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IP Update: Recent Patents, Patent Applications Awarded to Alnylam, Alcon, City of Hope, and More

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Title: Nanogenomics for Medicine: siRNA Engineering

Patent Number: 8,222,220

Filed: May 12, 2009

Lead Inventor: Ancha Baranova, George Mason University

The invention, the patent's abstract states, comprises “materials and methods for the delivery of siRNA and the production of nanoparticles useful for the delivery of siRNA. Methods of treating a disease or disorder using the nanoparticles described herein also are disclosed.”


Title: Modulation of Gene Expression through Endogenous Small RNA Targeting of Gene Promoters

Patent Number: 8,222,221

Filed: June 3, 2009

Lead Inventor: David Corey, University of Texas

“Gene expression can be selectively regulated by endogenous miRNAs that target promoters of genes,” the patent's abstract states. “Altering of the activity of these promoter-targeting miRNAs with single-stranded complementary oligonucleotides that bind the miRNA causes modulation of expression of the target gene. Endogenous miRNAs that modulate expression of target genes can be identified by evaluating an endogenous miRNA for complementarity to a target gene promoter; and determining that the complementary miRNA modulates expression of the target gene.”


Title: Compositions and Methods for Inhibiting Expression of the PCSK9 Gene

Patent Number: 8,222,222

Filed: Sept. 4, 2009

Lead Inventor: Pamela Tan, Alnylam Pharmaceuticals

“The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of the PCSK9 gene comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length ... and which is substantially complementary to at least a part of the PCSK9 gene,” the patent's abstract states. “The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by PCSK9 gene expression and the expression of the PCSK9 gene using the pharmaceutical composition; and methods for inhibiting expression of a PCSK9 gene in a cell.”


Title: Cell Type-Specific Aptamer-siRNA Delivery System for HIV-1 Therapy

Patent Number: 8,222,226

Filed: May 16, 2011

Lead Inventor: John Rossi, City of Hope

The invention relates to “compositions and methods for delivery of siRNA to specific cells or tissue,” according to the patent's abstract. “More particularly, the … invention relates to compositions and methods for cell type-specific delivery of anti-HIV siRNAs via fusion to an anti-gp120 aptamer.”


Title: RNAi-Mediated Inhibition of Histamine Receptor H1-Related Conditions

Patent Number: 8,222,227

Filed: June 30, 2011

Lead Inventor: John Yanni, Alcon

The patent, its abstract states, claims RNA for the “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: microRNA Molecules

Patent Number: 8,222,394

Filed: Jan. 23, 2009

Lead Inventor: Thomas Tuschl, Max Planck Institute

“In Caenorhabditis elegans, lin-4 and let-7 enclode 22- and 21-nucleotide RNAs, respectively, that function as key regulators of developmental timing,” the patent's abstract states. “Because the appearance of these short RNAs is regulated during development, they are also referred to as small temporal RNAs. We show that many more [small] RNAs, termed microRNAs, exist in invertebrates and vertebrates, and that some of these novel RNAs, similar to let-7 stRAN, are also highly conserved. This suggests that sequence-specific post-transcriptional regulatory mechanisms mediated by small RNAs are more general than previously appreciated.”


Title: siRNA Targeting Kinase Short Domain Receptor

Patent Number: 8,222,395

Filed: Sept. 3, 2009

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'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 nucleotide sequences for KDR.”


Title: siRNA Targeting Proto-Oncogene MET

Patent Number: 8,222,396

Filed: Sept. 8, 2010

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'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 nucleotide sequences for MET.”


Title: siRNA Phospholipid Conjugate

Application Number: 20120177723

Filed: July 14, 2010

Lead Inventor: Vladimir Torchilin, Northeastern Univeristy

The invention, the patent application's abstract states, comprises “siRNA-conjugated liposomes and micelles, methods of making such conjugates, and methods of using such conjugates, such as for the delivery of siRNA to cells to reduce expression of target polypeptides in such cells.”


Title: Modulation of Human Cytomegalovirus Replication by microRNA-132, microRNA-145, and microRNA-212

Application Number: 20120178791

Filed: Sept. 7, 2011

Lead Inventor: Timothy Kowalik, University of Massachusetts

The invention relates to “miR-145, miR-132, miR-212, and the genes or gene products regulated by these miRNAs,” according to the patent application. In cells infected with HCMV, “miR-145 is down-regulated … [which] modulates expression of miR-145 target genes, including IRS-I. Transfection of cells with a miR-145 agent, such as a miR-145 mimetic, reduces HCMV replication and protein expression.”

The abstract notes that miR-132 and miR-212 are up-regulated in cells infected with HCMV, which modulates expression of their target genes, including MeCP2 and RICS. “Transfection of cells with a miR-132 and/or a miR-212 antagonist reduces HCMV replication and protein expression. Accordingly, the invention provides methods of attenuating HCMV replication by modulating, for example, miR-145, miR-132, and/or miR-212, and targets thereof. Also provided are methods of detecting an HCMV infection, and compositions and kits useful for attenuating HCMV replication.”


Title: Nucleotide-Cochleate Compositions and Methods of Use

Application Number: 20120178793

Filed: March 19, 2012

Lead Inventor: Raphael Mannino, BioDelivery Sciences

The invention, the patent application's abstract states, relates to cochleate compositions that include a nucleotide. “The nucleotide may generally be bound via a linker to a component of the cochleate, or to a lipophilic tail. Additionally or alternatively, the nucleotide may be associated with a transfection agent. The ... invention also includes methods for making and using the compositions provided herein.”


Title: RNAi-Mediated Inhibition of RHO Kinase for Treatment of Ocular Disorder

Application Number: 20120178794

Filed: March 26, 2012

Lead Inventor: Jon Chatterton, Alcon

“RNA interference is provided for inhibition of Rho kinase mRNA expression for treating patients with ocular disorders, particularly for treating intraocular pressure, ocular hypertension, and glaucoma,” the patent application's abstract states. “Rho kinase mRNA targets include mRNA for ROCK1 and ROCK2.”


Title: RNAi-Mediated Inhibition of Frizzled-Related Protein-1 for Treatment of Glaucoma

Application Number: 20120178795

Filed: March 26, 2012

Lead Inventor: Abbot Clark, Novartis

“RNA interference is provided for inhibition of frizzled-related protein-1 mRNA expression, in particular, for treating patients having glaucoma or at risk of developing glaucoma,” according to the patent application's abstract.

The Scan

Shape of Them All

According to BBC News, researchers have developed a protein structure database that includes much of the human proteome.

For Flu and More

The Wall Street Journal reports that several vaccine developers are working on mRNA-based vaccines for influenza.

To Boost Women

China's Ministry of Science and Technology aims to boost the number of female researchers through a new policy, reports the South China Morning Post.

Science Papers Describe Approach to Predict Chemotherapeutic Response, Role of Transcriptional Noise

In Science this week: neural network to predict chemotherapeutic response in cancer patients, and more.