Clinical Study of CRISPR/Cas9 Gene Editing Therapy for Primary Open-Angle Glaucoma with Elevated Eye Pressure and MYOC Mutation

What this Trial Is About

Researchers are evaluating the safety, tolerability, and early effectiveness of a gene editing therapy called CRISPR/Cas9 Instantaneous Gene Editing Therapy (BD113 virus-like particle, BD113vLVP) in adults with primary open-angle glaucoma (POAG) who have high eye pressure and a mutation in the MYOC gene. This study aims to understand how well BD113vLVP works to lower eye pressure and how the treatment is processed in the body. The trial is open-label and includes patients with varying vision status in their affected eye. Participants are divided into two groups: Group 1 includes POAG patients with no vision in the treated eye regardless of MYOC mutation status, while Group 2 includes patients with vision and confirmed MYOC gene mutation. Each participant will receive a single dose of BD113vLVP by injection into the eye's anterior chamber. The study involves regular follow-up visits over one year to assess safety and the impact on eye pressure. Throughout the year, participants will undergo tests to monitor eye-related side effects, changes in eye pressure, visual acuity, visual fields, and retinal nerve fiber layer thickness. Blood and eye fluid samples will be analyzed for protein levels related to the treatment and immune responses. Safety evaluations include monitoring for any systemic side effects and ocular malignancies. Participants agree to long-term safety follow-up after the one-year study period.

CRISPR/Cas9 Instantaneous Gene Editing Therapy to Intraocular Hypertensive POAG With MYOC Mutation