Title: Polymeric Carrier for Delivery of Small Interfering RNA
Patent Number: 7,740,880
Filed: March 3, 2006
Lead Inventor: Won Jong Kim, University of Utah
"A carrier for delivering small interfering RNA into cells includes a cholesterol residue covalently bonded to oligoarginine," the patent's abstract states. "Mixing the siRNA with the carrier produces a complex-containing composition … [while] contacting a cell with the complex-containing composition results in delivery of the siRNA into the cell. Delivery of an siRNA targeted to vascular endothelial growth factor is a treatment for cancer. Methods of making the carrier and complex are also disclosed."
Title: Therapeutic Uses of Inhibitors of RTP801
Patent Number: 7,741,299
Filed: Aug. 16, 2005
Lead Inventor: Elena Feinstein, Quark Pharmaceuticals (Silence Therapeutics)
The invention, the patent's abstract states, "provides novel molecules, compositions, methods, and uses for treating microvascular disorders, eye diseases, and respiratory conditions based upon inhibition of the RTP801 gene and/or protein."
Title: Regulation of Oncogenes by microRNAs
Patent Number: 7,741,306
Filed: Oct. 22, 2007
Lead Inventor: Frank Slack, Yale University
The patent, its abstract states, claims "naturally occurring miRNAs that regulate human oncogenes and methods of use thereof. … Suitable nucleic acids for use in the methods and compositions described herein include, but are not limited to, pri-miRNA, pre-miRNA, mature miRNA , or fragments of variants thereof that retain the biological activity of the mature miRNA and DNA encoding a pri-miRNA, pre-miRNA, mature miRNA, fragments, or variants thereof; or regulatory elements of the miRNA. The compositions are administered to a subject prior to administration of a cytotoxic therapy in an amount effective to sensitize cells or tissues to be treated to the effects of the cytotoxic therapy."
Title: siRNA Targeting Gremlin
Patent Number: 7,741,470
Filed: May 28, 2009
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 CKSF1B1."
Title: Compositions and Methods for Selective Inhibition of VEGF
Application Number: 20100151007
Filed: Dec. 4, 2009
Lead Inventor: Phillip Frost, Opko Health
The invention, the patent application's abstract states, comprises "siRNA compositions and methods useful for inhibiting expression of vascular endothelial growth factor isoforms. Such compositions and methods further involve siRNA capable of selectively targeting angiogenic VEGF isoforms while selectively sparing anti-angiogenic isoforms. Diseases which involve angiogenesis stimulated by over-expression of VEGF, such as diabetic retinopathy, age-related macular degeneration, and many types of cancer, can be treated by administering small interfering RNAs."
Title: siRNA Targeting BCL2L1
Application Number: 20100152064
Filed: Jan. 15, 2010
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."
Title: RNA Interference-Mediated Inhibition of Cyclic Nucleotide Type 4 Phosphodiesterase Gene Expression Using Short Interfering Nucleic Acid
Application Number: 20100152278
Filed: May 2, 2008 PCT Filed: May 2, 2008
Lead Inventor: Vasant Jadhav, Sirna Therapeutics (Merck)
The invention, the patent application's abstract states, "relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases, and conditions that respond to the modulation of cyclic nucleotide type 4 phosphodiesterase gene expression and/or activity, including PDE4B1, PDE4B2, and PDE4B3 gene expression and/or activity. The … invention is also directed to compounds, compositions, and methods relating to traits, diseases, and conditions that respond to the modulation of expression and/or activity of genes involved in cyclic nucleotide type 4 phosphodiesterase gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases, and conditions, including but not limited to IL-6, IL-1, IL-8, IL-15, TNF-alpha, and matrix metalloproteinases such as MMP-1, MMP -2, MMP-3, MMP-9, and MMP-12.
"Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules … capable of mediating RNA interference against cyclic nucleotide type 4 phosphodiesterase gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle formulations of such small nucleic acid molecules," the abstract adds. The invention also relates to "small nucleic acid molecules … that can inhibit the function of endogenous RNA molecules or RNAi pathway components … or that can inhibit the function of RISC to modulate PDE4B gene expression. ... Such small nucleic acid molecules are useful, for example, in providing compositions to prevent, inhibit, or reduce inflammatory, respiratory, and autoimmune diseases, traits, and conditions, and/or other disease states associated with PDE4B gene expression or activity in a subject or organism."
Title: RNAi Inhibition of Serum Amyloid A for Treatment of Glaucoma
Application Number: 20100152279
Filed: Feb. 25, 2010
Lead Inventor: Abbot Clark, Alcon
The patent application, its abstract states, claims "RNA interference … for inhibition of serum amyloid A mRNA expression in glaucomas involving SAA expression."
Title: Modulation of SID-1 Expression
Application Number: 20100152280
Filed: Oct. 24, 2008
Lead Inventor: Kenneth Dobie, Isis Pharmaceuticals
The patent application, its abstract states, claims "compounds, compositions, and methods … for modulating the expression of SID-1. The compositions comprise oligonucleotides targeted to nucleic acid encoding SID-1. Methods of using these compounds for modulation of SID-1 expression and for diagnosis and treatment of diseases and conditions associated with expression of SID-1 are provided."
Title: Use of Double-Stranded RNA Hairpin Duplexes in Gene Silencing
Application Number: 20100152282
Filed: Jan. 29, 2008 PCT Filed: Jan. 29, 2008
Lead Inventor: Patrick Arbuthnot, University of Witwatersrand
The invention, the patent application'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; and 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 duplex, it adds, is capable of "inhibiting expression of the target gene. RNA polynucleotides and … duplexes transcribed by the DNA polynucleotide are also described, as is a method for producing the … duplexes," which can be used in gene silencing.
Title: siRNA Kinase and Methods of Use
Application Number: 20100154072
Filed: May 29, 2007 PCT Filed: May 29, 2007
Lead Inventor: Javier Martinez, Boehringer Ingelheim
"The invention is based on the finding that Clp1 has the activity of an RNA kinase," the patent application's abstract states. "Clp1 is therefore useful as an enhancer of siRNA activity. It can be used … in kits or expressed in cell lines. Clp1 transgenic and Clp1 knock-out non-human animals are useful for studying the function of siRNAs. Clp1 is also useful in gene therapy to enhance the efficacy of therapeutic siRNAs."