Sangamo Therapeutics. has been granted a patent for nucleases designed to modify the CFTR gene, along with methods for creating cells and animal models. The patent includes a claim for a fusion molecule that combines a functional domain with a DNA-binding domain targeting specific sites. GlobalData’s report on Sangamo 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 Sangamo Therapeutics, Zinc-finger nucleases was a key innovation area identified from patents. Sangamo Therapeutics's grant share as of June 2024 was 48%. Grant share is based on the ratio of number of grants to total number of patents.
Nucleases for cftr gene alteration and model generation
The granted patent US12043650B2 outlines a series of claims related to a fusion molecule that combines a functional domain with a DNA-binding domain, specifically designed to target and modify cystic fibrosis transmembrane conductance regulator (CFTR) genes. The claims include a polynucleotide encoding this fusion molecule, as well as various vectors—both viral and non-viral—that incorporate the polynucleotide. Notably, the patent specifies the use of an adeno-associated virus (AAV) vector and describes compositions and isolated cells containing the fusion molecule. Additionally, the patent details methods for modifying CFTR genes in isolated cells, including the introduction of zinc finger nucleases to cleave and alter the CFTR gene, with potential modifications involving insertions or deletions.
Further claims elaborate on the methods for correcting specific mutations within the CFTR gene, such as the AF508 mutation, by integrating donor nucleotide sequences that restore the gene to a wild-type sequence. The patent also covers genetically modified cells that express functional CFTR proteins following the integration of exogenous sequences. These cells may include lung or stem cells. Moreover, the patent describes methods for creating mutant CFTR genes by mutating wild-type genes using the specified nucleases. Lastly, a kit containing the polynucleotide is also included in the claims, emphasizing the practical applications of this technology in genetic modification and potential therapeutic interventions for cystic fibrosis.
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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
