This is a Phase 1/2 study, multi-center, dose-escalation interventional study of BS01 in subjects with GA secondary to dry AMD. Part 1 is an open label dose-escalation study; Part 2 is a dose-expansion study with dose(s) selected from Part 1 based on a benefit/risk assessment, and an untreated (sham injection) group to allow for a controlled comparison of efficacy and safety.
This is a seamless Phase 1/2 study in up to 10 patients for Phase 1 and 30 patients Phase 2 in patients with GA secondary to dry AMD.
Signed informed consent obtained before screening.
Men or women between 50 and 85 years of age inclusive at the time of signing the informed consent.
Geographic atrophy with some macula foveal involvement secondary to dry AMD.
Total GA area ≥ 5 and ≤ 17.5 mm2 (2 and 7 disk areas respectively), based on Heidelberg Region Finder or equivalent automated software.
Previous therapeutic radiation in the region of the SE.
Previous treatment with any ocular or systemic gene transfer product.
Any treatment with an investigational agent in the past 60 days for any condition.
Women who are pregnant or nursing.
Parallel
BS01
Biological/Vaccinea recombinant adeno-associated virus vector expressing ChronosFP (AAV2-CAGChronosFP)
Sham procedure control
Other interventionSham procedure without needle
[Phase 1] Number of the dose limiting toxicities (DLTs), incident and severity of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
[Phase 2] Change from baseline in BCVA (Best Corrected Visual Acuity)
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