Beam Therapeutics. has filed a patent for a novel Cas9 protein optimized for human cells, derived from Lachnospira bacterium. The engineered Cas9 can be used for nucleic acid targeting and manipulation, including as a base editor and for treating human diseases. GlobalData’s report on Beam 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 Beam Therapeutics, CRISPR genome editing was a key innovation area identified from patents. Beam 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.

Engineered cas9 protein for nucleic acid targeting and manipulation

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

A recently filed patent (Publication Number: US20230279373A1) describes an engineered Cas9 protein that has been modified from Lachnospira Cas9. The modified Cas9 protein has at least 80% identity to the original Lachnospira Cas9 protein. The patent claims that this engineered Cas9 protein has various properties and capabilities.

One of the claims states that the Cas9 protein has nickase activity, meaning it can cleave a single strand of DNA. The protein also contains amino acid mutations in specific domains, such as PAM Interacting, HNH, and/or RuvC. Additionally, the Cas9 protein may have a nuclear localization sequence (NLS) and/or a FLAG, HIS, or HA tag. It is also capable of recognizing a specific PAM sequence in the target DNA.

The patent also describes an engineered Cas9 fusion protein, which combines the Cas9 protein with a histone demethylase, a transcriptional activator, or a deaminase. The deaminase can be either a cytosine deaminase or an adenosine deaminase.

Methods for cleaving and modifying target nucleic acids in eukaryotic cells are also claimed in the patent. These methods involve contacting the cells with the engineered Cas9 protein and an RNA guide or a nucleic acid encoding the RNA guide. The RNA guide contains a direct repeat sequence and a spacer sequence that can hybridize to the target nucleic acid. The Cas9 protein can bind to the RNA guide and cause a break in the target nucleic acid sequence or edit the target nucleic acid sequence.

The patent also describes a base editor, which is a fusion protein of the Cas9 protein and an adenosine deaminase or a cytidine deaminase domain. The base editor can be targeted to specific nucleobases in a polynucleotide sequence to effect alterations.

Overall, this patent describes various engineered Cas9 proteins and their applications in cleaving and modifying target nucleic acids in eukaryotic cells. It also includes methods for treating disorders or diseases by administering the engineered Cas9 system to a subject. The patent also covers base editing methods using the fusion protein of Cas9 and a deaminase domain.

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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