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IP Update: Jan 27, 2011

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Title: Oligonucleotide Non-Viral Delivery Systems

Patent Number: 7,875,449

Filed: Sept. 14, 2007

Lead Inventor: Per Artur Sven Artursson, FMC Biopolymer

"Low molecular weight chitosan oligomers were able to self-assemble siRNA into nanosized particles, provide protection against enzymatic degradation, and mediate gene silencing that is stable over a long period of time in vitro," the patent's abstract states. "The control of structural variables in formulating complexes of siRNA with low molecular weight chitosans provides an efficient alternative delivery system for siRNA in vitro and in vivo."


Title: Compositions and Methods for Inhibiting Expression of XBP-1 Gene

Patent Number: 7,875,711

Filed: April 17, 2009

Lead Inventor: Kevin Fitzgerald, Alnylam Pharmaceuticals

"The invention relates to a double-stranded ribonucleic acid targeting X-Box Protein 1 and methods of using the dsRNA to inhibit expression of XBP-1," the patent's abstract states.


Title: Oligomeric Compounds Comprising 4'-Thionucleosides for Use in Gene Modulation

Patent Number: 7,875,733

Filed: Sept. 20, 2004

Lead Inventor: Balkrishen Bhat, Isis Pharmaceuticals

The invention "provides modified oligomeric compounds and compositions of oligomeric compounds for use in the RNA interference pathway of gene modulation," the patent's abstract states. "The modified oligomeric compounds include siRNA and asRNA having at least one affinity modification."


Title: RNA Interference-Mediating Small RNA Molecules

Application Number: 20110014123

Filed: Dec. 2, 2009

Lead Inventor: Thomas Tuschl, Max Planck Institute

"Double-stranded RNA induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference," the patent application's abstract states.
"Using a Drosophila in vitro system, we demonstrate that [19 to 23 nucleotide-long] 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," it adds. "Furthermore, we provide evidence that the direction of dsRNA processing determines whether sense or antisense target RNA can be cleaved by the produced siRNA complex."


Title: Compositions and Methods to Treat Asthma

Application Number: 20110014133

Filed: Aug. 13, 2010

Inventor: Michael Grunstein, Children's Hospital of Philadelphia

The invention provides "compositions and methods for the treatment of asthma," the patent application's abstract states. "The compositions can be, for example, siRNA directed to CD23. The invention also provides a method of treating asthma with a formulation for in vivo delivery of a CD23 siRNA to inhibit IgE binding in a patient."


Title: Compositions and Methods for Inhibiting or Inducing Adipogenesis

Application Number: 20110014275

Filed: Aug. 30, 2010

Inventor: Dechun Li, Saint Louis University

The invention, the patent application's abstract states, relates to "compositions and methods which utilization nuclear receptors for regulating adipogenesis in cells. Specifically, the invention is directed to compositions [that] regulate transcription factor PPAR gamma, and enhance or inhibit the transcription of genes responsible for directing cell differentiation towards a pathway of adipogenesis."

More specifically, the invention involves "a novel polypeptide co-activator of PPAR gamma and fragments thereof [that] possess co-activator or co-repressor activity," the abstract adds. "Also related are nucleotide sequences [that] express these polypeptides … [and] an interfering RNA that may be used to inhibit adipogenesis."


Title: Solution-Based Methods for RNA Expression Profiling

Application Number: 20110015080

Filed: Aug. 27, 2010

Lead Inventor: Todd Golub, Massachusetts Institute of Technology

The invention is relates to "novel high-throughput, low-cost, and flexible solution-based methods for RNA expression profiling, including expression of microRNAs and mRNAs," according to the patent application's abstract.


Title: High-Throughput Methods for Functionally Determining RNA Interference Efficiency

Application Number: 20110015093

Filed: Oct. 24, 2008

Lead Inventor: Christof Fellman, Cold Spring Harbor Laboratory

The invention comprises "a single construct combining a sequence encoding an RNAi molecule, a sequence encoding a reporter, and a target sequence specific for the RNAi molecule," the patent application's abstract states. "The construct can be used to determine the potency of the encoded RNAi molecule in a direct and unbiased way. These results can be used to inform the design of potent RNAi molecules of various types and can be extended to several other applications, including [the] generation of tiled libraries comprising every possible RNAi molecule-encoding sequence for a given gene target; large-scale parallel validation of RNAi molecules targeting many genes to generate validated RNAi molecule-encoding libraries; experimental comparison of design algorithms and strategies; and investigation of RNAi biology in target site mutagenesis assays by screening pools containing single nucleotide changes in target sites and/or in the RNAi molecule to identify the most relevant sequence characteristics of potent RNAi-target site predictions."


Title: Methods and Compositions for Treatment of Cancer and Other Angiogenesis-Related Diseases

Application Number: 20110015249

Filed: July 3, 2008

Lead Inventor: Frank Xie, Intradigm

The invention, the patent application's abstract states, "provides nucleic acid molecules that modulate the expression of molecules in the angiopoietin/Tie2 signaling pathway. Methods of using the nucleic acid molecules are also provided."


Title: Compositions and Methods for Inhibiting Expression of Eg5 Gene

Application Number: 20110015250

Filed: April 5, 2010

Lead Inventor: David Bumcrot, Alnylam Pharmaceuticals

"The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of the Eg5 gene comprising an antisense strand having a nucleotide sequence which is less than 30 nucleotides in length, generally 19 [to] 25 nucleotides in length, and which is substantially complementary to at least a part of the Eg5 gene," the patent application's abstract states. "The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier, methods for treating diseases caused by Eg5 expression and the expression of the Eg5 gene using the pharmaceutical composition, and methods for inhibiting the expression of the Eg5 gene in a cell."


Title: RNA Interference-Mediated Inhibition of Human Immunodeficiency Virus Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20110015251

Filed: May 11, 2010

Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)

"This invention relates to compounds, compositions, and methods useful for modulating human immunodeficiency virus gene expression using short interfering nucleic acid molecules," the patent application's abstract states. "This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of human immunodeficiency virus gene expression and/or activity by RNA interference using small nucleic acid molecules. … The small nucleic acid molecules are useful in the treatment of HIV infection, AIDS, and/or disease and conditions related to HIV infection and/or AIDS in a subject or organism."


Title: Lipid-Formulated dsRNA Targeting the PCSK9 Gene

Application Number: 20110015252

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 application's abstract states.

The Scan

Review of Approval Process

Stat News reports the Department for Health and Human Services' Office of the Inspector General is to investigate FDA's approval of Biogen's Alzheimer's disease drug.

Not Quite Right

A new analysis has found hundreds of studies with incorrect nucleotide sequences reported in their methods, according to Nature News.

CRISPR and mRNA Together

Time magazine reports on the use of mRNA to deliver CRISPR machinery.

Nature Papers Present Smartphone Platform for DNA Diagnosis of Malaria, Mouse Lines for Epigenomic Editing

In Nature this week: a low-cost tool to detect infectious diseases like malaria, and more.