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IP Update: Recent RNAi-Related Patent Applications

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Title: Methods for Treating Eye Disorders

Application Number: 20110229557

Filed: Oct. 22, 2009

Lead Inventor: Elena Feinstein, Quark Pharmaceuticals

The invention, the patent application's abstract states, “relates to compositions and methods for inhibiting loss of a retinal ganglion cell in a subject [by] non-invasively applying to the surface of the eye of the subject an ophthalmic composition comprising a therapeutically effective amount of at least one siRNA which down regulates expression of a target gene associated with loss of the retinal ganglion cell, thereby inhibiting loss of the retinal ganglion cell in the subject. The methods of the invention also relate to the use of chemically modified siRNA compounds possessing structural motifs which down-regulate the expression of human genes expressed in retinal tissue in the mammalian eye.”


Title: Hollow Silica Nanospheres and Methods of Making Same

Application Number: 20110229576

Filed: Aug. 13, 2008

Lead Inventor: William Trogler, University of California, Oakland

The invention, the patent application's abstract states, comprises “hollow nanospheres and methods of making and using the same. The methods and compositions of the disclosure are useful for drug delivery and gene transfer.”


Title: Releasable Cationic Lipids for Nucleic Acids Delivery Systems

Application Number: 20110229581

Filed: Nov. 17, 2009

Lead Inventor: Hong Zhao, Enzon Pharmaceuticals

The invention, the patent application's abstract states, “is directed to releasable cationic lipids and nanoparticle compositions for the delivery of nucleic acids and methods of modulating an expression of a target gene using the same. In particular, the invention relates to cationic lipids, including an acid labile linker, and nanoparticle compositions containing the same.”


Title: Compositions and Methods for Material Transfer into Cells

Application Number: 20110229972

Filed: March 21, 2011

Inventor: Victor Ruiz-Velasco, Pennsylvania State University

The invention comprises “methods for material transfer into a cell ... [including] electroporation of the cell in the presence of the material, such as nucleic acids, and 2,3-butanedione monoxime; and lipofection with a complex of a lipid-based carrier, such as liposomes, and the material, in the presence of 2,3-butanedione monoxime,” according to the patent application's abstract.


Title: Nanoparticle-Based Gene Delivery Systems

Application Number: 20110229966

Filed: March 18, 2010

Lead Inventor: Min Su Han, Chung-Ang University

The invention provides “a gene-delivery system containing nanoparticles,” the patent application's abstract states. More specifically, it “provides a gene delivery system containing a nanomaterial; an oligonucleotide as an universal binding partner covalently linked to the surface of the nanomaterial; and ... cargo comprising a complementary oligonucleotide containing a nucleotide sequence complementary to the universal binding partner as a binding counter-partner, and an inhibitory molecule having a nucleotide sequence complementary to a target gene of interest to be inhibited or an inducible molecule having a nucleotide sequence of a target gene of interest to be expressed."

The invention is capable of delivering aptamers, siRNAs, shRNAs, micromRNAs, and antisense oligonucleotides into cells, it adds.


Title: Low-Density Lipoprotein Receptor-Mediated siRNA Delivery

Application Number: 20110230410

Filed: June 1, 2011

Inventor: Jon Chatterton, Alcon

“The invention provides interfering RNA molecule-ligand conjugates useful as a delivery system for delivering interfering RNA molecules to a cell in vitro or in vivo,” the patent application's abstract states. “The conjugates comprise a ligand that can bind to a low-density lipoprotein receptor, or LDLR, family member. Therapeutic uses for the conjugates are also provided.”


Title: Releasable Cationic Lipids for Nucleic Acids Delivery Systems

Application Number: 20110230420

Filed: Nov. 17, 2009

Lead Inventor: Hong Zhao, Enzon Pharmaceuticals

The invention is “directed to nucleic acid-delivery systems and methods of modulating an expression of a target gene using the same,” the patent application's abstract states. “In particular, the invention relates to nucleic acid conjugates containing an endosomal release-promoting moiety. The nucleic acid conjugates further contain a nuclear localization signal moiety, and/or a cell targeting moiety.”


Title: Compositions and Methods for Inhibiting Expression of the PCSK9 Gene

Application Number: 20110230542

Filed: Sept. 4, 2009

Lead Inventor: Pamela Tan, Alnylam Pharmaceuticals

“The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of the PCSK9 gene comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19 [to] 5 nucleotides in length, and which is substantially complementary to at least a part of the PCSK9 gene,” the patent application's abstract states. “The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier [and] methods for treating diseases caused by PCSK9 gene expression and the expression of the PCSK9 gene using the pharmaceutical composition.”


Title: Oligoribonucleotide Inhibitors of NRF2 and Methods of Use Thereof for Treatment of Cancer

Application Number: 20110230543

Filed: Oct. 8, 2010

Inventor: Elena Feinstein, Quark Pharmaceuticals

“The invention provides novel double-stranded oligoribonucleotides that inhibit the Nrf2 gene,” the patent application's abstract states. “The invention also provides a pharmaceutical composition comprising one or more such oligoribonucleotides, and a vector capable of expressing the oligoribonucleotide. The ... invention also relates to methods and compositions for treating or preventing the incidence or severity of a cancerous disease, particularly various lung cancers.”


Title: dsRNA Compositions and Methods for Treating HPV Infection

Application Number: 20110230546

Filed: May 26, 2011

Lead Inventor: John Benson, Alnylam Pharmaceuticals

“The invention relates to a double-stranded ribonucleic acid for treating human papillomavirus infection,” the patent application's abstract states. “The dsRNA comprises an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19 [to] 25 nucleotides in length, and which is substantially complementary to at least a part of an HPV target gene selected from among HPV E1, HPV E6, and the human E6AP gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by HPV infection and the expression of the E6AP gene using the pharmaceutical composition; and methods for inhibiting the expression of the HPV target genes in a cell.”

The Scan

J&J Booster Support

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To Keep the Cases Moving

The president of the UK Royal College of Pathologists tells the Financial Times that more investment is needed to tackle a backlog of cases.

NAS Expels Archaeologist

Science reports Luis Jaime Castillo Butters' expulsion is the first of an international member from the US National Academy of Sciences.

PLOS Papers on Angelman Syndrome-Like Cases, Salmonella Paratyphi A, SARS-CoV-2 in Brazil

In PLOS this week: exome sequencing analysis of Angelman syndrome-like cases, genetic epidemiology of Salmonella Paratyphi A, and more.