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IP Update: Recent RNAi- and microRNA-Related Patents and Patent Applications: Oct 13, 2011


Title: Lipid-Containing Formulations

Patent Number: 8,034,376

Filed: March 26, 2008

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

The invention, the patent's abstract states, comprises “compositions and methods useful in administering nucleic acid based therapies, for example association complexes such as liposomes and lipoplexes.”

Title: microRNA-Based Methods and Compositions for the Diagnosis, Prognosis, and Treatment of Acute Myeloid Leukemia

Patent Number: 8,034,560

Filed: Jan. 29, 2008

Inventor: Carlo Croce, Ohio State University

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

Title: RNAi Modulation of MLL-AF4 and Uses Thereof

Patent Number: 8,034,793

Filed: Feb. 4, 2010

Lead Inventor: Olaf Heidenreich, Alnylam Pharmaceuticals

“The invention relates to compositions and methods for modulating the expression of the MLL-AF4 fusion gene, and more particularly to the down-regulation of MLL-AF4 by chemically modified oligonucleotides,” according to the patent's abstract.

Title: iRNA Agents Targeting CCR5-Expressing Cells and Uses Thereof

Patent Number: 8,034,921

Filed: Nov. 21, 2007

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

“The invention relates to iRNA agents that preferably include a modification that targets CC chemokine receptor 5,” the patent's abstract states. “The invention also relates to methods of making and using such modified iRNA agents.”

Title: Compositions and Methods for Modulating Activity of Capped Small RNAs

Patent Number: 8,034,922

Filed: Aug. 21, 2009

Lead Inventor: Mark Kay, Stanford University

The invention comprises “compositions and methods for modulating transcription by RNA polymerases,” the patent's abstract states.

Title: Method of Using Gold Nanorods-siRNA Complexes

Patent Number: 8,035,016

Filed: Sept. 8, 2009

Lead Inventor: Earl Bergey, State University of New York

The patent, its abstract states, claims “methods and compositions for inhibiting expression of one or more target genes. The compositions contain RNA polynucleotides that can inhibit expression of a target gene via RNA interference [that are] electrostatically complexed with surface-functionalized gold nanorods. The RNA polynucleotides are not covalently bound to the surface-functionalized GNRs. The method involves inhibiting expression of a target gene in an individual. The method is performed by administering to the individual an effective amount of a composition containing surface-functionalized GNRs electrostatically complexed with RNA polynucleotides, such as siRNA, that can inhibit expression of the target gene via RNAi. The siRNA is not covalently bound to the surface-functionalized GNRs.”

Title: RNA Interference Target for Treating AIDS

Application Number: 20110243904

Filed: June 2, 2008

Lead Inventor: Tong Cheng, Xiamen University

The patent application, its abstract states, claims an “RNA interference target sequence targeting HIV for the treatment of AIDS. Based on the target sequence, recombinant expression vectors, packaging vectors, and cells were constructed, which express a siRNA ... [an] miRNA … a ribozyme, and/or an antisense oligonucleotide targeting HIV. Also provided is the use of the recombinant expression vectors, packaging vectors and recombinant cells in the manufacture of a medicament for the treatment of AIDS.”

Titles: RNA Sequence-Specific Mediators of RNA Interference

Application Numbers: 20110244446, 20110244568, 20110245318

Filed: Oct. 4, 2010

Lead Inventor: Thomas Tuschl, Max Planck Institute

The inventions relate to a “Drosophila in vitro system ... used to demonstrate that dsRNA is processed to RNA segments 21 [to] 23 nucleotides in length,” the patent applications' abstracts state. “Furthermore, when these 21 [to] 23 [nucleotide] fragments are purified and added back to Drosophila extracts, they mediate RNA interference in the absence of long dsRNA. Thus, these 21 [to] 23 [nucleotide] fragments are the sequence-specific mediators of RNA degradation.”

Title: microRNA Compositions and Methods for the Treatment of Myelogenous Leukemia

Application Number: 20110244024

Filed: Aug. 11, 2008

Lead Inventor: Florian Kuchenbauer, British Columbia Cancer Agency

“The invention provides methods, uses, kits, and compositions comprising a therapeutically effective amount of the microRNA miR-223 for treating myelogenous leukemia in a subject in need of such treatment,” the patent application's abstract states. “The invention further comprises methods encompassing the use of miR-223 for promoting the differentiation of a leukemia stem cell that is resistant to a differentiating agent, and a method of screening for candidate compounds capable of treating a myeloid leukemia by comparison of the therapeutic activity of the candidate compound with the therapeutic activity of miR-233.”

Title: siRNA-Mediated Gene Silencing

Application Number: 20110244561

Filed: Nov. 23, 2010

Lead Inventor: Beverly Davidson, University of Iowa

The invention is directed to “small interfering RNA molecules targeted against an allele of interest, and methods of using these siRNA molecules,” the patent application's abstract states.

Title: RNA Interference Suppression of Neurodegenerative Diseases and Methods of Uses Thereof

Application Number: 20110244562

Filed: Dec. 23, 2010

Lead Inventor: Beverly Davidson, University of Iowa

The invention is directed to “RNA interference molecules targeted against a nucleic acid sequence that encodes polyglutamine repeat diseases, and methods of using these RNAi molecules,” the patent application's abstract states.

Title: RNAi-Mediated Inhibition of Rho Kinase for Treatment of Ocular Disorders

Application Number: 20110245319

Filed: Nov. 5, 2010

Lead Inventor: Jon Chatterton, Alcon

“RNA interference is provided for inhibition of Rho kinase mRNA expression for treating patients with ocular disorders, particularly for treating intraocular pressure, ocular hypertension, and glaucoma,” the patent application's abstract states. “Rho kinase mRNA targets include mRNA for ROCK1 and ROCK2.”

Title: Nuclease-Resistant Double-Stranded Ribonucleic Acid

Application Number: 20110245320

Filed: March 2, 2011

Lead Inventor: Hans-Peter Vornlocher, Alnylam Pharmaceuticals

“This invention relates to modified double-stranded oligoribonucleic acid having improved stability in cells and biological fluids, and methods of making and identifying dsRNA having improved stability, and of using the dsRNA to inhibit the expression or function of a target gene,” the patent application's abstract states.

The Scan

Germline-Targeting HIV Vaccine Shows Promise in Phase I Trial

A National Institutes of Health-led team reports in Science that a broadly neutralizing antibody HIV vaccine induced bnAb precursors in 97 percent of those given the vaccine.

Study Uncovers Genetic Mutation in Childhood Glaucoma

A study in the Journal of Clinical Investigation ties a heterozygous missense variant in thrombospondin 1 to childhood glaucoma.

Gene Co-Expression Database for Humans, Model Organisms Gets Update

GeneFriends has been updated to include gene and transcript co-expression networks based on RNA-seq data from 46,475 human and 34,322 mouse samples, a new paper in Nucleic Acids Research says.

New Study Investigates Genomics of Fanconi Anemia Repair Pathway in Cancer

A Rockefeller University team reports in Nature that FA repair deficiency leads to structural variants that can contribute to genomic instability.