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USPTO Publishes One Patent, Nine Patent Applications Related to RNAi: Aug 2, 2007

Title: Bioinformatically Detectable Group of Novel Regulatory Genes and Uses Thereof
Number: 7,250,496
Filed: Dec. 6, 2002
Inventor: Isaac Bentwich, Rosetta Genomics
The invention relates, the patent’s abstract states, to a “group of novel genes, here identified as genomic address messenger or GAM genes, and a second group of novel operon-like genes, here identified as genomic record or GR genes. GAM genes selectively inhibit translation of known target genes, many of which are known to be involved in various diseases,” the abstract adds. “Nucleic acid molecules are provided respectively encoding 20,600 GAM genes, and 6635 GR genes, as are vectors and probes both comprising the nucleic acid molecules, and methods and systems for detecting GAM and GR genes and specific functions and utilities thereof, for detecting expression of GAM and GR genes, and for selectively enhancing and selectively inhibiting translation of the respective target genes thereof.”

Title: Modified Polynucleotides for Use in RNA Interference
Number: 20070173476
Filed: Jan. 4, 2007
Lead Inventor: Devin Leake, Dharmacon (Thermo Fisher Scientific)
The patent application, its abstract states, claims “methods and compositions for performing RNA interference comprising a wide variety of stabilized siRNAs suitable for use in serum-containing media and for in vivo applications, such as therapeutic applications. … These siRNAs permit effective and efficient applications of RNA interference to applications such as diagnostics and therapeutics through the use of one or more modifications including orthoesters, terminal conjugates, modified linkages and 2' modified nucleotides.
“Uniquely modified siRNAs have been developed that reduce off-target effects incurred in gene-silencing,” the abstract adds.

Title: Double-Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
Number: 20070173475, 20070173474, 20070172948
Filed: Dec. 1, 2006
Lead Inventor: Balkrishen Bhat, Eli Lilly
The invention provides “double-stranded compositions wherein each strand is modified to have a motif defined by positioning of beta-D-ribonucleosides and sugar modified nucleosides,” the patent applications’ abstracts state. “More particularly, the present compositions comprise one strand having a gapped motif and another strand having a gapped motif, a hemimer motif, a blockmer motif, a fully modified motif, a positionally modified motif or an alternating motif. At least one of the strands has complementarity to a nucleic acid target.
“The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes,” the abstracts add. “In some embodiments, the compositions of the … invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The … invention also provides methods for modulating gene expression.”

Title: RNA Interference-Mediated Inhibition of Proprotein Convertase Subtilisin Kexin 9 Gene Expression Using Chemically Modified Short Interfering Nucleic Acid
Number: 20070173473
Filed: July 17, 2006
Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)
The patent application, according to its abstract, claims “compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases, and conditions that respond to the modulation of proprotein convertase subtilisin kexin 9 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 PCSK9 gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases, and conditions.
“Specifically, the invention relates to double stranded nucleic acid molecules … capable of mediating RNA interference against PCSK9 gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle LNP 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 that can inhibit the function of RISC to modulate PCSK9 gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous. … Such small nucleic acid molecules … are useful, for example, in providing compositions to prevent, inhibit, or reduce metabolic diseases traits and conditions, including but not limited to hyperlipidemia, hypercholesterolemia, cardiovascular disease, atherosclerosis, hypertension, diabetes, insulin resistance, obesity, and/or other disease states, conditions, or traits associated with PCSK9 gene expression or activity in a subject or organism.”

Title: Methods and Reagents for the Treatment of Apoptosis-Related Disorders
Number: 20070173472
Filed: Nov. 20, 2006
Lead Inventor: Jeff MacKeigan, Novartis
The invention, the patent application’s abstract states, “relates to novel uses and reagents relating to apoptosis-related disorders. In particular, modulation of PI3K class II-alpha activity is shown to influence apoptosis, and PI3K class II-alpha is thought to be cellular survival factors. Based on these findings, the … invention provides therapeutic methods and pharmaceutical compositions useful for the treatment of apoptosis-related disorders. In addition, PI3K class II-alpha is provided as a target for the development of therapeutics. Accordingly, the invention provides screening methods for identifying candidate compounds that modulate PI3K class II-alpha activity and may therefore be used to treat apoptosis-related disorders such as cardiovascular disorders, autoimmune disorders, neurodegenerative disorders, and cancer.”

Title: RNA Interference-Mediated Inhibition of Cholesteryl Ester Transfer Protein Gene Expression Using Chemically Modified Short Interfering Nucleic Acid
Number: 20070173467
Filed: Aug. 19, 2004 PCT Filed: Aug. 19, 2004
Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)
The invention relates to “compounds, compositions, and methods useful for modulating cholesteryl ester transfer protein … gene expression using short interfering nucleic acid molecules,” the patent application’s abstract states. “This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of CETP gene expression and/or activity by RNA interference using small nucleic acid molecules.”

Title: siRNA-Mediated Gene Silencing of Synuclein
Number: 20070172462
Filed: March 21, 2007
Lead Inventor: Martha Bohn, Children’s Memorial Research Center (Northwestern University)
The invention “is directed to small interfering RNAs that down regulate expression of a synuclein gene and methods of using the small interfering RNAs,” according to the patent application’s abstract.

Title: Dry Powder Compositions for RNA Influenza Therapeutics
Number: 20070172430
Filed: Jan. 15, 2007
Lead Inventor: Luis Brito, Nastech Pharmaceutical
The patent application, its abstract states, claims “a dry powder formulation for mucosal, intranasal, inhalation, or pulmonary delivery, which may include one or more siRNAs or Dicer-active precursors thereof targeted to a transcript involved in infection by, or replication or production of, an influenza virus.”

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