Title: Liposomal Formulations Comprising Dihydrosphingomyelin and Uses Thereof
Patent Number: 7,811,602
Filed: May 16, 2005
Lead Inventor: Pieter Cullis, Tekmira Pharmaceuticals
The invention "includes novel liposomes comprising dihydrosphingomyelin," the patent's abstract states. "The invention also includes compositions comprising these liposomes and a therapeutic agent, in addition to methods and kits for delivering a therapeutic agent or treating a disease [such as] a cancer using these compositions."
Title: Antisense microRNA and Uses Therefore
Patent Number: 7,812,003
Filed: Aug. 1. 2008
Lead Inventor: Stephen Safe, Texas A&M Health Center
The patent, its abstract states, claims "methods to suppress specificity protein activity in a cell associated with a cell proliferative disease. The methods are effective to inhibit a microRNA in the cell using an antisense microRNA oligonucleotide, which results in an increase in expression of a specificity protein suppressor gene thereby inducing [specificity protein] degradation, apoptosis, or growth arrest by releasing inhibitors of G2/M or inhibition. Also provided are methods of treating a cancer using the antisense microRNA oligonucleotide.
"In addition, the … invention provides antisense microRNA-27a oligonucleotides useful in the methods described herein," the abstract adds.
Title: 2'-Fluoro-Substituted Oligomeric Compounds and Compositions for Use in Gene Modulations
Patent Number: 7,812,149
Filed: Nov. 4, 2003
Lead Inventor: Thazha Prakash, Isis Pharmaceuticals
The invention, the patent's abstract states, relates to "compositions comprising first and second oligomers … wherein at least a portion of the first oligomer is capable of hybridizing with at least a portion of the second oligomer; at least a portion of the first oligomer is complementary to and capable of hybridizing to a selected target nucleic acid; and at least one of the first or second oligomers includes a modified sugar and/or backbone modification.
"In some embodiments the modification is a 2'-F substituent group on a sugar moiety," the abstract states. "Oligomer/protein compositions are also provided comprising an oligomer complementary to and capable of hybridizing to a selected target nucleic acid and at least one protein comprising at least a portion of an RNA-induced silencing complex, wherein at least one nucleoside of the oligomer has a modified sugar and/or backbone modification."
Title: RNAi Modulation of Aha and Therapeutic Uses Thereof
Patent Number: 7,812,150
Filed: May 18, 2007
Lead Inventor: Rainer Constien, Alnylam Pharmaceuticals
"The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of an Aha 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 an Aha gene," the patent'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 Aha1 expression and the expression of an Aha gene using the pharmaceutical composition; and methods for inhibiting the expression of an Aha gene in a cell."
Title: RNAi Therapeutic for Respiratory Virus Infection
Application Number: 20100254945
Filed: April 7, 2006 PCT Filed: April 7, 2006
Lead Inventor: Qing Ge, Nastech Pharmaceutical (Marina Biotech)
The patent application, its abstract states, claims "a double-stranded siRNA molecule that inhibits production of a respiratory virus, wherein each strand of said siRNA molecule is about 15 to about 50 nucleotides, and wherein one strand of said siRNA molecule comprises a nucleic acid sequence identical to a conserved site, or a variant thereof, within the nucleic acid sequence of the respiratory virus, and uses thereof."
Title: Remedy for the Treatment of Cardiovascular Diseases or Disorders
Application Number: 20100255041
Filed: Oct. 5, 2009
Lead Inventor: Martin Bergmann, Max Delbruck Center for Molecular Medicine
The patent application, its abstract states, claims "the use of inhibitors of beta-catenin expression or activity or modulators down-regulating beta-catenin expression or activity for the treatment of cardiovascular diseases or disorders and their use in the treatment of cardiovascular diseases and disorders, such as heart failure syndrome. The use of those agents resulted in particular in cardiomyocyte differentiation of endogenous cardiac stem cells."
Title: Efficient Nuclear Delivery of Antisense Oligonucleotides or siRNA In Vitro or In Vivo by Nano-Transforming Polymersomes
Application Number: 20100255112
Filed: Nov. 14, 2007 PCT Filed: Nov. 14, 2007
Lead Inventor: Dennis Discher, University of Pennsylvania
The invention, the patent application's abstract states, comprises "a biocompatible polyethylene oxide-based polymersome system for the delivery of oligonucleotides, including antisense RNA, siRNA, and RNAi, to a cell or tissue target, and method of use therefore, wherein the method comprises encapsulating the oligonucleotide in a biodegradable neutral, nano-transforming polymersome delivery vehicle and delivering the encapsulated oligonucleotide to the cell or tissue target in vitro or in vivo, particularly for treating a disease, such cancer or cellular hyperproliferation.
"The degradable polymersome and the oligonucleotides stably encapsulated therein are taken up passively by cells and delivered into endolysosomes, wherein the polymersomes decompose at a known rate at a known pH, thereby releasing encapsulated oligonucleotides in a controlled manner within the cell and facilitating delivery of antisense oligonucleotide or siRNA or RNAi into the nucleus of the cell target," the abstract adds.
Title: RNAi Modulation of MLL-AF4 and Uses Thereof
Application Number: 20100256218
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 application's abstract.
Title: microRNA Scaffolds, Non-Naturally Occurring microRNAs, and Methods for Optimizing Non-Naturally Occurring microRNAs
Application Number: 20100256222
Filed: May 22, 2008 PCT Filed: May 22, 2008
Lead Inventor: Melissa Kelley, Dharmacon (Thermo Fisher Scientific)
The invention, the patent application's abstract states, comprises "non-naturally occurring miR-196a-2 miRNAs and non-naturally occurring miR-204 miRNAs. The non-naturally occurring miRNAs of the disclosure have mature strand sequences distinct from their endogenous counterparts. The disclosure also provides methods of selecting mature strand sequences that function optimally in non-naturally occurring miR-196a-2 miRNAs. The methods and compositions of the disclosure may be used to mediate gene silencing via the RNAi pathway."
Title: Oligonucleotides for Modulating Target RNA Activity
Application Number: 20100256223
Filed: June 13, 2008 PCT Filed: June 13, 2008
Inventor: Thorleif Moller, Mirrx Therapeutics
The invention "relates to oligonucleotides for modulation of target RNA activity," the patent application's abstract states. "Thus, the invention provides oligonucleotides that bind to microRNA binding sites of target RNA. The oligonucleotides may activate RNase H or RNAi. In a preferred embodiment, the oligonucleotides prevent a microRNA from binding to its binding site of the target RNA and thereby prevent the microRNA from regulating the target RNA. Such oligonucleotides have uses in research and development of new therapeutics."
Title: microRNA-Based Methods and Compositions for the Diagnosis and Treatment of Colon Cancer-Related Diseases
Application Number: 20100257618
Filed: July 12, 2007 PCT Filed: July 12, 2007
Lead Inventor: Carlo Croce, Ohio State University
The invention, the patent application's abstract states, "provides novel methods and compositions for the diagnosis and treatment of colon cancers. The invention also provides methods of identifying inhibitors of tumorigenesis."