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IP Update: Jul 29, 2010

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

Patent Number: 7,763,590

Filed: March 7, 2003

Lead Inventor: Roland Kreutzer, Alnylam Pharmaceuticals

The invention, the patent'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: Small Interference RNA Gene Therapy

Patent Number: 7,763,722

Filed: Jan. 15, 2004 PCT Filed: Jan. 15, 2004

Lead Inventor: Lung-Ji Chang, University of Florida

"Gene expression is inhibited in a cell by introducing into the cell a lentiviral vector encoding an siRNA specific for the gene," the patent's abstract states. "Lentiviral vectors encoding siRNA specific for a cancer-associated gene inhibited expression of the gene and caused cell death after being introduced into cancer cells. Viral replication in HIV-infected cells was inhibited after introducing a lentiviral vector encoding siRNA specific for HIV genes into the cells."


Title: dsRNA for Treating Viral Infection

Application Number: 20100183704

Filed: Sept. 18, 2009

Lead Inventor: Jason Borawski, Novartis

"The invention relates to double-stranded ribonucleic acids targeting gene expression carbamoyl-phosphate synthetase 2, aspartate transcarbamylase, and dihydroorotase, and their use for treating infection by positive-stranded RNA viruses such as hepatitis C virus," the patent application's abstract states. "Each dsRNA comprises an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19 [to] 25 nucleotides in length, and which is substantially complementary to at least a part of the CAD target mRNA. A plurality of such dsRNA may be employed to provide therapeutic benefit.

"The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier, and including a delivery modality such as fully encapsulated liposomes or lipid complexes," the abstract states. "The invention further includes methods for treating diseases caused by positive stranded RNA virus infection using the pharmaceutical compositions; and methods for inhibiting the propagation of positive-stranded RNA viruses in and between cells."


Title: Methods and Compositions Relating to Labeled RNA Molecules That Reduce Gene Expression

Application Number: 20100184039

Filed: Sept. 28, 2009

Lead Inventor: Lance Ford, Life Technologies

The invention comprises "methods and compositions involving labeled, double-stranded RNA, including siRNA, capable of triggering RNA-mediated interference in a cell," the patent application's abstract states. "Compositions of the invention include labeled dsRNA for RNAi, which may be a single strand of RNA that base-pairs with itself, or two separate RNA strands. In some embodiments, the label is fluorescent. The … invention further concerns methods for preparing such compositions and kits for implementing such methods. Other methods of the invention include ways of using labeled dsRNA for RNAi."


Title: Nucleic Acids Involved in Viral Infection

Application Number: 20100184205

Filed: Dec. 5, 2007 PCT Filed: Dec. 5, 2007

Lead Inventor: Isaac Bentwich, Rosetta Genomics

The patent application, its abstract states, claims "isolated viral and human nucleic acids associated with viral infection and various nucleic acid molecules relating thereto or derived therefrom. The nucleic acids may be useful for the prevention, treatment, and diagnosis of viral infections."


Title: Methods for Producing Interfering RNA Molecules in Mammalian Cells and Therapeutic Uses for Such Molecules

Application Number: 20100184207

Filed: Feb. 22, 2010

Lead Inventor: John Rossi, City of Hope

The invention, the patent application's abstract states, comprises "methods for producing interfering RNA molecules in mammalian cells. … Therapeutic uses for the expressed molecules, including inhibiting expression of HIV, are also provided."


Title: Compositions and Methods for Inhibiting Gene Silencing by RNA Interference

Application Number: 20100184209

Filed: Feb. 16, 2007 PCT Filed: Feb. 16, 2007

Lead Inventor: Annaleen Vermeulen, Dharmacon (Thermo Fisher Scientific)

The invention "provides compositions and methods for inhibiting gene silencing by the RNAi pathway," the patent application's abstract states. "The RNAi inhibitors of the invention have a reverse complement region to the target molecule of interest in association with at least one flanking region coupled to either at the 3' or 5' end of the [reverse complement] region. The flanking regions can be single-stranded or can have one or more regions of double-stranded nucleic acid with or without a hairpin loop.

"The RNAi inhibitors described herein can inhibit endogenous targets, including but not limited to microRNAs, or piRNAs, or can be used to inhibit the effects of exogenously introduced molecules, such as synthetic siRNAs, siRNAs expressed from vector constructs, or siRNAs generated by enzymatic methods. Inhibition is specific, potent, prolonged, and can be performed on a single target or multiple targets simultaneously," the abstract adds.


Title: Compositions and Methods for Modulating Angiogenesis

Application Number: 20100184821

Filed: July 30, 2007 PCT Filed: July 30, 2007

Lead Inventor: Joshua Mendell, Johns Hopkins University

"The invention generally features compositions and methods that are useful for modulating angiogenesis," the patent application's abstract states.

The application specifically claims "an inhibitory nucleic acid molecule complementary to at least a portion of a microRNA nucleic acid molecule of the mir-17-92 cluster," which can thereby reduce angiogenesis.


Title: dsRNA for Treating Viral Infection

Application Number: 20100184823

Filed: July 4, 2008 PCT Filed: July 4, 2008

Lead Inventor: Mark Aron Labow, Novartis

"The invention relates to double-stranded ribonucleic acids targeting gene expression of phosphatidylinositol 4-kinase, in particular human phosphatidylinositol 4-kinase, catalytic, beta polypeptide, or human phosphatidylinositol 4-kinase, catalytic, alpha polypeptide, and their use for treating infection by positive stranded RNA viruses such as hepatitis C virus," the patent application's abstract states. "Each dsRNA comprises an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19 [to] 25 nucleotides in length, and which is substantially complementary to at least a part of the PIK4CB or PIK4CA target mRNA. A plurality of such dsRNA may be employed to provide therapeutic benefit.

"The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier, and including a delivery modality such as fully encapsulated liposomes or lipid complexes," it adds. "The invention further includes methods for treating diseases caused by positive-stranded RNA virus infection using the pharmaceutical compositions; and methods for inhibiting the propagation of positive stranded RNA viruses in and between cells."


Title: RNA Interference-Meditated Inhibition of Interleukin and Interleukin Receptor Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20100184824

Filed: Jan. 7, 2010

Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)

"This invention relates to compounds, compositions, and methods useful for modulating interleukin and/or interleukin receptor gene expression using short interfering nucleic acid molecules," the patent application's abstract states. "This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of interleukin and/or interleukin receptor gene expression and/or activity by RNA interference using small nucleic acid molecules."


Title: RNA Interference-Meditated Inhibition of Protein Tyrosine Phosphatase-1B Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20100184825

Filed: Feb. 25, 2010

Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)

"This invention relates to compounds, compositions, and methods useful for modulating protein tyrosine phosphatase-1B gene expression using short interfering nucleic acid molecules," according to the patent application's abstract. "This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of PTP-1B gene expression and/or activity by RNA interference using small nucleic acid molecules. ... Such small nucleic acid molecules are useful, for example, for treating, preventing, inhibiting, or reducing obesity, insulin resistance, diabetes in a subject or organism, and for any other disease, trait, or condition that is related to or will respond to the levels of PTP-1B in a cell or tissue, alone or in combination with other treatments or therapies."


Titles: Methods and Compositions for Enhancing the Efficacy and Specificity of RNA Silencing

Application Numbers: 20100184828, 20100184827, 20100184826

Filed: March 23/29, 2010

Lead Inventor: Phillip Zamore, University of Massachusetts

The inventions, the patent applications' abstracts state, provide "methods of enhancing the efficacy and specificity of RNA silencing … [as well as] compositions for mediating RNA silencing. In particular, the [inventions provide] siRNAs, siRNA-like molecules, shRNAs, vectors and transgenes having improved specificity and efficacy in mediating silencing of a target gene. Therapeutic methods are also featured."


Title: RNAi Modulation of SCAP and Therapeutic Uses Thereof

Application Number: 20100184829

Filed: March 29, 2010

Lead Inventor: Juergen Soutschek, Alnylam Pharmaceuticals

"The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of a SCAP gene comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19 [to] 25 nucleotides in length, and which is substantially complementary to at least a part of a SCAP 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 human SCAP expression and the expression of a SCAP gene using the pharmaceutical composition; and methods for inhibiting the expression of a SCAP gene in a cell."


Title: Primary microRNA Expression Cassette

Application Number: 20100184840

Filed: Nov. 30, 2009

Lead Inventor: Patrick Arbuthnot, University of the Witwatersrand

"This invention relates to inhibition of hepatitis gene expression," the patent application's abstract states. "More specifically, the invention relates to a method of using RNA sequences to inhibit hepatitis B and C virus replication. Expression cassettes that include DNA sequences derived from endogenous microRNAs are used in the method and are transcribed by Pol II promoters, and then processed to generate sequences that are specific to target hepatitis virus sequences. The RNAi effecter sequences can target the selected hepatitis virus sequences resulting in gene silencing or transcriptional inhibition of the hepatitis virus gene. The expression cassettes may be delivered in vitro or in vivo to host cells. A pharmaceutical composition containing the expression cassettes is also claimed."


Title: Extended Dicer-Substrate Agents and Methods for the Specific Inhibition of Gene Expression

Application Number: 20100184841

Filed: Dec. 18, 2009

Inventor: Bob Brown, Dicerna Pharmaceuticals

"The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid in an amount effective to reduce expression of a target gene in a cell," according to the patent application's abstract. "The dsNAs of the invention possess a pattern of deoxyribonucleotides — in most embodiments, the pattern comprises at least one deoxyribonucleotide-deoxyribonucleotide base pair — designed to direct the site of Dicer enzyme cleavage within the dsNA molecule. Deoxyribonucleotides of the dsNA molecules of the invention are located within a region of the dsNA that can be excised via Dicer cleavage to generate an active siRNA agent that no longer contains the deoxyribonucleotide pattern.

"Such DNA-extended Dicer-substrate siRNAs were demonstrated to be more effective RNA inhibitory agents than corresponding double-stranded RNA-extended DsiRNAs," the abstract notes. "DsiRNA agents were also found to tolerate guide strand mismatches."


Title: Ultraconserved Regions Encoding ncRNAs

Application Number: 20100184842

Filed: Aug. 4, 2008 PCT Filed: Aug. 4, 2008

Inventor: Carlo Croce, Ohio State University

The invention, the patent application's abstract states, relates to methods for differentiating human cancers that use "one or more transcribed ultraconserved regions' expression profiles where the association between the genomic location of UCRs and the analyzed cancer-related genomic elements is highly statistically significant and comparable to that reported for miRNAs."


Title: Cell-Based RNA Interference and Related Methods and Compositions

Application Number: 20100186097

Filed: July 6, 2009

Lead Inventor: Scott Lowe, Cold Spring Harbor Laboratory

"The invention provides, among other things, methods for performing RNA interference in stem cells and methods for using the stem cells in vivo," according to the patent application's abstract.


Title: Methods and Compositions for Post-Transcriptional Gene Silencing

Application Number: 20100186102

Filed: Aug. 21, 2008 PCT Filed: Aug. 21, 2008

Inventor: Alexzander Asea, Texas A&M

The invention, the patent application's abstract states, comprises "an isolated double-stranded ribonucleic acid molecule that inhibits the expression of a target gene [wherein] the dsRNA comprises two strands of nucleotides wherein a first strand has a length of from 19 to 28 consecutive nucleotides and is substantially identical to a sequence in the target gene and wherein a second strand is substantially complementary to the first strand; and a binding moiety that binds a 3' end of the first strand to a 5' end of the second strand."

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

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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.