ProQR Therapeutics has been granted a patent for antisense oligonucleotides designed to edit specific adenosine nucleotides in target RNA within eukaryotic cells. The oligonucleotides facilitate deamination by ADAR enzymes and are characterized by specific structural requirements to avoid forming intramolecular structures. 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 July 2024 was 26%. Grant share is based on the ratio of number of grants to total number of patents.
Antisense oligonucleotides for targeted rna editing
The granted patent US12018257B2 outlines a novel antisense oligonucleotide (AON) designed to target and form a double-stranded complex with alpha1-antitrypsin (A1AT)-encoding RNA within cells. This AON facilitates the deamination of a specific adenosine in the target RNA by utilizing an endogenous ADAR enzyme. Key features of the AON include its complementarity to the target RNA region containing the adenosine, the presence of a deoxyribose with a 2'-H group opposite the target adenosine, and the absence of any non-complementary segments that could form intramolecular stem-loop structures. The patent also specifies that the AON can address the c.1096G>A mutation in the SERPINA1 gene, which is linked to A1AT deficiency.
Further claims detail the structural characteristics of the AON, including the potential for mismatches, wobbles, or bulges with the target RNA, and specific nucleotide modifications such as 2'-O-methyl and 2'-O-methoxyethyl substitutions. The AON may also incorporate phosphorothioate linkages and can vary in length from 18 to 50 nucleotides. Additionally, the patent describes a pharmaceutical composition that includes the AON and a suitable carrier, as well as a method for treating A1AT deficiency in affected individuals through the administration of the AON. This innovative approach aims to provide a targeted therapeutic strategy for managing A1AT deficiency, a condition that can lead to serious health complications.
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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
