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Four New RNAi-Related Patent Applications Published By US Patent Office: Mar 5, 2004

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Title: Composition and Method for Inhibiting Expression of a Target Gene. Number: 20040038921. Filed: Aug. 11, 2003. Lead Inventor: Roland Kreutzer, Ribopharma (Alnylam).

According to the patent application’s abstract, the invention “relates to pharmaceutical compositions comprising a double-stranded oligoribonucleic acid having a nucleotide sequence which is substantially identical to at least part of a target gene in a mammalian cell and which is less than 25 nucleotides in length, together with a pharmaceutically acceptable carrier.”

These pharmaceutical compositions, the abstract states, “are useful for inhibiting the expression of a target gene, as well as for treating diseases caused by expression of the target gene, in a mammal at very low dosages.

The invention also relates to methods for inhibiting the expression of a target gene in a mammal, as well as methods for treating diseases caused by expression of the gene.”

This patent application is the US counterpart of an issued European patent that is currently the subject of an ongoing dispute between Ribopharma and seven biopharmaceutical companies (see RNAi News, 1/16/2004).


Title: Essential Downstream Component of the Wingless Signaling Pathway and Therapeutic and Diagnostic Applications Based Thereon. Number: 20040038901. Filed: Sept. 22, 2003. Lead Inventor: Konrad Basler, Universitat Zurich.

The patent application, its abstract states, covers “an isolated polypeptide comprising a peptide [that] consists of a legless homology domain, wherein the peptide inhibits Lgs function in colon cancer cells.”

The application specifically claims a double-stranded RNA sequence, “comprising RNA interfering activities,” that is able to “induce degradation of Lgs single-stranded RNA.” Also claimed is “a therapeutic method comprising the use of Lgs proteins, homologs thereof, functional homologs, nucleic acids and/or fragments thereof for the treatment of disorders of cell fate.”


Title: Potentiation of Cancer Therapies by ZNF217 Inhibition. Number: 20040038322. Filed: Jan. 22, 2003. Lead Inventor: Colin Collins, University of California, San Francisco.

The patent application, its abstract states, “provides methods, reagents, and kits for treating cancer. Accordingly, the … methods can be used to monitor the efficacy of a cancer treatment and to treat cancer, e.g. by inhibiting the expression and/or activity of ZNF217 in a neoplastic cell.”

The application specifically claims a ZNF217 inhibitor that “comprises an siRNA nucleic acid.”


Title: Methods and Compositions Relating to Gene Silencing. Number: 20040038278. Filed: July 18, 2003. Lead Inventor: George Tzertzinis, New England Biolabs.

According to the patent application’s abstract, the invention is “a method for obtaining a mixture of heterogenous short double-stranded RNA (hsiRNA) molecules suitable for use in gene silencing by subjecting large double-stranded RNA to enzymatic cleavage under specified conditions.”

The resultant dsRNA mixture “consistently includes enhanced representation of fragments having a size of 21-022 nucleotides absent any fractionation step,” the abstract notes. “The fragments contain sequences that collectively span the entire length of the large double-stranded RNA from which they are derived.”

The patent application also includes methods for analyzing the siRNA fragments for gene silencing efficacy, as well as a method for “preparing and cloning DNA encoding selected siRNA, hsiRNA mixtures, or hairpin sequences to provide a continuous supply of gene silencing reagent derived from any long double-stranded RNA,” states the abstract.

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