Title: Lipid-Encapsulated Interfering RNA
Patent Number: 7,799,565
Filed: June 7, 2005
Lead Inventor: Ian MacLachlan, Protiva Biotherapeutics (Tekmira Pharmaceuticals)
The invention, the patent's abstract states, comprises "lipid-based formulations for delivering … nucleic acid-lipid particles comprising an interference RNA molecule to a cell, and assays for optimizing the delivery efficiency of such lipid-based formulations."
Title: Method for Delivery Across the Blood-Brain Barrier
Application Number: 20100233084
Filed: May 22, 2007 PCT Filed: May 22, 2007
Lead Inventor: Manjunath Narasimhaswamy, Immune Disease Institute (Harvard University)
The invention provides "compositions and methods useful for delivering agents to target cells or tissues, for example nerve cells and other cells in the central nervous system," the patent application's abstract states. "The compositions and methods are useful for delivering agents across the blood-brain barrier. The … invention also provides methods of using the compositions provided by the … invention to deliver agents, for example therapeutic agents for the treatment of neurologically related disorders."
The application specifically claims the delivery of RNAi agents such as siRNAs and shRNAs.
Title: Compositions and Methods for the Delivery of Biologically Active RNAs
Application Number: 20100233141
Filed: March 15, 2010
Lead Inventor: Kevin Polach, EGEN
The invention, the patent application's abstract states, comprises "novel compounds, compositions, and methods for the delivery of biologically active RNA molecules to cells. Specifically, the invention provides novel nucleic acid molecules, polypeptides, and RNA-protein complexes useful for the delivery of biologically active RNAs to cells and polynucleotides encoding the same.
"The invention also provides vectors for expressing said polynucleotides," the abstract notes. "In addition, the invention provides cells and compositions comprising the novel compounds and vectors, which can be used as transfection reagents. The invention further provides methods for producing said compounds, vectors, cells, and compositions. Additionally, vectors and methods for delivering biologically active RNA molecules to cells and/or tissues are provided.
"The novel compounds, vectors, cells, and compositions are useful, for example, in delivering biologically active RNA molecules to cells to modulate target gene expression in the diagnosis, prevention, amelioration, and/or treatment of diseases, disorders, or conditions in a subject or organism," it adds.
Titles: In Vivo Production of Small Interfering RNAs That Mediate Gene Silencing
Application Numbers: 20100233810, 20100234448
Filed: March 23 & 19, 2010
Lead Inventor: Phillip Zamore, University of Massachusetts
"The invention provides engineered RNA precursors that when expressed in a cell are processed by the cell to produce targeted small interfering RNAs that selectively silence targeted genes … using the cell's own RNA interference pathway," the patent applications' abstracts state. "By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vivo with appropriate regulatory sequences, expression of the engineered RNA precursors can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells."
Title: Diagnosis and Treatment of Cancers with microRNA Located In or Near Cancer-Associated Chromosomal Features
Application Number: 20100234241
Filed: April 26, 2010
Lead Inventor: Carlo Croce, Thomas Jefferson University
According to the patent application's abstract, "microRNA genes are highly associated with chromosomal features involved in the etiology of different cancers. The perturbations in the genomic structure or chromosomal architecture of a cell caused by these cancer-associated chromosomal features can affect the expression of the miR genes located in close proximity to that chromosomal feature.
"Evaluation of miR gene expression can therefore be used to indicate the presence of a cancer-causing chromosomal lesion in a subject," it adds. "As the change in miR gene expression level caused by a cancer-associated chromosomal feature may also contribute to cancerigenesis, a given cancer can be treated by restoring the level of miR gene expression to normal microRNA expression profiling [or the change in expression] can be used to diagnose cancer and predict whether a particular cancer is associated with an adverse prognosis. The identification of specific mutations associated with genomic regions that harbor miR genes in CLL patients provides a means for diagnosing CLL and possibly other cancers."
Title: Patterns of Known and Novel Small RNAs in Human Cervical Cancer
Application Number: 20100234445
Filed: June 30, 2009
Lead Inventor: Weng Onn Lui, Stanford University
The patent application, its abstract states, claims "small RNA sequences that are differentially expressed in" squamous cell carcinoma of the cervix.
"The sequences find use in diagnosis of cancer, and classification of cancer cells according to expression profiles," it states. "The methods are useful for detecting cervical cancer cells, facilitating diagnosis of cervical cancer and the severity of the cancer … in a subject, facilitating a determination of the prognosis of a subject, and assessing the responsiveness of the subject to therapy."
Title: RNAi Modulation of Bcr-Abl Fusion Genes and Uses Thereof
Application Number: 20100234446
Filed: June 27, 2009
Lead Inventor: Philipp Hadwiger, Alnylam Pharmaceuticals
"The invention relates to compositions and methods for modulating the expression of Bcr-Abl, and more particularly to the down-regulation of Bcr-Abl mRNA and Bcr-Abl protein levels by oligonucleotides via RNA interference," the patent application's abstract states.
Title: Modulation of Glucagon Receptor Expression
Application Number: 20100234447
Filed: March 16, 2010
Inventor: Susan Freier, Isis Pharmaceuticals
The patent application, its abstract states, claims "compounds, compositions and methods … for modulating the expression of glucagon receptor. The compositions comprise oligonucleotides targeted to nucleic acid encoding glucagon receptor. Methods of using these compounds for modulation of glucagon receptor expression and for diagnosis and treatment of disease associated with expression of glucagon receptor are provided."
Title: siRNA Targeting Gremlin
Application Number: 20100234582
Filed: April 7, 2010
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 CKSF1B1."
Title: siRNA Targeting Hypoxia-Inducible Factor 1
Application Number: 20100234583
Filed: April 12, 2010
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."
Title: Zinc Finger Nuclease- and RNA Interference-Mediated Inactivation of Viral Genomes
Application Number: 20100235936
Filed: March 27, 2008 PCT Filed: March 27, 2008
Lead Inventor: Anton McCaffrey, University of Iowa
"Embodiments of the … invention provide methods for targeted inactivation of viral genomes," the patent application's abstract states. "In one embodiment, zinc-finger proteins in which DNA binding sites are altered such that they recognize and bind different, desired DNA sequences contained in hepatitis B virus and that include nuclease domains are used for inactivation. Other embodiments for targeted inactivation of viral genomes use small nucleic acid molecules, such as short microRNA molecules or short hairpin RNA molecules capable of mediating RNA interference against the hepatitis B virus."