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IP Update: Recent Patents, Patent Applications Awarded to Alnylam, Quark, UMass, and More

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Title: Compositions and Methods for Modulating Ghrelin-Mediated Conditions

Patent Number: 8,309,530

Filed: Feb. 4, 2010

Lead Inventor: Eva Szentirmai, Washington State University

“In certain aspects, the preproghrelin gene, but not the ghrelin receptor, is required for normal integration of thermoregulation and sleep in mice," the patent's abstract states. As such, the invention comprises “methods for modulation of thermoregulation and other ghrelin-mediated conditions [by] ... administration of an inhibitor of ghrelin expression … [including siRNAs] for treatment of obesity and for modulation of thermoregulation.”


Title: Therapeutic Uses of Inhibitors of RTP801

Patent Number: 8,309,532

Filed: June 17, 2010

Lead Inventor: Elena Feinstein, Quark Pharmaceuticals

The invention, the patent's abstract states, “provides novel molecules, compositions, methods, and uses for treating microvascular disorders, eye diseases, and respiratory conditions based upon inhibition of the RTP801 gene and/or protein.”


Title: Allele-Specific RNA Interference

Patent Number: 8,309,533

Filed: Feb. 21, 2011

Inventor: Zoushang Xu, University of Massachusetts

“Human diseases caused by dominant, gain-of-function mutations develop in heterozygotes bearing one mutant and one wild-type copy of a gene,” the patent's abstract states. “Because the wild-type gene often performs important functions, whereas the mutant gene is toxic, any therapeutic strategy must selectively inhibit the mutant while retaining wild-type gene expression. The ... invention includes methods of specifically inhibiting the expression of a mutant allele, while preserving the expression of a co-expressed wild-type allele using RNAi. ... The invention also includes small interfering RNAs and small hairpin RNAs that selectively suppress mutant, but not wild-type, expression of copper zinc superoxide dismutase, which causes inherited amyotrophic lateral sclerosis. The ... invention further provides asymmetric siRNAs and shRNAs with enhanced efficacy and specificity and mediating RNAi.”


Title: Compositions and Methods for Short Interfering Nucleic Acid Inhibition of Nav1.8

Patent Number: 8,309,703

Filed: May 28, 2010

Lead Inventor: Sameer Goregaoker, Merck

“The invention provides short interfering nucleic acids, either single-stranded or double-stranded, that cause RNAi-induced degradation of mRNA from the Nav1.8 sodium channel gene … pharmaceutical compositions comprising such short interfering nucleic acids; recombinant vectors comprising such short interfering nucleic acids; a method for inhibiting translation of an mRNA; a method for inhibiting expression of a polypeptide; a method for blocking the membrane potential in a cell; a method for blocking the sodium current in a cell; and a method for inhibiting chronic pain,” the patent's abstract states.


Title: Methods and Compositions for Enhancing the Efficacy and Specificity of RNAi

Patent Number: 8,309,704

Filed: June 2, 2004

Lead Inventor: Phillip Zamore, University of Massachusetts

The invention, the patent's abstract states, “provides methods of enhancing the efficacy and specificity of RNAi. The invention also provides compositions for mediating RNAi. In particular, the invention provides siRNAs, shRNAs, vectors, and transgenes having improved specificity and efficacy in mediating silencing of a target gene. Therapeutic methods are also featured.”


Title: Methods and Compositions for Enhancing the Efficacy and Specificity of RNA Silencing

Patent Number: 8,309,705

Filed: March 29, 2010

Lead Inventor: Phillip Zamore, University of Massachusetts

The invention provides “methods of enhancing the efficacy and specificity of RNA silencing,” according to the patent's abstract. “The invention also provides compositions for mediating RNA silencing. In particular, the invention provides siRNAs, siRNA-like molecules, shRNAs, vectors, and transgenes having improved specificity and efficacy in mediating silencing of a target gene. Therapeutic methods are also featured.”


Title: Templated Nanoconjugates

Application Number: 20120282186

Filed: Nov. 1, 2010

Lead Inventor: Chad Mirkin, Northwestern University (AuraSense Therapeutics)

The patent application, its abstract states, is “directed to compositions comprising templated nanoconjugates and methods of their use.”


Title: Compositions and Methods Related to miRNA in Diabetic Conditions

Application Number: 20120282326

Filed: Dec. 16, 2010

Lead Inventor: Subrata Chakrabarti, University of Western Ontario

The invention, the patent application's abstract states, “relates to methods of treating a disorder associated with glucose-mediated cell damage in a subject comprising administering to the subject an agent that modulates the expression of one or more miRNAs in a damaged cell or cells of the subject. The ... invention also relates to compositions for treating a disorder associated with glucose-mediated cell damage comprising an agent that modulates the expression of one or more miRNAs in a damaged cell or cells. The invention also relates to methods of diagnosing a disorder associated with glucose-mediated cell damage in a subject, including diagnosis of diabetic retinopathy.”


Title: Lipid-Formulated Compositions and Methods for Inhibiting Expression of Eg5 and VEGF Genes

Application Number: 20120282341

Filed: Sept. 10, 2010

Lead Inventor: David Bumcrot, Alnylam Pharmaceuticals

“This invention relates to compositions containing double-stranded ribonucleic acid in a SNALP formulation, methods of using the compositions to inhibit the expression of the Eg5/KSP and VEGF, and methods of using the compositions to treat pathological processes mediated by Eg5/KSP and VEGF expression, such as cancer,” according to the patent application's abstract.


Title: siRNA Targeting Amyloid Beta Precursor Protein

Application Number: 20120283142

Filed: July 18, 2012

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 nucleotide sequences for APP.”


Title: siRNA Compounds Comprising Terminal Substitutions

Application Number: 20120283309

Filed: May 21, 2012

Lead Inventor: Sharon Avkin-Nachum, Quark Pharmaceuticals

“The invention relates to modified siRNA compounds [that] down-regulate target gene expression, to pharmaceutical compositions comprising such compounds, and to methods of treating and/or preventing the incidence or severity of various diseases or conditions associated with the genes and/or symptoms associated with such diseases or conditions,” according to the patent application's abstract.


Title: microRNA Signatures Associated with Human Chronic Lymphocytic Leukemia and Uses Thereof

Application Number: 20120283310

Filed: June 28, 2012

Lead Inventor: Carlo Croce, Ohio State University

The patent application, its abstract states, claims “methods and compositions for the diagnosis, prognosis and/or treatment of leukemia associated diseases.”


Title: siRNA Targeting Glucagon Receptor

Application Number: 20120283311

Filed: July 5, 2012

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 nucleotide sequences for GCGR.”


Title: Reduction of Off-Target RNA Interference Activity

Application Number: 20120283424

Filed: July 18, 2012

Lead Inventor: Beverly Davidson, University of Iowa

The invention is directed to “RNA interference molecules targeted against a nucleic acid sequence, and methods of using these RNAi molecules to reduce off-target toxicity,” the patent application's abstract states.

The Scan

Booster Push

New data shows a decline in SARS-CoV-2 vaccine efficacy over time, which the New York Times says Pfizer is using to argue its case for a booster, even as the lower efficacy remains high.

With Help from Mr. Fluffington, PurrhD

Cats could make good study animals for genetic research, the University of Missouri's Leslie Lyons tells the Atlantic.

Man Charged With Threatening to Harm Fauci, Collins

The Hill reports that Thomas Patrick Connally, Jr., was charged with making threats against federal officials.

Nature Papers Present Approach to Find Natural Products, Method to ID Cancer Driver Mutations, More

In Nature this week: combination of cryogenic electron microscopy with genome mining helps uncover natural products, driver mutations in cancer, and more.