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IP Update: Recent Patents, Patent Applications Awarded to Quark, Merck, Tekmira, and More


Title: Genetic Inhibition of Double-Stranded RNA

Patent Number: 8,283,329

Filed: Sept. 28, 2007

Lead Inventor: Andrew Fire, Carnegie Institution

The patent, its abstract states, claims “a process ... of introducing an RNA into a living cell to inhibit gene expression of a target gene in that cell. The process may be practiced ex vivo or in vivo. The RNA has a region with double-stranded structure. Inhibition is sequence-specific in that the nucleotide sequences of the duplex region of the RNA and of a portion of the target gene are identical. The ... invention is distinguished from prior art interference in gene expression by antisense or triple-strand methods.”

Title: Methods to Regulate miRNA Processing by Targeting Lin-28

Patent Number: 8,283,331

Filed: Oct. 8, 2008

Lead Inventor: Richard Gregory, Children's Medical Center

The invention, the patent's abstract states, relates “generally to methods to regulate microRNA biogenesis, in particular the regulation of the processing of pri-miRNA to mature miRNA by Lin-28 and/or variants such as Lin-28B. In particular, the ... invention relates to methods and compositions comprising at least one agent which inhibits Lin-28 function or activity and/or expression to increase the processing of pri-mRNA to mature miRNA. More specifically, one aspect of the invention is directed to treating and/or preventing cancer in a subject by administering an agent that inhibits Lin-28 activity or expression to a subject, preferably a human subject.”

Title: Lipid Formulations for Nucleic Acid Delivery

Patent Number: 8,283,333

Filed: June 30, 2010

Lead Inventor: Ed Yaworski, Protiva Biotherapeutics (Tekmira Pharmaceuticals)

The invention, the patent's abstract states, relates to “novel, serum-stable lipid particles comprising one or more active agents or therapeutic agents, methods of making the lipid particles, and methods of delivering and/or administering the lipid particles. More particularly, the ... invention provides serum-stable nucleic acid-lipid particles comprising a nucleic acid, methods of making the [particles], and methods of delivering and/or administering the [particles]. In particular embodiments, the ... invention provides tumor-directed lipid particles that preferentially target solid tumors. The tumor-directed formulations of the ... invention are capable of preferentially delivering a payload such as a nucleic acid to cells of solid tumors compared to non-cancerous cells.”

Title: Short Hairpin RNAs for Inhibition of Gene Expression

Patent Number: 8,283,460

Filed: Oct. 14, 2009

Lead Inventor: Qing Ge, Somagenics

The invention, the patent's abstract states, comprises “methods, compositions, and kits that include small hairpin RNA useful for inhibition of gene expression, such as viral-mediated gene expression.”

Title: Multiple-Promoter Expression Cassettes for Simultaneous Delivery of RNAi Agents

Patent Number: 8,283,461

Filed: March 12, 2010

Lead Inventor: Petrus Roelvink, Benitec

The invention provides “multiple-promoter expression cassettes for simultaneous delivery of RNAi, preferably to mammalian cells in vivo,” according to the patent's abstract.

Title: Group of Nucleic Acid Fragments for Prevention of HIV Infection or AIDS and the Usage Thereof

Patent Number: 8,283,462

Filed: Jan. 10, 2011

Lead Inventor: Zhiwen Zhou, Beijing Solobio Genetechnology Company

“The invention provides a group of nucleic acid fragments ... for [the] prevention of HIV infection or AIDS and the usage thereof,” the patent's abstract states. “In the invention, a series of RNA fragments, which are highly homogenous to all the published HIV gene sequences, were obtained by homology [comparison]. The double-stranded [RNAs] derived from these fragments can effectively inhibit the expression of the HIV genes. The RNA transcribed by plasmid also can suppress the expression of the HIV in the cell. After the adenovirus or associated virus [that] carry DNA corresponding above RNA infect the cell, the transcription dsRNA can inhibit the expression of the HIV genes.”

Title: Conjugated Lipomers and Uses Thereof

Application Number: 20120251560

Filed: March 23, 2012

Lead Inventor: James Dahlman, Massachusetts Institute of Technology

“The invention comprises inventive conjugated polyethyleneimine polymers and conjugated aza-macrocycles,” the patent application's abstract states. “Also provided are compositions comprising the inventive conjugated lipomers, and methods of preparation and use.”

Title: Bi-Functional shRNA Targeting Stathmin 1 and Uses Thereof

Application Number: 20120251617

Filed: March 1, 2012

Lead Inventor: Donald Rao, Gradalis

The invention includes “bi-functional shRNAs capable of reducing an expression of a stathmin 1 gene, wherein at least one target site sequence of the bi-functional RNA molecule is located within the stathmin 1 gene … [and the] RNA molecule is capable of activating a cleavage-dependent and a cleavage-independent RNA-induced silencing complex for reducing the expression level of stathmin 1,” according to the patent application's abstract.

Title: microRNA-29a, b, c as a Tumor Suppressor and Sensitizing Agent for Chemotherapy

Application Number: 20120251619

Filed: April 2, 2012

Inventor: Preethi Gunaratne, University of Houston

The invention provides “a method of improving a therapeutic response to a cancer treatment in a subject [by] administering an effective amount of an agent that enhances the expression of microRNA-29 or an agent that mimics the effects of microRNA-29,” according to the patent application's abstract. “Further provided is a method of treating a cancer in a subject in need of such treatment comprising the step of administering an effective amount of a microRNA-29 or an agent that enhances the expression of microRNA-29.”

Title: Method for Rapidly Evaluating Performance of Short Interfering RNA with Novel Chemical Modifications

Application Number: 20120252027

Filed: Dec. 9, 2010

Lead Inventor: Gabor Butora, Merck

The invention comprises a “method for the rapid evaluation of novel sugar modifications to be used in siRNA synthesis, including the rapid evaluation of chemical modification patterns within the siRNA to effectuate increased stability and ultimately increased efficacy of a siRNA therapeutic,” the patent application's abstract states.

Title: Method of Therapy and Diagnosis of Atherosclerosis

Application Number: 20120252867

Filed: June 3, 2011

Lead Inventor: Suh-Hang Juo, Kaohsiung Medical University

The invention comprises a method of treating atherosclerosis by providing let-7g, an analogue thereof, or modified let-7g to inhibit the expression of lectin-like oxidized low-density lipoprotein receptor-1, as well as the binding of LOX-1 to oxidized low-density lipoprotein, the patent application's abstract states. Also claimed is a method of diagnosing atherosclerosis by measuring levels of let-7g in serum or plasma samples of organisms.

Title: Diagnosis and Treatment of Chronic Lymphocytic Leukemia

Application Number: 20120252871

Filed: March 16, 2012

Lead Inventor: David Galas, Institute for Systems Biology

The patent application, its abstract states, claims microRNA levels in plasma as markers for “indicating the presence of CLL in a subject, as well as suggesting routes of therapeutic treatment.”

Title: RNAi Modulation of ApoB and Uses Thereof

Application Number: 20120252872

Filed: April 19, 2012

Lead Inventor: Juergen Soutschek, Alnylam Pharmaceuticals

“The invention relates to compositions and methods for modulating the expression of apolipoprotein B and, more particularly, to the down-regulation of apolipoprotein B by chemically modified oligonucleotides,” the patent application's abstract states.

Title: siRNA Targeting Interleukin-1 Receptor-Associated Kinase 4

Application Number: 20120252873

Filed: June 6, 2012

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: Methods for Delivery of siRNA to the Spinal Cord and Therapies Arising Therefrom

Application Number: 20120252874

Filed: Nov. 8, 2010

Lead Inventor: Elena Feinstein, Quark Pharmaceuticals

The patent application, its abstract states, relates “at least in part to methods for the administration of small interfering RNAs to the spinal cord of a human or animal patient, and also to a method of treatment for spinal cord injury and other diseases and disorders of the CNS. In particular, the application discloses methods to deliver an siRNA compound locally, directly, and without the need for transduction vehicles and formulations in effective doses to the injured spinal cord to promote recovery of CNS function and or attenuation of allodynia.”

Title: Methods and Compositions for Treating Diseases, Disorders, or Injury of the CNS

Application Number: 20120252875

Filed: Dec. 9, 2010

Lead Inventor: Elena Feinstein, Quark Pharmaceuticals

The invention relates to “non-invasive methods for treating diseases, disorders, and injury to the central nervous system, and in particular to otic compositions and to methods of use thereof,” according to the patent application's abstract.

Title: Novel Cationic Lipids with Short Lipid Chains for Oligonucleotide Delivery

Application Number: 20120253032

Filed: Dec. 20, 2010

Lead Inventor: Mark Cameron, Merck

The invention comprises “novel cationic lipids with short lipid chains that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides to facilitate the cellular uptake and endosomal escape, and to knockdown target mRNA both in vitro and in vivo while decreasing inflammatory toxicities,” the patent application's abstract states.

Title: Method of Regulating Gene Expression

Application Number: 20120255042

Filed: Jan. 23, 2012

Lead Inventor: Bryan Cullen, Duke University

The invention relates, in general, to “gene expression and, in particular, to a method of inhibiting the expression of a target gene and to constructs suitable for use in such a method,” the patent application's abstract states.

Title: microRNA as a Cancer Progression Predictor and Its Use for Treating Cancer

Application Number: 20120255043

Filed: March 29, 2011

Lead Inventor: Shih-Hwa Chiou, Taipei Veterans General Hospital

The invention, the patent application's abstract states, is based on “the findings that a novel function for miR142-3p [is] in the regulation of Sox2, adenylyl cyclase 9, and CD133 expression ...[as well as] the overall stemness of recurrent [glioblastoma multiforme] cells [and cancer stem cells], and that miR142-3p modulated tumor-initiating properties in recurrent [glioblastoma multiforme]. The ... invention consequently supports the development of novel miRNA-based strategies for brain tumor treatment.”

Title: Inducible Small RNA Expression Constructs for Targeted Gene Silencing

Application Number: 20120255045

Filed: April 3, 2012

Lead Inventor: Thomas Tuschl, Max Planck

“The invention relates to vectors for the inducible expression of RNA molecules in eukaryotic” organisms, the patent application's abstract states.

The Scan

Researchers Develop Polygenic Risk Scores for Dozens of Disease-Related Exposures

With genetic data from two large population cohorts and summary statistics from prior genome-wide association studies, researchers came up with 27 exposure polygenic risk scores in the American Journal of Human Genetics.

US Survey Data Suggests Ancestry Testing Leads Way in Awareness, Use of Genetic Testing Awareness

Although roughly three-quarters of surveyed individuals in a Genetics in Medicine study reported awareness of genetic testing, use of such tests was lower and varied with income, ancestry, and disease history.

Coral Genome Leads to Alternative Amino Acid Pathway Found in Other Non-Model Animals

An alternative cysteine biosynthesis pathway unearthed in the Acropora loripes genome subsequently turned up in sequences from non-mammalian, -nematode, or -arthropod animals, researchers report in Science Advances.

Mosquitos Genetically Modified to Prevent Malaria Spread

A gene drive approach could be used to render mosquitos unable to spread malaria, researchers report in Science Advances.