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IP Update: Recent Patents, Patent Applications Awarded to Alnylam, Northwestern, Arrowhead, and More


Title: Liposome for Liver-Specific Delivery and Release of Therapeutic Nucleic Acids or Drugs

Patent Number: 8,318,199

Filed: June 1, 2010

Lead Inventor: Meehyein Kim, Mogam Biotechnology Research Institute

The invention, the patent's abstract states, comprises a nanoscale composite “able to specifically deliver therapeutic agents such as therapeutic nucleic acids or drugs to the liver and selectively release them into hepatic cells to manifest potent therapeutic effects of the therapeutic agents. The composite may be comprised of an apolipoprotein A-1 and a liposome-forming material. A composition containing the composite and a pharmaceutically acceptable carrier is disclosed.”

Title: Compositions and Methods for Altering RNAi

Patent Number: 8,318,688

Filed: Dec. 19, 2006

Lead Inventor: Richard Carthew, Northwestern University

The invention relates to “compositions and methods for altering RNAi,” the patent's abstract states. “In particular, the ... invention provides systems and methods for identifying regulators of RNAi. … [as well as] methods of identifying inhibitors of RNAi regulators and methods of using the same. Compositions and methods of the ... invention find use in research, therapeutic, and clinical applications.”

Title: siRNA-Based Cancer Treatment

Patent Number: 8,318,689

Filed: Feb. 28, 2008

Lead Inventor: Annick Harel-Bellan, French National Center for Scientific Research

The invention, the patent's abstract states, comprises “a double-strand oligonucleotide, including two complementary oligonucleotide sequences forming a hybrid.” Each of the sequences includes at one end “one-to-five unpaired nucleotides forming single-strand ends extending beyond the hybrid, one of the oligonucleotide sequences being substantially complementary to a target sequence belonging to a DNA or RNA molecule to be specifically repressed, the target sequence belonging to a DNA or RNA molecule of a gene coding an angiogenic factor.”

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

Patent Number: 8,318,693

Filed: Sept. 2, 2009

Lead Inventor: Dinah Sah, Alnylam Pharmaceuticals

“The invention relates to a double-stranded ribonucleic acid targeting a mutant epidermal growth factor receptor and methods of using the dsRNA to inhibit expression of mutant EGFR,” the patent's abstract states.

Title: Triggered RNAi

Patent Number: 8,318,921

Filed: Feb. 29, 2008

Lead Inventor: Niles Pierce, California Institute of Technology

The invention, the patent's abstract states, “relates to methods and compositions for triggering RNAi.” Specifically, the invention comprises “a method of silencing a target gene … [by] providing an initiator to a cell comprising a detection target and a silencing target gene, wherein the detection target is different from the silencing target gene; providing a first substrate monomer to the cell, wherein the first substrate monomer comprises a silencing target complement region that is substantially complementary to a portion of the silencing target gene, and an initiator complement region that is substantially complementary to a portion of the initiator; and providing a second substrate monomer to the cell, wherein the second substrate monomer comprises a silencing target region that is substantially complementary to the silencing target complement region.”

The binding of the detection target to the initiator “initiates formation of an inactivator double-stranded RNA, which silences the silencing target gene,” it adds.

Title: Immunostimulatory siRNA Molecules

Patent Number: 8,318,924

Filed: Nov. 2, 2010

Lead Inventor: Michael Gantier, Monash University

The invention relates to “a double-stranded siRNA molecule that is capable of silencing gene expression, as well as inducing an immune response,” the patent's abstract states. “The molecule comprises a sense strand and an antisense strand, wherein the antisense strand comprises a first nucleotide sequence that is specifically complementary to mRNA transcribed from a target gene, and the sense strand comprises a second nucleotide sequence that is substantially or perfectly complementary to the antisense strand with the exception that the second nucleotide sequence comprises at least one immunostimulatory motif comprising two or more non-complementary nucleotides. Such a molecule may be utilized in a method of treating or preventing a disease or condition in a subject.”

Title: RNAi Therapeutic for Treatment of Hepatitis C Infection

Patent Number: 8,318,925

Filed: Feb. 25, 2011

Inventor: Hengli Tang, Florida State University

The patent, its abstract states, claims “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. … 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, encapsulated lipid particles, such as liposomes. Methods for treating, managing inhibiting, preventing … HCV infection using such siRNA and shRNAs and compositions comprising same are also provided.”

Title: Anti-microRNA Oligonucleotide Molecules

Patent Number: 8,318,926

Filed: Jan. 9, 2012

Lead Inventor: Thomas Tuschl, Rockefeller University

“The invention relates to isolated anti-microRNA molecules,” according to the patent's abstract. “In another embodiment, the invention relates to an isolated microRNA molecule. In yet another embodiment, the invention provides a method for inhibiting microRNP activity in a cell.”

Title: Compositions for Targeted Delivery of siRNA

Patent Number: 8,313,772

Filed: Feb. 22, 2011

Lead Inventor: David Rozema, Arrowhead Research

The invention, the patent's abstract states, “is directed compositions for targeted delivery of RNA interference polynucleotides to hepatocytes in vivo. Targeted RNAi polynucleotides are administered together with co-targeted delivery polymers. Delivery polymers provide membrane penetration function for movement of the RNAi polynucleotides from outside the cell to inside the cell. Reversible modification provides physiological responsiveness to the delivery polymers.”

Title: Cancer Cell-Specific Cell Proliferation Inhibitors

Patent Number: 8,314,073

Filed: Nov. 17, 2005

Lead Inventor: Motoki Takagi, Genecare Research

“The ... inventors discovered that although suppressing expression of the RecQ1 gene, a RecQ helicase family gene, shows suppressive effects on cell proliferation in cancer cells, such effects are not observed in [normal] human TIG3 cells,” the patent's abstract states. “Hence, the ... inventors discovered that siRNAs against [the] RecQ1 gene have cancer cell-specific cell proliferation-suppressing effects that are mediated by suppression of the expression of said gene.”

Title: Compositions and Methods for Inhibiting Expression of Huntingtin Gene

Patent Number: 8,314,075

Filed: Oct. 4, 2011

Lead Inventor: Dinah Sah, Alnylam Pharmaceuticals

“The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of the huntingtin gene, comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the ... gene,” the patent'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 the expression of the [huntingtin] gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.”

Title: Methods, Compositions, and Kits for Detection of microRNA

Patent Number: 8,314,220

Filed: Jan. 26, 2007

Lead Inventor: Rebecca Mullinax, Agilent Technologies

The invention, the patent's abstract states, provides “methods, nucleic acids, compositions, and kits for detecting [microRNAs] in samples. The methods comprise designing mRNA-specific primers, adding a polyA tail to the miRNA, and using reverse transcription and amplification to detect the miRNA. The nucleic acids, compositions, and kits typically comprise some or all of the components necessary to practice the methods of the invention.”

Title: Oligonucleotide Complex Compositions and Methods of Use as Gene Alteration Tool

Patent Number: 8,314,226

Filed: Dec. 19, 2007

Lead Inventor: David Tabatzdze, Massachusetts General Hospital

The invention, the patent's abstract states, comprises “compositions and methods of treatments of cells ... for altering the phenotype of a cell by administering an oligonucleotide complex to the cell, the complex having two strands and chemical modifications.”

Title: Hydroxymethyl Substituted RNA Oligonucleotides and RNA Complexes

Patent Number: 8,314,227

Filed: May 18, 2009

Inventor: Jesper Wengel, RiboTask (Marina Biotech)

The invention comprises “RNA oligonucleotides or complexes of RNA oligonucleotides … containing at least one, but optionally more than one, hydroxymethyl substituted nucleotide monomer,” the patent's abstract states. “The RNA complexes of the invention may be useful for therapeutic applications, diagnostic applications, or research applications.”

Title: siRNA Targeting Tie-2

Patent Number: 8,314,226

Filed: Nov. 8, 2010

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.”

Title: Structure and Use of 5' Phosphate Oligonucleotides

Application Number: 20120288476

Filed: May 8, 2012

Lead Inventor: Gunther Hartmann, University of Bonn

“Oligonucleotides bearing free, uncapped 5' phosphate group(s) are recognized by RIG-I, leading to the induction of type I IFN, IL-18 and IL-1 beta production,” the patent application's abstract states. “Bacterial RNA also induces type I IFN production, [while] 5' phosphate oligonucleotides and bacterial RNA can be used for inducing an anti-viral response or an anti-bacterial response … and for treating various disorders and diseases such as viral infections, bacterial infections, parasitic infections, tumors, allergies, autoimmune diseases, immunodeficiencies, and immunosuppression. Single-stranded 5' triphosphate RNA can be used for inducing an anti-viral response, an anti-bacterial response, or an anti-tumor response … in a target cell-specific manner.”

Title: Click Nanoparticle Conjugates

Application Number: 20120288935

Filed: Sept. 22, 2010

Lead Inventor: Chad Mirkin, Northwestern University (AuraSense Therapeutics)

The invention, the patent application's abstract states, comprises “nanoparticles modified with an agent through a triazole linkage … [as well as] methods of preparing modified nanoparticles and methods of using these modified nanoparticles.”

Title: Use of RNA Interference for Treating or Reducing Pain

Application Number: 20120289578

Filed: July 14, 2011

Lead Inventor: Ming-Dar Tsai, Fu-Jen Catholic University

“A use of a nucleic acid molecule mediating RNA interference for treating or reducing pain is disclosed,” the patent application's abstract states. “The nucleic acid molecule … is used for effectively inhibiting expression of bradykinin B2 receptor, treating or reducing pain, and preparing a pharmaceutical composition for reducing neuropathic pain.”

Title: Lipid-Formulated Compositions and Methods for Inhibiting Expression of Transthyretin

Application Number: 20120294905

Filed: Nov. 3, 2010

Inventor: Dinah Sah, Alnylam Pharmaceuticals

“The invention relates to lipid formulated double-stranded ribonucleic acid targeting a transthyretin gene, and methods of using the dsRNA to inhibit expression of TTR,” the patent application's abstract states.

Title: Sustained-Release Nucleic Acid Matrix Compositions

Application Number: 20120294944

Filed: Jan. 18, 2011

Lead Inventor: Noam Emanuel, Polypid

The invention, the patent application's abstract states, “provides compositions for extended release of a nucleic acid agent [and] a biodegradable polymer. The ... invention also provides methods of producing the matrix compositions and methods for using the matrix compositions to provide controlled release of the nucleic acid agent.”

Title: Novel Lipids and Compositions for Intracellular Delivery of Biologically Active Compounds

Application Number: 20120295832

Filed: May 8, 2012

Lead Inventor: Rainer Constien, Arrowhead Research

The invention, according to the patent application's abstract, relates to “novel amino-lipids, compositions comprising such amino-lipids, and methods of producing them. In addition, lipid nanoparticles comprising the novel amino-lipids and a biologically active compound are provided, as well as methods of production and their use for intracellular drug delivery.”

Title: microRNA-Based Methods and Compositions for the Diagnosis, Prognosis, and Treatment of Tumor Involving Chromosomal Rearrangements

Application Number: 20120295949

Filed: Aug. 31, 2010

Lead Inventor: Joern Bullerdiek, Qiagen

The invention comprises “novel methods and compositions for the diagnosis, prognosis, and treatment of tumor involving a chromosomal rearrangement, in particular a tumor or neoplasia of the thyroid gland,” the patent application's abstract states. “In addition, methods of identifying anti-tumor agents are described.”

The Scan

Expanded Genetic Testing Uncovers Hereditary Cancer Risk in Significant Subset of Cancer Patients

In Genome Medicine, researchers found pathogenic or likely pathogenic hereditary cancer risk variants in close to 17 percent of the 17,523 patients profiled with expanded germline genetic testing.

Mitochondrial Replacement Therapy Embryos Appear Largely Normal in Single-Cell 'Omics Analyses

Embryos produced with spindle transfer-based mitochondrial replacement had delayed demethylation, but typical aneuploidy and transcriptome features in a PLOS Biology study.

Cancer Patients Report Quality of Life Benefits for Immune Checkpoint Inhibitors

Immune checkpoint inhibitor immunotherapy was linked in JAMA Network Open to enhanced quality of life compared to other treatment types in cancer patients.

Researchers Compare WGS, Exome Sequencing-Based Mendelian Disease Diagnosis

Investigators find a diagnostic edge for whole-genome sequencing, while highlighting the cost advantages and improving diagnostic rate of exome sequencing in EJHG.