ProQR Therapeutics has been granted a patent for nucleic acid molecules designed to facilitate pseudouridylation of target RNA in mammalian cells. The molecules contain a guide region that interacts with a target uridine, enabling conversion to pseudouridine by a mammalian enzyme. GlobalData’s report on ProQR 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 ProQR Therapeutics, Nucleic acid active pharmaceutical ingredient was a key innovation area identified from patents. ProQR Therapeutics's grant share as of January 2024 was 25%. Grant share is based on the ratio of number of grants to total number of patents.
Nucleic acid molecule for converting uridine to pseudouridine
A recently granted patent (Publication Number: US11866702B2) discloses a nucleic acid molecule designed to convert a target uridine in a mammalian cell's RNA into pseudouridine. The molecule comprises a guide region that forms a partially double-stranded complex with the target RNA, facilitating interaction with a mammalian pseudouridylation enzyme for the conversion process. The patent also covers modifications to the nucleic acid molecule, including non-natural nucleosides and inter-nucleosidic linkages, to enhance its functionality compared to wild type H/ACA snoRNA.
Furthermore, the patent includes claims related to the positioning of the nucleic acid molecule within an intron sequence, vector systems for expression, and methods for converting uridine to pseudouridine in cells. The invention aims to address genetic disorders associated with specific mutations in the target RNA, such as Premature Termination Codons (PTC). The patent also encompasses vectors containing the nucleic acid molecule, pharmaceutical compositions utilizing the molecule, and methods for administering the molecule to mammalian cells, particularly human cells, for therapeutic purposes. Overall, the patent provides a comprehensive framework for utilizing the nucleic acid molecule to modulate RNA function and potentially treat genetic disorders at the molecular level.
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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
