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IP Update: Recent Patents, Patent Applications Awarded to Alnylam, Nitto Denko, Silence, and More


Title: Method to Trigger RNA Interference

Patent Number: 8,476,422

Filed: Aug. 24, 2011

Lead Inventor: James Carrington, Oregon State University

The invention, the patent’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: Method and Medicament for Inhibiting the Expression of a Given Gene

Application Number: 20130164366

Filed: Oct. 19, 2012

Lead Inventor: Roland Kreutzer, Alnylam Pharmaceuticals

The invention, the patent application’s abstract states, “relates to an isolated RNA that mediates RNA interference of an mRNA to which it corresponds and a method of mediating RNA interference of mRNA of a gene in a cell or organism using the isolated RNA.”

Title: Single-use System for Sterilely Producing Lipid-Nucleic Acid Particles

Application Number: 20130164400

Filed: Nov. 5, 2012

Lead Inventor: Victor Knopov, Nitto Denko

The invention comprises a “process of forming lipid-nucleic acid nanoparticles simply and reproducibly under aseptic conditions comprising single-use components.”

Title: Compositions and Methods for the Delivery of Biologically Active RNAs

Application Number: 20130164845

Filed: Dec. 24, 2012

Lead Inventor: Kevin Polach, EGEN

The patent application, its abstract states, claims “novel compounds, compositions, and methods for the delivery of biologically active RNA molecules to cells. Specifically, the invention provides novel nucleic acid molecules, polypeptides, and RNA-protein complexes useful for the delivery of biologically active RNAs to cells and polynucleotides encoding the same. The invention also provides vectors for expressing said polynucleotides. In addition, the invention provides cells and compositions comprising the novel compounds and vectors, which can be used as transfection reagents. The invention further provides methods for producing said compounds, vectors, cells, and compositions. Additionally, vectors and methods for delivering biologically active RNA molecules to cells and/or tissues are provided. The novel compounds, vectors, cells, and compositions are useful, for example, in delivering biologically active RNA molecules to cells to modulate target gene expression in the diagnosis, prevention, amelioration, and/or treatment of diseases, disorders, or conditions in a subject or organism.”

Title: Lipids, Lipid Complexes, and Use Thereof

Application Number: 20130165381

Filed: Jan. 16, 2013

Lead Inventor: Oliver Keil, Silence Therapeutics

The invention, the patent application states, is related to a method of treating cancer and/or a cardiovascular disease by administering a pharmaceutically active compound such as a nucleic acid and a lipid carrier of a pre-defined formula.

Title: Invertebrate microRNAs

Application Number: 20130165498

Filed: Jan. 22, 2013

Lead Inventor: Edwards Allen, Monsanto

“This invention provides plants having resistance to invertebrate pests,” the patent application’s abstract states. “More specifically, this invention discloses a non-natural transgenic plant cell expressing at least one invertebrate miRNA in planta for suppression of a target gene of an invertebrate pest or of a symbiont associated with the invertebrate pest. Also provided are recombinant DNA constructs for expression of at least one invertebrate miRNA in planta, a non-natural transgenic plant containing the non-natural transgenic plant cell of this invention, a non-natural transgenic plant grown from the non-natural transgenic plant cell of this invention, and non-natural transgenic seed produced by the non-natural transgenic plants, as well as commodity products produced from a non-natural transgenic plant cell, plant, or seed of this invention. This invention further provides a method of suppressing at least one target gene of an invertebrate pest of a plant or of a symbiont associated with the invertebrate, including providing a plant including the non-natural transgenic plant cell of this invention, wherein the invertebrate is the invertebrate pest, the recombinant DNA is transcribed in the non-natural transgenic plant cell to the recombinant miRNA precursor, and when the invertebrate pest ingests the recombinant miRNA precursor, the at least one target gene is suppressed.”

Title: Methods and Compositions for Prevention or Treatment of RSV Infection

Application Number: 20130165499

Filed: Feb. 27, 2013

Lead Inventor: Akshay Vaishnaw, Alnylam Pharmaceuticals

The patent application, its abstract states, claims “methods and compositions … for the prevention or treatment of RSV infection in a human. The methods include administering one or more doses of a composition comprising an siRNA. The dose can be formulated for topical or parenteral administration. Topical administration includes administration as a nasal spray, or by inhalation of respirable particles or droplets.”

Title: RNA Interference-mediated Inhibition of Prolyl Hydroxylase Domain 2 Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20130165500

Filed: Feb. 22, 2013

Lead Inventor: Brandon Ason, Merck

The invention, the patent application’s abstract states, “relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases, and conditions that respond expressed/synthetic to the modulation of PHD2 gene expression and/or activity, and/or modulate a beta-catenin gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules … capable of mediating or that mediate RNA interference against PHD2 gene expression.”

Title: Diagnostic, Prognostic, and Therapeutic Uses of miRs in Adaptive Pathways and Disease Pathways

Application Number: 20130165502

Filed: Dec. 11, 2012

Lead Inventor: Clay Marsh, Ohio State University

The patent application, its abstract states, claims “methods and compositions for the diagnosis, prognosis, and treatment of various adaptive and/or disease pathways by examining samples containing one or more [miRNAs] therein, and by formulating therapeutic agents therefrom.”

The Scan

Positive Framing of Genetic Studies Can Spark Mistrust Among Underrepresented Groups

Researchers in Human Genetics and Genomics Advances report that how researchers describe genomic studies may alienate potential participants.

Small Study of Gene Editing to Treat Sickle Cell Disease

In a Novartis-sponsored study in the New England Journal of Medicine, researchers found that a CRISPR-Cas9-based treatment targeting promoters of genes encoding fetal hemoglobin could reduce disease symptoms.

Gut Microbiome Changes Appear in Infants Before They Develop Eczema, Study Finds

Researchers report in mSystems that infants experienced an enrichment in Clostridium sensu stricto 1 and Finegoldia and a depletion of Bacteroides before developing eczema.

Acute Myeloid Leukemia Treatment Specificity Enhanced With Stem Cell Editing

A study in Nature suggests epitope editing in donor stem cells prior to bone marrow transplants can stave off toxicity when targeting acute myeloid leukemia with immunotherapy.