Title: Materials and Methods for Treatment of Allergic Disease
Filed: Feb. 25, 2005 PCT Filed: Feb. 25, 2005
Lead Inventor: William Walker, Allerna Therapeutics
The invention, the patent's abstract states, comprises "siRNA molecules … [that] target the transcription factor STAT6 and repress the expression of STAT6 mRNA, STAT6 protein, and STAT6 function in lung cells. The use of STAT6 specific siRNA molecules in the treatment of allergic diseases of the respiratory tract such as asthma and rhinitis is disclosed."
Title: Cancer-Related Nucleic Acids
Filed: May 4, 2007 PCT Filed: May 4, 2007
Lead Inventor: Ranit Aharonov, Rosetta Genomics
The patent application, its abstract states, claims "polynucleotides … [namely] miRNAs, miRNA precursors, and associated nucleic acids … associated with specific types of cancers.
The application also describes "methods and compositions … that can be used for diagnosis, prognosis, and treatment of various cancers," the abstract adds.
Title: sVEGFR-2 and Its Role in Lymphangiogenesis Modulation
Filed: Dec. 18, 2008
Lead Inventor: Jayakrishna Ambati, University of Kentucky
The invention, the patent application's abstract states, relates to "nucleic acid molecules comprising a nucleotide sequence of sVEGF-2, proteins encoded by those sequences, and antibodies that bind to the protein. Also disclosed are methods for inhibiting or enhancing expression or activity of sVEGFR-2 and methods for inhibiting graft rejection, particularly cornea graph rejection … [as well as] methods for inhibiting lymphangiogenesis and lymphatic endothelial cell proliferation by administering an effective amount of sVEGFR-2 and methods for treating lymphedema by inhibiting the activity of sVEGFR-2."
The application specifically claims siRNAs capable of reducing levels of sVEGFR-2.
Title: RNA Silencing Compositions and Methods for the Treatment of Huntington's Disease
Filed: Jan. 5, 2009
Lead Inventor: Neil Aronin, University of Massachusetts
The invention "relates to the discovery of an effective treatment for a variety of Huntington's disease," the patent application's abstract states. "The … invention utilizes RNA silencing technology against single nucleotide polymorphisms in the huntingtin gene encoding the dominant, gain-of-function mutant Huntington protein, thereby resulting in an effective treatment for the gain-of-function disease."
Title: Cell-Penetrating Peptide Conjugates for Delivering Nucleic Acids into Cells
Filed: June 16, 2008
Lead Inventor: Bertrand Alluis, Diatos
"The invention provides cell penetrating peptide-nucleic acid conjugates having the formula P-L-N, wherein P is a cell penetrating peptide, N is a nucleic acid, preferably an oligonucleotide and more preferably a siRNA, and L is a hydrophilic polymer, preferably a polyethylene glycol-based linker linking P and N together," the patent application's abstract states. "Compositions, methods of use and methods for producing such conjugates are also disclosed."
Title: Identification of JAK/STAT Pathway-Modulating Genes by Genome-Wide RNAi Screening
Filed: June 14, 2006 PCT Filed: June 14, 2006
Lead Inventor: Michael Boutros, German Cancer Research Center
The invention, according to the patent application's abstract, "relates to a method for identifying a compound capable of modulating the activity of the JAK/STAT pathway and to the use of different JAK/STAT pathway components as a target for the modulation of the activity of the JAK/STAT pathway. Moreover, the … invention is concerned with a method for modulating the activity of the JAK/STAT pathway … [and] pertains to a pharmaceutical composition and to the use of different JAK/STAT pathway components and/or effector molecules thereof for the manufacture of such composition for the diagnosis, prevention, or treatment of a JAK/STAT pathway-associated disorder."
Title: RNA Sequence-Specific Mediators of RNA Interference
Filed: July 19, 2007
Lead Inventor: Thomas Tuschl, Max Planck
The invention, the patent application's abstract states, "relates to a Drosophila in vitro system [that] was used to demonstrate that dsRNA is processed to RNA segments 21-23 nucleotides in length. Furthermore, when these 21-23 [nucleotide] fragments are purified and added back to Drosophila extracts, they mediate RNA interference in the absence of long dsRNA. Thus, these 21-23 [nucleotide] fragments are the sequence-specific mediators of RNA degradation.
"A molecular signal, which may be their specific length, must be present in these 21-23 [nucleotide] fragments to recruit cellular factors involved in RNAi," the abstract notes. "This … invention encompasses these 21-23 [nucleotide] fragments and their use for specifically inactivating gene function. 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."
Title: siRNA Delivery into Mammalian Nerve Cells
Filed: Jan. 10, 2008
Lead Inventor: Gordon Mitchell, University of Wisconsin, Madison
The invention, the patent application's abstract states, "relates to methods of affecting expression of a target gene, suitably brain-derived neurotrophic factor or related genes, in a nerve cell in the central nervous system of a mammal. The method includes formulating and delivering an siRNA composition to a target site on the mammal to affect expression of the target gene in the nerve cell, wherein the target site is cerebrospinal space or muscle tissue innervated by a nerve cell, to down-regulate the target gene. Also disclosed are kits for use in practicing the novel methods of in vivo siRNA delivery into target cells and gene regulation."
Title: Interfering RNA Molecules
Filed: Aug. 28, 2008
Lead Inventor: Klaus Giese, Silence Therapeutics
The invention is related to a "ribonucleic acid comprising a [blunt-ended] double-stranded structure whereby … the first strand comprises a first stretch of contiguous nucleotides … at least partially complementary to a target nucleic acid," the patent application's abstract states. "The second strand comprises a second stretch of contiguous nucleotides … at least partially identical to a target nucleic acid."
Title: Processes and Reagents for Sulferization of Oligonucleotides
Filed: Jan. 9, 2009
Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals
The invention, the patent application's abstract states, "relates to processes and reagents for oligonucleotide synthesis and purification. One aspect of the … invention relates to compounds useful for activating phosphoramidites in oligonucleotide synthesis … [while another relates] to a method of preparing oligonucleotides via the phosphoramidite method using an activator of the invention.
"Another aspect of the … invention relates to sulfur-transfer agents," it adds. "In a preferred embodiment, the sulfur-transfer agent is a 3-amino-1,2,4-dithiazolidine-5-one. Another aspect of the … invention relates to a method of preparing a phosphorothioate by treating a phosphite with a sulfur-transfer reagent of the invention. In a preferred embodiment, the sulfur-transfer agent is a 3-amino-1,2,4-dithiazolidine-5-one."
According to the abstract, another aspect of the invention relates to "compounds that scavenge acrylonitrile produced during the deprotection of phosphate groups bearing ethylnitrile protecting groups. In a preferred embodiment, the acrylonitrile scavenger is a polymer-bound thiol. Another aspect of the … invention relates to agents used to oxidize a phosphite to a phosphate. In a preferred embodiment, the oxidizing agent is sodium chlorite, chloramine, or pyridine-N-oxide."
Yet another aspect of the invention relates to "methods of purifying an oligonucleotide by annealing a first single-stranded oligonucleotide and second single-stranded oligonucleotide to form a double-stranded oligonucleotide, and subjecting the double-stranded oligonucleotide to chromatographic purification," according to the abstract. "In a preferred embodiment, the chromatographic purification is high-performance liquid chromatography."
Title: Cre-Lox-Based Gene-Knockdown Constructs and Methods of Use Thereof
Filed: March 12, 2008
Lead Inventor: Patrick Stern, Massachusetts Institute of Technology
The invention "relates to vectors, compositions, and methods for conditional, Cre-Lox-regulated RNA interference," the patent application's abstract states. "The vectors allow for spatial and temporal control of miRNA expression in vivo."