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IP Update: Recent Patents, Patent Applications Awarded to Biomics, Tekmira, Arrowhead Research, and More

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Title: Reversible siRNA-Based Silencing Mutant and Endogenous Wild-Type Huntingtin Gene and Its Application for the Treatment of Huntington's Disease

Patent Number: 8,217,018

Filed: June 18, 2008

Lead Inventor: Valerie Perrin, Atomic and Alternative Energies Commission

The invention, the patent's abstract states, comprises “isolated double-stranded short interfering nucleic acid molecules [that can inhibit] the expression of endogenous wild-type and exogenous human mutant huntingtin genes in cells of a non-human mammal ... expressing both said huntingtin genes, and their application for the treatment of Huntington's disease, as well as to study Huntington's disease in rodent models.”


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

Patent Number: 8,217,020

Filed: Nov. 19, 2010

Lead Inventor: Peter Sarnow, Stanford University

The patent, 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, [for example], an HCV-mediated disease condition.”


Title: Methods of Inhibiting Multiple Cytochrome P450 Genes with siRNA

Patent Number: 8,217,161

Filed: April 22, 2009

Inventor: William Baldwin, Clemson University

The invention, the patent's abstract states, “relates to siRNAs that are targeted to RNAs encoding two or more enzymes of a subfamily of cytochrome P450 enzymes, along with vectors, cells, and kits comprising the siRNAs. The invention further relates to methods of decreasing expression of two or more CYP subfamily genes in a non-human animal, animals in which expression of two or more CYP subfamily genes has been decreased, and methods of using such animals to study the function of cytochrome P450 enzymes.”


Title: siRNA Targeting Interleukin-1 Receptor-Associated Kinase

Patent Number: 8,217,162

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'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: Application of Highly Conserved Domain Sequences from Viral Genome as Template to Design Therapeutic sliRNAs

Patent Number: 8,217,163

Filed: Sept. 20, 2010

Lead Inventor: York Yuan Yuan Zhu, Biomics Biotechnologies

“This invention relates to the application of the highly conserved sequences of viral genome, especially from a highly conserved domain of enteroviral genome, as templates to design target small ligand RNAs,” according to the patent's abstract. “The resulting sliRNAs are therapeutically active ingredients in the treatment of the related diseases caused by pathological angiogenesis.”


Title: Treatment of Collagen Gene-Related Diseases by Inhibition of Natural Antisense Transcript to a Collagen Gene

Application Number: 20120171170

Filed: June 16, 2010

Lead Inventor: Joseph Collard, CuRNA (Opko Health)

The invention, the patent application's abstract states, “relates to antisense oligonucleotides that modulate the expression of and/or function of a collagen gene, in particular, by targeting natural antisense polynucleotides of a collagen gene. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of collagen genes.”


Title: Virus Discovery by Sequencing and Assembly of Virus-Derived siRNAs, miRNAs, and piRNAs

Application Number: 20120172237

Filed: June 1, 2010

Lead Inventor: Shou-Wei Ding, University of California, Oakland

The patent application, its abstract states, claims ”methods and systems for identifying viral nucleic acids in a sample … [as well as] methods for viral genome assembly and viral discovery using small inhibitory RNAs, microRNAs, and/or Piwi-interacting RNAs ... isolated or sequenced from invertebrate organisms … and/or plants, fungi, or algae.”


Title: Novel Triaykyl Cationic Lipids and Methods of Use Thereof

Application Number: 20120172411

Filed: Sept. 16, 2011

Lead Inventor: James Heyes, Protiva Biotherapeutics (Tekmira Pharmaceuticals)

The invention comprises “compositions and methods for the delivery of therapeutic agents to cells,” according to the patent application's abstract. “In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the ... invention are highly potent, thereby allowing effective knockdown of a specific target protein at relatively low doses. In addition, the compositions and methods of the ... invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.”


Title: Short Hairpin RNA for Gene Knockdown of NR1 Subunit of the N-Methyl-D-Aspartate Receptor for Its Application of Pharmaceutics

Application Number: 20120172410

Filed: Feb. 15, 2011

Inventor: Ping-Heng Tan, I-Shou University

The invention comprises a short hairpin RNA for the knockdown of the NR1 subunit of N-methyl-D-aspartate receptor, the patent application's abstract states. The shRNA comprises “a ... fragment sharing homologous nucleotides among the NR1 subunit of NMDA receptor; a … fragment having complementary sequence to the first fragment; and a connecting fragment having any base in repeated arrangement and connecting to the first and second fragments.”

The application also claims “a method of treatment for pathological pain by applying the shRNA ... to subcutaneous tissues of living organisms,” it adds.


Title: In Vivo Polynucleotide Delivery Conjugates Having Enzyme-Sensitive Linkages

Application Number: 20120172412

Filed: Dec. 23, 2011

Lead Inventor: David Rozema, Arrowhead Research

The invention, the patent application's abstract states, “is directed compositions for delivery of RNA interference polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active polyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membrane activity of the polymer while reversibility provides physiological responsiveness. The reversibly modified polyamines are further covalently linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate.”


Title: Methods and Compositions of Diagnosing Heart Failure

Application Number: 20120172251

Filed: Sept. 16, 2010

Inventor: Brian Lowes, University of Colorado, Denver

The invention relates to “diagnostic and prognostic assays and kits for determining whether a heart failure patient will respond to a pharmacotherapy,” the patent application's abstract states. “Methods of the invention include determining the expression level of biomarkers [including microRNAs] that are differentially expressed in a heart failure patient that responds to a pharmacotherapy compared to a heart failure patient that does not respond to a pharmacotherapy.”


Title: Method for the Diagnosis and/or Prognosis of Acute Renal Damage

Application Number: 20120172248

Filed: Sept. 3, 2010

Lead Inventor: Maria Garcia Bermejo, Hospital Universitario Ramon y Cajal

“The invention relates to a method for the diagnosis and/or prognosis of acute renal damage by analyzing the level of expression of at least one microRNA selected from miR-127, miR-126, miR-210, and miR-101,” the patent application's abstract states.

The Scan

For Better Odds

Bloomberg reports that a child has been born following polygenic risk score screening as an embryo.

Booster Decision Expected

The New York Times reports the US Food and Drug Administration is expected to authorize a booster dose of the Pfizer-BioNTech SARS-CoV-2 vaccine this week for individuals over 65 or at high risk.

Snipping HIV Out

The Philadelphia Inquirer reports Temple University researchers are to test a gene-editing approach for treating HIV.

PLOS Papers on Cancer Risk Scores, Typhoid Fever in Colombia, Streptococcus Protection

In PLOS this week: application of cancer polygenic risk scores across ancestries, genetic diversity of typhoid fever-causing Salmonella, and more.