RecruitingNot ApplicableNCT06465537

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

A Clinical Study on CRISPR/Cas9 Instantaneous Gene Editing Therapy to Primary Open-angle Glaucoma With Elevated Intraocular Pressure and MYOC Gene Mutation

Sponsor

Shanghai BDgene Co., Ltd.

Enrollment

9 participants

Start Date

Jun 10, 2024

Study Type

INTERVENTIONAL

Conditions

Primary Open Angle Glaucoma

Summary

This study is intented to evaluate the safety, tolerability and preliminary efficacy of CRISPR/Cas9 Instantaneous Gene Editing Therapy (BD113 virus-like particle, also BD113vLVP) in patients with primary open-angle glaucoma (POAG) with elevated intraocular pressure and MYOC gene mutation. The main objectives to evaluate the safety and tolerability BD113vLVP) in POAG patients with intraocular hypertension and MYOC mutation, and secondary objectives is to explore the preliminary efficacy and the metabolism characteristics of BD113vLVP in participants.

Eligibility

Min Age: 18 YearsMax Age: 65 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a CRISPR gene-editing therapy injected into the eye to treat a hereditary form of glaucoma caused by a mutation in the MYOC gene. It aims to directly fix the genetic defect that causes elevated eye pressure and vision loss. **You may be eligible if...** - You are between 18 and 65 years old - You have been diagnosed with primary open-angle glaucoma caused by the MYOC gene mutation, with at least 1 year of medical history - Your organs are functioning well and you are willing to attend long-term follow-up visits **You may NOT be eligible if...** - You do not carry the MYOC gene mutation - You have other types of glaucoma not related to this gene - You are unwilling to commit to long-term follow-up Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

GENETICBD113vVLP

CRISPR/Cas9 gene editing technology, called BD113vVLP (also BD113 virus-like particle) which is a developing product of gene therapy from modified third-generation integrated defective lentivirus, can deliver gRNA/Cas9 ribonucleoprotein complex (RNP). It works to knock out or knock down the mutated MYOC gene. The BD113vVLP is administrated by intracamerally injecton (sigle-dose: 4ug/p24) for each target interventional eye.