Intellia Therapeutics has been granted a patent for compositions and methods to treat amyloidosis associated with TTR (ATTR) by editing the TTR gene. The method involves administering a composition containing specific RNA-guided DNA binding agents. 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 May 2024 was 4%. Grant share is based on the ratio of number of grants to total number of patents.

Treating amyloidosis associated with ttr using gene editing

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

A recently granted patent (Publication Number: US11965165B2) discloses a method for treating amyloidosis associated with TTR (ATTR) by administering a composition to a subject in need. The composition includes an RNA-guided DNA binding agent or a nucleic acid encoding it, along with a single guide RNA with specific sequences. This method aims to treat ATTR by targeting the TTR gene and inducing a double-stranded break (DSB) or modifying the gene, ultimately reducing TTR serum concentration and preventing the accumulation of amyloids or amyloid fibrils in the subject's body. The composition's components are designed to achieve these therapeutic effects by utilizing specific sequences and modification patterns of nucleotides, including phosphorothioate (PS) linkages and 2'-O-Me modifications.

Furthermore, the patent also covers methods for inducing DSB within the TTR gene or modifying it, targeting specific mutations associated with ATTR, such as V30, T60, or V122 mutations. By delivering a composition containing a SpyCas9 or a nucleic acid encoding it, along with a single guide RNA with a defined sequence, this method aims to induce precise genetic modifications to address amyloidosis associated with TTR. The patent highlights the potential of this approach to edit the TTR gene, reduce serum TTR levels, and prevent amyloid deposition in various tissues, offering a promising therapeutic strategy for individuals with ATTR and related conditions.

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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