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IP Update: Recent Patents, Patent Applications Awarded to Rosetta; Novartis; Alnylam; and More

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Title: Method of Diagnosing Poor Survival Prognosis Colon Cancer Using microRNA-21

Patent Number: 8,084,199

Filed: July 12, 2007

Lead Inventor: Carlo Croce, Ohio State University

The invention provides “novel methods and compositions for the diagnosis and treatment of colon cancers,” the patent's abstract states. “The invention also provides methods of identifying inhibitors of tumorigenesis.”


Title: Bioinformatically Detectable Group of Novel Regulatory Oligonucleotides and Uses Thereof

Patent Number: 8,084,598

Filed: Jan. 29, 2004

Inventor: Isaac Bentwich, Rosetta Genomics

“The invention relates to a first group of novel oligonucleotides, here identified as genomic address messenger, or GAM, oligonucleotides, and a second group of novel operon-like polynucleotides, here identified as genomic record, or GR, polynucleotides,” the patent's abstract states. “GAM oligonucleotides selectively inhibit translation of known target genes, many of which are known to be involved in various diseases. Nucleic acid molecules are provided ... encoding 2147 GAM oligonucleotides and 313 GR polynucleotides, as are vectors and probes both comprising the nucleic acid molecules.” Also claimed are “methods and systems for detecting GAM oligonucleotides and GR polynucleotide, and specific functions and utilities thereof for detecting expression of GAM oligonucleotides and GR polynucleotides, and for selectively enhancing and selectively inhibiting translation of the respective target genes thereof.”


Title: Methods and Compositions for the Specific Inhibition of Gene Expression by Double-Stranded RNA

Patent Number: 8,084,599

Filed: March 15, 2005

Lead Inventor: John Rossi, City of Hope (Integrated DNA Technologies)

“The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene,” the patent's abstract states. “More particularly, the invention is directed to compositions that contain double-stranded RNA, and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery.”


Title: Short Interfering Ribonucleic Acid with Improved Pharmacological Properties

Patent Number: 8,084,600

Filed: May 2, 2007

Lead Inventor: Francois Jean-Charles Natt, Novartis

The invention, the patent's abstract states, comprises “short interfering ribonucleic acid for oral administration [with] siRNA comprising two separate RNA strands that are complementary to each other over at least 15 nucleotides, wherein each strand is 49 nucleotides or less, and wherein at least one of which strands contains at least one chemical modification.”


Title: Cross-Linking Agent, Cross-Linking Method, Method for Controlling Gene Expression, and Method of Examining Gene Function

Patent Number: 8,084,625

Filed: Feb. 27, 2006

Lead Inventor: Toshiaki Furuta, Wako Pure Chemical Industries

The invention, the patent's abstract states, “provides a cross-linking agent [that has] photodegradable protective groups at two ends to cross-link double-stranded nucleic acid; a nucleic acid and a protein or a polypeptide; or proteins or polypeptides.”

The invention also comprises “a method for cross-linking a double-stranded RNA or the like using the same; a method for regulating gene expression, which can control the expression of a target gene at an arbitrary timing and location; and a method for examining a gene function,” the abstract states. “Cross-linking between double-stranded nucleic acids between a nucleic acid and a protein or a polypeptide, or between proteins or polypeptides … can be easily formed. … In addition, the cross-linking can also be easily removed so that the expression of a target gene can be easily controlled at an arbitrary timing and location with high efficiency. Hence, as a result, function examination and/or identification of a gene that is expressed at a specific timing and location can be performed. In addition, the RNAi effect of a double-stranded RNA that cannot be easily inhibited by a conventional caged compound can be inhibited, and the expression of a target gene can be easily controlled at an arbitrary timing and location.”


Title: DNA Virus microRNA and Methods for Inhibiting Same

Patent Number: 8,088,902

Filed: Oct. 19, 2004

Lead Inventor: Sebastien Pfeffer, Rockefeller University

“The invention relates to isolated nucleic acid molecules comprising the sequence of a human cytomegalovirus microRNA,” the patent's abstract states. “In another embodiment, the invention relates to single-stranded DNA virus microRNA molecules comprising the sequence of a human cytomegalovirus microRNA. The invention also relates to the anti-DNA virus microRNA molecules.”


Title: Compositions and Methods for Therapy and Diagnosis of Cancer

Patent Number: 8,088,913

Filed: Dec. 7, 2006

Lead Inventor: Ugur Sahin, Ganymed Pharmaceuticals

The invention, the patent's abstract states, is “directed to siRNA molecules which specifically target and cause RNAi-induced degradation of mRNA from TPTE genes so that the protein product of the TPTE gene is not produced or is produced in reduced amounts. The siRNA compounds and compositions of the invention are useful for treating diseases which require inhibition of TPTE expression for their treatment, in particular cancer pathologies. The ... invention also includes methods which make [it] possible to assess and/or prognose the metastatic behavior of a cancer disease and/or the occurrence of a relapse of cancer.”


Title: microRNA and Methods for Inhibiting Same

Patent Number: 8,088,914

Filed: July 6, 2009

Lead Inventor: Markus Stoffel, Rockefeller University

“The invention relates to isolated DNA or RNA molecules comprising at least ten contiguous bases having a sequence in a pancreatic islet microRNA,” according to the patent's abstract. “In another embodiment, the invention relates to isolated single stranded pancreatic islet microRNA molecules or anti-pancreatic islet microRNA molecules.”


Title: Functional and Hyperfunctional siRNA

Patent Number: 8,090,542

Filed: Nov. 14, 2003

Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)

“Efficient sequence-specific gene silencing is possible through the use of siRNA technology,” the patent'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.”


Titles: Novel Lipids and Compositions for the Delivery of Therapeutics

Application Numbers: 20110311583; 20110311582

Filed: Nov. 10, 2009

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

The inventions, the patent applications' abstracts state, relate to “lipids that are advantageously used in lipid particles for the in vivo delivery of therapeutic agents to cells.”


Title: RNAi-Based Selection System

Application Number: 20110311985

Filed: Feb. 9, 2010

Inventor: Peter Hahn, Qiagen

The invention provides a “novel RNAi-based selection system for selecting host cells that have incorporated an expression vector,” the patent application's abstract states.


Title: Diagnostic and Prognostic Use of Human Bladder Cancer-Associated microRNAs

Application Number: 20110312516

Filed: March 18, 2011

Lead Inventor: Torben Orntoft, Catalyst Assets

The invention “is based, at least in part, upon discovery of a number of miRNAs having expression that significantly correlates with bladder cancer, including certain stages or types of bladder cancer, as well as with bladder cancer survival and/or responsiveness to bladder cancer therapies,” the patent application's abstract states. “Accordingly, the ... invention features the identification and use of miRNAs to detect, diagnose, and/or predict the course, progression, or therapy responsiveness of bladder cancer. Kits for performing such assessments, and for administering therapeutic agents to subjects diagnosed with bladder cancer or certain forms of bladder cancer using the methods of the invention, are also featured.”


Title: Engineered Tunable Nanoparticles for Delivery of Therapeutics, Diagnostics, and Experimental Compounds and Related Compositions for Therapeutic Use

Application Number: 20110312877

Filed: Feb. 26, 2009

Lead Inventor: Mark Berninger, Aparna Biosciences

The invention comprises “biomedical nanoparticles ... based on new engineered modular carrier macromolecules, on engineered macromolecules or associated entities providing an internal nanoparticle structure, and compositions for minimizing non-specific binding of the nanoparticles while enabling efficient and convenient targeting to cells and tissues,” according to the patent application's abstract. “These nanoparticles may be used to deliver atomic or molecular or associated entities which are useful for diagnostics, primarily in vivo imaging, for therapeutics, for vaccines, or for experimental research. Nanoparticles comprising combinations of active entities such as gene inhibitors with gene expression cassettes or imaging agents with therapeutic agents, and polyamide compounds useful for treatment of microbial infections are also disclosed.”


Title: HDL Particles for Delivery of Nucleic Acids

Application Number: 20110312899

Filed: Nov. 17, 2009

Lead Inventor: Anil Sood, UNT Health Sciences Center

The invention comprises “high-density lipoprotein-nucleic acid particles, wherein the particles include an apolipoprotein; a nucleic acid component comprising a therapeutic nucleic acid segment; and a polypeptide comprising a positively charged region, wherein the positively-charged region of the polypeptide associates with the nucleic acid component,” the patent application's abstract states. “Also disclosed are pharmaceutical compositions that include an apolipoprotein; a nucleic acid component comprising a therapeutic nucleic acid segment; and a polypeptide comprising a positively charged region. Methods that concern the particles and pharmaceutical compositions of the present invention are also set forth, as well as kits.”


Title: SNALP Formulations Containing Polyoxazoline-Dialkyloxypropyl Conjugates

Application Number: 20110313017

Filed: Jan. 13, 2011

Inventor: James Heyes, Protiva Biotherapeutics (Tekmira Pharmaceuticals)

The invention, the patent application's abstract states, claims “polyoxaline-dialkyloxypropyl conjugates, SNALP compositions comprising POZ-DAA conjugates, and methods of using such SNALP compositions to introduce a therapeutic agent, such as a nucleic acid, into a cell.”


Title: Treatment of Intestinal Conditions

Application Number: 20110313016

Filed: March 14, 2006

Lead Inventor: Ana Jimenez, Sylentis

The invention comprises “methods and compositions for the treatment of intestinal disorders, such as IBD and Crohn's disease,” according to the patent application's abstract. “Preferred compositions include siNA. Also disclosed is a method of specifically targeting siNA to treat intestinal disorders by intrarectal administration of siNA compounds.”


Title: UsiRNA Compositions

Application Number: 20110313020

Filed: Dec. 3, 2009

Lead Inventor: Michael Templin, Marina Biotech

The invention comprises “double-stranded RNA complexes having one or more hydroxymethyl substituted nucleomonomers in the passenger strand of an RNA complex,” the patent application's abstract states. “RNA complexes of the disclosure may be useful for therapeutic applications, diagnostic applications, or research applications.”


Title: RNAi Modulation of RSV and Therapeutic Uses Thereof

Application Number: 20110313023

Filed: June 14, 2011

Inventor: Rachel Meyers, Alnylam Pharmaceuticals

The invention is “based on the in vivo demonstration that RSV can be inhibited through intranasal administration of iRNA agents, as well as by parenteral administration of such agents,” the patent application's abstract states. “Further, it is shown that effective viral reduction can be achieved with more than one virus being treated concurrently. Based on these findings, the present invention provides general and specific compositions and methods that are useful in reducing RSV mRNA levels, RSV protein levels, and viral titers in a subject.”


Title: RNA Interference-Mediated Inhibition of Proprotein Convertase Subtilisin Kexin 9 Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20110313024

Filed: July 25, 2011

Lead Inventor: Leonid Beigelman, 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 proprotein convertase subtilisin kexin 9 gene expression and/or activity,” the patent application's abstract states. “Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules... capable of mediating RNA interference against PCSK9 gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle formulations of such small nucleic acid molecules.”


Title: Methods and Compositions Involving miRNA and miRNA Inhibitor Molecules

Application Number: 20110313025

Filed: July 25, 2011

Lead Inventor: David Brown, Asuragen

The invention comprises “methods and compositions for introducing miRNA activity or function into cells using synthetic nucleic acid molecules,” the patent application's abstract states. “Moreover, the ... invention concerns methods and compositions for identifying miRNAs with specific cellular functions that are relevant to therapeutic, diagnostic, and prognostic applications wherein synthetic miRNAs and/or miRNA inhibitors are used in library screening assays.”


Title: iRNA Agents Targeting CCR5-Expressing Cells and Uses Thereof

Application Number: 20110313144

Filed: Aug. 23, 2011

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

“The invention relates to iRNA agents that preferably include a modification that targets CC chemokine receptor 5,” the patent application's abstract states. “The invention also relates to methods of making and using such modified iRNA agents.”


Title: Method to Trigger RNA Interference

Application Number: 20110314571

Filed: Aug. 24, 2011

Lead Inventor: James Carrington, Oregon State University

The invention, the patent application's 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.”


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

Application Number: 20110318838

Filed: Sept. 12, 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 application's abstract. “More particularly, the present invention relates to compositions and methods for cell type-specific delivery of anti-HIV siRNAs via fusion to an anti-gp120 aptamer.”


Title: siRNA Targeting Histamine Receptor H1

Application Number: 20110319296

Filed: Aug. 10, 2011

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.”


Title: siRNA Targeting Gremlin

Application Number: 20110319297

Filed: Aug. 23, 2011

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 CKSF1B1.”


Title: Cell Specifically Effective Molecules on the Basis of siRNA and Application Kits for the Production Thereof and Use Thereof

Application Number: 20110319342

Filed: March 12, 2010

Lead Inventor: Tobias Poehlmann, BianoScience

The invention comprises “a biologically inactivated [cell-specific] effective molecule for biologically inactive transfection into a target cell to inhibit expression of genes in the target cell after biological activation of the molecule,” according to the patent application's abstract.


Title: siRNA Targeting Cyclin-Dependent Kinase 4

Application Number: 20110319474

Filed: Aug. 17, 2011

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.”

The Scan

US Supports Patent Waivers

NPR reports that the Biden Administration has announced its support for waiving intellectual property protections for SARS-CoV-2 vaccines.

Vaccines Versus Variants

Two studies find the Pfizer-BioNTech SARS-CoV-2 vaccine to be effective against viral variants, and Moderna reports on booster shots to combat variants.

CRISPR for What Ails You

The Wall Street Journal writes that CRISPR-based therapies could someday be used to treat common conditions like heart attacks.

Nature Papers Review Integration of Single-Cell Assay Data, Present Approach to Detect Rare Variants

In Nature this week: review of ways to integrate data from single-cell assays, and more.