Intellia Therapeutics. has filed a patent for compositions and methods to edit the KLKB1 gene, including introducing double-stranded breaks. The patent also covers compositions and methods for treating hereditary angioedema (HAE). The claim specifies the requirements for a guide RNA sequence. GlobalData’s report on Intellia 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 Intellia Therapeutics, CRISPR genome editing was a key innovation area identified from patents. Intellia Therapeutics's grant share as of September 2023 was 3%. Grant share is based on the ratio of number of grants to total number of patents.

Gene editing method for treating hereditary angioedema (hae)

Source: United States Patent and Trademark Office (USPTO). Credit: Intellia Therapeutics Inc

A recently filed patent (Publication Number: US20230295587A1) describes a guide RNA that can be used in various applications related to gene editing and therapeutic treatments. The guide RNA consists of a guide sequence that is at least 95%, 90%, or 85% identical to specific sequences (SEQ ID NOs: 15, 8, and 41), or it can be selected from these sequences. The guide sequence is composed of at least 17, 18, 19, or 20 contiguous nucleotides from the specified sequences. Additionally, the guide RNA may include a nucleotide sequence (SEQ ID NO: 202) and/or a nucleotide sequence selected from SEQ ID NOs: 170, 171, 172, and 173, which are positioned 3' of the guide sequence. The guide RNA can also have a 3' tail and may contain modifications such as 5' end modifications, 3' end modifications, or modifications in a hairpin region. These modifications can include 2'-O-methyl (2'-O-Me) modified nucleotides, phosphorothioate (PS) bonds between nucleotides, or 2'-fluor (2'F) modified nucleotides.

The patent also covers compositions that include the guide RNA, either alone or in combination with an RNA-guided DNA binding agent or nucleic acid encoding such an agent. The RNA-guided DNA binding agent can be Cas9, specifically S. pyogenes Cas9. The compositions may be associated with a lipid nanoparticle (LNP) containing an ionizable lipid, such as (9Z,12Z)-3-((4,4-bis(octyloxy)butanoyl)oxy)-2-((((3-(diethylamino)propoxy)carbonyl)oxy)methyl)propyl octadeca-9,12-dienoate. The patent also includes a pharmaceutical composition that combines the guide RNA with a pharmaceutical excipient or carrier.

Furthermore, the patent describes a method for various applications, including inducing breaks in a specific gene (KLKB1), reducing expression of KLKB1, treating hereditary angioedema (HAE), treating or preventing angioedema associated with HAE, reducing bradykinin production and accumulation, reducing bradykinin-induced swelling, preventing angioedema obstruction of the airway, or preventing asphyxiation. This method involves contacting a cell with the guide RNA.

Overall, this patent provides a comprehensive description of guide RNA sequences, compositions, and methods that can be utilized in gene editing and therapeutic applications.

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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