Sangamo Therapeutics. has filed a patent for zinc finger fusion proteins that target alpha-synuclein gene to treat neurodegenerative diseases like Parkinson’s and Alzheimer’s. The fusion protein includes a zinc finger protein domain and a transcription repressor domain. 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 January 2024 was 46%. Grant share is based on the ratio of number of grants to total number of patents.
Zinc finger fusion protein to treat neurodegenerative diseases
A fusion protein patent application (Publication Number: US20240018203A1) describes a fusion protein comprising a zinc finger protein (ZFP) domain and a transcription repressor domain. The ZFP domain binds to a specific target region of the human alpha-synuclein gene (SNCA gene), with the fusion protein capable of repressing SNCA gene expression by varying degrees, ranging from 40% to 99%, while minimizing off-target binding or activity. The fusion protein includes six zinc fingers in the ZFP domain and a KRAB domain amino acid sequence from human KOX1 in the transcription repressor domain. The patent also covers a nucleic acid construct containing the fusion protein coding sequence linked to a transcription regulatory element, a recombinant virus incorporating this construct, and a pharmaceutical composition with the recombinant virus and a carrier.
Furthermore, the patent application outlines methods for inhibiting alpha-synuclein expression in human brain cells, particularly neurons, glial cells, ependymal cells, or neuroepithelial cells, which could be beneficial for patients with synucleinopathies like Parkinson's disease, Lewy body dementia, Alzheimer's disease, or multiple system atrophy. The methods involve introducing the fusion protein into the cells directly or via a recombinant virus, such as an adeno-associated virus (AAV) of serotype 9. Additionally, the patent application covers a treatment method for synucleinopathies in patients by administering a recombinant AAV encoding the fusion protein through various injection methods, including intravenous, intrathecal, or intracerebroventricular routes, targeting specific brain regions affected by the disease.
To know more about GlobalData’s detailed insights on Sangamo Therapeutics, buy the report here.
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