Verve Therapeutics. has been granted a patent for compositions and methods aimed at gene editing to lower LDL cholesterol levels. The invention focuses on targeting the PCSK9 gene using an mRNA-based editor and guide RNA, potentially addressing cardiovascular disease effectively and safely. GlobalData’s report on Verve Therapeutics gives a 360-degree view of the company including its patenting strategy. Buy the report here.
According to GlobalData’s company profile on Verve Therapeutics, CRISPR nucleases was a key innovation area identified from patents. Verve Therapeutics's grant share as of June 2024 was 12%. Grant share is based on the ratio of number of grants to total number of patents.
Gene editing compositions to lower ldl cholesterol levels
The granted patent US12029795B2 outlines a novel composition for gene editing, specifically targeting the PCSK9 gene. The composition includes an mRNA that encodes a base editor protein, which consists of a DNA binding domain and a deaminase. The mRNA must exhibit at least 95% sequence identity to a specified sequence (SEQ ID NO: 2192). Additionally, the composition incorporates a guide RNA that features a tracr sequence for binding the base editor protein and a spacer sequence that aligns with a protospacer on the PCSK9 gene. The claims further specify variations in sequence identity, GC content, and structural components of the mRNA and guide RNA, enhancing the precision and efficacy of the gene editing process.
Further claims detail the specific characteristics of the mRNA and guide RNA, including the potential for chemical modifications to the guide RNA and the ratios of guide RNA to mRNA. The patent also describes the intended outcomes of the gene editing, such as inducing nucleobase alterations that could lead to frame shifts, premature stop codons, or aberrant transcripts in the PCSK9 gene. Notably, the alterations may occur at splice donor or acceptor sites, with specific reference to sequences that demonstrate significant identity to established sequences. This patent represents a significant advancement in the field of genetic engineering, particularly in the context of therapeutic interventions targeting the PCSK9 gene.
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