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IP Update: Recent Patents, Patent Applications Awarded to OSU, Alnylam, Regeneron, and More

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Title: microRNA Fingerprints During Human Megakaryocytopoiesis

Patent Number: 8,354,224

Filed: June 27, 2011

Lead Inventor: Carlo Croce, Ohio State University

The invention, the patent's abstract states, “provides novel methods and compositions for the diagnosis, prognosis, and treatment of cancer and myeloproliferative disorders. The invention also provides methods of identifying anti-cancer agents.”


Title: miR-25-Based Methods for the Diagnosis and Prognosis of Acute Myeloid Leukemia

Patent Number: 8,354,229

Filed: Oct. 7, 2011

Lead Inventor: Carlo Croce, Ohio State University

The invention provides “novel methods and compositions for the diagnosis, prognosis and treatment of acute myeloid leukemia,” the patent's abstract states. “The invention also provides methods of identifying anti-AML agents.”


Title: Anti-Aging microRNAs

Patent Number: 8,354,384

Filed: June 23, 2006

Lead Inventor: Frank Slack, Yale University

“Methods and compositions for modulating aging genes or their targets for the treatment or prevention of senescence or symptoms thereof have been developed based on the discovery of naturally occurring inhibitory nucleic acids, in particular lin-4 miRNA, that down-regulate genes involved in senescence, lifespan, or age-related disorders,” the patent's abstract states. “Representative aging genes include, but are not limited to lin-4, lin-14, let-7, lin-28, egl-35, and lin-42. Methods for identifying modulators of aging genes and targets of aging genes are also provided. The disclosed compositions are useful as diagnostics. These can be used in assays to compare genes in normal individuals, with those who are aging well or who demonstrate early senescence, and with those who have age-related disorders such as Parkinson's and Alzheimer's [diseases]. The genes can be used to study the pathways and mechanisms involved in aging and age-related disorders. These genes can be used as drug targets, or in drug design, to develop drugs that can inhibit one or more characteristics of senescence or age-related disorders. These compositions should be effective therapies for treating or slowing the effects of one or more symptoms or characteristics of age-related disorders resulting from activation or over-expression of aging genes. Compositions that alter the expression of particular aging genes affecting the insulin-like signal pathway are described. Suitable compositions described herein include, inhibitory nucleic acids and small molecules, in particular miRNA.”


Title: Methods and Compositions for Delivering siRNA in Mammalian Cells

Patent Number: 8,354,387

Filed: Feb. 23, 2006

Lead Inventor: Gilles Divita, French National Center for Scientific Research

The invention comprises a complex comprised of a peptide carrier and an siRNA, the patent's abstract states.


Title: miRNA-Regulated Differentiation-Dependent Self-Deleting Cassette

Patent Number: 8,354,389

Filed: Aug. 13, 2010

Lead Inventor: David Frendewey, Regeneron Pharmaceuticals

The patent, its abstract states, claims “targeting constructs and methods of using them … for differentiation-dependent modification of nucleic acid sequences in cells and in non-human animals. Targeting constructs comprising a promoter operably linked to a recombinase are provided, wherein the promoter drives transcription of the recombinase in a differentiated cell but not an undifferentiated cell. Promoters include Blimp1, Prm1, Gata6, Gata4, Igf2, Lhx2, Lhx5, and Pax3. Targeting constructs with a cassette flanked on both sides by recombinase sites can be removed using a recombinase gene operably linked to a 3'-UTR that comprises a recognition site for an miRNA that is transcribed in undifferentiated cells but not in differentiated cells. The constructs may be included in targeting vectors, and can be used to automatically modify or excise a selection cassette from an ES cell, a non-human embryo, or a non-human animal.”


Title: Compositions and Methods for Inhibiting Expression of a Gene from the Ebola Virus

Patent Number: 8,354,390

Filed: April 25, 2011

Lead Inventor: Sina Bavari, Alnylam Pharmaceuticals

“The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of a gene from the Ebola virus,” the patent's abstract states.


Title: Inactivated Microorganisms Containing Double-Stranded RNA Molecules, Their Use as Pesticides, and Methods for Their Preparation

Application Number: 20130011372

Filed: Sept. 17, 2010

Lead Inventor: Assunta Tortora, Arterra Bioscience

The invention relates to “a new group of pesticides consisting of inactivated microorganisms containing double-strand RNA molecules corresponding to receptor genes coupled with G proteins whose functioning is vital for phytophagous invertebrates or for infesting or in any case harmful organisms for the health of human beings and domestic animals, and to a method for the preparation of said microorganisms,” according to the patent application's abstract.


Title: Targeted Delivery of siRNA

Application Number: 20130011441

Filed: Jan. 6, 2012

Lead Inventor: Jeffrey Hollinger, Carnegie Mellon University

The invention, the patent application's abstract states, relates to “nano-structured bioconjugates and nano-structured network hydrogels used to deliver nucleic acids to targeted biological locations. The ... invention further relates to methods of treating clinical conditions using the nano-structured bioconjugates and nano-structured network hydrogels.”


Title: Methods, Compositions, and Kits Comprising Linker Probes for Quantifying Polynucleotides

Application Number: 20130011840

Filed: Sept. 12, 2012

Lead Inventor: Caifu Chen, Life Technologies

The invention, the patent application's abstract states, is “directed to methods, reagents, kits, and compositions for identifying and quantifying target polynucleotide sequences. A linker probe comprising a 3' target specific portion, a loop, and a stem is hybridized to a target polynucleotide and extended to form a reaction product that includes a reverse primer portion and the stem nucleotides. A detector probe, a specific forward primer, and a reverse primer can be employed in an amplification reaction wherein the detector probe can detect the amplified target polynucleotide based on the stem nucleotides introduced by the linker probe. In some embodiments a plurality of short [microRNAs] are queried with a plurality of linker probes, wherein the linker probes all comprise a universal reverse primer portion, a different 3' target specific portion, and different stems. The plurality of queried miRNAs can then be decoded in a plurality of amplification reactions.”


Title: Methods for Reducing Protein Levels in a Cell

Application Number: 20130011920

Filed: Jan. 17, 2011

Inventor: Pengbo Zhou, Cornell University

The invention, the patent application's abstract states, “provides a method of reducing levels of at least one target protein in a cell. The cell is contacted with a first agent … [that] reduces synthesis of the target protein … [and a] second agent [that] accelerates degradation of the target protein. The first agent may contact the cell before, after, or simultaneously with the second agent. The first agent and the second agent may be in separate delivery vehicles, or in a single delivery vehicle. The first agent may be an RNAi molecule … [and] the second agent may be a chimeric polypeptide containing a ubiquitin ligase polypeptide and a target protein interacting domain. The ubiquitin ligase polypeptide can be an E3 ubiquitin ligase, including, but not limited to, an SCF polypeptide, a HECT polypeptide and a UBR1 polypeptide.”


Title: Nucleic Acid Compounds for Inhibiting Gene Expression and Uses Thereof

Application Number: 20130011922

Filed: Dec. 15, 2011

Lead Inventor: Steven Quay, Marina Biotech

The invention, the patent application's abstract states, relates to “RNA molecules, for example, meroduplex ribonucleic acid molecules and blunt ended double-stranded ribonucleic acid molecules, capable of decreasing or silencing expression of a target gene. ... Also provided are methods of decreasing expression of a target gene in a cell or in a subject to treat a disease or condition associated with the target gene.”


Title: Marker for Diagnosis of Breast Cancer, Test Method, and Test Kit

Application Number: 20130012412

Filed: Sept. 14, 2012

Lead Inventor: Masahiko Kuroda, Miracure

The invention comprises a “marker, a test method, and a test kit which can detect the onset of breast cancer that cannot be detected by palpation or mammography examination or breast cancer in an early stage,” the patent application's abstract states. The marker is a microRNA found in serum or plasma at a “significantly reduced level after the onset of breast cancer, or during or after an early stage of breast cancer.”


Title: RNA Interference-Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid

Application Number: 20130012567

Filed: May 15, 2012

Lead Inventor: James McSwiggen, Merck

The invention concerns “methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications,” according to the patent application's abstract. “Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules … capable of mediating RNA interference against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.”


Title: GNAQ-Targeted dsRNA Compositions and Methods for Inhibiting Expression

Application Number: 20130012570

Filed: Sept. 13, 2012

Lead Inventor: Jared Gollob, Alnylam Pharmaceuticals

“The invention relates to a double-stranded ribonucleic acid targeting a G-alpha q subunit of a heterotrimeric G gene, and methods of using the dsRNA to inhibit expression of GNAQ,” according to the patent application's abstract.


Title: Organic Compositions to Treat Beta-ENaC-Related Diseases

Application Number: 20130012571

Filed: Sept. 13, 2012

Lead Inventor: Antonin de Fougerolles, Alnylam Pharmaceuticals (Novartis)

The invention relates to “RNAi agents useful in methods of treating Beta-ENaC-related diseases such as cystic fibrosis, pseudohypoaldosteronism type 1, Liddle's syndrome, hypertension, alkalosis, hypokalemia, and obesity-associated hypertension, using a therapeutically effective amount of a RNAi agent to Beta-ENaC,” the patent application's abstract states.


Title: Compositions and Methods for Inhibiting Expression of GSK-3 Genes

Application Number: 20130012572

Filed: Sept. 13, 2012

Lead Inventor: Dinah Sah, Alnylam Pharmaceuticals

“The invention relates to a double-stranded ribonucleic acid targeting glycogen synthase kinase-3 and methods of using the dsRNA to inhibit expression of GSK-3,” according to the patent application's abstract.

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.