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USPTO Publishes 11 RNAi-Related Patent Applications, August 2007

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Title: RNA Probes
 
Number: 20070184464
 
Filed: Oct. 12, 2006
 
Lead Inventor: Azeddine Si-Ammour, Novartis
 
The invention, the patent application’s abstract states, “is based in part on the generation of a double-stranded RNA molecule substantially covering the whole transcribed region of a gene, and cleaving this using an RNA endonuclease to generate small RNA molecules which are already or may be subsequently labeled. The invention provides small labeled ribonucleic acid fragments for use as probes to detect potentially small interfering ribonucleic acid fragments produced in vivo. The invention also provides uses of said small labeled RNA fragments and kits suitable for preparing said small labeled RNA fragments.”
 

 
Title: siRNA-Based Methods for Treating Alzheimer’s Disease
 
Number: 20070185042
 
Filed: Aug. 9, 2004 PCT Filed: Aug. 9, 2004
 
Lead Inventor: Li-Huei Tsai, Brigham and Women’s Hospital
 
The invention, according to the patent application’s abstract, “relates to siRNA molecules derived from BACE1 and BACE2 genes. Accordingly, provided herein are siRNA molecules comprising a nucleotide sequence consisting essentially of a BACE1 or BACE2 gene. Also provided are methods for reducing the level of BACE1 protein in a cell. Further provided are methods for preparing a pharmaceutical composition comprising an siRNA with a pharmaceutically acceptable carrier.”
 

 
Title: RNA Interference-Mediated Inhibition of NOGO and NOGO Receptor Gene Expression Using Short Interfering Nucleic Acid
 
Number: 20070185043
 
Filed: Aug. 20, 2004 PCT Filed: Aug. 20, 2004
 
Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)
 
“This invention relates to compounds, compositions, and methods useful for modulating NOGO and/or NOGO receptor 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 NOGO and/or NOGO receptor gene expression and/or activity by RNA interference using small nucleic acid molecules. In particular, the … invention features small nucleic acid molecules … and methods used to modulate the expression of NOGO and/or NOGO receptor genes, such as NOGO-A, NOGO-B, NOGO-C, NOGO-66 receptor, NI-35, NI-220, NI-250, myelin-associated glycoprotein, tenascin-R, and NG-2.”
 

 
Title: Double-Stranded Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
 
Number: 20070185046, 20070185047
 
Filed: Dec. 1, 2006
 
Lead Inventor: Balkrishen Bhat, Eli Lilly
 
The inventions, the patent applications’ abstracts state, provide “double-stranded compositions wherein each strand is modified to have a motif defined by positioning of beta-D-ribonucleosides and sugar modified nucleosides. More particularly, the … compositions comprise one strand having an alternating motif and another strand having a hemimer motif, a blockmer motif, a fully modified motif or a positionally modified 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. In preferred embodiments the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The [inventions also provide] methods for modulating gene expression.”
 

 
Title: RNA Interference-Mediated Inhibition of Histone Deacetylase Gene Expression Using Short Interfering Nucleic Acid
 
Number: 20070185049
 
Filed: Nov. 2, 2006
 
Lead Inventor: Vasant Jadhav, Sirna Therapeutics (Merck)
 
The invention “relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases, and conditions that respond to the modulation of histone deacetylase gene expression and/or activity,” the patent application’s abstract states. “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 HDAC 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 or that mediate RNA interference against HDAC gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle formulations of such small nucleic acid molecules.
 
The abstract adds that the invention also relates to “small nucleic acid molecules … that can inhibit the function of endogenous RNA molecules, such as endogenous HDAC microRNA … or endogenous HDAC short interfering RNA … or that can inhibit the function of RISC … to modulate HDAC gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs … including cocktails of such small nucleic acid molecules and lipid nanoparticle formulations of such small nucleic acid molecules.”
 

 
Title: Double-Stranded RNA and Method of Use for Inhibiting Expression of a Fusion Gene
 
Number: 20070185050
 
Filed: Jan. 22, 2007
 
Lead Inventor: Olaf Heidenreich, Alnylam Europe (Alnylam)
 
The invention, according to the patent application’s abstract, “relates to the specific inhibition of expression of a fusion gene in mammals using a short double-stranded RNA. The dsRNA is approximately 19-24 nucleotides in length and has a nucleotide sequence which is complementary to at least a part of the target gene. The dsRNAs of the … invention are useful for treating diseases caused by chromosomal aberrations, particularly malignant diseases such as lymphoma and leukemia.”
 

 
Title: siRNA Targeting HtrA Serine Peptidase 1
 
Number: 20070185317
 
Filed: March 28, 2007
 
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 HtrA1.”
 

 
Title: siRNA Targeting Connexin 43
 
Number: 20070185318
 
Filed: March 30, 2007
 
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 connexin 43 (gap junction protein alpha 1).”
 

 
Title: siRNA Targeting Insulin-Like Growth Factor 1 Receptor
 
Number: 20070185319
 
Filed: April 3, 2007
 
Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)
 
According to the patent application’s abstract, “efficient sequence specific gene silencing is possible through the use of siRNA technology. 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 insulin-like growth factor 1 receptor.”
 

 
Title: siRNA Targeting Cell Division Cycle 20 Homolog
 
Number: 20070185320
 
Filed: April 9, 2007
 
Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)
 
“Efficient sequence specific gene silencing is possible through the use of siRNA technology,” according to the patent application’s abstract. “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 CDC20.”

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