Alnylam Pharmaceuticals has been granted a patent for RNAi agents that target the TMPRSS6 gene. These agents can be used to inhibit the expression of TMPRSS6 and treat disorders associated with iron overload, such as ß-thalassemia or hemochromatosis. The patent claims a specific double-stranded RNAi agent with modified nucleotide sequences. GlobalData’s report on Alnylam Pharmaceuticals gives a 360-degree view of the company including its patenting strategy. Buy the report here.
According to GlobalData’s company profile on Alnylam Pharmaceuticals, siRNA gene silencing was a key innovation area identified from patents. Alnylam Pharmaceuticals's grant share as of September 2023 was 36%. Grant share is based on the ratio of number of grants to total number of patents.
Patent granted for double-stranded rnai agent targeting tmprss6 gene
A recently granted patent (Publication Number: US11725209B2) discloses a double stranded RNAi agent for inhibiting the expression of TMPRSS6 in a cell. The agent consists of a sense strand and an antisense strand, each comprising specific modified nucleotide sequences. The sense strand is 21 nucleotides long, while the antisense strand is 23 nucleotides long.
The patent also claims that the double stranded RNAi agent can further include a ligand, which can be one or more GalNAc derivatives attached through a bivalent or trivalent branched linker. The ligand can be attached to the 3' end of the sense strand.
The patent describes a pharmaceutical composition comprising the double stranded RNAi agent, which can be used to inhibit TMPRSS6 expression in a cell. The method involves contacting the cell with the RNAi agent and maintaining it for a sufficient time to degrade the mRNA transcript of the TMPRSS6 gene, thereby inhibiting its expression.
Furthermore, the patent discloses a method of treating a subject with a TMPRSS6 associated disorder by administering a therapeutically effective amount of the double stranded RNAi agent or the pharmaceutical composition. The subject can be a human and may have conditions such as hereditary hemochromatosis, ß-thalassemia, or erythropoietic porphyria, or disorders associated with iron overload.
The patent also mentions the administration of an iron chelator in combination with the RNAi agent for treating the subject.
Overall, this granted patent provides a novel double stranded RNAi agent and its use in inhibiting the expression of TMPRSS6, as well as methods for treating subjects with TMPRSS6 associated disorders. The inclusion of a ligand and the potential for combination therapy with an iron chelator adds to the potential therapeutic applications of this invention.
To know more about GlobalData’s detailed insights on Alnylam Pharmaceuticals, buy the report here.
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