A Phase 12 Study of BS01 Eye Injection to Test Safety and Effectiveness in Patients Aged 50 to 85 With Geographic Atrophy from Dry Age-Related Macular Degeneration

What this Trial Is About

Researchers are studying BS01, a gene therapy delivered by intravitreal injection, in people aged 50 to 85 with geographic atrophy GA caused by dry age-related macular degeneration AMD. This Phase 12 multi-center trial aims to assess the safety and effectiveness of BS01. The study includes an initial dose-escalation phase followed by a dose-expansion phase to compare the selected dose against a sham injection control group. The trial has two parts Part 1 is an open-label dose-escalation study involving up to 10 patients to determine the best dose based on benefit and risk. Part 2 will involve up to 30 patients randomized to receive either the selected doses of BS01 or a sham injection without needle, allowing controlled comparison of safety and efficacy. BS01 is a recombinant adeno-associated virus vector expressing ChronosFP. Participants will undergo eye exams and imaging to monitor geographic atrophy area and visual acuity. The study will evaluate adverse events for safety over 12 months in Phase 1 and measure changes in best corrected visual acuity BCVA over 12 months in Phase 2. Assessments include fundus photography, fundus autofluorescence, and optical coherence tomography. Participants will be monitored for treatment side effects and disease progression throughout the study period.

A Phase 1/2 Study to Evaluate the Safety and Efficacy of Intravitreal Administration of BS01 in Patients With Geographic Atrophy Secondary to Dry AMD