular Degeneration; GA