Intellia Therapeutics has been granted a patent for compositions and methods to treat amyloidosis associated with TTR (ATTR) by editing the TTR gene. The method involves administering a composition containing specific RNA-guided DNA binding agents. GlobalData’s report on Intellia Therapeutics gives a 360-degree view of the company including its patenting strategy. Buy the report here.
According to GlobalData’s company profile on Intellia Therapeutics, CRISPR genome editing was a key innovation area identified from patents. Intellia Therapeutics's grant share as of April 2024 was 4%. Grant share is based on the ratio of number of grants to total number of patents.
Treating amyloidosis associated with ttr gene using compositions
A recently granted patent (Publication Number: US11965165B2) discloses a method for treating amyloidosis associated with TTR (ATTR) by administering a composition to a subject in need. The composition includes an RNA-guided DNA binding agent or a nucleic acid encoding it, along with a single guide RNA containing specific sequences and modifications. This method aims to treat ATTR by inducing a double-stranded break within the TTR gene or modifying it, ultimately reducing TTR serum concentration and preventing the accumulation of amyloids or amyloid fibrils in the subject.
Furthermore, the patent claims encompass various aspects of the method, including reducing serum TTR levels by at least 50%, editing the TTR gene in a population of cells, and reducing amyloid deposition in specific tissues. The composition may also include additional components like mRNA encoding the RNA-guided DNA binding agent, Cas9, or be associated with a lipid nanoparticle. The method is designed to target different manifestations of ATTR, such as familial amyloid polyneuropathy or cardiomyopathy, based on the subject's genetic profile. Overall, the patent outlines a comprehensive approach to treating ATTR by leveraging innovative genetic editing techniques and specific RNA sequences to address the underlying causes of the disease.
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