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IP Update: Recent Patents, Patent Applications Awarded to Merck, Alnylam, CSHL, and More


Title: Enhanced Biotherapeutic Production Using Inhibitory RNA

Patent Number: 8,273,722

Filed: July 11, 2008

Lead Inventor: James Ladine, Dharmacon (Thermo Fisher Scientific)

The invention, the patent's abstract states, comprises “compositions, kits, systems, equipment, and protocols [that] utilize synthetic siRNA having a delivery-facilitating moiety in improved bioprocesses that enhance the production of biomaterials. The siRNA can target genes associated with ... deleterious vector derived genes; genes that confer non-optimal growth or differentiation properties to the cells; genes that can influence heterogeneity or [the] post-translational modification pattern of the desirable gene product; genes that highly express non-desired proteins; genes that express proteins which interfere with purification of the desired protein; and other genes that can interfere with the bioprocess.”

Title: RNA Interference-Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid

Patent Number: 8,273,866

Filed: Nov. 24, 2003

Lead Inventor: James McSwiggen, Merck

The invention, the patent's abstract states, “concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules … capable of mediating RNA interference against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.”

Title: Transducible Delivery of siRNA by dsRNA Binding Domain Fusions to PTD/CPPS

Patent Number: 8,273,867

Filed: Feb. 9, 2007

Lead Inventor: Steven Dowdy, University of California

The invention relates to “fusion polypeptides and constructs useful in delivering anionically charged nucleic acid molecules including diagnostics and therapeutics to a cell or subject,” according to the patent's abstract. “The fusion constructs include a protein transduction domain and a nucleic acid binding domain, or a protein transduction domain and a nucleic acid that is coated with one or more nucleic acid binding domains sufficient to neutralize an anionic charge on the nucleic acid. Also provided are methods of treating disease and disorders such as cell proliferative disorders.”

Title: Compositions and Methods for Inhibiting Viral Replication

Patent Number: 8,273,868

Filed: Dec. 4, 2009

Lead Inventor: John Matthias, Alnylam Pharmaceuticals

The invention relates to “a double-stranded ribonucleic acid having a nucleotide sequence [that] is less than 30 nucleotides in length and … substantially identical to at least a part of a 3'-untranslated region of a [plus] strand RNA virus, such as HCV, as well as pharmaceutical compositions comprising the dsRNA, together with a pharmaceutically acceptable carrier,” the patent's abstract states. “The pharmaceutical compositions are useful for treating infections and diseases caused by the replication or activity of the [plus] strand RNA virus, as well as methods for inhibiting viral replication.”

Title: Lipid Formulated dsRNA Targeting the PCSK9 Gene

Patent Number: 8,273,869

Filed: June 15, 2010

Lead Inventor: Kevin Fitzgerald, Alnylam Pharmaceuticals

“This invention relates to composition and methods using lipid formulated siRNA targeted to a PCSK9 gene,” the patent's abstract states.

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

Patent Number: 8,273,870

Filed: July 6, 2011

Lead Inventor: Roland Kreutzer, Alnylam Pharmaceuticals

The invention, the patent's abstract states, relates to “a double-stranded ribonucleic acid for inhibiting the expression of a mutant gene, comprising a first strand that has a complementary region that is complementary to at least a portion of an RNA transcript of at least part of the mutant target gene and a second strand of the dsRNA complementary or substantially complementary to the first strand. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target mutant gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene.”

Title: Structurally Designed shRNAs

Patent Number: 8,273,871

Filed: Oct. 4, 2011

Lead Inventor: Gregory Hannon, Cold Spring Harbor Laboratory

The invention, the patent's abstract states, relates to an “improved design of shRNA based on structural mimics of miR-451 precursors. These miR-451 shRNA mimics are channeled through a novel small RNA biogenesis pathway, require Ago2 catalysis and are processed by Drosha but are independent of Dicer processing. This miRNA pathway feeds active elements only into Ago2 because of its unique catalytic activity. These data demonstrate that this newly identified small RNA biogenesis pathway can be exploited in vivo to produce active molecules.”

Title: Novel siRNA Structure for Minimizing Off-Target Effects and Relaxing Saturation of RNAi Machinery and the Use Thereof

Application Number: 20120238017

Filed: Dec. 8, 2008

Lead Inventor: Dong Ki Lee, Sungkyunkwan University

The invention, the patent application's abstract states, “relates to a novel siRNA structure and the use thereof. More particularly, the invention relates to a double-stranded small interfering RNA molecule comprising a 19 [to] 21 nucleotide antisense strand and a 15 [to] 19 [nucleotide] sense strand having a sequence complementary to the antisense sequence, wherein the 5' end of the antisense strand has a blunt end and the 3' end of the antisense strand has an overhang, and to a method for silencing the expression of a target gene using the siRNA molecule.”

Title: Inducible Systems and Methods for Controlling siRNA Expression

Application Number: 20120238022

Filed: March 20, 2012

Lead Inventor: Hoshang Unwalla, City of Hope

The invention comprises “an inducible system and methods for controlling expression of siRNA,” the patent application's abstract states. “An inducible system for producing siRNA only in the presence of HIV TAT, and methods for inhibiting HIV-1 gene expression in cells comprising such inducible system also are provided.”

Title: Monoconjugated Chitosans as Delivery Agents for Small Interfering Nucleic Acids

Application Number: 20120238735

Filed: Sept. 16, 2010

Lead Inventor: Samuel McManus, Nektar Therapeutics

The patent application, its abstract states, claims “conjugates ... having a single water-soluble, non-peptidic polymer, such as a poly-ethylene oxide, attached to a chitosan. Complexes of small interfering nucleic acids formed with such conjugates, as well as small interfering nucleic acids attached to such conjugates, are also provided. Related methods, intermediates, and compositions are also provided.”

The Scan

Study Points to Tuberculosis Protection by Gaucher Disease Mutation

A mutation linked to Gaucher disease in the Ashkenazi Jewish population appears to boost Mycobacterium tuberculosis resistance in a zebrafish model of the lysosomal storage condition, a new PNAS study finds.

SpliceVault Portal Provides Look at RNA Splicing Changes Linked to Genetic Variants

The portal, described in Nature Genetics, houses variant-related messenger RNA splicing insights drawn from RNA sequencing data in nearly 335,700 samples — a set known as the 300K-RNA resource.

Automated Sequencing Pipeline Appears to Allow Rapid SARS-CoV-2 Lineage Detection in Nevada Study

Researchers in the Journal of Molecular Diagnostics describe and assess a Clear Labs Dx automated workflow, sequencing, and bioinformatic analysis method for quickly identifying SARS-CoV-2 lineages.

UK Team Presents Genetic, Epigenetic Sequencing Method

Using enzymatic DNA preparation steps, researchers in Nature Biotechnology develop a strategy for sequencing DNA, along with 5-methylcytosine and 5-hydroxymethylcytosine, on existing sequencers.