CRISPR Therapeutics has been granted a patent for methods and compositions that utilize gene editing to treat diseases related to angiopoietin-like 3 (ANGPTL3). The patented method involves administering nanoparticles containing specific guide RNA and SpCas9 to achieve over 50% editing efficiency in the liver. GlobalData’s report on CRISPR 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 CRISPR Therapeutics, CRISPR genome editing was a key innovation area identified from patents. CRISPR Therapeutics's grant share as of July 2024 was 13%. Grant share is based on the ratio of number of grants to total number of patents.
Gene editing for angptl3-related diseases using nanoparticles
The granted patent US12037616B2 outlines a novel method for treating diseases or disorders related to Angiopoietin-like 3 (ANGPTL3) through the administration of nanoparticles. These nanoparticles are complexed with a guide RNA (gRNA) specifically targeting the ANGPTL3 gene and a nucleic acid encoding the SpCas9 protein. The gRNA, which includes a defined sequence (SEQ ID NO: 13) and a spacer sequence (SEQ ID NO: 20), is designed to achieve an editing efficiency exceeding 50% in the liver. The method aims to reduce ANGPTL3 expression and protein concentration in the subject's plasma by at least 20%, with potential reductions of up to 70% observed one month post-administration. The treatment is particularly relevant for subjects with elevated triglyceride levels and other lipid-related disorders.
Additionally, the patent specifies that the method can be administered as a single dose of nanoparticles, with various dosage options ranging from 0.1 mg/kg to 1.0 mg/kg of total nucleic acids. The nanoparticles are characterized as lipid nanoparticles, which may contain a combination of neutral lipids, charged lipids, and polymers. The treatment targets a range of ANGPTL3-related conditions, including metabolic diseases, cardiovascular diseases, and specific disorders such as obesity, diabetes, and dyslipidemia. The claims also highlight the potential for significant reductions in non-high-density lipoprotein (non-HDL) lipids and apolipoprotein B (ApoB) levels, further emphasizing the therapeutic implications of this innovative approach.
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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
