Researchers Submit Patent Application, "Polynucleotides Encoding Ornithine Transcarbamylase For The Treatment Of Urea Cycle Disorders", for Approval (USPTO 20240247239).

Researchers from Moderna TX Inc. have submitted a patent application for a potential treatment for urea cycle disorders (UCD), a rare genetic disorder that affects the body's ability to process ammonia. The application focuses on the use of mRNA therapeutics to deliver functional ornithine transcarbamylase (OTC) protein to target cells, with the goal of improving treatment options for UCD. The invention also includes the use of lipid nanoparticles as a delivery method for the mRNA encoding OTC. The patent application addresses the need for improved treatments for UCD and provides detailed claims and methods for the use of the pharmaceutical composition in treating ornithine transcarbamylase deficiency (OTCD). [Extracted from the article]