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IP Update: Mar 24, 2011

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Title: Prevention and Treatment of Acute Renal Failure and Other Kidney Diseases by Inhibition of p53 by siRNA

Patent Number: 7,910,566

Filed: Jan. 4, 2008

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 acute renal failure or other kidney diseases comprising administering to the patient the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the patient.”


Title: RNAi Therapeutic for Treatment of Hepatitis C Infection

Patent Number: 7,910,722

Filed: July 3, 2008

Inventor: Hengli Tang, Florida State University

The invention, the patent's abstract states, comprises “small interfering RNAs or small hairpin RNA and compositions comprising same ... that specifically target human cyclophilin A to effectively inhibit hepatitis C infection in a cell. Such siRNA and shRNAs may have a length of from about 19 to about 29 contiguous nucleotides corresponding to a specific region of human cyclophilin A cDNA of from about nucleotide 155 to about nucleotide 183 having particular potency against [cyclophilin A] and HCV. Such siRNA and shRNAs may be formulated as naked compositions or as pharmaceutical compositions.

“DNA polynucleotides, plasmids, and viral or non-viral vectors are also provided that encode siRNA or shRNA molecules, which may be delivered directly to cells or in combination with known delivery agents, such as lipids, polymers, [or] encapsulated lipid particles, such as liposomes,” the abstract adds.


Title: RNA Interference-Mediated Inhibition of Fos Gene Expression Using Short Interfering Nucleic Acid

Patent Number: 7,910,724

Filed: Aug. 4, 2008

Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)

“This invention relates to compounds, compositions, and methods useful for modulating c-Fos gene expression using short interfering nucleic acid molecules,” the patent'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 c-Fos gene expression and/or activity by RNA interference using small nucleic acid molecules … [which are] useful in the treatment of cancer, proliferative diseases or conditions, inflammatory diseases or conditions, allergic diseases or conditions, infectious diseases or conditions, autoimmune diseases or conditions, or transplantation/allograft rejection in a subject or organism.”


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

Patent Number: 7,910,725

Filed: Jan. 6, 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'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: Methods and Compositions Involving Chitosan Particles

Application Number: 20110064664

Filed: Oct. 8, 2008

Lead Inventor: Gabriel Lopez-Berenstein, University of Texas

The invention, the patent application's abstract states, comprises “nanoparticles for the delivery of a therapeutic agent or a diagnostic agent to a subject that include a chitosan and a polyphosphate, wherein the weight ratio of the chitosan to the polyphosphate is about 1 or greater and the weight ratio of the polyphosphate to the therapeutic agent or diagnostic agent is about 15 or less. Also disclosed are nanoparticles that include a chitosan and an inhibitor of enhancer of Zeste homologue 2.”


Titles: RNA Interference-Mediating Small RNA Molecules

Application Numbers: 20110065109 & 20110065773

Filed: July 19 & Jan. 6, 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,” the patent applications' abstracts state. “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: Compositions Comprising Human PCSK9 and Apolipoprotein B siRNA and Methods of Use

Application Number: 20110065644

Filed: March 9, 2009

Lead Inventor: Frank Xie, Intradigm (Silence Therapeutics)

The invention “provides siRNA nucleic acid molecules that inhibit PCSK9 or apolipoprotein B expression,” the patent application's abstract states. “Methods of using the nucleic acid molecules are also provided.”


Title: Double-Stranded RNA and Method of Use for Inhibiting Expression of a Fusion Gene

Application Number: 20110065777

Filed: Oct. 26, 2010

Lead Inventor: Olaf Heidenreich, University of Tuebingen (Roche)

“Specific inhibition of expression of a fusion gene in mammals occurs using a short, double-stranded ribonucleic acid molecule,” the patent application's abstract states. “The dsRNA comprises two separate non-linked RNA strands, an S1 strand and a complementary strand. The strands are 20 to 23 nucleotides in length, and the S1 strand is complementary to the fusion junction of the AML-1/MTG-8 fusion gene. The dsRNA also comprises at least 3 nucleotides on each side of the fusion junction. The dsRNA can be introduced into and maintained in a mammalian cell under conditions and for a time sufficient to obtain degradation of mRNA of the fusion gene to inhibit expression of the fusion gene. The dsRNAs and methods described are useful for treating diseases caused by chromosomal aberrations, particularly malignant diseases such as lymphoma and leukemia.”


Title: RNA Interference-Mediated Inhibition of Muscarinic Colinergic Receptor Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20110065778

Filed: Nov. 19, 2010

Lead Inventor: James McSwiggen, Merck

The invention relates to “compounds, compositions, and methods useful for modulating the expression of genes associated with respiratory and pulmonary disease, such as cholinergic muscarinic receptor genes, 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 cholinergic muscarinic receptor genes, or other genes involved in pathways of cholinergic muscarinic receptor gene expression and/or activity by RNA interference using small nucleic acid molecules … and methods used to modulate the expression of M3 muscarinic acetylcholine receptor or cholinergic receptor muscarinic 3.”


Title: Methods and Compositions for the Specific Inhibition of Gene Expression by Double-Stranded RNA

Application Number: 20110065908

Filed: Nov. 29, 2010

Lead Inventor: John Rossi, City of Hope (Integrated DNA Technologies)

“The invention provides compositions and methods for selectively reducing the expression of a gene product from a desired target gene, as well as treating diseases caused by expression of the gene,” the patent application's abstract states. “The method involves introducing into the environment of a cell an amount of a double-stranded RNA such that a sufficient portion of the dsRNA can enter the cytoplasm of the cell to cause a reduction in the expression of the target gene. The dsRNA has a first oligonucleotide sequence that is between 26 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of from about 19 to about 23 nucleotides is complementary to a nucleotide sequence of the RNA produced from the target gene.”

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