Skip to main content
Premium Trial:

Request an Annual Quote

IP Update: Dec 2, 2010

Premium

Title: RNAi Inhibition of CTGF for Treatment of Ocular Disorders

Patent Number: 7,838,507

Filed: Oct. 12, 2009

Lead Inventor: Allan Shepard, Alcon

The invention, the patent's abstract states, comprises the use of RNAi "for inhibition of connective tissue growth factor mRNA expression in ocular disorders involving CTGF expression. Ocular disorders involving aberrant CTGF expression include glaucoma, macular degeneration, diabetic retinopathy, choroidal neovascularization, proliferative vitreoretinopathy and wound healing. Such disorders are treated by administering interfering RNAs."


Title: siRNA Silencing of Filovirus Gene Expression

Patent Number: 7,838,658

Filed: Oct. 20, 2006

Lead Inventor: Ian MacLachlan, Protiva Biotherapeutics (Tekmira Pharmaceuticals)

The invention, the patent's abstract states, "provides siRNA molecules that target filovirus gene expression and methods of using such siRNA molecules to silence filovirus gene expression. The … invention also provides nucleic acid-lipid particles that target filovirus gene expression comprising an siRNA that silences filovirus gene expression, a cationic lipid, and a non-cationic lipid."


Titles: microRNA Molecules

Patent Numbers: 7,838,660, 7,838,661, 7,838,662, 7,838,663, 7,838,664

Filed: Aug. 31, 2009

Lead Inventor: Thomas Tuschl, Max Planck Institute

"In Caenorhabditis elegans, lin-4 and let-7 encode 22- and 21-nucleotide RNAs, respectively, that function as key regulators of developmental timing," the patents' abstracts state. "Because the appearance of these short RNAs is regulated during development, they are also referred to as small temporal RNAs. We show that many more 21- and 22-nt expressed RNAs, termed microRNAs, exist in invertebrates and vertebrates, and that some of these novel RNAs, similar to let-7 stRAN, are also highly conserved. This suggests that sequence-specific post-transcriptional regulatory mechanisms mediated by small RNAs are more general than previously appreciated."


Title: Delivery of DNA or RNA Via Gap Junctions from Host Cells to Target Cells and a Cell-Based Delivery System for Antisense or siRNA

Patent Number: 7,842,673

Filed: Dec. 17, 2004 PCT Filed: Dec. 17, 2004

Lead Inventor: Peter Brink, Columbia University

The invention, the patent's abstract states, comprises "a method of delivering an oligonucleotide or a plasmid expressing an oligonucleotide into a target cell comprises introducing an oligonucleotide into a donor cell, particularly a stem cell, and contacting the target cell with the donor cell under conditions permitting the donor cell to form a gap junction with the target cell, whereby the oligonucleotide or a product of the oligonucleotide is delivered into the target cell from the donor cell."


Title: Methods of Treatment of Acute Renal Failure

Patent Number: 7,842,674

Filed: July 10, 2007

Lead Inventor: Elena Feinstein, Quark Pharmaceuticals

"The invention relates to a double-stranded compound, preferably an oligoribonucleotide, which down-regulates the expression of a human p53 gene," the patent's abstract states. "The invention also relates to a pharmaceutical composition comprising the compound, or a vector capable of expressing the oligoribonucleotide compound, and a pharmaceutically acceptable carrier … [and] contemplates a method of treating a patient suffering from alopecia or acute renal failure or other diseases comprising administering to the patient the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the patient. The alopecia may be induced by chemotherapy or radiotherapy, and the patient may be suffering from cancer, in particular breast cancer."


Title: Bioinformatically Detectable Group of Novel Regulatory Bacterial and Bacterial-Associated Oligonucleotides and Uses Thereof

Patent Number: 7,842,800

Filed: April 2, 2004

Inventor: Isaac Bentwich, Rosetta Genomics

The invention, the patent's abstract states, "relates to a first group of novel bacterial and human associated oligonucleotides, here identified as genomic address messenger … [oligonucleotides], and a second group of novel operon-like bacterial and human polynucleotides, here identified as genomic record … [polynucleotides]. GAM oligonucleotides selectively inhibit translation of known target genes, many of which are known to be involved in various bacterial diseases. Nucleic acid molecules are provided respectively encoding 6,444 GAM precursors oligonucleotides, and 726 GR polynucleotides, as are vectors and probes both comprising the nucleic acid molecules, and methods and systems for detecting GAM oligonucleotides and GR polynucleotides and specific functions and utilities thereof, for detecting expression of GAM oligonucleotides and GR polynucleotides, and for selectively enhancing and selectively inhibiting translation of the respective target genes thereof."


Title: Compositions and Methods for Inhibiting Expression of a Mutant Gene

Application Number: 20100291194

Filed: June 16, 2010

Lead Inventor: Roland Kreutzer, Alnylam Pharmaceuticals

The invention, the patent application's abstract states, "relates to a double-stranded ribonucleic acid for inhibiting the expression of a mutant gene, comprising a complementary RNA strand having a complementary region that is substantially complementary to a portion of the mutant gene, and which is partially complementary to the corresponding wild-type gene. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier.

"The pharmaceutical compositions are useful for inhibiting the expression of a target mutant gene, as well as for treating diseases caused by expression of the target gene," the abstract adds. "The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene."


Title: Alternative Export Pathways for Vector-Expressed RNA Interference

Application Number: 20100291673

Filed: Nov. 28, 2007 PCT Filed: Nov. 28, 2007

Lead Inventor: Scott Harper, University of Iowa

The invention comprises "nucleic acid molecules containing a loop sequence designed to circumvent exportin-5 mediated export, and methods using these novel molecules," according to the patent application's abstract.


Title: Methods and Compositions for Selecting siRNA of Improved Functionality

Application Number: 20100291681

Filed: June 11, 2010

Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)

"Efficient sequence-specific gene silencing is possible through the use of siRNA technology," the patent application's abstract states. "By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed."


Title: Targeting of RNA with External Guide Sequences

Application Number: 20100292099

Filed: Aug. 22, 2008 PCT Filed: Aug. 22, 2008

Lead Inventor: David Dreyfus, Keren Pharmaceutical

The invention comprises "compositions and methods for modulating gene expression by using EGS to target miRNA," the patent application's abstract states. Another aspect of the invention
"is the use of EGS to target mitochondrial RNA. MiRNA targets may include immature or mature forms of miRNA, such as miRNA over-expressed in diseases such as cancer."


Title: microRNAs That Regulate Muscle Cell Proliferation and Differentiation

Application Number: 20100292297

Filed: Dec. 12, 2006 PCT Filed: Dec. 12, 2006

Lead Inventor: Da-Zhi Wang, University of North Carolina, Chapel Hill

The invention comprises "methods and compositions for modulating gene expression in myocytes," the patent application's abstract states.


Title: Novel siRNA Structures

Application Number: 20100292301

Filed: Feb. 28, 2008

Lead Inventor: Elena Feinstein, Quark Pharmaceuticals

The invention comprises "novel compounds, compositions, methods, and uses for treating microvascular disorders, eye diseases, and respiratory conditions based upon inhibition of a target gene," the patent application's abstract states. "More specifically, the … invention relates to positional motifs of modified ribonucleotides useful in the design of siRNA compounds. In particular, the ribonucleotides include modified internucleotide linkages and/or modified sugar moieties. These novel siRNA compounds may be used therapeutically to treat a variety of diseases and indications."


Title: RNAi Modulation of HIF-1 and Therapeutic Uses Thereof

Application Number: 20100292305

Filed: April 2, 2010

Lead Inventor: Akin Akinc, Alnylam Pharmaceuticals

The invention relates to "compounds, compositions, and methods useful for modulating the expression of HIF-1 alpha, such as by the mechanism of RNA interference," the patent application's abstract states. "The compounds and compositions include iRNA agents that can be unmodified or chemically modified."


Title: microRNA Molecules

Application Number: 20100292308

Filed: Jan. 23, 2009

Lead Inventor: Thomas Tuschl, Max Planck Institute

"In Caenorhabditis elegans, lin-4 and let-7 encode 22- and 21-nucleotide RNAs, respectively, that function as key regulators of developmental timing," the patent application's abstract states. "Because the appearance of these short RNAs is regulated during development, they are also referred to as small temporal RNAs. We show that many more 21- and 22-nt expressed RNAs, termed microRNAs, exist in invertebrates and vertebrates, and that some of these novel RNAs, similar to let-7 stRAN, are also highly conserved. This suggests that sequence-specific post-transcriptional regulatory mechanisms mediated by small RNAs are more general than previously appreciated."


Title: microRNA Scaffolds and Non-Naturally Occurring microRNAs

Application Number: 20100292310

Filed: May 22, 2008 PCT Filed: May 22, 2008

Lead Inventor: Melissa Kelley, Dharmacon (Thermo Fisher Scientific)

The invention comprises "a non-naturally occurring miRNA having a stem-loop structure comprising a scaffold derived from a first endogenous miRNA, a mature strand derived from a second endogenous miRNA, and a star strand sequence that is at least partially complementary to the mature strand sequence," the patent application's abstract states. The invention "also provides a non-naturally occurring miRNA having a stem-loop structure comprising a scaffold derived from an endogenous miRNA, a mature strand designed to be at least partially complementary to a target RNA, and a star strand sequence that is at least partially complementary to the mature strand sequence. The methods and compositions of the disclosure may be used to mediate gene silencing via the RNAi pathway."


Title: Modified iRNA Agents

Application Number: 20100292455

Filed: Feb. 26, 2010

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

"The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose," the patent application's abstract states. "The inclusion of such a monomer can allow for modulation of a property of the iRNA agent into which it is incorporated, e.g., by using the non-ribose moiety as a point to which a ligand or other entity, e.g., a lipophilic moiety. e.g., cholesterol, is directly, or indirectly, tethered. The invention also relates to methods of making and using such modified iRNA agents."


Title: RNA Interference-Mediating Small RNA Molecules

Application Number: 20100292456

Filed: June 4, 2010

Lead Inventor: Thomas Tuschl, Max Planck Institute

"Double-stranded RNA induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference," according to the patent application's abstract. "Using a Drosophila in vitro system, we demonstrate that [19 to 23 nucleotide-long] short RNA fragments are the sequence-specific mediators of RNAI. The short interfering RNAs are generated by an RNase III-like processing reaction from long dsRNA. Chemically synthesized siRNA duplexes with overhanging 3' ends mediate efficient target RNA cleavage in the lysate, and the cleavage site is located near the center of the region spanned by the guiding siRNA. Furthermore, we provide evidence that the direction of dsRNA processing determines whether sense or antisense target RNA can be cleaved by the produced siRNP complex."


Title: Tumor-Suppressor Gene Screening Using RNA Interference Libraries and Method of Treatment

Application Number: 20100297010

Filed: May 16, 2008 PCT Filed: May 16, 2008

Lead Inventor: Anka Bric, Cold Spring Harbor Laboratory

The invention, the patent application's abstract states, "is directed to methods of identifying tumor-suppressor genes in vivo, tumor suppressors thus found, methods of treatment taking advantage of the identified tumor suppressors, methods of and kits for diagnosis of cancer using the identified tumor suppressor, and pharmaceutical composition comprising an identified tumor suppressor or modulators thereof."


Title: Means for Delivery of Nucleic Acids Active for Gene Silencing Using Synthetic Polymers

Application Number: 20100297756

Filed: Dec. 12, 2008 PCT Filed: Dec. 12, 2008

Lead Inventor: Abdennajj Adib

"The invention relates to a composition useful as transfection agent, comprising polyamines modified by aromatic amino acids and small double-strand or single-strand RNA active for RNA interference," the patent application's abstract states.


Title: Oligonucleotide Compositions with Enhanced Efficiency

Application Number: 20100298408

Filed: Dec. 3, 2009

Lead Inventor: Tod Woolf, Life Technologies

The oligonucleotide compositions of the invention, the patent application's abstract states, "make use of combinations of oligonucleotides. In one aspect, the invention features an oligonucleotide composition including at least [two] different oligonucleotides targeted to a target gene. This invention also provides methods of inhibiting protein synthesis in a cell and methods of identifying oligonucleotide compositions that inhibit synthesis of a protein in a cell."


Title: Compositions and Methods for Inhibiting Expression of Huntingtin Gene

Application Number: 20100298405

Filed: April 2, 2009

Lead Inventor: Dinah Sah, Alnylam Pharmaceuticals

"The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of the huntingtin gene comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the … gene," the patent application's abstract states. "The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the [huntingtin] gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell."


Title: Compositions Comprising STAT3 siRNA and Methods of Use Thereof

Application Number: 20100298409

Filed: Sept. 17, 2008 PCT Filed: Sept. 17, 2008

Lead Inventor: Frank Xie, Intradigm

The invention comprises "nucleic acid molecules that inhibit STAT3 expression," the patent application's abstract states. "Methods of using the nucleic acid molecules are also provided."


Title: Lipid-Modified Double-Stranded RNA Having Potent RNA Interference Effect

Application Number: 20100298411

Filed: Oct. 24, 2008 PCT Filed: Oct. 24, 2008

Lead Inventor: Takanori Kubo, Otsuka Pharmaceutical

The invention comprises "a novel double-stranded RNA that has high nuclease resistance and high cellular uptake efficiency, and that is capable of producing an excellent RNA interference effect," according to the patent application's abstract. The invention also provides "a lipid-modified double-stranded RNA comprising a sense strand having a nucleotide sequence complementary to a target sequence, and an antisense strand having a nucleotide sequence complementary to the sense strand, the double-stranded RNA being capable of inhibiting the expression of the target gene, the sense strand having a lipid linked to at least one of the first to sixth nucleotides from the 5' end side directly or via a linker."


Title: Preventing Hyaluronan-Mediated Tumorigenetic Mechanisms Using Intronic RNAs

Application Number: 20100298416

Filed: Oct. 29, 2008 PCT Filed: Oct. 29, 2008

Lead Inventor: Shao-Yao Ying, University of Southern California

"Patterns of microRNA expression are correlated to the degrees of tumor cell differentiation in human prostate cancer," the patent application's abstract states. "MiRNAs can complementarily bind to either oncogenes or tumor-suppressor genes, resulting in targeted gene silencing and thus changes of cellular tumorigenecity.

"Patterns of microRNA expression are correlated to the degrees of tumor cell differentiation in human prostate cancer," the patent application's abstract states. "MiRNAs can complementarily bind to either oncogenes or tumor-suppressor genes, resulting in targeted gene silencing and thus changes of cellular tumorigenicity.

"Using miRNA microarray analysis, [eight] down-regulated and [three] up-regulated known miRNAs in androgen-independent human prostate cancer cell lines, such as LNCaP C4-2B and PC3, compared to those androgen-dependent cell lines, such as LNCaP and PC3-AR9, were consistently detected," it adds. "Fluorescent in situ hybridization assays in human prostate cancer tissue arrays containing sixty patients at different stages also showed the same miRNA expression patterns in hormone-refractory prostate carcinomas compared to androgen-sensitive non-cancerous prostate epithelium. In vitro tumorigenicity assays using one of the identified miRNAs, mir-146a, were performed to provide validation of its function in prostate cancer. Gain-of-function transfection of mir-146a markedly suppressed its targeted ROCK1 gene expression in androgen-independent PC3 cells, consequently resulting in reduced cancer cell proliferation, invasion, and metastasis to human bone marrow endothelial cell monolayers. Since ROCK1 is the key kinase for activating hyaluronan-mediated HRPC transformation in vivo and in PC3 cells, mir-146a should function as a tumor-suppressor gene in modulating the ROCK1-associated tumorigenicity."


Titles: Method and Devices for Improved Efficiency of RNA Delivery to Cells

Application Numbers: 20100298697, 20100298762

Filed: May 18, 19 2010

Lead Inventor: Deepak Ramesh Thakker, Medtronic

The invention provides "a method for improving efficiency of RNA delivery to cells," the patent applications' abstracts state. "The method comprises applying a low-strength electric field to the cells and then after a certain time period, administering the ribonucleic acid sequence to the cells. Devices, kits, and RNA molecules suitable for delivery and devices suitable for practicing the disclosed methods are also provided."


Title: Reversible siRNA-Based Silencing of Mutant and Endogenous Wild-Type Huntingtin Gene and Its Application for the Treatment of Huntington's Disease

Application Number: 20100299768

Filed: June 18, 2008 PCT Filed: June 18, 2008

Lead Inventor: Valerie Perrin, Atomic and Alternative Energies Commission

The invention, the patent application's abstract states, comprises "isolated double-stranded short interfering nucleic acid molecules inhibiting the expression of endogenous wild-type and exogenous human mutant huntintin genes in cells of a non-human mammal [that] are expressing both … huntingtin genes, and their application for the treatment of Huntington's disease, as well as to study Huntington's disease in rodent models."


Title: Means and Methods for shRNA-Mediated Conditional Knockdown of Genes

Application Number: 20100299771

Filed: Sept. 17, 2008 PCT Filed: Sept. 17, 2008

Lead Inventor: Ralf Kuhn, German Research Center for Environmental Health

The invention, the patent application's abstract states, relates to a method of transcribing an shRNA molecule in a cell.

"Further, the invention relates to a genetically engineered non-human animal and a method to produce said transgenic non-human animal … [as well as] a cell genetically engineered with the DNA molecule of the invention and a method of simultaneously knocking down two genes in a cell," the abstract states. Also claimed is a "method of identifying a combination of two target genes as a potential drug target and the use of the DNA molecule of the invention for the preparation of a composition for gene therapy."

The Scan

WHO Seeks Booster Pause

According to CNN, the World Health Organization is calling for a moratorium on administering SARS-CoV-2 vaccine boosters until more of the world has received initial doses.

For Those Long Legs

With its genome sequence and subsequent RNAi analyses, researchers have examined the genes that give long legs to daddy longlegs, New Scientist says.

September Plans

The New York Times reports that the US Food and Drug Administration is aiming for early September for full approval of the Pfizer-BioNTech SARS-CoV-2 vaccine.

Nucleic Acids Research Papers on Targeting DNA Damage Response, TSMiner, VarSAn

In Nucleic Acids Research this week: genetic changes affecting DNA damage response inhibitor response, "time-series miner" approach, and more.