Alnylam Pharmaceuticals. has been granted a patent for RNAi agents targeting the Serpina1 gene. These double-stranded RNA agents are designed to inhibit Serpina1 expression, offering potential treatment methods for diseases associated with this gene, particularly liver disorders. 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 July 2024 was 36%. Grant share is based on the ratio of number of grants to total number of patents.
Rnai agents targeting serpina1 gene for disease treatment
The patent US12071619B2 describes a double-stranded ribonucleic acid (dsRNA) agent designed to inhibit the expression of the serine peptidase inhibitor, clade A member 1 (Serpina1) in cells. The dsRNA agent consists of a sense strand and an antisense strand, each ranging from 19 to 30 nucleotides in length. The antisense strand is characterized by at least 19 contiguous nucleotides that differ by no more than three nucleotides from specified sequences. Notably, all nucleotides in both strands are modified, and at least one strand is conjugated to a ligand, specifically one or more GalNAc derivatives. The claims also detail various structural modifications, including phosphorothioate or methylphosphonate linkages, and the potential for mismatches in the seed region of the antisense strand.
Additionally, the patent outlines methods for utilizing the dsRNA agent to inhibit Serpina1 expression by contacting cells with the agent and allowing sufficient time for degradation of the Serpina1 mRNA transcript. The claims further specify the lengths of the sense and antisense strands, the presence of 3' overhangs, and the types of nucleotide modifications that can be employed. The invention also encompasses isolated cells containing the dsRNA agent and pharmaceutical compositions that include the agent, indicating its potential application in therapeutic contexts. Overall, the patent presents a comprehensive approach to targeting Serpina1 expression through engineered dsRNA technology.
To know more about GlobalData’s detailed insights on Alnylam Pharmaceuticals, buy the report here.
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