Arcturus Therapeutics. has filed a patent for a modified human OTC protein to treat OTC deficiency. The protein is produced from codon optimized mRNA for effective treatment. The patent also includes codon optimized mRNA sequences for OTC deficiency treatment. GlobalData’s report on Arcturus Therapeutics gives a 360-degree view of the company including its patenting strategy. Buy the report here.

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According to GlobalData’s company profile on Arcturus Therapeutics, Nanoparticle drug conjugates was a key innovation area identified from patents. Arcturus Therapeutics's grant share as of January 2024 was 33%. Grant share is based on the ratio of number of grants to total number of patents.

Modified human otc protein for treating otc deficiency

Source: United States Patent and Trademark Office (USPTO). Credit: Arcturus Therapeutics Holdings Inc

The patent application (Publication Number: US20240002815A1) discloses a polynucleotide encoding an ornithine transcarbamylase (OTC) protein with specific amino acid sequences and enzymatic activity. The polynucleotide can contain specific sequences, including a 3' poly A tail, a 5' untranslated region (5'UTR) from Arabidopsis thaliana, and a 3' UTR with selected sequences. Additionally, the polynucleotide may include a Kozak sequence, a 5' cap structure, and modifications to reduce uracil nucleobases or incorporate modified uridine analogs. The patent also covers pharmaceutical compositions containing the polynucleotide in lipid formulations for treating OTC deficiency in patients.

Furthermore, the patent application includes methods for treating OTC deficiency by administering the disclosed pharmaceutical compositions to patients. It also encompasses vectors containing the polynucleotide and mRNA encoding the OTC protein with specific sequences and enzymatic activity. The mRNA may include modified uridine analogs or specific uridine nucleotide modifications. Overall, the patent application aims to provide novel polynucleotides, pharmaceutical compositions, and methods for treating OTC deficiency, utilizing specific sequences and modifications to enhance therapeutic efficacy.

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData Patent Analytics tracks bibliographic data, legal events data, point in time patent ownerships, and backward and forward citations from global patenting offices. Textual analysis and official patent classifications are used to group patents into key thematic areas and link them to specific companies