Title: Pharmaceutical Composition Comprising Anti-miRNA Antisense Oligonucleotide
Patent Number: 8,163,708
Filed: March 30, 2009
Lead Inventor: Joacim Elmen, Santaris Pharma
The invention, the patent's abstract states, relates to ”pharmaceutical compositions comprising short single-stranded oligonucleotides, of length of between 8 and 26 nucleobases, [and] which are complementary to human microRNAs selected from the group consisting of miR-19b, miR-21, miR-122a, miR-155 and miR-375. The short oligonucleotides are particularly effective at alleviating miRNA repression in vivo. It is found that the incorporation of high affinity nucleotide analogues into the oligonucleotides results in highly effective anti-microRNA molecules which appear to function via the formation of almost irreversible duplexes with the miRNA target, rather than RNA cleavage-based mechanisms such as [ones] associated with RNaseH or RISC.”
Title: TAK1-D-Mediated Induction of Cell Death in Human Cancer Cells by Specific Sequence Short Double-Stranded RNAs
Patent Number: 8,163,709
Filed: Jan. 28, 2009
Lead Inventor: Reinhard Kodyn, University of Texas
“The splicing variant D of the TAK1 gene is activated by short double-stranded RNAs in a sequence-specific manner,” the patent's abstract states. “Activation of TAK1-D leads to the downstream activation of the p38 MAPK and of SAPK/JNK, but not the NF-kappa B pathway. The current invention therefore provides a method of inducing apoptosis and/or cell cycle arrest in a cancer cell comprising contacting said cell with an agonist of Tak1-D function.
“The invention further provides a method of modulating inflammation and the treatment of cancer by the administration of an agonist of Tak1-D function or expression,” the abstract states. “In yet another aspect, the invention provides a method of inducing p38 MAPK and SAPK/JNK signaling in a cell comprising contacting said cell with an agonist of Tak1-D function or expression.”
Title: Compositions and Methods for Inhibiting Expression of the HAMP Gene
Patent Number: 8,163,711
Filed: April 9, 2010
Lead Inventor: Tomoko Nakayama, Alnylam Pharmaceuticals
The invention, the patent's abstract states, “relates to a double-stranded ribonucleic acid for inhibiting the expression of the HAMP gene comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19 [to] 25 nucleotides in length, and which is substantially complementary to at least a part of the HAMP gene.
“The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by HAMP gene expression, and the expression of the HAMP gene using the pharmaceutical composition,” the abstract adds.
Title: Devices, Systems, and Methods for Improving Memory and/or Cognitive Function through Brain Delivery of siRNA
Application Number: 20120093916
Filed: Sept. 23, 2011
Inventor: William Kaemmerer, Medtronic
The invention, the patent application's abstract states, “relates to devices, systems, and methods for improving memory and/or cognitive function by brain delivery of compositions of small interfering RNA or vectors containing the DNA encoding for small interfering RNA. Such compositions can be administered using devices, systems, and methods for direct delivery of the compositions to the brain, or using devices, systems, methods of delivery, and compositions that deliver small interfering RNA or vectors containing the DNA encoding the small interfering RNA across the blood-brain barrier. The ... invention also provides valuable small interfering RNA vectors and methods for reduction of BACE1 levels in the hippocampus, cerebral cortex, or other regions of the brain that have beneficial effects on improving memory and/or cognitive function in a subject.”
Title: Novel Lipids and Compositions for the Delivery of Therapeutics
Application Number: 20120095075
Filed: Aug. 16, 2011
Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals
The invention, the patent application's abstract states, comprises “lipids that are advantageously used in lipid particles for the in vivo delivery of therapeutic agents to cells.”
Title: Compositions and Methods for Inhibiting Expression of Huntingtin Gene
Application Number: 20120095076
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 [huntingtin] gene,” the patent application'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 and Compositions Related to Modified Guanine Bases for Controlling Off-Target Effects in RNA Interference
Application Number: 20120095077
Filed: March 23, 2010
Lead Inventor: Cynthia Burrows, University of Utah
The invention, the patent application's abstract states, comprises “compositions and methods related to modified nucleobases. Also disclosed are compositions and methods related to modified interfering RNAs … [as well as] compositions and methods related to modified guanine bases for controlling off-target effects in RNA interference.”
Title: Methods for Producing Interfering RNA Molecules in Mammalian Cells and Therapeutic Uses for Such Molecules
Application Number: 20120095080
Filed: Dec. 13, 2011
Lead Inventor: John Rossi, City of Hope
The patent application, its abstract states, claims “methods for producing interfering RNA molecules in mammalian cells. ... Therapeutic uses for the expressed molecules, including inhibiting expression of HIV, are also provided.”
Title: Use of Interfering RNA for Treating an HIV Infection
Application Number: 20120095082
Filed: June 28, 2010
Lead Inventor: Vincent Calvez, Pierre and Marie Curie University
The invention relates to “interfering RNAs, in particular miRNAs, capable of specifically blocking the replication of a strain of the HIV virus that is resistant to an antiretroviral compound, and use thereof for treating infections by this type of virus,” according to the patent application's abstract.
Title: Compositions and Methods for the Specific Inhibition of Gene Expression by Nucleic Acid Containing a Dicer Substrate and a Receptor-Binding Region
Application Number: 20120095200
Filed: Nov. 17, 2011
Inventor: Bob Brown, Dicerna Pharmaceuticals
“The invention features compositions and methods that are useful for reducing the expression or activity of a specified gene in a eukaryotic cell, involving contacting a cell with an isolated nucleic acid containing a Dicer substrate and a receptor binding region in an amount effective to reduce expression of a target gene in a cell,” the patent application's abstract states.