CRISPR Therapeutics has filed a patent application for a method to treat Amyotrophic Lateral Sclerosis (ALS) by editing the C9ORF72 gene in human cells using genome editing techniques. The method involves modifying the transcription start site of exon1a to address the genetic cause of ALS. 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 January 2024 was 12%. Grant share is based on the ratio of number of grants to total number of patents.

Genome editing method for treating amyotrophic lateral sclerosis (als)

Source: United States Patent and Trademark Office (USPTO). Credit: CRISPR Therapeutics AG

A patent application (Publication Number: US20230392134A1) describes a method for editing the C9ORF72 gene in human cells using genome editing techniques. The method involves introducing site-directed DNA endonucleases to create double-strand breaks within or near the first exon of the C9ORF72 gene. These breaks result in modifications that render the transcription start site non-functional, leading to the deletion of exon1a or the expanded hexanucleotide repeat associated with ALS/FTD. Various configurations of double-strand breaks are specified, targeting specific regions of the gene to achieve the desired modifications. The method also involves the use of specific DNA endonucleases, such as Cas9 or Cpf1, along with guide ribonucleic acids (gRNAs) to facilitate the editing process.

Furthermore, the patent application details the use of modified polynucleotides, RNAs, and gRNAs in the editing process, as well as the formulation of DNA endonucleases and gRNAs into lipid nanoparticles or viral vectors for delivery into cells. The method aims to reduce the expression of hexanucleotide repeat-containing transcripts of the C9ORF72 gene compared to unedited mutant cells. Specific nucleotide sequences for gRNAs are provided, along with recombinant expression vectors encoding the necessary components for gene editing. The system described in the patent application offers a precise and targeted approach to editing the C9ORF72 gene, potentially providing a valuable tool for research and therapeutic applications related to ALS/FTD and other conditions associated with this gene.

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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