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IP Update: Recent Patents, Patent Applications Awarded to Alnylam, Tekmira, Opko, and More

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Title: Enhancement of Influenza Gene Expression

Patent Number: 8,227,188

Filed: Sept. 3, 2010

Lead Inventor: Antonin de Fougerolles, Alnylam Pharmaceuticals

“The invention relates to compositions and methods for modulating the expression of influenza viral genes, and more particularly to the down-regulation of influenza viral genes by chemically modified oligonucleotides,” the patent's abstract states.


Title: Silencing of CSN5 Gene Expression Using Interfering RNA

Patent Number: 8,227,443

Filed: Oct. 13, 2010

Lead Inventor: Ian MacLachlan, Protiva Biotherapeutics (Tekmira Pharmaceuticals)

The invention, the patent's abstract states, “provides compositions comprising nucleic acids that target CSN5 gene expression and methods of using such compositions to silence CSN5 gene expression. More particularly, the ... invention provides unmodified and chemically modified interfering RNA molecules which silence CSN5 gene expression and methods of use thereof. ... The ... invention also provides nucleic acid-lipid particles that target CSN5 gene expression comprising an interfering RNA molecule, a cationic lipid, a non-cationic lipid, and optionally a conjugated lipid that inhibits aggregation of particles.”


Title: Compositions and Methods for Inhibition of VEGF

Patent Number: 8,227,444

Filed: Dec. 6, 2010

Inventor: Nadine Dejneka, Opko Health

The invention, the patent's abstract states, comprises ”siRNA compositions and methods useful for inhibiting expression of vascular endothelial growth factor isoforms. Diseases which involve angiogenesis stimulated by over-expression of VEGF, such as diabetic retinopathy, age-related macular degeneration, and many types of cancer, can be treated by administering small interfering RNAs as disclosed.”


Title: Alternative Export Pathways for Vector-Expressed RNA Interference

Patent Number: 8,227,592

Filed: Nov. 28, 2007

Lead Inventor: Scott Harper, University of Iowa

The invention, the patent's abstract states, relates to “nucleic acid molecules containing a loop sequence designed to circumvent exportin-5 mediated export, and methods using these novel molecules.”


Title: Novel Lipid Formulations for Nucleic Acid Delivery

Application Number: 20120183581

Filed: Oct. 5, 2011

Lead Inventor: Edward Yaworski, Protiva Biotherapeutics (Tekmira Pharmaceuticals)

The invention, the patent application's abstract states, comprises “novel, stable lipid particles comprising one or more active agents or therapeutic agents, methods of making the lipid particles, and methods of delivering and/or administering the lipid particles. More particularly, the ... invention provides stable nucleic acid-lipid particles comprising a nucleic acid, methods of making the SNALP, and methods of delivering and/or administering the SNALP.”


Title: Design and Construction of Bi-Functional Short Hairpin RNA

Application Number: 20120183955

Filed: Feb. 1, 2012

Inventor: Donald Rao, Gradalis

The invention comprises “a method for designing a bi-shRNA expression cassette encoding a bi-shRNA [by] selecting one or more target site sequences; providing a backbone sequence comprising a first and a second stem-loop structure; inserting a first passenger strand and a second passenger strand; and providing for synthesis of the bi-shRNA expression cassette,” according to the patent application's abstract.


Title: Lipid Formulation

Application Number: 20120183602

Filed: Jan. 25, 2012

Lead Inventor: Jianxin Chen, Alnylam Pharmaceuticals

The patent application, its abstract states, comprises a cationic lipid of a specific formulation and methods of use.


Title: Modulation of miRNA Activity

Application Number: 20120184457

Filed: Aug. 6, 2010

Lead Inventor: Eric Miska, University of Cambridge

The patent application, its abstract states, comprises “a method of identifying a compound which modulates miRNA activity.” The method involves “determining the ability of a test compound to alter the polyuridylation activity of a ZCCHC polypeptide wherein a test compound, which alters the polyuridylation activity, is useful in modulating miRNA activity; or determining the ability of a test compound to alter the binding of a ZCCHC polypeptide to a LIN28 polypeptide, wherein a test compound which alters said binding may be useful in modulating miRNA activity; or determining the ability of a test compound to bind to a ZCCHC polypeptide, wherein a test compound which binds to the ZCCHC polypeptide may be useful in modulating miRNA activity.”


Title: siRNA Targeting Connective Tissue Growth Factor

Application Number: 20120184463

Filed: Feb. 14, 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 CTGF.”


Title: Novel Human microRNAs Associated with Cancer

Application Number: 20120184603

Filed: March 30, 2012

Lead Inventor: Thomas Litman, Exiqon

“The invention provides new sequences for human microRNAs associated with cancer, which may be used as molecular markers for cancer diagnostics or as therapeutic targets or agents,” the patent application's abstract states.


Title: Short RNA Mimetics

Application Number: 20120184726

Filed: Sept. 13, 2010

Inventor: Kevin Forbes, Sigma-Aldrich

The invention, the patent application's abstract states, “provides synthetic oligonucleotides that mimic the function of short RNAs such as, for example, microRNAs or short interfering RNAs. In particular, the synthetic oligonucleotides comprise a duplex region comprising an unpaired bulge in one of the strands.”

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