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.
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 July 2024 was 47%. 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 that contain 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, potentially correcting mutations such as the AF508 variant.
Further claims elaborate on the methods of integrating transgenes into the CFTR gene, which may involve inserting or deleting sequences to restore functional CFTR protein expression. The patent also covers genetically modified cells that have undergone these modifications, including lung cells and stem cells. Moreover, it describes a method for creating a cell or cell line with a mutant CFTR gene by mutating a wild-type gene using the specified nuclease. Lastly, the patent includes provisions for a kit containing the polynucleotide, as well as isolated cells that incorporate this polynucleotide, indicating a comprehensive approach to gene editing and potential therapeutic applications for cystic fibrosis.
To know more about GlobalData’s detailed insights on Sangamo Therapeutics, buy the report here.
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