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IP Update: Recent Patents, Patent Applications Awarded to Alnylam, Merck, BASF, and More

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Title: RNA Sequence-Specific Mediators of RNA Interference

Patent Number: 8,420,391

Filed: Oct. 4, 2010

Lead Inventor: Thomas Tuschl, Max Planck Institute

The invention, the patent’s abstract states, relates to a Drosophila in vitro system [that] was used to demonstrate that dsRNA is processed to RNA segments 21 [to] 23 nucleotides in length. Furthermore, when these … fragments are purified and added back to Drosophila extracts, they mediate RNA interference in the absence of long dsRNA. Thus, these … fragments are the sequence-specific mediators of RNA degradation. A molecular signal, which may be their specific length, must be present in these … fragments to recruit cellular factors involved in RNAi.”

The invention, the abstract adds, “encompasses these … fragments and their use for specifically inactivating gene function. The use of these fragments or chemically synthesized oligonucleotides of the same or similar nature enables the targeting of specific mRNAs for degradation in mammalian cells, where the use of long dsRNAs to elicit RNAi is usually not practical, presumably because of the deleterious effects of the interferon response. This specific targeting of a particular gene function is useful in functional genomic and therapeutic applications.”


Title: MAT II Beta Subunit RNAi and Therapeutic Methods Using Same

Patent Number: 8,420,616

Filed: April 7, 2009

Lead Inventor: Malak Kotb, University of Cinncinnati

The invention, the patent’s abstract states, relates to “methods and compositions for inhibiting the expression of MAT II beta subunit in a subject via RNAi by administering siRNA or shRNA. … In some embodiments, the methods and compositions … [relate] to the treatment of cancer,” namely leukemia.


Title: Therapeutic Compositions

Patent Number: 8,420,799

Filed: July 16, 2010

Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

“This application relates to therapeutic siRNA agents, and methods of making and using the agents,” the patent’s abstract states.


Title: Models of Malignant Brain Cancer and Therapeutic siRNAs Against Oncogenic Signaling Pathways, and Methods and Kits for Uses Therefor

Patent Number: 8,420,884

Filed: Oct. 16, 2009

Inventor: Alain Charest, Tufts Medical Center

The patent, its abstract states, claims “methods for screening compounds to treat an oncological disorder regulated through a tumor-inducing pathway. … The compounds are administered to non-human animal subjects having a disease model, so that the subjects display pathology symptoms that correspond to the oncological disorder in humans. … The methods further involve analyzing tumor formation in subjects administered the compound and comparing appearance and amount of tumors in the subjects administered the compound with control subjects not administered the compound. Also included are a vector for engineering a disease model and a kit for its use.”


Title: Treating Cancer by Modulating Mammalian Sterile 20-Like Kinase 3

Application Number: 20130089538

Filed: June 9, 2011

Lead Inventor: Hauke Hermann Karl Cornils, Novartis

The invention relates to a “method for modulating miRNA … [in which] a modulator of XRN1 is used,” according to the patent application’s abstract. “Also provided are uses of said method for therapeutical purposes, reagents … [and] screening methods.”


Title: microRNA-130a,b as a Tumor Suppressor and Sensitizing Agent for Chemotherapy

Application Number: 20130089597

Filed: April 2, 2012

Lead Inventor: Preethi Gunaratne, University of Houston

The invention provides a “method of improving a therapeutic response to a cancer treatment … [by] administering an effective amount of an agent that enhances the expression of microRNA-130 or an agent that mimics the effects of microRNA-130,” the patent application’s abstract states. “Further provided is a method of treating a cancer in a subject in need of such treatment comprising the step of administering an effective amount of a microRNA-130 or an agent that enhances the expression of microRNA-130.”


Title: Modulation of Epidermal Growth Factor Receptor Ligands

Application Number: 20130090366

Filed: Nov. 24, 2010

Lead Inventor: Peter Leedman, Western Australian Institute for Medical Research

The invention, the patent application’s abstract states, relates to a “method for modulating the expression and/or activity of an epidermal growth factor receptor ligand in a cell or tissue [that involves] contacting the cell or tissue with a miR-7 miRNA, a pre-cursor or variant thereof, a miRNA comprising a seed region comprising the sequence GGAAGA, or an antagonist of any such miRNA.”


Title: Agent for Suppressing Expression of Dominant Allele

Application Number: 20130090373

Filed: June 17, 2011

Lead Inventor: Hirohiko Hohjoh, National Center of Neurology and Psychiatry

The patent application, its abstract states, claims an “agent for selectively suppressing the expression of a dominant allele while allowing expression of wild-type or desired alleles and methods for using the agent. … The RNAi agent has a structure obtained by assigning a dominant point mutation in the targeted allele as a standard point, setting a base length from the standard point to the 5' end to a predetermined length, and introducing one mismatch base differing from the target sequence to a predetermined position downstream from the standard point.”


Title: Novel Low Molecular Weight Cationic Lipids for Oligonucleotide Delivery

Application Number: 20130090372

Filed: May 31, 2011

Lead Inventor: Brian Budzik, Merck

The invention comprises “novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides,” the patent application’s abstract states. The invention provides a “cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The … invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.”


Title: Nematode-Resistant Transgenic Plants

Application Number: 20130091598

Filed: Feb. 16, 2011

Lead Inventor: Aaron Wiig, BASF Plant Science

The invention, the patent application’s abstract states, relates to a “double-stranded RNA compositions and transgenic plants capable of inhibiting expression of plants genes, and methods associated therewith. Specifically, the invention relates to the use of RNA interference to inhibit expression of a target plant gene [such as] a plant a CLASP1 gene, an aspartic proteinase delta subunit gene, a secreted protein 1 gene, a lectin receptor kinase-like gene, a pectin methylesterase-like gene, and an N PY1 gene; and relates to the generation of plants that have increased resistance to parasitic nematodes.”

The Scan

Interfering With Invasive Mussels

The Chicago Tribune reports that researchers are studying whether RNA interference- or CRISPR-based approaches can combat invasive freshwater mussels.

Participation Analysis

A new study finds that women tend to participate less at scientific meetings but that some changes can lead to increased involvement, the Guardian reports.

Right Whales' Decline

A research study plans to use genetic analysis to gain insight into population decline among North American right whales, according to CBC.

Science Papers Tie Rare Mutations to Short Stature, Immunodeficiency; Present Single-Cell Transcriptomics Map

In Science this week: pair of mutations in one gene uncovered in brothers with short stature and immunodeficiency, and more.