ProQR Therapeutics has been granted a patent for antisense oligonucleotides designed to edit specific adenosine nucleotides in target RNA within eukaryotic cells. These 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 June 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 ADAR enzyme that is naturally present in the cell. 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 portions that could form intramolecular stem-loop structures. The patent also specifies that the AON can include various modifications, such as mismatches, wobbles, and bulges, as well as specific nucleotide substitutions like 2'-O-methyl and 2'-O-methoxyethyl.
Additionally, the patent claims the AON's application in treating A1AT deficiency, particularly targeting the c.1096G>A mutation in the SERPINA1 gene, which is known to cause this deficiency. The AON can be formulated into a pharmaceutical composition alongside a pharmaceutically acceptable carrier, and the method for treatment involves administering the AON to affected individuals. The claims further detail the structural characteristics of the AON, including the potential for specific nucleotide linkages and lengths, thereby providing a comprehensive framework for its therapeutic use in addressing A1AT deficiency.
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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
