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

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Title: Method of Diagnosing Acute Lymphomic Leukemia Using miR-146a

Patent Number: 8,372,586

Filed: Oct. 17, 2011

Inventor: Carlo Croce, Ohio State University

The invention, the patent's abstract states, comprises “compositions and methods for reducing the proliferation of ALL cancer cells through targeted interactions with ALL1 fusion proteins.”


Title: Methods and Compositions for the Specific Inhibition of KRAS by Asymmetric Double-Stranded RNA

Patent Number: 8,372,816

Filed: April 5, 2010

Inventor: Bob Brown, Dicerna Pharmaceuticals

“This invention relates to compounds, compositions, and methods useful for reducing KRAS target RNA and protein levels via use of Dicer-substrate siRNA agents possessing asymmetric end structures,” the patent's abstract states.


Title: Small Interfering RNA for Gene Knockdown of the Subcutaneous N-Methyl-D-Aspartate Receptor NR1 Subunit and Its Application on Pharmaceutics

Patent Number: 8,372,817

Filed: May 14, 2010

Inventor: Ping-Heng Tan, I-Shou University

The patent, its abstract states, claims “a small interfering RNA for gene knockdown of the N-methyl-D-aspartate receptor NR1 subunit … [as well as a] method of using the small interfering RNA [by] applying the small interfering RNA on subcutaneous tissues [to temporarily] interfere with the genetic expression of the NMDA receptor NR1 subunit in [the] hypoderm.” Also claimed is the use of the siRNA to treat inflammatory or chronic pain.


Title: RNA Interference-Mediating Small RNA Molecules

Patent Number: 8,372,968

Filed: Aug. 7, 2009

Lead Inventor: Thomas Tuschl, Mack Planck Institute

“Double-stranded RNA induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference,” the patent's abstract states. “Using a Drosophila in vitro system, we demonstrate that 19-23 [nucleotide] short RNA fragments are the sequence-specific mediators of RNAi. The short interfering RNAs are generated by an RNase III-like processing reaction from long dsRNA. Chemically synthesized siRNA duplexes with overhanging 3' ends mediate efficient target RNA cleavage in the lysate, and the cleavage site is located near the center of the region spanned by the guiding siRNA. Furthermore, we provide evidence that the direction of dsRNA processing determines whether sense or antisense target RNA can be cleaved by the produced siRNP complex.”


Title: RNA Interference Methods Using DNA-RNA Duplex Constructs

Patent Number: 8,372,969

Filed: Oct. 25, 2010

Lead Inventor: Shao-Yao Ying, University of Southern California

The invention, the patent's abstract states, “provides novel compositions and methods for suppressing the function or activity of a targeted gene through a novel intracellular piRNA-mediated RNAi mechanism using RNA-DNA duplex constructs. The invention further provides novel methods and compositions for generating or producing RNA-DNA duplex agents, whose quantity is high enough to be used for the invention's gene silencing transfection and possibly in therapeutic applications. This improved RNA-polymerase chain reaction method utilizes thermocycling steps of promoter-linked DNA or RNA template synthesis, in vitro transcription, and then reverse transcription to bring up the amount of RNA-DNA duplexes up to two thousand folds within one round of the above procedure for using in D-RNAi-directed gene silencing.”


Title: Oligonucleotide Compounds Comprising Non-Nucleotide Overhangs

Application Number: 20130035368

Filed: Jan. 6, 2011

Lead Inventor: Sharon Avkin-Nachum, Quark Pharmaceuticals

“The invention relates to siRNA compounds comprising one non-nucleotide moiety covalently attached to at least one of the sense or antisense strands to down-regulate the expression of human genes,” according to the patent application's abstract. “The invention also relates to pharmaceutical compositions comprising such compounds and a pharmaceutically acceptable carrier and to methods of treating and/or preventing the incidence or severity of various diseases or conditions associated with the target genes and/or symptoms associated with such diseases or conditions.”


Title: Double-Stranded Compositions Comprising Differentially Modified Strands for use in Gene Modulation

Application Number: 20130035369

Filed: Aug. 3, 2012

Lead Inventor: Balkrishen Bhat, Isis Pharmaceuticals

The invention, the patent application's abstract states, provides “double-stranded compositions wherein the first strand is modified to have a particular motif and the second strand is a modified selected motif. More particularly, the ... compositions comprise an antisense strand that is modified to have a positional/full motif and the sense strand is modified to have an alternating motif, a hemimer motif, a blockmer motif, a gapped motif, a positional motif, a positional/full motif, or a fully modified motif. Each strand further comprises one or more phosphorothioate internucleoside linkage[s]. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes.”


Title: Lipid-Formulated dsRNA Targeting the PCSK9 Gene

Application Number: 20130035371

Filed: Aug. 7, 2012

Lead Inventor: Kevin Fitzgerald, Alnylam Pharmaceuticals

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


Title: microRNA Induction of Pluripotent Stem Cells and Uses Thereof

Application Number: 20130035374

Filed: March 25, 2011

Inventor: Edward Morrisey, University of Pennsylvania

The invention, the patent application's abstract states, relates to “compositions and methods for inducing the formation of an induced pluripotential stem cell from a somatic cell. … The compositions comprise miR 302-367 cluster and valproic acid. Further disclosed are methods for treatment of a disease or condition in a subject through the use of the compositions.”

The Scan

For Better Odds

Bloomberg reports that a child has been born following polygenic risk score screening as an embryo.

Booster Decision Expected

The New York Times reports the US Food and Drug Administration is expected to authorize a booster dose of the Pfizer-BioNTech SARS-CoV-2 vaccine this week for individuals over 65 or at high risk.

Snipping HIV Out

The Philadelphia Inquirer reports Temple University researchers are to test a gene-editing approach for treating HIV.

PLOS Papers on Cancer Risk Scores, Typhoid Fever in Colombia, Streptococcus Protection

In PLOS this week: application of cancer polygenic risk scores across ancestries, genetic diversity of typhoid fever-causing Salmonella, and more.