ProQR Therapeutics has been granted a patent for an oligonucleotide that targets a specific mutation in the CEP290 gene, reducing the inclusion of aberrant exons in mRNA. The sequence is modified with 2′-O-methyl ribose 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 February 2024 was 35%. 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

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

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 avenues for therapeutic interventions in genetic disorders.

Furthermore, the patent also covers a pharmaceutical composition that includes the aforementioned oligonucleotide. By incorporating this specialized oligonucleotide into a pharmaceutical formulation, the composition may provide a targeted and precise mechanism for modulating splice site selection in the context of the identified genetic mutation. This development underscores the ongoing advancements in the field of genetic medicine, where tailored molecular interventions hold promise for addressing genetic abnormalities at a fundamental level. The patent's focus on a specific mutation within the CEP290 gene highlights the potential for personalized treatments that target the underlying genetic causes of certain disorders, paving the way for more effective and precise therapeutic strategies in the realm of genetic medicine.

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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