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IP Update: Recent Patents, Patent Applications Awarded to Alnylam, Tekmira, OSU, and More

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Title: Dock-and-Lock Complexes for Delivery of Interference RNA

Patent Number: 8,491,914

Filed: Dec. 9, 2010

Lead Inventor: Chien-Hsing Chang, IBC Pharmaceuticals

The patent, its abstract states, relates to “compositions and methods of use of targeted delivery complexes for delivery of siRNA to a disease-associated cell, tissue, or pathogen. The targeted delivery complex comprises a targeting molecule, such as an antibody or fragment thereof, conjugated to one or more siRNA carriers. In preferred embodiments the siRNA carrier is a dendrimer or protamine and the targeting molecule is an anti-cancer antibody, such as hRS7. More preferably, the antibody or fragment is rapidly internalized into the target cell to facilitate uptake of the siRNA. Most preferably, the targeted delivery complex is made by the DNL technique. The compositions and methods are of use to treat a variety of disease states, such as cancer, autoimmune disease, immune dysfunction, cardiac disease, neurologic disease, inflammatory disease, or infectious disease.”


Title: microRNA Fingerprints during Human Megakaryocytopoiesis

Patent Number: 8,492,083

Filed: Aug. 27, 2012

Lead Inventor: Carlo Croce, Ohio State University

The patent, its abstract states, claims “a method of decreasing expression of HOXA1 in a subject having a cancer and/or myeloproliferative disorder associated with overexpression of a HOXA1 gene product.” Through the method, “an effective amount of at least one miR-10a gene product or an isolated variant or biologically active fragment thereof is administered to the subject [and is] sufficient to decrease expression of the HOXA1 gene product.”


Title: microRNA-Mediated Modulation of Colony Stimulating Factors

Patent Number: 8,492,357

Filed: July 24, 2009

Lead Inventor: Jesper Worm, Santaris Pharma

The invention, the patent’s abstract states, “relates to the modulation of immunoregulatory proteins, including cytokines, such as colony stimulatory factors via the use of microRNA-155 modulators.”


Title: Lipid Formulations for Nucleic Acid Delivery

Patent Number: 8,492,359

Filed: Oct.5, 2011

Lead Inventor: Edward Yaworski, Protiva Biotherapeutics (Tekmira Pharmaceuticals)

The invention, the patent’s abstract states, relates to “novel, stable lipid particles comprising one or more active agents or therapeutic agents, methods of making the lipid particles, and methods of delivering and/or administering the lipid particles. More particularly, the … invention provides stable nucleic acid-lipid particles comprising a nucleic acid, methods of making the [particles], and methods of delivering and/or administering” the particles.


Title: System and Method for Identification of microRNA Target Sites and Corresponding Targeting microRNA Sequences

Patent Number: 8,494,784

Filed: June 9, 2008

Lead Inventor: Tien Huynh, IBM

The patent, its abstract states, claims a “method for determining whether a nucleotide sequence contains a microRNA binding site and which microRNA will bind thereto. … For example, in one aspect of the invention … [the] method … [involves generating] one or more patterns … by processing a collection of known mature microRNA sequences. The reverse complement of each generated pattern is then computed. One or more attributes are then assigned to the reverse complement of the one or more generated patterns. The one or more patterns that correspond to a reverse complement having one or more assigned attributes that satisfy at least one criterion are thereafter subselected. Each subselected pattern is then used to analyze the nucleotide sequence, such that a determination is made whether the nucleotide sequence contains a microRNA binding site and which microRNA sequence will bind thereto.”


Title: Transgenic Plants Modified for Reduced Cadmium Transport, Derivative Products, and Related Methods

Application Number: 20130183738

Filed: Feb. 19, 2013

Lead Inventor: Alec Hayes, Philip Morris

The patent application, its abstract states, claims “transgenic tobacco plants and derivative seeds genetically modified to impede the transport of cadmium from the root system to aerial portions of transgenic plants by reducing the expression levels of HMA-related transporters. Various embodiments are directed to transgenic tobacco plants genetically modified to stably express a RNAi construct encoding RNAi polynucleotides that enable the degradation of endogenous NtHMA RNA variants. Reduced expression of NtHMA transporters in transgenic plants results in substantially reduced content of cadmium in the leaf lamina.”


Title: Dual-targeting siRNA Agents

Application Number: 20130184324

Filed: Sept. 22, 2010

Lead Inventor: Kevin Fitzgerald, Alnylam Pharmaceuticals

“The invention relates to dual targeting siRNA agents targeting a PCSK9 gene and a second gene, and methods of using dual targeting siRNA agents to inhibit expression of PCSK9 and to treat PCSK9-related disorders,” the patent application’s abstract states.


Title: Compositions and Methods for Inhibiting Expression of Factor VII Gene

Application Number: 20130184326

Filed: Sept. 14, 2012

Lead Inventor: Antonin de Fougerolles, Alnylam Pharmaceuticals

“The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of the factor VII gene,” according to the patent application’s abstract.


Title: Compositions and Methods for Inhibiting Expression of Mutant EGFR Gene

Application Number: 20130184329

Filed: Oct. 24, 2012

Lead Inventor: Dinah Sah, Alnylam Pharmaceuticals

“The invention relates to a double-stranded ribonucleic acid targeting a mutant epidermal growth factor receptor and methods of using the dsRNA to inhibit expression of mutant EGFR,” the patent application’s abstract states.


Title: Method to Trigger RNA Interference

Application Number: 20130185818

Filed: March 14, 2013

Lead Inventor: James Carrington, Oregon State University

The patent application, its abstract states, comprises “a method to generate siRNAs in vivo … [as well as] constructs and compositions useful in the method. The method does not depend on the use of DNA or synthetic constructs that contain inverted duplications or dual promoters so as to form perfect or largely double-stranded RNA. Rather, the method depends on constructs that yield single-stranded RNA transcripts, and exploits endogenous or in vivo-produced miRNAs or siRNAs to initiate production of siRNAs. The miRNAs or siRNAs guide cleavage of the transcript and set the register for production of siRNAs encoded adjacent to the initiation cleavage site within the construct. The method results in specific formation of siRNAs of predictable size and register relative to the initiation cleavage site. The method can be used to produce specific siRNAs in vivo for inactivation or suppression of one or more target genes or other entities, such as pathogens.”

The Scan

Study Finds Sorghum Genetic Loci Influencing Composition, Function of Human Gut Microbes

Focusing on microbes found in the human gut microbiome, researchers in Nature Communications identified 10 sorghum loci that appear to influence the microbial taxa or microbial metabolite features.

Treatment Costs May Not Coincide With R&D Investment, Study Suggests

Researchers in JAMA Network Open did not find an association between ultimate treatment costs and investments in a drug when they analyzed available data on 60 approved drugs.

Sleep-Related Variants Show Low Penetrance in Large Population Analysis

A limited number of variants had documented sleep effects in an investigation in PLOS Genetics of 10 genes with reported sleep ties in nearly 192,000 participants in four population studies.

Researchers Develop Polygenic Risk Scores for Dozens of Disease-Related Exposures

With genetic data from two large population cohorts and summary statistics from prior genome-wide association studies, researchers came up with 27 exposure polygenic risk scores in the American Journal of Human Genetics.