Shanghai BDgene Technology Co., Ltd has completed efficacy and preliminary safety studies for BD111, its CRISPR-Cas9 in vivo-based therapy for herpes simplex virus (HSV)-related keratitis (HSK). The company plans to file for Investigational New Drug (IND) approval and was awarded orphan drug designation (ODD) status in the US last week.
Therapy Mechanism and Innovation
BD111 is an in vivo gene-editing therapy developed using BDgene’s proprietary BDmRNA delivery technology. It is the second human clinical research project to apply CRISPR-Cas9 gene-editing treatment globally. The therapy directly targets and cuts the HSV-1 genome using CRISPR gene-editing tools, aiming to remove the viral genome and achieve a cure for HSK.
Advantages of BD111
The therapy delivers Cas9 mRNA, which remains in the body for a short time, reducing the risk of immune response and off-target effects. It cuts the virus genome without altering human genes, with no off-target effects detected in human genome studies.
Market and Clinical Significance
Herpes simplex virus keratitis, caused by HSV-1 infection, is the most common infectious blinding disease. Current antiviral drugs only inhibit viral replication but cannot clear the latent viral genome, leading to recurrent episodes and potential blindness. BD111 offers a potential cure with a single injection, representing a significant advancement in the treatment of this condition.-Fineline Info & Tech
