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IP Update: Dec 31, 2009

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Title: Oligoribonucleotide Derivatives for Specific Inhibition of Gene Expression

Patent Number: 7,635,769

Filed: Sept. 22, 2005

Lead Inventor: Eugen Uhlmann, Sanofi-Aventis

The invention, the patent's abstract states, "relates to oligoribonucleotide derivatives [that] have a 2'5'-linked oligoribonucleotide residue without a 5'-phosphate residue on the 3' end, and to the use thereof for specific inhibition of gene expression."


Title: siRNA Targeting Protein Kinase N-3

Patent Number: 7,635,770

Filed: July 24, 2007

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

"Efficient sequence-specific gene silencing is possible through the use of siRNA technology," the patent'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 PKN-3."


Title: siRNA Targeting Amyloid Beta Precursor Protein

Patent Number: 7,635,771

Filed: Oct. 29, 2007

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

"Efficient sequence-specific gene silencing is possible through the use of siRNA technology," the patent'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 APP."


Title: siRNA Targeting Insulin-Like Growth Factor 1 Receptor

Patent Number: 7,638,621

Filed: April 3, 2007

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

"Efficient sequence-specific gene silencing is possible through the use of siRNA technology," according to the patent's abstract. "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 insulin-like growth factor 1 receptor."


Title: siRNA Targeting Intercellular Adhesion Molecule 1

Patent Number: 7,638,622

Filed: Oct. 26, 2007

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

"Efficient sequence-specific gene silencing is possible through the use of siRNA technology," the patent'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 ICAM1."


Title: Method for Molecular Delivery into Cells Using Nanotube Spearing

Application Number: 20090311767

Filed: April 21, 2006 PCT Filed: April 21, 2006

Inventor: Thomas Chiles, Boston College

The invention comprises "a method of delivering a macromolecule to a cell [by] engaging the macromolecule to a nanotube [made of] a magnetic particle, placing the nanotubes into a sample wherein the sample comprises a plurality of cells, and applying a force to the sample wherein the force causes the nanotubes to collide with the cells and thereby spear the cells," the patent application's abstract states. "The method further discloses applying a second force [that] drives the nanotubes into the cell … [as well as] a method of transfecting and/or transducing a cell [by] engaging a macromolecule to a nanotube, placing the nanotubes into communication with the cells, and applying a force to the nanotubes which causes the nanotubes to collide with the cells and thereby pierce the cellular membrane. Once inside the cell, the macromolecule may disengage from the nanotube."

The application specifically claims the use of the nanotubes to deliver RNAi molecules.


Title: Levels and/or Sustainability of DNA-Based Gene Expression

Application Number: 20090311786

Filed: Jan. 29, 2007 PCT Filed: Jan. 29, 2007

Lead Inventor: Andrew Fire, Carnegie Institution of Washington

"The invention encompasses methods for improving the level and/or sustainability of expression for a target nucleic acid in a eukaryotic cell comprising modifying the target nucleic acid to introduce or to comprise signals that limit or constrain the positions of nucleosome cores, and introducing the modified target nucleic acid into the eukaryotic cell, wherein the modified target nucleic acid has improved levels and/or sustainability of expression compared to original unmodified nucleic acid," the patent application's abstract states.


Title: Multiple-Compartment Eukaryotic Expression Systems

Application Number: 20090311788

Filed: Oct. 3, 2008

Lead Inventor: Catherine Pachuk, Nucleonics (Alnylam Pharmaceuticals)

The patent application, its abstract states, claims "method and constructs for expressing heterologous sequences of interest in eukaryotic cells using multiple-compartment expression systems. These systems, which may be comprised of a single construct or multiple constructs, utilize at least two different promoters which are each active within a different sub-cellular compartment of the same eukaryotic cell. The system and constructs of the invention are particularly useful for achieving enhanced in vivo expression of RNA molecules capable of modulating the expression of target genes."


Title: Cancer Therapy Using Bcl-xL-Specific siNA

Application Number: 20090312393

Filed: June 22, 2007 PCT Filed: June 22, 2007

Lead Inventor: Laurent Poulain, Centre Regional de Lutte Contre le Cancer

"The invention relates to a double-stranded short interfering nucleic acid molecule specific to the Bcl-xL transcript, comprising a sense and an antisense region, wherein the sense region comprises [a predefined] nucleotide sequence or a sequence having at least 70 percent identity, preferably at least 80 percent identity, more preferably at least 90 percent identity with said sequence, and the antisense region comprises a nucleotide sequence that is complementary to the sense region, and its use for treating cancer."


Title: Protection Against and Treatment of Age-Related Macular Degeneration

Application Number: 20090312394

Filed: June 13, 2007 PCT Filed: June 13, 2007

Inventor: Anne Elizabeth Hughes, Queen's University of Belfast

The invention, the patent application's abstract states, comprises "methods and reagents in relation to the diagnosis, protection, and treatment of age-related macular degeneration. In particular, the methods and reagents [relate] to RNA [and] … provide a strong protection to a subject against development of AMD."

The application specifically claims an RNAi inhibitor of CFHL1 and/or CFHL3.


Title: RNAi Modulation of apoB and Uses Thereof

Application Number: 20090312397

Filed: March 9, 2009

Lead Inventor: Juergen Soutschek, Alnylam Pharmaceuticals

"The invention relates to compositions and methods for modulating the expression of apolipoprotein B, and more particularly to the down-regulation of apolipoprotein B by chemically modified oligonucleotides," according to the patent application's abstract.


Title: Encapsulated Nanoparticles for Drug Delivery

Application Number: 20090312402

Filed: May 20, 2009

Lead Inventor: Christopher Contag, Stanford University

The invention, the patent application's abstract states, comprises "compositions and methods … for preparing nanosized … nucleic acids, including agents formulated for target-specific drug delivery. In some embodiments, the nanoparticles comprise a polymer coating, which can provide for controlled delivery, targeting, controlled release, and the like. In other embodiments, the nanoparticles comprise a target specific tag for targeting the nanoparticles to a site of interest, e.g., tissue, cell, etc."


Title: Oligonucleotides Comprising a Ligand Tethered to a Modified or Non-Natural Nucleobase

Application Number: 20090312531

Filed: July 31, 2009

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

The invention, the patent application's abstract states, "relates to a double-stranded oligonucleotide comprising at least one ligand tethered to an altered or non-natural nucleobase. In certain embodiments, the non-natural nucleobase is difluorotolyl, nitropyrrolyl, or nitroimidazolyl. In [other] embodiments, the ligand is a steroid or aromatic compound … [while in another] only one of the two oligonucleotide strands comprising the double-stranded oligonucleotide contains a ligand tethered to an altered or non-natural nucleobase."

In other embodiments, "both of the oligonucleotide strands comprising the double-stranded oligonucleotide independently contain a ligand tethered to an altered or non-natural nucleobase," the abstract states. In others embodiments, "the oligonucleotide strands comprise at least one modified sugar moiety.

"Another aspect of the … invention relates to a single-stranded oligonucleotide comprising at least one ligand tethered to an altered or non-natural nucleobase," it adds. "In certain embodiments, the non-natural nucleobase is difluorotolyl, nitropyrrolyl, or nitroimidazolyl … [while in other] … embodiments, the ligand is a steroid or aromatic compound. In certain embodiments, the ribose sugar moiety that occurs naturally in nucleosides is replaced with a hexose sugar, polycyclic heteroalkyl ring, or cyclohexenyl group. [And in still other] embodiments, at least one phosphate linkage in the oligonucleotide has been replaced with a phosphorothioate linkage."


Title: RNA Interference and Disease Resistance in Avians

Application Number: 20090313712

Filed: April 1, 2009

Lead Inventor: Leandro Christmann, Synageva BioPharma

"The invention relates to transgenic avians whose genome contains nucleotide sequences [that] encode therapeutic polynucleotides [corresponding] to one or more certain sequences in the genome of an avian pathogen," the patent application's abstract states.


Title: Compositions Comprising Cardiac Stem Cells Over-Expressing Specific microRNAs and Methods of Their Use in Repairing Damaged Myocardium

Application Number: 20090317369

Filed: June 9, 2009

Lead Inventor: Toru Hosoda, Brigham and Women's Hospital

"The invention provides compositions comprising modified stem cells containing a transgene that affects the expression of at least one gene that inhibits or promotes cardiomyogenesis," the patent application's abstract states. "In particular, the invention discloses compositions comprising cardiac stem cells, wherein said cardiac stem cells comprise a transgene encoding a microRNA. The compositions of the invention find use in the treatment of cardiovascular disorders, such as myocardial infarction. Methods of repairing damaged myocardium in a subject using the modified stem cells are also disclosed."


Title: Compositions and Methods to Monitor RNA Delivery to Cells

Application Number: 20090317802

Filed: Dec. 8, 2006 PCT Filed: Dec. 8, 2006

Lead Inventor: Sangeeta Bhatia, Massachusetts Institute of Technology

The patent application, its abstract states, claims "methods and compositions for tracking or monitoring uptake of siRNA by mammalian cells. … The methods and compositions may be used to monitoring the silencing activity of the internalized siRNA. The compositions contain an siRNA, an optically or magnetically detectable nanoparticle such as a quantum dot, and, optionally, a transfection reagent. Cells are contacted with an siRNA and an optically or magnetically detectable nanoparticle, optionally in the presence of a transfection reagent. Detection of internalized nanoparticles is indicative of siRNA uptake.

"The invention allows analysis, identification, processing, etc., of cells that have efficiently taken up siRNA," the abstract adds. "In one embodiment, cells are sorted into at least two populations based on the amount of siRNA taken up."


Title: microRNA Profile in Human Saliva and Its Use for Detection of Oral Cancer

Application Number: 20090317820

Filed: May 19, 2009

Lead Inventor: David Wong, University of California, Oakland

The invention, the patent application's abstract states, "provides for the first time the detection of [a] microRNA in human saliva and the correlation between such microRNA and oral cancers. The … invention therefore provides methods and kits for diagnosing oral cancers by examining pertinent [miRNAs] in saliva."


Title: Receptor-Mediated Delivery: Compositions and Methods

Application Number: 20090317855

Filed: July 26, 2007 PCT Filed: July 26, 2007

Lead Inventor: Ernst Lengyel, University of Chicago

The invention, according to the patent application's abstract, comprises "compositions and methods for delivering an agent to a cell [using] a prolactin receptor. … Also provided is a method of inhibiting a breast, ovarian, or prostate cancer cell … [by] contacting the cell with a complex comprising a prolactin receptor ligand linked to at least one of an RNAi-inducing agent, a polynucleotide sequence encoding a polypeptide, an miRNA, a cytotoxic moiety, a chemotherapeutic moiety, a radioactive moiety, or a nanoparticle. Methods of detecting a cancer cell expressing a prolactin receptor are also provided."


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

Application Number: 20090317907

Filed: Dec. 30, 2008

Lead Inventor: Christine Esau, Regulus Therapeutics

The invention, the patent application's abstract states, comprises "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: Small Interfering RNA Specific for HCV and Therapeutic Agent for Hepatitis C Comprising the Same

Application Number: 20090318531

Filed: May 30, 2006 PCT Filed: May 30, 2006

Lead Inventor: Meehyein Kim, Mogam Biotechnology Research Institute

The invention "relates to a therapeutic reagent for hepatitis C comprising HCV-specific short interfering RNA as an effective ingredient," the patent application's abstract states. "The siRNA of the invention is a double-stranded RNA specific for the nucleotide sequence of HCV which induces viral RNA degradation in mammalian cells and thereby inhibits HCV protein expression and replication. The method of the invention, which includes the step of administrating the synthetic siRNA or a DNA vector encoding the RNA, is thus effective for the treatment of HCV carrier by inhibiting HCV gene expression and replication."


Title: Annexin A9 Biomarker and Therapeutic Target in Epithelial Cancer

Application Number: 20090318533

Filed: March 26, 2009

Lead Inventor: Zhi Hu, University of California, Oakland

"Amplification of the ANXA9 gene in human chromosomal region 1q21 in epithelial cancers indicates a likelihood of both in vivo drug resistance and metastasis, and serves as a biomarker indicating these aspects of the disease," according to the patent application's abstract. "ANXA9 can also serve as a therapeutic target. Interfering RNAs and shRNA adapted to inhibit ANXA9 expression, when formulated in a therapeutic composition, and delivered to cells of the tumor, function to treat the epithelial cancer."


Title: Beta-TrCP1, Beta-TrCP2, and RSK1 or RSK2 Inhibitors and Methods of Sensitizing Target Cells to Apoptosis

Application Number: 20090318535

Filed: June 4, 2009

Lead Inventor: Michele Pagano, New York University

"The invention relates to modulating BimEL levels to sensitize cancer cells to cell death or apoptosis," the patent application's abstract states. "In certain embodiments, the invention relates to increasing BimEL levels. In certain embodiments, the invention relates to inhibitors of at least one of beta-TrCP1/2 or RSK1/2 proteins that sensitize tumor cells to chemotherapy-induced death or apoptosis. Additionally, the invention relates to cancer therapies, diagnostics, and methods for identifying novel drugs or drug candidates for increasing BimEL levels."

The application specifically claims siRNAs targeting beta-TrCP1, beta-TrCP2, RSK1, or RSK2.


Title: Silencing of TGF-Beta Type II Receptor Expression by siRNA

Application Number: 20090318537

Filed: July 20, 2009

Lead Inventor: Nalin Kumar, University of Illinois at Chicago

"The … application is directed to siRNA-based silencing of the type II receptor of TGF-beta siRNAs that target this receptor [and] abrogate the receptor protein and transcript," the patent application's abstract states. "TGF-beta-mediated processes such as fibronectin assembly and cell migration also are inhibited and the molecules of the invention are efficacious in reducing the inflammatory response and matrix deposition. These findings show that siRNAs can be successfully delivered both in vitro and in vivo to regulate the TGF-beta type II receptor level and modulate wound response. Methods and compositions exploiting the findings of the … invention have a wide-ranging application, extending from treatment of disorders of the eye to other organs and tissues throughout the body."


Title: Nucleic Acids for Expressing a Polynucleotide of Interest in Mammalian Cancer Cells

Application Number: 20090318540

Filed: Sept. 12, 2007 PCT Filed: Sept. 12, 2007

Lead Inventor: Jamilla Faivre, Institut National de la Sante et de la Recherche Medicale

"The invention relates to regulatory polynucleotides derived from rat and human AFP promoter sequences and from rat and human HIP/PAPI promoter sequences," the patent application's abstract states. The "regulatory polynucleotides are useful for expressing nucleic acids of interest specifically in mammalian cancer cells, including mammalian cancer liver cells."


Title: Oligonucleotides Comprising a Non-Phosphate Backbone Linkage

Application Number: 20090318676

Filed: July 1, 2009

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

"One aspect of the … invention relates to a ribonucleoside substituted with a phosphonamidite group at the 3'-position," the patent application's abstract states. "In certain embodiments, the phosphonamidite is an alkyl phosphonamidite.

"Another aspect of the … invention relates to a double-stranded oligonucleotide comprising at least one non-phosphate linkage," it notes. "Representative non-phosphate linkages include phosphonate, hydroxylamine, hydroxylhydrazinyl, amide, and carbamate linkages. In certain embodiments, the non-phosphate linkage is a phosphonate linkage. In certain [other] embodiments, a non-phosphate linkage occurs in only one strand. In [still other] embodiments, a non-phosphate linkage occurs in both strands. In [other] embodiments, a ligand is bound to one of the oligonucleotide strands comprising the double-stranded oligonucleotide. In [other] embodiments, a ligand is bound to both of the oligonucleotide strands comprising the double-stranded oligonucleotide. In [yet other] embodiments, the oligonucleotide strands comprise at least one modified sugar moiety.

"Another aspect of the present invention relates to a single-stranded oligonucleotide comprising at least one non-phosphate linkage," the abstract adds. "Representative non-phosphate linkages include phosphonate, hydroxylamine, hydroxylhydrazinyl, amide, and carbamate linkages. In certain embodiments, the non-phosphate linkage is a phosphonate linkage. In certain [other] embodiments, a ligand is bound to the oligonucleotide strand. In [other] embodiments, the oligonucleotide comprises at least one modified sugar moiety."

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