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IP Update: Dec 3, 2009

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Title: RNAi Inhibition of CTGF for Treatment of Ocular Disorders

Number: 7,622,454

Filed: Dec. 19, 2005

Lead Inventor: Allan Shepard, Alcon

"RNA interference is provided for inhibition of connective tissue growth factor mRNA expression in ocular disorders involving CTGF expression," the patent's abstract states. "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 of the … invention."


Title: Genetic Inhibition by Double-Stranded RNA

Number: 7,622,633

Filed: Oct. 30, 2002

Lead Inventor: Andrew Fire, Carnegie Institution

"A process is provided of introducing an RNA into a living cell to inhibit gene expression of a target gene in that cell," the patent's abstract states. "The process may be practiced ex vivo or in vivo.

"The RNA has a region with double-stranded structure," the abstract notes. "Inhibition is sequence-specific in that the nucleotide sequences of the duplex region of the RNA and of a portion of the target gene are identical. The … invention is distinguished from prior art interference in gene expression by antisense or triple-strand methods."


Title: Compositions for DNA-Mediated Gene Silencing

Number: 7,625,750

Filed: Dec. 26, 2002

Lead Inventor: Jiwu Wang, Allele Biotechnology & Pharmaceuticals

The invention, the patent's abstract states, comprises "DNA compositions that mediate gene silencing via RNA interference. … The DNA compositions include an RNA polymerase III promoter, which drives expression of a nucleotide sequence encoding an intermediate small interfering RNA molecule, and RNA polymerase III terminator."


Title: Compositions and Methods for Treating Diseases Associated with T-box and N-Myc

Number: 7,625,874

Filed: April 11, 2007

Lead Inventor: Sylvia Evans, University of California, Oakland

"The invention … provides a mode of treating a disease associated with the regulation of [the] T-Box and N-Myc [genes], which includes cancers and heart disease in human and other subjects, by identifying and administering a compound which modulates T-Box or N-Myc function," the patent's abstract states. "The invention also provides polynucleotides, polypeptides, vectors, cells, tissues, and organisms useful in the identification and treatment of metabolic syndrome. A number of desirable cell proliferation and senescence regulating aspects are achieved by various embodiments of the … invention."

The patent specifically claims the use of siRNAs against a TBX2 gene.


Title: Single-Stranded and Double-Stranded Oligonucleotides Comprising a 2-Arylpropyl Moiety

Number: 7,626,014

Filed: April 27, 2005

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

The invention "provides single-stranded and double-stranded oligonucleotides comprising at least one aralkyl ligand that improves the pharmacokinetic properties of the oligonucleotides," the patent's abstract states. "The aralkyl ligands of the … invention include naproxen, ibuprofen, and derivatives thereof. The … invention also provides methods for modulating gene expression using the modified oligonucleotide compounds and compositions comprising those modified oligonucleotides."


Title: Therapeutic Uses of Inhibitors of RTP801L

Number: 7,626,015

Filed: June 8, 2007

Lead Inventor: Elena Feinstein, Quark Pharmaceuticals

The invention "provides novel molecules, compositions, methods, and uses for treating microvascular disorders, eye diseases, respiratory conditions, and hearing disorders based upon inhibition of the RTP801L gene and/or protein," the patent's abstract states.


Title: Compositions and Methods Using RNA Interference for Control of Nematodes

Number: 7,622,301

Filed: Feb. 22, 2005

Lead Inventor: Peifeng Ren, BASF Plant Science

The invention "concerns double-stranded RNA compositions and transgenic plants capable of inhibiting expression of essential genes in parasitic nematodes, and methods associated therewith," the patent's abstract states.


Title: dsRNA as Insect Control Agent

Number: 20090285784

Filed:
Jan. 12, 2007 PCT Filed: Jan. 12, 2007

Lead Inventor: Romaan Raemaekers, Devgen

The invention "concerns methods for controlling insect infestation via RNAi-mediated gene silencing, whereby the intact insect cells are contacted with a double-stranded RNA from outside the insect cells, and whereby the double-stranded RNA is taken up by the intact insect cells," the patent application's abstract states. "In one particular embodiment, the methods of the invention are used to alleviate plants from insect pests. Alternatively, the methods are used for treating and/or preventing insect infestation on a substrate or a subject in need of such treatment and/or prevention. Suitable insect target genes and fragments thereof, dsRNA constructs, recombinant constructs, and compositions are disclosed."


Title: Random RNAi Libraries, Methods of Generating Same, and Screening Methods Utilizing Same

Number: 20090285788

Filed: March 6, 2007 PCT Filed: March 6, 2007

Lead Inventor: Robert Wilson, University of Pennsylvania

"This invention provides expression vectors for a ribonucleic acid molecule comprising a double-stranded region of random sequence, sets and libraries of same, methods of generating same, and methods for identifying an RNA therapeutic or RNA molecule that has an ability to affect a biological parameter, for identifying a drug target for a disease or disorder of interest, and for identifying a variant of an RNA molecule that has an altered ability to affect a biological parameter of interest," according to the patent application's abstract.


Title: Cationic Lipids and Uses Thereof

Number: 20090285881

Filed: April 16, 2009

Lead Inventor: Prasad Dande, Abbott Laboratories

The invention, the patent application's abstract states, comprises "cationic lipids, cationic lipid-based drug delivery systems, ways to make them, and methods of treating diseases using them."


Title: Identification of Gastrointestinal, Pancreatic, and Cancer Stem Cell Markers and Methods of Use Thereof

Number: 20090285883

Filed: May 15, 2009

Lead Inventor: Courtney Houchen, University of Oklahoma

"DCAMKL-1 has been identified as a biomarker for stem cells, as well as cancer stem cells," according to the patent application's abstract. "Methods of detecting the presence of at least one stem cell, methods of isolating stem cells, and methods of inhibiting growth of cancer cells utilizing DCAMKL-1 are disclosed herein."

The application specifically claims the use of siRNAs to silence DCAMKL-1.


Title: microRNA Expression Profiling and Uses Thereof

Number: 20090286242

Filed: Dec. 10, 2008

Lead Inventor: Greg Hannon, Cold Spring Harbor Laboratory

The invention, the patent application's abstract states, comprises "methods and reagents for obtaining microRNA expression profiles in selected cell populations or sub-populations, such as stem cell or progenitor cell populations, and using such microRNA expression profiles for cell characterization, isolation/purification, and/or reinforcement of cell fate specification, both in research and development, and in therapeutic applications. Also provided are methods of identifying and isolating mammary progenitor cells using miRNA sensor constructs."


Title: Methods and Compositions for Use in Preparing siRNAs

Number: 20090286287

Filed: June 3, 2009

Lead Inventor: Jason Myers, Stanford University

The patent application, its abstract states, claims "methods and compositions for producing siRNAs … from dsRNAs. … 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] 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. Also provided are kits for use in practicing the subject invention."


Title: Novel Oligonucleotide Compositions and Probe Sequences Useful for Detection and Analysis of microRNAs and Their Target mRNAs

Number: 20090286753

Filed: April 4, 2008

Lead Inventor: Sakari Kauppinen, Exiqon

"The invention relates to ribonucleic acids and oligonucleotide probes useful for detection and analysis of microRNAs and their target mRNAs, as well as small interfering RNAs," according to the patent application's abstract.


Title: Compositions and Methods for Delivering RNAi Using Lipoproteins

Number: 20090286851

Filed: April 29, 2009

Lead Inventor: Akin Akinc, Alnylam Pharmaceuticals

"This invention relates to new compositions comprising at least one of a single- or double-stranded oligonucleotide, where said oligonucleotide has been conjugated to a lipophile and to which the conjugated oligonucleotide has been preassembled with lipoproteins," the patent application's abstract states. "These compositions are effective in delivering oligonucleotides to mammalian tissue where they effect gene silencing."


Title: RNA Containing Modified Nucleosides and Methods of Use Thereof

Number: 20090286852

Filed: Aug. 21, 2006 PCT Filed: Aug. 21, 2006

Lead Inventor: Katalin Kariko, University of Pennsylvania

"This invention provides RNA, oligoribonucleotide, and polyribonucleotide molecules comprising pseudouridine or a modified nucleoside, gene-therapy vectors comprising same, methods of synthesizing same, and methods for gene replacement, gene therapy, gene-transcription silencing, and the delivery of therapeutic proteins to tissue in vivo, comprising the molecules," according to the patent application's abstract. The invention also "provides methods of reducing the immunogenicity of RNA, oligoribonucleotide, and polyribonucleotide molecules."


Titles: Oligomeric Compounds and Compositions for Use in Modulation of Small Non-Coding RNAs

Numbers: 20090286969, 20090291906, 20090291907

Filed: Dec. 30, 2008

Lead Inventor: Christine Esau, Regulus Therapeutics

The patent applications, according to their abstracts, claim "compounds, compositions, and methods … for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds, targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs are also provided."


Title: Silencing of Polo-Like Kinase Expression Using Interfering RNA

Number: 20090291131

Filed: Dec. 23, 2008

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

The invention "provides compositions comprising interfering RNA that target polo-like kinase 1 expression and methods of using such compositions to silence PLK-1 expression," the patent application's abstract states. "More particularly, the … invention provides unmodified and chemically modified interfering RNA molecules which silence PLK-1 expression and methods of use thereof."

The invention also provides "serum-stable nucleic acid-lipid particles comprising an interfering RNA molecule described herein, a cationic lipid, and a non-cationic lipid, which can further comprise a conjugated lipid that inhibits aggregation of particles," the abstract notes. It also provides "methods of silencing PLK-1 gene expression by administering an interfering RNA molecule described herein to a mammalian subject … [and] methods of identifying and/or modifying PLK-1 interfering RNA having immunostimulatory properties. Methods for sensitizing a cell such as a cancer cell to the effects of a chemotherapy drug comprising sequentially delivering PLK-1 interfering RNA followed by the chemotherapy drug are also provided."


Title: siRNA Targeting Transducin Beta-Like 3

Number: 20090291497

Filed: July 1, 2009

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 including those directed to nucleotide sequences for TBL3."


Title: Improved Methods Controlling Gene Expression

Number: 20090293148

Filed: April 13, 2006 PCT Filed: April 13, 2006

Lead Inventor: Peifeng Ren, BASF Plant Science

The invention "provides agents capable of controlling gene expression … [specifically] sequences of naturally occurring, tissue-specifically expressed microRNAs," the patent application's abstract states. "The invention further provides for transgenic expression constructs comprising sequences encoding said microRNAs. By incorporation of the microRNA encoding sequence, the expression from said expression construct is specifically silenced in the tissue where the naturally occurring microRNA is naturally expressed. Thereby, the expression profile resulting from the promoter is modulated and leakiness is reduced.

"The invention further provides for a method for modulating transgenic expression by incorporating sequences encoding said microRNAs into transgenic expression constructs," the abstract adds. "The compositions and methods of the invention can be used to enhance performance of agricultural relevant crops and for therapy, prophylaxis, research and diagnostics in diseases and disorders [that] afflict mammalian species."

The Scan

For Better Odds

Bloomberg reports that a child has been born following polygenic risk score screening as an embryo.

Booster Decision Expected

The New York Times reports the US Food and Drug Administration is expected to authorize a booster dose of the Pfizer-BioNTech SARS-CoV-2 vaccine this week for individuals over 65 or at high risk.

Snipping HIV Out

The Philadelphia Inquirer reports Temple University researchers are to test a gene-editing approach for treating HIV.

PLOS Papers on Cancer Risk Scores, Typhoid Fever in Colombia, Streptococcus Protection

In PLOS this week: application of cancer polygenic risk scores across ancestries, genetic diversity of typhoid fever-causing Salmonella, and more.