Head and neck tumors are the sixth most common malignant tumor worldwide, with a gradually increasing incidence, and may develop in multiple locations, including the neck, otorhinolaryngology, and the oral and maxillofacial regions. There is a lack of effective clinical treatments for head and neck tumors, and no new drugs have been approved for the treatment of radioactive xerostomia in the past 20 years.
RiboX Therapeutics, a biotechnology company focused on the discovery and development of fully engineered circRNA therapeutics, recently announced that its investigational therapeutic for radioactive xerostomia, RXRG001, has received IND approval from the FDA and will soon be launched in the US in a Phase 1/2a clinical trial, SPRINX-1. This significant development signifies that the circRNA drug has officially entered the clinical development stage.
RXRG001 is the world's first circRNA therapeutic licensed by the FDA to enter clinical trials and the world's first circRNA drug approved for clinical studies in radioactive xerostomia.
RXRG001 is encapsulated by lipid nanoparticles (LNPs), which are delivered by LNPs that express the water channel protein AQP1 in salivary glands to enhance water permeability, thereby increasing the flow and velocity of saliva into the oral cavity and improving patients' symptoms of dry mouth.
Preclinical studies have shown that RXRG001 exhibits long-lasting efficacy with excellent tolerability and safety. With a significant increase in salivary flow rate after a single dose and effects lasting up to four weeks, RXRG001 has the potential to be an effective and long-lasting treatment option for patients with radioactive xerostomia.
circRNA is a class of circular single-stranded RNA formed by backsplicing without 5' -end cap and 3' -end poly tail, and connected by covalent bonds to form a loop. As a new generation of RNA therapy, circRNA can overcome the limitations of existing mRNA therapies and has great potential application value.
circRNAs exhibit high protein expression efficiency, and can promote higher levels of total protein translation and regulate function.
CircRNAs have no free ends, are resistant to nucleic acid exonucleases, and have greater storage and intracellular stability.
The production costs of circRNAs are lower than that of linear mRNA because it does not need to be capped, tailing or modified.
circRNAs evade RIG1 and TOLL-like receptor recognition, and have low immunogenicity and high safety.
This innovative therapy from RiboX not only brings new hope to patients with radioactive xerostomia, but also opens up a fresh direction for the future development of RNA drugs. We can foresee that circRNA is likely to become another generation of nucleic acid drugs.
Driven by innovation, Protheragen-ING AI-Pharma focuses on the AI platform to develop nucleic acid drug program development, including circRNA drug design and delivery system research. We optimize the design of circRNAs through AI algorithms, simplify the sequence optimization design of circRNAs, and improve cyclization efficiency, stability and protein translation. If you are interested in our services, please contact us to learn how our AI technology can support circRNA drug development.
Related Services:
Therapeutic Applications of Small Nucleic Acid Drugs
Small Nucleic Acid Drugs CDMO
Advanced Delivery Systems for Small Nucleic Acid Therapeutics
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