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 base 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 July 2024 was 11%. 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 the mRNA sequence identity, GC content, and structural components of the base editor protein, including regions for adenine tRNA deaminase, Cas9 nickase, and nuclear localization.
Further claims detail the characteristics of the guide RNA, including potential chemical modifications and the specific nucleobase alterations it can induce in the PCSK9 gene. These alterations may lead to significant changes in the gene's transcript, such as frame shifts, premature stop codons, or aberrant transcripts. The patent also specifies the identity requirements for the protospacer and the guide RNA's spacer sequence, ensuring precise targeting for effective gene editing. Overall, the patent presents a comprehensive framework for a gene editing composition that could have implications in therapeutic applications related to PCSK9 and associated conditions.
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