ProQR Therapeutics has filed a patent for antisense oligonucleotides capable of editing specific nucleotides in RNA. The oligonucleotides do not form hairpin structures and contain cytidine or uridine opposite the target adenosine. The patent claims have been canceled. 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 September 2023 was 25%. Grant share is based on the ratio of number of grants to total number of patents.

Specific editing of target nucleotide in eukaryotic cell rna

Source: United States Patent and Trademark Office (USPTO). Credit: ProQR Therapeutics NV

A recently filed patent (Publication Number: US20230279392A1) describes an antisense oligonucleotide (AON) that can form a double-stranded complex with a specific RNA target in cells. The AON is designed to facilitate the deamination of a target adenosine present in the RNA by an ADAR enzyme naturally found in the cell. The AON is complementary to a specific region of the target RNA that includes the target adenosine. Importantly, the nucleotide in the AON that is opposite the target adenosine contains a deoxyribose with a 2'-H group. Additionally, the AON does not include a portion that is both non-complementary to the target RNA region and capable of forming an intramolecular stem-loop structure.

One specific application of this AON is in the treatment of A1AT deficiency, a condition caused by a specific mutation in the human SERPINA1 gene. The target adenosine in this case corresponds to the c.1096G>A mutation. The AON can form a mismatch with the target adenosine and may contain 1 to 10 mismatches, wobbles, and/or bulges with the complementary target RNA region. The AON may also include an inosine nucleotide directly 5' of the nucleotide opposite the target adenosine. Furthermore, the AON can contain a Central Triplet, where the middle nucleotide is the one opposite the target adenosine, and the Central Triplet consists of three DNA nucleotides.

The AON may also include nucleotides with 2'-O-methyl (2'-OMe) or 2'-O-methoxyethyl (2'-MOE) substitutions, as well as phosphorothioate linkages. The length of the AON can vary, ranging from 18 to 50 nucleotides. Importantly, the AON does not contain a 5'-terminal O6-benzylguanosine.

The patent also covers a pharmaceutical composition that includes the AON described above and a pharmaceutically acceptable carrier. This composition can be used for the treatment of A1AT deficiency in human subjects. The method involves administering the AON to individuals who require treatment for A1AT deficiency.

In summary, this patent describes an antisense oligonucleotide (AON) that can form a complex with a specific RNA target in cells, facilitating the deamination of a target adenosine by an ADAR enzyme. The AON has potential applications in the treatment of A1AT deficiency, and the patent also covers a pharmaceutical composition and a method for treating this condition using the AON.

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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