Title: Enhanced Biotherapeutic Production Using Inhibitory RNA
Patent Number: 8,273,722
Filed: July 11, 2008
Lead Inventor: James Ladine, Dharmacon (Thermo Fisher Scientific)
The invention, the patent's abstract states, comprises “compositions, kits, systems, equipment, and protocols [that] utilize synthetic siRNA having a delivery-facilitating moiety in improved bioprocesses that enhance the production of biomaterials. The siRNA can target genes associated with ... deleterious vector derived genes; genes that confer non-optimal growth or differentiation properties to the cells; genes that can influence heterogeneity or [the] post-translational modification pattern of the desirable gene product; genes that highly express non-desired proteins; genes that express proteins which interfere with purification of the desired protein; and other genes that can interfere with the bioprocess.”
Title: RNA Interference-Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid
Patent Number: 8,273,866
Filed: Nov. 24, 2003
Lead Inventor: James McSwiggen, Merck
The invention, the patent's abstract states, “concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules … capable of mediating RNA interference against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.”
Title: Transducible Delivery of siRNA by dsRNA Binding Domain Fusions to PTD/CPPS
Patent Number: 8,273,867
Filed: Feb. 9, 2007
Lead Inventor: Steven Dowdy, University of California
The invention relates to “fusion polypeptides and constructs useful in delivering anionically charged nucleic acid molecules including diagnostics and therapeutics to a cell or subject,” according to the patent's abstract. “The fusion constructs include a protein transduction domain and a nucleic acid binding domain, or a protein transduction domain and a nucleic acid that is coated with one or more nucleic acid binding domains sufficient to neutralize an anionic charge on the nucleic acid. Also provided are methods of treating disease and disorders such as cell proliferative disorders.”
Title: Compositions and Methods for Inhibiting Viral Replication
Patent Number: 8,273,868
Filed: Dec. 4, 2009
Lead Inventor: John Matthias, Alnylam Pharmaceuticals
The invention relates to “a double-stranded ribonucleic acid having a nucleotide sequence [that] is less than 30 nucleotides in length and … substantially identical to at least a part of a 3'-untranslated region of a [plus] strand RNA virus, such as HCV, as well as pharmaceutical compositions comprising the dsRNA, together with a pharmaceutically acceptable carrier,” the patent's abstract states. “The pharmaceutical compositions are useful for treating infections and diseases caused by the replication or activity of the [plus] strand RNA virus, as well as methods for inhibiting viral replication.”
Title: Lipid Formulated dsRNA Targeting the PCSK9 Gene
Patent Number: 8,273,869
Filed: June 15, 2010
Lead Inventor: Kevin Fitzgerald, Alnylam Pharmaceuticals
“This invention relates to composition and methods using lipid formulated siRNA targeted to a PCSK9 gene,” the patent's abstract states.
Title: Compositions and Methods for Inhibiting Expression of a Mutant Gene
Patent Number: 8,273,870
Filed: July 6, 2011
Lead Inventor: Roland Kreutzer, Alnylam Pharmaceuticals
The invention, the patent's abstract states, relates to “a double-stranded ribonucleic acid for inhibiting the expression of a mutant gene, comprising a first strand that has a complementary region that is complementary to at least a portion of an RNA transcript of at least part of the mutant target gene and a second strand of the dsRNA complementary or substantially complementary to the first strand. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target mutant gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene.”
Title: Structurally Designed shRNAs
Patent Number: 8,273,871
Filed: Oct. 4, 2011
Lead Inventor: Gregory Hannon, Cold Spring Harbor Laboratory
The invention, the patent's abstract states, relates to an “improved design of shRNA based on structural mimics of miR-451 precursors. These miR-451 shRNA mimics are channeled through a novel small RNA biogenesis pathway, require Ago2 catalysis and are processed by Drosha but are independent of Dicer processing. This miRNA pathway feeds active elements only into Ago2 because of its unique catalytic activity. These data demonstrate that this newly identified small RNA biogenesis pathway can be exploited in vivo to produce active molecules.”
Title: Novel siRNA Structure for Minimizing Off-Target Effects and Relaxing Saturation of RNAi Machinery and the Use Thereof
Application Number: 20120238017
Filed: Dec. 8, 2008
Lead Inventor: Dong Ki Lee, Sungkyunkwan University
The invention, the patent application's abstract states, “relates to a novel siRNA structure and the use thereof. More particularly, the invention relates to a double-stranded small interfering RNA molecule comprising a 19 [to] 21 nucleotide antisense strand and a 15 [to] 19 [nucleotide] sense strand having a sequence complementary to the antisense sequence, wherein the 5' end of the antisense strand has a blunt end and the 3' end of the antisense strand has an overhang, and to a method for silencing the expression of a target gene using the siRNA molecule.”
Title: Inducible Systems and Methods for Controlling siRNA Expression
Application Number: 20120238022
Filed: March 20, 2012
Lead Inventor: Hoshang Unwalla, City of Hope
The invention comprises “an inducible system and methods for controlling expression of siRNA,” the patent application's abstract states. “An inducible system for producing siRNA only in the presence of HIV TAT, and methods for inhibiting HIV-1 gene expression in cells comprising such inducible system also are provided.”
Title: Monoconjugated Chitosans as Delivery Agents for Small Interfering Nucleic Acids
Application Number: 20120238735
Filed: Sept. 16, 2010
Lead Inventor: Samuel McManus, Nektar Therapeutics
The patent application, its abstract states, claims “conjugates ... having a single water-soluble, non-peptidic polymer, such as a poly-ethylene oxide, attached to a chitosan. Complexes of small interfering nucleic acids formed with such conjugates, as well as small interfering nucleic acids attached to such conjugates, are also provided. Related methods, intermediates, and compositions are also provided.”