In the global race for a Herpes cure, China is taking the lead. For the first time, transient gene-editing of the herpes simplex virus type 1 (HSV-1) using mRNA-based CRISPR was shown to cure herpes keratitis in mice. Chinese researchers reported in a paper published January 11, 2021 of Nature Biotechnology.
In a paper published May 2021, the team from Shanghai showed therapeutic efficacy in three different Herpes Simplex Keratitis (HSK) mouse models and human-derived corneas. The approach, an HSV-1-erasing lentiviral particle (referred to as HELP), was shown in these pre-clinical studies to significantly block HSV-1 replication and HSK occurrence in three different infection models, and eliminated the viral reservoir in the ganglia.
Shanghai BD Gene is now currently in a phase 1/2 clinical study studying the HELP approach in humans with final study results expected May 2022. This study, CRISPR/Cas9 mRNA Instantaneous Gene Editing Therapy Assisted Corneal Transplantation in the Treatment of Refractory Viral Keratitis, may facilitate the development of therapeutics targeting other viruses, including HSV-2, (HPV) and Hepatitis B (HBV). This is the first clinical study of CRISPR in the antiviral field and is Chinas first completely self-developed original gene therapy vector.
Patients worldwide are highly anticipating “first time in humans” clinical study results. Early results show that study subjects are effectively cured of the virus within 4-10 months and company representatives are expecting final results to show HELP is the worlds first proven treatment to cure for Herpes Keratitis. HELP is giving much hope to HSV carriers worldwide.
Dr. Cai Yujia, the corresponding author of the study, said: “Realizing the instantaneous delivery of gene editing enzymes in the body is the dream of our gene therapy scientists. With such a vector, we can maximize the safety of gene editing therapy, which also means The in vivo treatment of gene editing is infinitely close to clinical application.”
In August 2021, Shanghai BD Gene signed R&D cooperation agreements to explore the application of BDGene core delivery technology PVP-mrna in other major diseases such as nervous system diseases. They have also added HSV-2 to their pipeline although company reps indicate the approach is different than HSK. HSK is easier to administer treatment via intrastromal injection to the eye. For HSV-2, administering the treatment is more challenging, but something Shanghai BD Gene is considering. Alternatively this team is considering a viral-like approach for HSV-2 using mRNA.
Patient Advocates are hopeful that China, without the lengthy red tape and regulatory processes of the FDA that can take decades, bringing this treatment to market can happen quickly. Estimates indicate once results are published and peer reviewed in May 2022 that this treatment could be in the clinic, and available on the market for patients treatment, within 5 years.