CRISPR Therapeutics has been granted a patent for treating Glycogen Storage Disease type 1a (GSD1a) using genome editing to modulate the expression of specific genes. The patent includes methods for editing specific exons of the glucose-6-phosphatase gene with high efficiency. GlobalData’s report on CRISPR Therapeutics gives a 360-degree view of the company including its patenting strategy. Buy the report here.
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 January 2024 was 12%. Grant share is based on the ratio of number of grants to total number of patents.
A recently granted patent (Publication Number: US11866727B2) discloses a method for editing specific exons of the glucose-6-phosphatase, catalytic (G6PC) gene in cells using guide ribonucleic acids (gRNAs). The gRNAs contain a spacer sequence of 10 to 30 nucleotides in length, corresponding to specific RNA sequences listed in the patent. These gRNAs are designed to have an editing efficiency of at least 10% and can target pathological variants associated with the G6PC gene.
The patent also covers the use of single-molecule guide RNAs (sgRNAs) as well as modified gRNAs or sgRNAs for editing the G6PC gene. The target cells for this gene editing technique are specified as liver cells. Additionally, the gRNAs are directed towards specific pathological variants such as rs80356479, rs80356486, rs606231368, among others. The spacer sequences in the gRNAs correspond to a range of full-length nucleic acid sequences provided in the patent, enabling precise editing of the G6PC gene in cells.
To know more about GlobalData’s detailed insights on CRISPR Therapeutics, buy the report here.
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