Title: iRNA Agents Targeting VEGF
Patent Number: 8,293,719
Filed: March 8, 2011
Lead Inventor: Antonin de Fougerolles, Alnylam Pharmaceuticals
The patent, its abstract states, claims “compounds, compositions, and methods useful for modulating the expression of vascular endothelial growth factor such as by the mechanism of RNA interference. The compounds and compositions include iRNA agents that can be unmodified or chemically-modified.”
Title: siRNA Targeting Beta Secretase
Patent Number: 8,293,887
Filed: July 1, 2011
Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)
“Efficient sequence-specific gene silencing is possible through the use of siRNA technology,” the patent's abstract states. “By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for BACE.”
Title: Use of Double-Stranded RNA Hairpin Duplexes in Gene Silencing
Patent Number: 8,293,974
Filed: Jan. 29, 2008
Lead Inventor: Patrick Arbuthnot, University of Witwatersrand
The invention, the patent's abstract states, comprises “a DNA polynucleotide ... having a modified sequence of a target gene, wherein any one type of nucleotide in the target gene sequence has been chemically modified to another type of nucleotide.” The polynucleotide also has “a complementary sequence of the unmodified target gene wherein either one of the modified sequence or the complementary sequence is in a reverse orientation to the other sequence and ... the RNA sequence transcribed from the DNA polynucleotide forms a duplex between the modified sequence and the complementary sequence so that a long double-stranded RNA duplex forms between the modified and complementary sequences with base pair mismatches where the nucleotides have been modified.
The dsRNA is “capable of inhibiting expression of the target gene … [and] can be used in gene silencing,” the abstract adds.
Title: Multiplex Dicer-Substrate RNA Interference Molecules Having Joining Sequences
Application Number: 20120263738
Filed: Feb. 24, 2012
Inventor: Bob Brown, Dicerna Pharmaceuticals
The invention, the patent application's abstract states, is “based, in part, upon the insight that compound DsiRNA agents can be generated using site-specific RNase H-cleavable double-stranded nucleic acid regions to attach ... one DsiRNA moiety to another DsiRNA moiety and/or one DsiRNA moiety to a functional group and/or payload. Because such double-stranded nucleic acid joining sequences are site-specifically RNase H-cleavable, the bi-functional molecule is cleaved into DsiRNAs bearing terminal ends that orient dicer cleavage.
“Detrimental impacts of administering a single double-stranded nucleic acid RNAi agent of longer than 30-35 nucleotides is minimized, as once administered to a subject or RNase H-containing cell, RNase H cleavage produces a shortened, active DsiRNA agents,” the abstract adds. “The invention provides bifunctional DsiRNA agents that are joined by double-stranded DNA extension joining sequences, which do not provoke RNase H cleavage.”
Title: microRNA Affinity Assay and Uses Thereof
Application Number: 20120264619
Filed: Oct. 18, 2010
Lead Inventor: Simon Spivack, Albert Einstein College of Medicine
The invention provides “methods and kits for determining which microRNAs bind to a target mRNA where the methods comprise … creating a bait sequence from the target mRNA, where the bait sequence comprises a label that binds to a binding agent; adding a mixture of microRNAs to the bait sequence; separating the microRNAs that bind to the bait sequence from those microRNAs that do not bind; and identifying the microRNAs that bind to the bait sequence, wherein the microRNAs identified are those that bind to the target mRNA,” according to the patent application's abstract.
Title: Medicament for the Treatment of Prevention of Liver Failure
Application Number: 20120264805
Filed: April 15, 2011
Lead Inventor: Amar Sharma, Hannover Medical School
The invention, the patent application's abstract states, comprises “small inhibitory RNA molecules … that through RNA interference reduce or prevent expression of the p53 up-regulated modulator of apoptosis. The siRNA molecules can be administered as a medicament to a patient suffering from an impaired liver function or from liver damage for the treatment of a functionally impaired liver, for delaying a deterioration of liver function, and/or for prevention of liver failure, especially in patients who suffer from a critical impairment of damage to the liver.”
Title: Method for Introducing siRNA into Cells by Photochemical Internalization
Application Number: 20120264807
Filed: Jan. 12, 2012
Lead Inventor: Sigurd Boe, PCI Biotech
The invention relates to “introducing an siRNA molecule into the cytosol of a cell [by] contacting said cell with an siRNA molecule, a carrier, and a photosensitizing agent; and irradiating the cell with light of a wavelength effective to activate the photosensitizing agent. ... Cells or a population of cells obtainable by the method, a composition containing an siRNA molecule and the carrier molecule, kits and therapeutic uses of the above are also provided.”
Title: RNAi-Mediated Inhibition of Histamine Receptor H1-Related Conditions
Application Number: 20120264809
Filed: June 11, 2012
Lead Inventor: John Yanni, Alcon
The patent application, its abstract states, claims “RNA interference … for inhibition of histamine receptor H1 mRNA expression, in particular, for treating patients having an HRH1-related condition or at risk of developing an HRH1-related condition such as allergic conjunctivitis, ocular inflammation, dermatitis, rhinitis, asthma, or allergy.”
Title: Compositions and Methods for Enhancing Cellular Uptake and Intracellular Delivery of Lipid Particles
Application Number: 20120264810
Filed: Sept. 22, 2010
Lead Inventor: Paulo Lin, University of British Columbia
The patent application, its abstract states, claims “compositions, methods, and compounds useful for enhancing the uptake of a lipid particle [in] a cell. … In particular embodiments, the methods of the invention include contacting a cell with a lipid particle and a compound that binds a Na+/K+ ATPase to enhance uptake of the lipid particle. ... Related compositions useful in practicing methods include lipid particles comprising a conjugated compound that enhances uptake of the lipid particles. ... The methods and compositions are useful in delivering a therapeutic agent to a cell.”
Title: siRNA Targeting Cyclin-Dependent Kinase Inhibitor 1B
Application Number: 20120264813
Filed: June 25, 2012
Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)
“Efficient sequence-specific gene silencing is possible through the use of siRNA technology,” the patent application's abstract states. “By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for CDKN1B.”