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IP Update: Recent Patents, Patent Applications Awarded to Regulus; Alnylam; OSU; and More

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Title: Oligomeric Compounds and Compositions for Use in Modulation of Small Non-Coding RNAs

Patent Number: 8,110,558

Filed: Dec. 31, 2008

Lead Inventor: Frank Bennett, Regulus Therapeutics

The invention comprises “compounds, compositions, and methods ... for modulating the expression and function of small non-coding RNAs,” the patent's abstract states. “The compositions comprise oligomeric compounds targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs, are also provided.”


Title: Therapeutic Compositions

Patent Number: 8,110,674

Filed: March 8, 2004

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

The patent, its abstract states, claims “therapeutic sRNA agents and methods of making and using” them.


Title: microRNAs in Never-Smokers and Related Materials and Methods

Application Number: 20120027753

Filed: Feb. 24, 2010

Lead Inventor: Carlo Croce, Ohio State University

The invention, the patent application's abstract states, comprises “novel methods and compositions for the diagnosis, prognosis, and treatment of lung cancer in never-smokers. The invention also provides methods of identifying anti-lung cancer agents.”


Title: Novel Lipids and Compositions for the Delivery of Therapeutics

Application Number: 20120027796

Filed: Nov. 10, 2009

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

The invention comprises “lipids that are advantageously used in lipid particles for the in vivo delivery of therapeutic agents to cells,” according to the patent application's abstract.


Title: Biodegradable Lipids for the Delivery of Active Agents

Application Number: 20120027803

Filed: June 3, 2011

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

“The ... invention relates to a cationic lipid having one or more biodegradable groups located in the mid- or distal section of a lipidic moiety of the cationic lipid,” the patent application's abstract states. “These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.”


Title: Methods and Compositions Relating to Polypeptides with RNase III Domains that Mediate RNA Interference

Application Number: 20120028312

Filed: Aug. 2, 2011

Lead Inventor: Lance Ford, Life Technologies

The invention, the patent application's abstract states, “concerns methods and compositions involving RNase III and polypeptides containing RNase III domains to generate RNA capable of triggering RNA-mediated interference in a cell. In some embodiments, the RNase III is from a prokaryote. RNase III activity will cleave a double-stranded RNA molecule into short RNA molecules that may trigger or mediate RNAi. Compositions of the invention include kits that include an RNase III domain-containing polypeptide.

“The ... invention further concerns methods using polypeptides with RNase III activity for generating RNA molecules that effect RNAi, including the generation of a number of RNA molecules to the same target,” the abstract adds.


Title: Multiple RNA Polymerase III Promoter Expression Constructs

Application Number: 20120028348

Filed: June 15, 2011

Lead Inventor: Catherine Pachuk, Nucleonics (Alnylam Pharmaceuticals)

The invention comprises “expression constructs [with] at least two different RNA polymerase III promoters, wherein each promoter is operably linked to a nucleic acid sequence encoding an RNA effector molecule,” the patent application's abstract states. “Further provided are expression constructs comprising multiple polymerase III promoters operably linked to sequences encoding short hairpin RNA molecules, which may comprise single and/or multiple fingers. The provided constructs are useful for in vivo delivery of RNA molecules effective in gene silencing, including of viral genes including HBV and HCV.”


Title: miR-29-Based Methods for the Diagnosis and Prognosis of Acute Myeloid Leukemia

Application Number: 20120028831

Filed: Oct. 7, 2011

Inventor: Carlo Croce, Ohio State University

The invention, the patent application's abstract states, “provides novel methods and compositions for the diagnosis, prognosis, and treatment of acute myeloid leukemia. The invention also provides methods of identifying anti-AML agents.”


Title: miR-182-, miR-191-, miR-199-Based Methods for the Diagnosis and Prognosis of Acute Myeloid Leukemia

Application Number: 20120028832

Filed: Oct. 7, 2011

Inventor: Carlo Croce, Ohio State University

The invention, the patent application's abstract states, “provides novel methods and compositions for the diagnosis, prognosis, and treatment of acute myeloid leukemia.”


Title: miR-25-Based Methods for the Diagnosis and Prognosis of Acute Myeloid Leukemia

Application Number: 20120028833

Filed: Oct. 7, 2011

Inventor: Carlo Croce, Ohio State University

The invention, the patent application's abstract states, “provides novel methods and compositions for the diagnosis, prognosis, and treatment of acute myeloid leukemia. The invention also provides methods of identifying anti-AML agents.”


Title: siRNA Targeting VEGFA and Methods for Treatment In Vivo

Application Number: 20120029051

Filed: July 26, 2011

Lead Inventor: Anja Smith, Alcon

“Vascular endothelial growth factor A is a chemical signal produced by cells that stimulates the growth of new blood vessels, and over-expression of VEGFA can lead to undesirable physiological conditions,” the patent application's abstract states. “Through the identification of new siRNA and modifications that improve the silencing ability of these siRNA in vivo, therapeutic compositions and methods have been invented to address the problems associated with this over-expression.”


Titles: Short Interfering Ribonucleic Acid for Oral Administration

Application Numbers: 20120029052; 20120029053

Filed: Aug. 26, 2011

Lead Inventor: Francois Jean-Charles Natt, Novartis

The inventions, the patent applications' abstracts state, comprise “short interfering ribonucleic acid for oral administration, [with the siRNA made of] 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: RNA Interference-Mediated Inhibition of GATA Binding Protein 3 Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20120029054

Filed: March 17, 2010

Lead Inventor: Victoria Pickering, Merck

The invention, the patent application's abstract states, “relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases, and conditions that respond to the modulation of GATA3 gene expression and/or activity, and/or modulate a GATA3 gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules ... that are capable of mediating or that mediate RNA interference against GATA3 gene expression.”


Title: Differentially Expressed microRNAs as Biomarkers for the Diagnosis and Treatment of Sjogren's Syndrome

Application Number: 20120029056

Filed: March 31, 2010

Lead Inventor: Ilias Alevizos, National Institutes of Health

The patent application, its abstract states, claims “a method of diagnosing a subject as having Sjogren's syndrome by measuring the level of at least one differentially expressed [miRNA] gene product. ... Also provided is a method of treating a patient with Sjogren's syndrome by administering to the patient a therapeutically effective amount of an agent that inhibits expression of a [miRNA] gene product that is up-regulated in the patient with Sjogren's syndrome relative to a control, or by administering to the patient a therapeutically effective amount of an isolated [miRNA] gene product that is down-regulated in the patient with Sjogren's syndrome relative to a control. A method of restoring salivary flow in a patient with Sjogren's syndrome is also provided.”


Title: RNA Sequence-Specific Mediators of RNA Interference

Application Number: 20120029061

Filed: Oct. 4, 2010

Lead Inventor: Thomas Tuschl, Max Planck Institute

The invention comprises 21- to 23-nucleotide-long RNA segments “and their use for specifically inactivating gene function,” the patent application's abstract states. “The use of these fragments, or chemically synthesized oligonucleotides of the same or similar nature, enables the targeting of specific mRNAs for degradation in mammalian cells, where the use of long dsRNAs to elicit RNAi is usually not practical, presumably because of the deleterious effects of the interferon response. This specific targeting of a particular gene function is useful in functional genomic and therapeutic applications.”

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