Title: Method of Treating Neurodegenerative Disease
Filed: Oct. 12, 2006
Inventor: David Bumcrot, Alnylam Pharmaceuticals
"Aspects featured in the invention relate to compositions and methods for inhibiting alpha-synuclein gene expression, such as for the treatment of neurodegenerative disorders," the patent's abstract states. "An anti-SNCA agent featured herein that targets the SNCA gene can have been modified to alter distribution in favor of neural cells."
The patent specifically claims the use of an RNAi agent to inhibit alpha-synuclein expression.
Title: Modified Polynucleotides for Reducing Off-Target Effects in RNA Interference
Filed: Dec. 22, 2004
Lead Inventor: Devin Leake, Dharmacon (Thermo Fisher Scientific/Merck)
The invention, the patent's abstract states, comprises "methods and compositions for performing RNA interference with decreased off-target effects. … The methods and compositions permit effective and efficient applications of RNA interference to applications such as diagnostics and therapeutics through the use of modifications to the siRNA. Uniquely modified siRNAs have been developed that reduce off-target effects incurred in gene silencing. The modifications comprise 2'-O-alkyl or mismatch modifications at specific positions on the sense and/or antisense strands."
Title: siRNA Targeting EPH Receptor A3
Filed: June 5, 2007
Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)
"Efficient sequence-specific gene silencing is possible through the use of siRNA technology," the patent'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 EPHA3."
Title: siRNA Targeting Casitas B Cell Lymphoma-B
Filed: July 23, 2007
Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)
"Efficient sequence-specific gene silencing is possible through the use of siRNA technology," the patent'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 CBL-B."
Title: Staple Type Oligonucleotide and Drug Comprising the Same
Filed: Sept. 29, 2004 PCT Filed: Sept. 29, 2004
Lead Inventor: Yasuo Kunugiza, Anges MG
"Conventional oligonucleotides are opened at both ends and thereby unstable," the patent's abstract states. "Their stability against catabolic enzymes is increased by phosphorothioate modification, but such phosphorothioate causes toxicity. The … invention provides oligonucleotides and medicaments in which these problems are improved."
Specifically, the invention comprises "transcription factor inhibitors, antisense oligonucleotides, and siRNAs … [and] provides agents for preventing, treating, or improving inflammation, autoimmune diseases, central diseases, reperfusion injury in ischaemic diseases, worsened prognosis after organ transplantation or organ surgery, or restenosis after" percutaneous transluminal coronary angioplasty, the abstract states. "Further … it provides agents for preventing, treating, or improving arthritis, dermatitis, nephritis, hepatitis, renal failure, cystitis, prostatitis, urethritis, ulcerative colitis, [Crohn's] disease, chronic rheumatoid arthritis, osteoarthritis, atopic dermatitis, contact dermatitis, psoriasis, cutaneous ulcer, or decubitus."
Title: Methods and Compositions for Prevention or Treatment of RSV Infection
Filed: Dec. 15, 2008
Lead Inventor: Akshay Vaishnaw, Alnylam Pharmaceuticals
The invention, according to the patent application's abstract, comprises "methods and compositions … for the prevention or treatment of RSV infection in a human … [including] administering one or more doses of a composition comprising an siRNA. The dose can be formulated for topical or parenteral administration. Topical administration includes administration as a nasal spray, or by inhalation of respirable particles or droplets.
"The siRNA comprises a sense strand of ALN-RSV01 and an antisense strand of ALN-RSV01," the abstract adds.
Title: Toll-Like Receptor Stimulation for Ocular Angiogenesis and Macular Degeneration
Filed: May 15, 2007 PCT Filed: May 15, 2007
Inventor: Jayakrishina Ambati, University of Kentucky
The patent application, its abstract states, claims "methods and compositions for the treatment or prevention of ocular angiogenesis and neovascularization. Administration of stimulators of the TLR3 and TLR7 receptors, TRIF … or IL-10 and IL-12 inhibits ocular angiogenesis. Furthermore, all siRNAs, both targeted and non-targeted, can inhibit ocular angiogenesis," it notes.
Title: Methods and Compositions for Increasing RNA Interference
Filed: Feb. 20, 2009
Lead Inventor: Romaan Raemaekers, Devgen
"The … invention relates to compositions containing a combination of a first active component, comprising one or more sulfated polysaccharides and/or glycosaminoglycans, and a second active component, comprising one or more RNAi-inducing molecules," the patent application's abstract states. Additionally, the invention relates to "methods for using these compositions to enhance double-stranded RNA-mediated gene silencing in pest or pathogen species. The invention further relates to methods for controlling pests or pathogens, methods for preventing pest infestations or pathogen infections, and methods for knocking down gene expression in pests or pathogens using the compositions and methods of the invention."
Title: Liver-Specific Nanocapsules and Methods of Using
Filed: April 27, 2007 PCT Filed: April 27, 2007
Lead Inventor: Betsy Kren, University of Minnesota (GeneSegues)
The invention comprises "liver-specific nanocapsules for specifically targeting liver cells … [as well as] methods of using such liver-specific nanocapsules to deliver one or more cargo moieties to the liver cells," according to the patent application's abstract.
Title: Compositions and Methods for Diagnosing and Treating Melanoma
Filed: March 24, 2009
Lead Inventor: Eva Hernando, New York University
The patent application, its abstract states, claims "compositions and methods for the diagnosis, prognosis, prevention, and treatment of melanoma or melanoma-associated symptoms. The compositions are microRNA molecules associated with melanoma, as well as various nucleic acid molecules relating thereto or derived therefrom."
Title: Carbohydrate Conjugates as Delivery Agents for Oligonucleotides
Filed: Dec. 4, 2008
Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals
The invention provides RNAi agents comprising at least one subunit of a specific formula, according to the patent application's abstract.
Title: Novel Human microRNAs Associated with Cancer
Filed: Oct. 19, 2007
Lead Inventor: Thomas Litman, Exiqon
"The invention provides new sequences for human microRNAs associated with cancer [that] may be used as molecular markers for cancer diagnostics or as therapeutic targets or agents," according to the patent application's abstract.
Title: Multi-Targeting Interfering RNAs and Methods of Their Use and Design
Filed: May 21, 2008
Lead Inventor: Laurent Pierre Rivory, Johnson & Johnson Research
"Interfering RNA molecules are now designed and produced with specificity for multiple binding sequences present in distinct genetic contexts in one or more pre-selected target RNA molecules," according to the patent application's abstract. These molecules "are used to modulate expression of the target sequences.
"Such a multi-targeting interfering RNA approach provides a powerful tool for gene regulation," the abstract adds.
Title: Effective Treatment of Ovarian Cancer Using Triciribine and Related Compounds
Filed: Sept. 8, 2008
Inventor: Jin Cheng, University of South Florida College of Medicine
"The invention … encompasses a number of miRNAs, which are altered in human ovarian cancer, with the most significantly deregulated miRNAs being [miRs] -214, -199a*, -200a, -100, [and] -12Sb, and [the] let-7 cluster," according to the patent application's abstract. "Frequent deregulation of miR-214, -199a*, -200a and -100 in ovarian cancers and their alterations are associated with high grade and late stage tumor. Significantly, miR-214 induces cell survival and cisplatin resistance through targeting 3'UTR of the PTEN, which leads to down regulation of PTEN protein and activation of Akt pathway.
"Inhibition of Akt using Akt inhibitor, API-2/triciribine, or introduction of PTEN cDNA lacking 3'UTR, largely abrogates miR-214-induced cell survival," it adds. "These findings indicate that deregulation of miRNAs is a recurrent event in human ovarian cancer, and that miR-214 induces cell survival and cisplatin resistance primarily through targeting the PTEN/Akt pathway."
Title: RNA Interference-Mediated Inhibition of Protein Tyrosine Phosphatase-1B Gene Expression Using Short Interfering Nucleic Acid
Filed: May 14, 2007
Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)
"This invention relates to compounds, compositions, and methods useful for modulating protein tyrosine phosphatase-1B 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 PTP-1B gene expression and/or activity by RNA interference using small nucleic acid molecules.
"Such small nucleic acid molecules are useful, for example, for treating, preventing, inhibiting, or reducing obesity, insulin resistance, diabetes in a subject or organism, and for any other disease, trait, or condition that is related to or will respond to the levels of PTP-1B in a cell or tissue, alone or in combination with other treatments or therapies," the abstract adds.
Title: Anti-Myosin Va siRNA and Skin Depigmentation
Filed: Dec. 12, 2006 PCT Filed: Dec. 12, 2006
Lead Inventor: Anne-Marie Schmitt-Milas, Pierre Fabre Dermo-cosmetique
The invention, the patent application's abstract states, "relates to novel isolated siRNAs comprising a sense RNA strand and a complementary antisense RNA strand, which together form an RNA duplex. [This structure is] characterized in that the sense RNA strand comprises a sequence which has at most one nucleotide that is distinct in relation to a fragment of 14 to 30 contiguous nucleotides of the nucleotide sequence of exon F of the gene encoding the myosin Va protein.
The invention also relates to "compositions comprising at least one such siRNA, and to the use of at least one such siRNA as a cosmetic or therapeutic agent for skin depigmentation."
Title: RNAi Modulation of RSV and Therapeutic Uses Thereof
Filed: Jan. 28, 2008
Lead Inventor: Rachel Meyers, Alnylam Pharmaceuticals
The invention, the patent application's abstract states, "is based on the in vivo demonstration that RSV can be inhibited through intranasal administration of iRNA agents as well as by parenteral administration of such agents. Further, it is shown that effective viral reduction can be achieved with more than one virus being treated concurrently.
"Based on these findings, the … invention provides general and specific compositions and methods that are useful in reducing RSV mRNA levels, RSV protein levels, and viral titers in a subject, e.g., a mammal, such as a human," the abstract notes. "These findings can be applied to other respiratory viruses."