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Keywords: Disease Treatment
Recently, the use of mRNA was validated to create vaccines against COVID-19; hence, there is significant potential for using them as vaccines and treatments for many other conditions. While mRNA drugs have led to a potential paradigm shift in disease treatment, they face challenges that impede the successful translation of these molecules into drugs. They are very large molecules, are intrinsically unstable, and prone to degradation by nucleases, and they can abnormally activate the immune system. While some of these challenges have been partially solved by chemical modification of mRNA and nanoparticle-mediated delivery, intracellular delivery and off-target effects of mRNA still represent a major hurdle. The novel mRNA technology developed by Clemson researchers overcomes the delivery and targeting challenges with existing mRNA, increasing stability and controlling release location helping fully exploit their effectiveness for many.
Delivery of mRNA therapeutics to treat chronic and degenerative diseases
This invention describes the methods and compositions of complex coacervates for stabilization and delivery of mRNA therapeutics by targeting elevated levels of reactive oxygen species (ROS). ROS overproduction has been implicated in the development of various chronic and degenerative diseases such as cancer, respiratory, neurodegenerative, and digestive diseases. The technology uses complex coacervation to improve mRNA delivery, involving the liquid-liquid charge-based phase separation of mRNA into dense liquid droplets. Coacervated mRNA protects the mRNA from enzymatic degradation, facilitates endocytosis, and enables targeted and ROS-triggered release through a novel chemical modification. The current data suggests the modality could be viable for delivering mRNA into cells and tissues with high ROS, like tumor microenvironments, or for radiotherapy.
• Novel mRNA delivery system that can target tissues and cells with high levels of ROS.
• Potential to improve the targeting safety of mRNA-based therapeutics for cancer and other chronic indications.
• Modified coacervates remain stable in low ROS conditions but activate in the presence of elevated ROS levels.
Proof of Concept
Provisional
2024-025
Dr. Jessica Larsen, Chloe Forenzo
Pushparajah Thavarajah
Business Development Associate
pthavar@clemson.edu
864.656.5708
Contact
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