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IP Update: Recent Patents, Patent Applications Awarded to Alnylam, Novartis, Isis, and More

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Title: Modified iRNA Agents

Patent Number: 8,344,125

Filed: Nov. 16, 2009

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

“The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose,” the patent's abstract states. “The inclusion of such a monomer can allow for modulation of a property of the iRNA agent into which it is incorporated. The invention also relates to methods of making and using such modified iRNA agents.”


Title: Compositions and Methods for Inhibiting Expression of XBP-1 Gene

Patent Number: 8,344,126

Filed: Dec. 16, 2010

Lead Inventor: Kevin Fitzgerald, Alnylam Pharmaceuticals

“The invention relates to a double-stranded ribonucleic acid targeting X-Box protein 1, and methods of using the dsRNA to inhibit expression of XBP-1,” the patent's abstract states.


Titles: Organic Compositions to Treat Beta-ENaC-Related Diseases

Patent Numbers: 8,344,127; 8,344,129; 8,344,130; & 8,344,131

Filed: April 20, 2011; Jan. 23, 2012

Lead Inventor: Antonin de Fougerolles, Alnylam Pharmaceuticals

The patents, their abstracts state, claim “RNAi agents useful in methods of treating beta-ENaC-related diseases such as cystic fibrosis, pseudohypoaldosteronism type 1, Liddle's syndrome, hypertension, alkalosis, hypokalemia, and obesity-associated hypertension, using a therapeutically effective amount of an RNAi agent to beta-ENaC.”


Title: Short Interfering Ribonucleic Acid for Oral Administration

Patent Number: 8,344,128

Filed: Aug. 26, 2011

Lead Inventor: Francois Jean-Charles Natt, Novartis

The invention, the patent's abstract states, relates to “short interfering ribonucleic acid for oral administration … [wherein the] siRNA compris[e] two separate RNA strands that are complementary to each other over at least 15 nucleotides, wherein each strand is 49 nucleotides or less, and wherein at least one of which strands contains at least one chemical modification.”


Title: Method of Diagnosing Poor Survival Prognosis Colon Cancer Using miR-181b

Patent Number: 8,338,102

Filed: Nov. 14, 2011

Lead Inventor: Carlo Croce, Ohio State University

The invention, the patent's abstract states, “provides novel methods and compositions for the diagnosis and treatment of colon cancers. In particular, the ... invention provides diagnostics and prognostics for colon cancer patients, wherein the methods related to measuring miR levels can predict poor survival. The invention also provides methods of identifying inhibitors of tumorigenesis.”


Title: Method of Diagnosing Poor Survival Prognosis Colon Cancer Using miR-106a

Patent Number: 8,338,103

Filed: Nov. 14, 2011

Lead Inventor: Carlo Croce, Ohio State University

The invention, the patent's abstract states, “provides novel methods and compositions for the diagnosis and treatment of colon cancers. In particular, the ... invention provides diagnostics and prognostics for colon cancer patients, wherein the methods related to measuring miR levels can predict poor survival. The invention also provides methods of identifying inhibitors of tumorigenesis.”


Title: Method of Diagnosing Poor Survival Prognosis Colon Cancer Using miR-103-2

Patent Number: 8,338,104

Filed: Nov. 15, 2011

Lead Inventor: Carlo Croce, Ohio State University

The invention, the patent's abstract states, “provides novel methods and compositions for the diagnosis and treatment of colon cancers. In particular, the ... invention provides diagnostics and prognostics for colon cancer patients, wherein the methods related to measuring miR levels can predict poor survival. The invention also provides methods of identifying inhibitors of tumorigenesis.”


Title: Method of Diagnosing Poor Survival Prognosis Colon Cancer Using miR-203

Patent Number: 8,338,105

Filed: Nov. 15, 2011

Lead Inventor: Carlo Croce, Ohio State University

The invention, the patent's abstract states, “provides novel methods and compositions for the diagnosis and treatment of colon cancers. In particular, the ... invention provides diagnostics and prognostics for colon cancer patients, wherein the methods related to measuring miR levels can predict poor survival. The invention also provides methods of identifying inhibitors of tumorigenesis.”


Title: Method of Diagnosing Poor Survival Prognosis Colon Cancer Using miR-29a

Patent Number: 8,338,106

Filed: Nov. 15, 2011

Lead Inventor: Carlo Croce, Ohio State University

The invention, the patent's abstract states, “provides novel methods and compositions for the diagnosis and treatment of colon cancers. In particular, the ... invention provides diagnostics and prognostics for colon cancer patients, wherein the methods related to measuring miR levels can predict poor survival. The invention also provides methods of identifying inhibitors of tumorigenesis.”


Title: Methods of Determining Potency of Chemically Synthesized Oligonucleotides

Patent Number: 8,338,185

Filed: Dec. 5, 2011

Lead Inventor: Walter Lima, Isis Pharmaceuticals

The patent, its abstract states, claims “methods for determining potency of RNAi agents. Such methods include, but are not limited to, cell-based and cell-free assays that measure binding of an RNAi agent with Ago2 or that measure Ago2 activity in the presence of such RNAi agents. Also provided are assays that determine potency of RNAi agents by assessing their ability to compete with other RNAi agents, including control RNAi agents, for binding and/or activation of Ago2.”


Title: SnoRNAi-Small Nucleolar RNA Degradation by RNA Interference in Trypanosomatids

Patent Number: 8,338,583

Filed: Aug. 19, 2004

Inventor: Shulamit Michaeli, Bar-Ilan University

The patent, its abstract states, claims “polynucleotides and a method suitable for down-regulation of small nuclear RNA [that] can be used to treat diseases associated with activity of small nuclear RNA. ... Specifically, the ... invention can be used to down-regulate snoRNA molecules or box H/ACA-containing RNA molecules which are involved in diseases such as cancer.”


Title: Nucleic Acid Complex and Nucleic Acid Delivery Composition

Patent Number: 8,338,584

Filed: Nov. 6, 2008

Lead Inventor: Hirofumi Takeuchi, Otsuka Pharmaceutical

The invention comprises “a nucleic acid complex with low toxicity and high safety that can persistently maintain a nucleic acid, such as siRNA or the like, in a cell,” the patent's abstract states. It also comprises “a nucleic acid delivery composition that can efficiently deliver the nucleic acid complex into a cell. [The] nucleic acid complex … can be obtained by forming a complex using a nucleic acid to be introduced into a cell and a highly branched cyclic dextrin. Moreover, when a carrier comprising a diacylphosphatidylcholine, cholesterol, and/or a derivative thereof, and an aliphatic primary amine is used as a nucleic acid delivery carrier to introduce the nucleic acid complex into a cell, the safety, the efficiency of intracellular delivery, and the persistence of the nucleic acid in the cell can be further improved.”


Title: Down-Regulating Gene Expression in Insect Pests

Application Number: 20120322660

Filed: May 2, 2012

Lead Inventor: Myriam Beghyn, Devgen

The invention, the patent application's abstract states, relates to “genetic control of infestation by insect pest species, particularly prevention and/or control of pest infestation of plants, using interfering ribonucleic acid molecules. Compositions and combinations containing the interfering RNA molecules of the invention for use in topical applications, for example in the form of insecticides,” are also provided.


Title: Compositions and Methods for Diagnosing and Treating Melanoma

Application Number: 20120322743

Filed: Aug. 8, 2012

Lead Inventor: Eva Hernando, New York University

The patent application, its abstract states, claims “compositions and methods for the diagnosis, prognosis, prevention, and treatment of melanoma or melanoma-associated symptoms. The compositions are microRNA molecules associated with melanoma, as well as various nucleic acid molecules relating thereto or derived therefrom.”


Title: Methods and Compositions for Enhancing the Efficacy and Specificity of Single and Double Blunt-Ended siRNA

Application Number: 20120322846

Filed: Oct. 11, 2011

Lead Inventor: Phillip Zamore, University of Massachusetts

The invention, the patent application's abstract states, “provides methods of enhancing the efficacy and specificity of RNAi using single or double blunt-ended siRNA. The invention also provides single and double-blunt ended siRNA compositions, vectors, and transgenes containing the same for mediating silencing of a target gene. Therapeutic methods are also featured.”


Title: Duplex Oligonucleotide Complexes and Methods for Gene Silencing by RNA Interference

Application Number: 20120322855

Filed: July 19, 2012

Lead Inventor: Christina Yamada, Dharmacon (Thermo Fisher Scientific)

The invention comprises “duplex oligonucleotide complexes [that] can be administered to a cell, tissue, or organism to silence a target gene without the aid of transfection reagents,” according to the patent application's abstract. “The duplex oligonucleotide complexes of the disclosure include a conjugate moiety that facilitates delivery to a cell, tissue, or organism.”


Title: Regulation of Macrophage Activation Using miR-125b

Application Number: 20120322854

Filed: June 18, 2012

Lead Inventor: Aadel Chaudhuri, California Institute of Technology

The invention, the patent application's abstract states, “relates to regulation of macrophage activation by delivering of miRNAs, for example, miR-125b or anti-miR-125b, to macrophages. … In some embodiments, macrophage activation can be elevated or reduced by administering miR-125b or anti-miR-125b oligonucleotides. Also disclosed are methods for promoting T cell activation and method for treating various disorders such as tumor and autoimmune diseases.”


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

Application Number: 20120322858

Filed: Aug. 24, 2012

Lead Inventor: Roland Kreutzer, Alnylam Pharmaceuticals

The invention, the patent application's abstract states, “relates to a double-stranded ribonucleic acid for inhibiting the expression of a mutant gene, comprising a first strand that has a complementary region that is complementary to at least a portion of an RNA transcript of at least part of the mutant target gene and a second strand of the dsRNA complementary or substantially complementary to the first strand. 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 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.”


Titles: microRNA Fingerprints During Human Megakaryocytopoiesis

Application Numbers: 20120322859, 20120329053

Filed: Aug. 27, 2012

Lead Inventor: Carlo Croce, Ohio State University

The patent applications, their abstracts state, claim “a method of decreasing expression of HOXA1 in a subject having a cancer and/or myeloproliferative disorder associated with over-expression of a HOXA1 gene product, where an effective amount of at least one miR-10a gene product or an isolated variant or biologically active fragment thereof is administered to the subject sufficient to decrease expression of the HOXA1 gene product.”


Title: Compositions and Methods for Selective Delivery of Oligonucleotide Molecules to Specific Neuron Types

Application Number: 20120322991

Filed: April 19, 2011

Lead Inventor: Andres Pablo Montefeltro, NLife Therapeutics

“The invention provides a conjugate comprising a nucleic acid ... complementary to a target nucleic acid sequence and [whose] expression prevents or reduces expression of the target nucleic acid; and a selectivity agent which is capable of binding with high affinity to a neurotransmitter transporter. The conjugates of the ... invention are useful for the delivery of the nucleic acid to a cell of interest and thus, for the treatment of diseases which require a down-regulation of the protein encoded by the target nucleic acid as well as for the delivery of imaging agents to the cells for diagnostic purposes.”


Title: Modified siRNA Molecules and Uses Thereof

Application Number: 20120328668

Filed: April 26, 2012

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

The invention comprises “chemically modified siRNA molecules and methods of using such siRNA molecules to silence target gene expression,” the patent application's abstract states. “Advantageously, the modified siRNA of the ... invention is less immunostimulatory than its corresponding unmodified siRNA sequence and retains RNAi activity against the target sequence. The ... invention also provides nucleic acid-lipid particles comprising a modified siRNA, a cationic lipid, and a non-cationic lipid, which can further comprise a conjugated lipid that inhibits aggregation of particles. The ... invention further provides methods of silencing gene expression by administering a modified siRNA to a mammalian subject. Methods for identifying and/or modifying an siRNA having immunostimulatory properties are also provided.”


Title: Method and Substances for Isolating miRNAs

Application Number: 20120329054

Filed: Aug. 29, 2012

Lead Inventor: Elliott Dawson, Bioventures

The invention, the patent application's abstract states, comprises a “capture probe suitable for use with a method for isolating miRNAs.” Also claimed are “a method for isolating an miRNA of interest from a sample … [and] a method for identifying an miRNA of interest.”


Title: Diagnostic for Lung Cancer Using miRNA

Application Number: 20120329060

Filed: March 14, 2011

Lead Inventor: Jayaprakash Karkera, Centofor Ortho Biotech

“The invention provides a method for diagnosis of lung cancer, in particular, non-small cell lung cancer, using circulating levels of miRNA,” according to the patent application's abstract. “In a particular embodiment, the ratio of miRNA-21 to miRNA-221 can be used to diagnosis the presence of lung cancer or to monitor the response of a lung cancer patient to treatment.”


Title: Down-Regulation of Gene Expression Using Artificial microRNAs

Application Number: 20120329159

Filed: Aug. 22, 2012

Inventor: Brian McGonigle, Pioneer Hi-Bred

The patent application, its abstract states, claims “isolated nucleic acid fragments comprising precursor miRNA, and artificial miRNAs and their use in down-regulating gene expression.”


Title: microRNA Expression Signature for Predicting Survival and Metastases in Hepatocellular Carcinoma

Application Number: 20120329672

Filed: Aug. 27, 2012

Lead Inventor: Carlo Croce, Ohio State University

The patent application, its abstract states, claims “microarrays, kits, methods, and compositions for the diagnosis, prognosis, and treatment of hepatocellular carcinoma. Also provided are methods of identifying anti-HCC agents.”


Title: Methods and Compositions for Reducing Viral Genome Amounts in a Target Cell

Application Number: 20120329856

Filed: June 21, 2012

Lead Inventor: Peter Sarnow, Stanford University

The patent application, its abstract states, claims “methods and compositions for reducing viral genome amounts in a target cell. ... In the subject methods, the activity of a miRNA is inhibited in a manner sufficient to reduce the amount of viral genome in the target cell. Also provided are pharmaceutical compositions, kits, and systems for use in practicing the subject methods. The subject invention finds use in a variety of applications, including the treatment of subjects suffering from a viral mediated disease condition.”


Title: Short Hairpin RNAs for Inhibition of Gene Expression

Application Number: 20120329857

Filed: Aug. 22, 2012

Lead Inventor: Qing Ge, SomaGenics

The patent application, its abstract states, claims “methods, compositions, and kits that include small hairpin RNA useful for inhibition of gene expression, such as viral-mediated gene expression.”


Title: microRNA Fingerprints During Human Megakaryocytopoiesis

Application Number: 20120329859

Filed: Aug. 27, 2012

Lead Inventor: Carlo Croce, Ohio State University

The invention comprises “a method of decreasing expression of MAFB in a subject having a cancer and/or myeloproliferative disorder associated with over-expression of a MAFB gene product where an effective amount of at least one miR-130a gene product or an isolated variant or biologically-active fragment thereof is administered to the subject sufficient to decrease expression of the MAFB gene product in the subject,” the patent application's abstract states.