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IP Update: Recent RNAi- and microRNA-Related Patents and Patent Applications: Sep 15, 2011

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Title: RNAi-Mediated Inhibition of Histamine Receptor H1-Related Conditions

Patent Number: 8,017,592

Filed: April 10, 2007

Lead Inventor: John Yanni, Alcon

“RNA interference is provided for inhibition of histamine receptor H1 mRNA expression, in particular, for treating patients having an HRH1-related condition or at risk of developing an HRH1-related condition such as allergic conjunctivitis, ocular inflammation, dermatitis, rhinitis, asthma, or allergy,” according to the patent's abstract.


Title: Adenoviral VA1 Pol III Promoter System for RNAi Expression

Patent Number: 8,017,759

Filed: July 30, 2003

Lead Inventor: John Rossi, City of Hope

The invention, the patent's abstract states, comprises “an adenoviral VA1 Pol III expression system for RNAi expression.”


Title: RNA Interference-Mediated Inhibition of Stearoyl-CoA Desaturase Gene Expression Using Short Interfering Nucleic Acid

Patent Number: 8,017,761

Filed: Aug. 15, 2008

Lead Inventor: James McSwiggen, Merck

“This invention relates to compounds, compositions, and methods useful for modulating stearoyl-CoA desaturase gene expression using short-interfering nucleic acid molecules,” the patent'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 stearoyl-CoA desaturase gene expression and/or activity by RNA interference using small nucleic acid molecules.”


Title: Modified iRNA Agents

Patent Number: 8,017,762

Filed: July 27, 2009

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

“The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group,” the patent's abstract states. “The tether, in turn, can be connected to a selected moiety ... a targeting or delivery moiety, or a moiety which alters a physical property. The cleavable linking group is one [that] is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent coupled by way of the cleavable linking group to a targeting agent ... but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent. The inclusion of such a monomer can allow for modulation of a property of the iRNA agent into which it is incorporated. ... The invention also relates to methods of making and using such modified iRNA agents.”


Title: Chemically Modified Oligonucleotides

Patent Number: 8,017,763

Filed: Sept. 17, 2009

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

The invention relates to "composition and methods for making and using chemically modified oligonucleotide agents for inhibiting gene expression,” the patent's abstract states.


Title: Therapeutic Uses of Inhibitors of RTP801L

Patent Number: 8,017,764

Filed: Oct. 29, 2009

Lead Inventor: Elena Feinstein, Quark Pharmaceuticals

The invention, the patent's abstract states, comprises “novel molecules, compositions, methods, and uses for treating microvascular disorders, eye diseases, respiratory conditions, and hearing disorders based upon inhibition of the RTP801L gene and/or protein.”


Title: RNA Interference-Mediated Treatment of Alzheimer's Disease Using Short Interfering Nucleic Acid

Patent Number: 8,017,765

Filed: Dec. 17, 2009

Lead Inventor: James McSwiggen, Merck

The invention, the patent's abstract states, “concerns methods and reagents useful in modulating BACE gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small nucleic acid molecules... capable of mediating RNA interference against beta-secretase, amyloid precursor protein, pin-1, presenillin 1, and/or presenillin 2 gene expression and/or activity. The small nucleic acid molecules are useful in the treatment of Alzheimer's disease and any other condition that responds to modulation of BACE, APP, pin-1, PS-1, and/or PS-2 expression or activity.”


Title: Lipids, Lipid Complexes, and Uses Thereof

Patent Number: 8,017,804

Filed: May 6, 2005

Lead Inventor: Oliver Keil, Silence Therapeutics

The invention is related to “cationic lipids, compositions containing the same, and use thereof as well as a method for transferring chemical compounds into cells,” the patent states.


Titles: Group of Nucleic Acid Fragments for Prevention of HIV Infection or AIDS and the Usage Thereof

Application Numbers: 20110217361, 20110217362

Filed: Jan. 10, 2011

Lead Inventor: Zhiwen Shou, Beijing Solobio Genetechnology

The invention, the patent applications' abstracts state, “provides a group of nucleic acid fragments ... for prevention of HIV infection or AIDS and the usage thereof. In the invention, a series of RNA fragments, which are highly homogenous to all the published HIV gene sequences, were obtained by homology [comparison]. The double-stranded RNA derived from these fragments can effectively inhibit the expression of the HIV genes. The RNA transcribed by [a] plasmid also can suppress the expression of the HIV in the cell.”


Title: Means for Inhibiting the Expression of ORC-1

Application Number: 20110217367

Filed: Sept. 23, 2009

Lead Inventor: Manfred Gossen, Silence Therapeutics

The invention, the patent application's abstract states, is “related to a nucleic acid molecule comprising a double-stranded structure … whereby the first strand comprises a ... stretch of contiguous nucleotides ... at least partially complementary to a target nucleic acid and ... the second strand comprises a ... stretch of contiguous nucleotides … at least partially complementary to the first stretch.”


Title: Cell Type-Specific Aptamer-siRNA Delivery System for HIV-1 Therapy

Application Number: 20110217769

Filed: May 16, 2011

Lead Inventor: John Rossi, City of Hope

The invention, according to the patent application's abstract, “relates to compositions and methods for delivery of siRNA to specific cells or tissue. More particularly, the ... invention relates to compositions and methods for cell type-specific delivery of anti-HIV siRNAs via fusion to an anti-gp120 aptamer.”


Title: Phosphorothioate Oligonucleotides and Non-Nucleosidic Phosphorothioates as Delivery Agents for iRNA Agents

Application Number: 20110218334

Filed: July 10, 2009

Lead Inventor: Martin Maier, Alnylam Pharmaceuticals

“The invention relates to use of single-stranded phosphorothioate oligonucleotides or non-nucleosidic phosphorothioate as vehicles or carriers to modulate the biodistribution of iRNA agents,” the patent application's abstract states.


Title: Gene Vector for Inducing Transgene-Specific Immune Tolerance

Application Number: 20110218234

Filed: Nov. 11, 2009

Lead Inventor: Andrea Annoni, San Raggaele del Monte Tabor Foundation

“A gene vector adapted for transient expression of a transgene in a peripheral organ cell comprising a regulatory sequence operably linked to a transgene wherein the regulatory sequence prevents or reduces expression of said transgene in hematopoietic lineage cells,” the patent application's abstract states.

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.