ProQR Therapeutics has been granted a patent for an oligonucleotide targeting a mutation in the CEP290 gene to reduce aberrant exon inclusion. The oligonucleotide has specific characteristics to prevent hairpin formation and includes 2′-O-methyl modifications and phosphorothioate linkages. 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 April 2024 was 26%. Grant share is based on the ratio of number of grants to total number of patents.
Oligonucleotide for reducing aberrant splice site selection in cep290 gene
A recently granted patent (Publication Number: US11920132B2) discloses an oligonucleotide designed to reduce splice site selection of an aberrant splice site associated with the c.2991+1655A>G mutation in intron 26 of the human CEP290 gene when expressed in a human cell. The oligonucleotide's sequence, as per SEQ ID NO: 7, is characterized by all ribose moieties being 2'-O-methyl modified and all internucleosidic linkages being phosphorothioate linkages. This innovative approach aims to address specific genetic mutations at the molecular level, potentially offering new therapeutic possibilities for genetic disorders linked to the CEP290 gene.
Furthermore, the patent also covers a pharmaceutical composition that includes the aforementioned oligonucleotide. By incorporating the oligonucleotide into a pharmaceutical formulation, the composition may provide a targeted and precise treatment strategy for individuals affected by the c.2991+1655A>G mutation in the CEP290 gene. This development highlights the ongoing efforts in the field of genetic medicine to explore novel therapeutic interventions that can address genetic abnormalities at the molecular level, potentially paving the way for more personalized and effective treatment options for genetic disorders.
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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
