Shanghai BDgene Technology Co., Ltd has announced that a clinical trial filing for its BD111, a CRISPR-Cas9 in vivo-based therapy for herpes simplex virus (HSV)-related keratitis (HSK), has been accepted for review by the Center for Drug Evaluation (CDE). This marks a significant step forward in the development of innovative treatments for HSK.
BD111: CRISPR-Cas9 Therapy for HSK
BD111 has been developed based on BDgene’s proprietary BDmRNA delivery technology, which is the only technology globally to apply Cas9 mRNA delivery by lentivirus. The mechanism of action involves using CRISPR gene editing tools to directly target and edit the HSV-1 genome, with the goal of removing the HSV-1 virus genome to treat HSK. This technology offers several advantages:
- Delivering Cas9 mRNA ensures that the gene enzyme stays in the body for a short time, reducing the risk of immune response and off-target effects.
- The virus genome is cut without altering human genes, ensuring no off-target effects on the human genome.
BD111 obtained orphan drug designation (ODD) status from the US FDA in June last year, highlighting its potential as a novel treatment for HSK.
Virus-Like Particle (VLP) Technology
Virus-like particle (VLP) technology combines the advantages of both viral and non-viral vectors, offering broad applications in in vivo gene editing therapy. Based on the principles of specific recognition of mRNA stem-loop structures and phage capsid proteins, VLP-mRNA constructed through virus engineering technology can safely and efficiently deliver CRISPR gene editing tools. This ensures instantaneous expression of gene editing enzymes in vivo, reduces the risk of off-target gene editing, and enhances the safety of gene editing drugs.-Fineline Info & Tech
