A CRISPR-based technology to treat cytomegalovirus (CMV) and other Viral Infections.

Value Proposition

• Novel Mechanism: Utilizes a Crispr/Cas9 system to target the viral genome to prevent production of the disease-causing protein 1E1.
• Widely Adaptable: Design allows modulation to fit a wide variety of other viral genomes to combat other infectious diseases.
• Therapeutic Potential: Administration of proposed technology to infected pregnant mothers would mitigate transmission to developing child, therefore eliminating congenital CMV infections.

Unmet Need

There is no proper treatment for CMV infections, only antivirals and prophylactic drugs exist for certain types of CMV infections currently. Current treatment regimen of antiviral and prophylaxis have high toxicity levels for various organs. Limited therapeutics are available for the prevention of congenital CMV.

Technology Description

JHU researchers have created a CRISPR/Cas9 system specifically designed to target and modify the cytomegalovirus (CMV) genome by inactivating the immediate early protein 1 (IE1), a key factor in viral infection and immune evasion. This innovative technology utilizes guide RNAs to introduce double-strand breaks at specific CMV genome sequences, disrupting IE1 production and limiting viral propagation. Pre-clinical studies demonstrate significant reductions in viral protein levels and loads, which is crucial for controlling infections, especially in vulnerable populations such as pregnant women. The versatile platform can also be adapted to target various viral pathogens, including HIV and hepatitis, and early findings indicate a favorable safety profile with minimized off-target effects, enhancing its suitability for therapeutic applications. It can be administered through systemic methods or localized injections, with ongoing research focused on optimizing delivery for effective uptake by infected cells. Additionally, this CRISPR/Cas9 system holds the potential to complement existing antiviral therapies, improving their efficacy by reducing viral loads and associated disease impacts, representing a significant advancement in CMV treatment and offering broad implications for combating various viral infections.

Stage of Development

• This CRISPR technology has been validated in pre-clinical mouse models to treat CMV infection and was shown to reduce the amount of viral protein produced.
• The CRISPR/Cas9 system is ready to proceed to phase I clinical trials.

Data Availability: Data available upon request.

Publications:

• Saito, A., Tankou, S., Ishii, K., Sakao-Suzuki, M., Oh, E. C., Murdoch, H., Namkung, H., Adelakun, S., Furukori, K., Fujimuro, M., Salomoni, P., Maul, G. G., Hayward, G. S., Tang, Q., Yolken, R. H., Houslay, M. D., Katsanis, N., Kosugi, I., Yang, K., Kamiya, A., … Sawa, A. (2025). DISC1-PML protein interaction for congenital CMV infection-induced cortical neural progenitor deficit: perturbance of host signaling via viral IE1. bioRxiv : the preprint server for biology, 2025.09.03.674025. https://doi.org/10.1101/2025.09.03.674025