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Six New RNA Interference-Related US Patent Applications Published

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Title: Compositions and Processes for Inhibiting Gene Expression Using Polynucleotides. Number: 20030224055. Filed: May 28, 2003. Lead Inventor: David Lewis, Mirus.

The patent application, its abstract states, covers “compositions … for the delivery of polynucleotides to cells for the purpose of inhibiting gene expression.

“Antisense polynucleotide-containing complexes are described,” according to the abstract. “The sale and serum stability, and small size of the complexes, permits delivery to cells in vitro and in vivo.”


Title: Screening Assays for Identifying Differentiation-Inducing Agents and Production of Differentiated Cells for Cell Therapy. Number: 20030224345. Filed: Aug. 26, 2002. Lead Inventor: Michael West, Advanced Cell Technology.

This patent application’s abstract states that the invention relates to assays used to screen growth factors, adhesion molecules, immunostimulatory molecules, extracellular matrix components, and other materials, “alone or in combination, simultaneously or temporally, for the ability to induce directed differentiation of pluripotent and multipotent stem cells.”

The patent application specifically claims a method for evaluating the differentiation of stem cells that includes exposing the cells to “one or more putative differentiating-inducing conditions,” which are “selected from the group consisting of growth factors, cytokines, tissue extracts, nucleic acids, factors involved in cell-components from tissue, media components, environmental conditions, and living cells that induce differentiation by cell-cell interactions.”

“Nucleic acids that may be tested,” the application adds, “include but are not limited to those that encode or block by antisense, ribozyme activity, or RNA interference.”


Title: High Throughput-Directed Evolution of Nucleic Acids by Rational Mutagenesis. Number: 20030224404. Filed: Feb. 24, 2003. Lead Inventor: Manuel Vega, Nautilus Biotech.

The patent application, its abstract states, covers “methods and systems for the high throughput-directed evolution of functional nucleic acid molecules, such as cis-acting elements, particularly those that act in complex biological settings.”

The method described involves using a “population of sets of target functional nucleic acid molecules that each comprise a target modified functional sequence of nucleotides,” according to the patent application.

“Target functional nucleic acid molecules include, but are not limited to, cis-acting regulatory sequences, antisense nucleic acid and RNA, including dsRNA,” it adds. “These include, for example, promoters, enhancers, silencers, protein-binding domains, ribosomal RNA and ribozymes and tRNA, interfering RNA (RNAi) and antisense molecules.”


Title: MBCATs as Modifiers of the Beta-Catenin Pathway and Methods of Use. Number: 20030224406. Filed: Feb. 28, 2003. Lead Inventor: Michael Costa, Exelixis.

According to the patent application’s abstract, “human MBCAT genes are identified as modulators of the beta-catenin pathway, and thus are therapeutic targets for disorders associated with defective beta-catenin function.”

The abstract adds that the application covers “methods for identifying modulators of beta-catenin, comprising screening for agents that modulate the activity of MBCAT.”

The patent application states that the inventors have identified the human orthologs for genes in C. elegans that modify the beta-catenin pathway. “The invention provides for methods of utilizing these beta-catenin modifier genes and polypeptides to identify MBCAT-modulating agents that are candidate therapeutic agents,” the application adds. Some “preferred MBCAT-modulating agents are nucleic acid modulators such as antisense oligomers and RNAi that repress MBCAT gene expression or product activity.”


Title: Methods and Compositions for Use in Preparing siRNAs. Number: 20030224432. Filed: April 30, 2003. Lead Inventor: Jason Myers, Stanford University.

The patent application covers “methods and compositions for producing siRNAs — e.g. in the form of a d-siRNA composition — from dsRNAs,” its abstract states.

“In the subject methods, a dsRNA is contacted with a composition that includes an activity that cleaves dsRNA into siRNAs, where the composition efficiently cleaves dsRNA into siRNAs,” the abstract adds. “SiRNAs produced by the subject methods find use in a variety of applications, particularly in applications where the specific reduction or silencing of a gene is desired.”

The patent application, the abstract notes, also provides for kits used to practice the invention.


Title: Gene SHINC-2 and Diagnostics and Therapeutic Uses Thereof. Number: 20030225023. Filed: April 10, 2003. Lead Inventor: Usha Kasid, Georgetown University.

According to the patent application’s abstract, the invention “provides a SHINC-2 polynucleotide, which can be a nucleic acid encoding all or a portion of a SHINC-2 protein, or a complementary polynucleotide or antisense polynucleotide.

“Desirably, the SHINC-2 polypeptide modulates apoptosis,” the abstract adds.

According to the abstract, aspects of the invention include diagnostic methods; a method for detecting or evaluating the prognosis of cancer; and a method of modulating, preventing, or treating cancer, tumor growth, and/or metastasis by administering an agent that modulates the expression and/or activity of SHINC-2.

The patent application notes that the aforementioned agent can be an antisense oligonucleotide, a ribozyme, or an RNAi molecule.


In other news, Alnylam said that it has launched a program to facilitate the out-licensing of technology covered under its RNAi patents and patent applications. See this issue’s Products & Services (p.2) for more details.

The Scan

Renewed Gain-of-Function Worries

The New York Times writes that the pandemic is renewing concerns about gain-of-function research.

Who's Getting the Patents?

A trio of researchers has analyzed gender trends in biomedical patents issued between 1976 and 2010 in the US, New Scientist reports.

Other Uses

CBS Sunday Morning looks at how mRNA vaccine technology could be applied beyond SARS-CoV-2.

PLOS Papers Present Analysis of Cervicovaginal Microbiome, Glycosylation in Model Archaea, More

In PLOS this week: functional potential of the cervicovaginal microbiome, glycosylation patterns in model archaea, and more.