Title: Compositions and Methods for Inhibiting Expression of a Target Gene
Patent Number: 7,829,693
Filed: March 7, 2003
Lead Inventor: Roland Kreutzer, Alnylam Pharmaceuticals
The invention, the patent's abstract states, "relates to a double-stranded ribonucleic acid having a nucleotide sequence which is substantially identical to at least a part of a target gene and which is no more than 49, preferably less than 25, nucleotides in length, and which comprises a complementary RNA strand having a 1 to 4 nucleotide overhang at the 3' end and a blunt 5' end.
"The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier," it adds. "The pharmaceutical compositions are useful for inhibiting the expression of a target gene, as well as for treating diseases caused by expression of the target gene, at low dosages. The invention also relates to methods for inhibiting the expression of a target gene, as well as methods for treating diseases caused by the expression of the gene."
Title: Treatment of a Neurodegenerative Disease Through Intracranial Delivery of siRNA
Patent Number: 7,829,694
Filed: May 25, 2004
Inventor: William Kaemmerer, Medtronic
The invention "provides devices, small interfering RNA, and methods for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent to a predetermined infusion site in a brain, and discharging through the discharge portion of the catheter a predetermined dosage of at least one substance capable of inhibiting production of at least one neurodegenerative protein," the patent's abstract states. The invention also provides "valuable small interfering RNA vectors, and methods for treating neurodegenerative disorders such as Alzheimer's disease, Parkinson's disease, Huntington's disease, spinocerebellar ataxia type 1, type 2, type 3, and/or dentatorubral-pallidoluysian atrophy."
Title: Specific GRP78 Expression-Inhibition RNAi Sequence, Medicine Thereof, and Method Thereof
Patent Number: 7,829,695
Filed: July 17, 2008
Lead Inventor: Ann-Joy Cheng, Chang Gung University
The invention comprises "a specific GRP78 expression-inhibition RNAi sequence, a medicine thereof, and a method thereof, wherein [a pre-defined] RNAi sequence … complementary to GRP78 forms a special hairpin structure inside cancer cells to specifically and effectively inhibit GRP78 expression and then inhibit the canceration process, including the growth, migration, invasion, and metastasis of cancer," the patent's abstract states.
Title: siRNA Targeting Amyloid Beta Precursor Protein
Patent Number: 7,829,696
Filed: Nov. 4, 2009
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: Down-Regulation of Mortalin by siRNA
Application Number: 20100278841
Filed: Sept. 25, 2008 PCT Filed: Sept. 25, 2008
Lead Inventor: Zvi Fishelson, Tel-Aviv University
The patent application, its abstract states, claims the "use of a siRNA molecule selected from [a specific] group … for treating a disease associated with a pathological cell population in a subject in need thereof … Pharmaceutical compositions and articles of manufacture comprising the siRNAs are also disclosed."
Title: Gene Silencing of the Brother of the Regulator of Imprinted Sites
Application Number: 20100279291
Filed: Aug. 11, 2008 PCT Filed: Aug. 11, 2008
Lead Inventor: Boris Reznik, Vendevia Group
The invention "provides methods and compositions useful for inhibiting expression of the gene encoding the transcription factor, Brother of the Regulatory of Imprinted Sites, by RNA interference," the patent application's abstract states. "Methods of the … invention can be used to silence BORIS in cancer cells, which results in apoptosis and may be useful for treating cancer in mammals. The methods of the invention directed to cancer therapy can be used alone or in combination with standard cancer treatments such as surgery, radiation, chemotherapy, and immunotherapy."
Title: microRNA Expression in Human Peripheral Blood Microvesicles and Uses Thereof
Application Number: 20100279292
Filed: Sept. 12, 2008 PCT Filed: Sept. 12, 2008
Lead Inventor: Clay Marsh, Ohio State University
The invention comprises "novel methods and compositions for the diagnosis, prognosis, and treatment of disorders by examining samples containing microvesicles and miRs therein," the patent application's abstract states.
Title: Polymeric Short Interfering RNA Conjugates
Application Number: 20100279408
Filed: Nov. 27, 2007 PCT Filed: Nov. 27, 2007
Inventor: Hong Zhao, Enzon Pharmaceuticals
The invention comprises "polymeric siRNA conjugates … [as well as] methods for down-regulation of gene expression in vivo and in vitro and for inhibition of the growth of cancer cells using the conjugates," the patent application's abstract states.
Title: siRNA Targeting Interleukin-1 Receptor-Associated Kinase 4
Application Number: 20100279896
Filed: April 30, 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 including those directed to nucleotide sequences for IRAK4."
Title: Compositions Comprising Human Integrin-Linked Kinase siRNA and Methods of Use Thereof
Application Number: 20100279919
Filed: Sept. 4, 2008 PCT Filed: Sept. 4, 2008
Inventor: Frank Xie, Intradigm
The invention comprises "nucleic acid molecules that inhibit ILK expression … [and] methods of using the nucleic acid molecules," the patent application's abstract states.
Title: Compositions and Methods to Treat Muscular and Cardiovascular Disorders
Application Number: 20100280094
Filed: Dec. 13, 2007 PCT Filed: Dec. 13, 2007
Lead Inventor: Iwan Beuvink, Novartis
The invention "relates to a novel microRNA, mir-208-2, implicated in muscular and cardiovascular disorders," the patent application's abstract states. The invention also relates to "oligonucleotide therapeutic agents and their use in the treatment of muscular and cardiovascular disorders resulting from dysregulation of mir-208-2."
Title: Therapeutic Agent for Wounds and Screening Method for the Same
Application Number: 20100280095
Filed: Nov. 5, 2009
Lead Inventor: Takashi Tanaka, Riken
The invention, the patent application's abstract states, "provides an agent for treating [a] wound, containing a substance that suppresses the expression or function of PDLIM2. The substance that suppresses the expression or function of PDLIM2 is preferably an siRNA or antisense nucleic acid capable of specifically suppressing the expression of PDLIM2, or an expression vector capable of expressing said polynucleotide.
"In addition, the … invention provides a method of screening for a substance capable of treating wounds, including determining whether or not a test substance is capable of suppressing the expression or function of PDLIM2," it adds.
Title: Treatment of Influenza
Application Number: 20100280096
Filed: July 2, 2008 PCT Filed: July 2, 2008
Lead Inventor: Misako Nakazawa, Kyorin Pharmaceutical
The invention "provides a double-stranded RNA which inhibits replication of influenza B viruses by RNA interference, in which the double-stranded RNA comprises an RNA having 19 to 25 nucleotides homologous with a part of an mRNA transcribed from a genomic RNA of the influenza B viruses and an antisense RNA thereof," according to the patent application's abstract.
Title: Compositions Comprising HIF-1 Alpha siRNA and Methods of Use Thereof
Application Number: 20100280097
Filed: Sept. 18, 2008 PCT Filed: Sept. 18, 2008
Inventor: Frank Xie, Intradigm
The invention comprises "nucleic acid molecules that inhibit HIF-1 alpha expression … [as well as] methods of using the nucleic acid molecules," the patent application's abstract states.
Title: Receptor-Targeted Oligonucleotides
Application Number: 20100280098
Filed: Oct. 6, 2008 PCT Filed: Oct. 6, 2008
Lead Inventor: Rudolph Juliano, University of North Carolina School of Medicine
The invention, the patent application's abstract states, comprises "oligonucleotide conjugates that include ligands that target cell receptors that mediate endocytosis. The ligands can include peptides and small molecules. The conjugates can include carrier macromolecules to which the ligands and oligonucleotides are attached, or conjugates where an oligonucleotide is attached to a ligand in the absence of a carrier macromolecule.
"The oligonucleotides can include therapeutic oligonucleotides, such as siRNA, antisense RNA and miRNA," the abstract adds. "The ligand can be an RGD peptide. Also disclosed … are methods of delivering the conjugates to cells in subjects."
Title: Method of Inhibiting Cancer Cell Proliferation, Proliferation Inhibitor, and Screening Method
Application Number: 20100280101
Filed: Dec. 26, 2008 PCT Filed: Dec. 26, 2008
Lead Inventor: Kimitoshi Kohno, University of Occupational and Environmental Health
The invention comprises "a method of inhibiting cancer cell proliferation by suppressing a function of ZNF143; a cancer cell proliferation inhibitor containing as an active ingredient a substance capable of inhibiting a function of ZNF143, like a ZNF143-specific siRNA; a prophylactic and/or therapeutic drug for cancer; a method of detecting cancer cells; a diagnostic reagent for cancer; a vector and transformant cell incorporating the vector; and a screening method for a substance possessing cancer cell proliferation inhibitory activity with the amount of inhibition of the binding of ZNF143 protein as an index," the patent application's abstract states.
Title: Double-Stranded Ribonucleic Acid with Increased Effectiveness in an Organism
Application Number: 20100280102
Filed: June 16, 2010
Lead Inventor: Claudia Woppman, Alnylam Pharmaceuticals
The invention, the patent application's abstract states, "relates to a method for the targeted selection of a double-stranded ribonucleic acid consisting of two single strands that exhibits increased effectiveness in inhibiting the expression of a target gene by means of RNA interference, wherein at least [one] end of the dsRNA comprises a nucleotide overhang of 1 to 4 unpaired nucleotides in length; wherein the unpaired nucleotide adjacent to the terminal nucleotide pair comprises a purine base; and wherein the terminal nucleotide pair on both ends of the dsRNA is a G-C pair, or at least two of the last four consecutive terminal nucleotide pairs are G-C pairs."