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IP Update: Recent Patents, Patent Applications Awarded to Medtronic, Novartis, Alnylam, and More

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Title: Methods and Means for Obtaining Modified Phenotypes

Patent Number: 8,183,217

Filed: July 13, 2005

Lead Inventor: Peter Waterhouse, Commonwealth Scientific and Industrial Research Organization

The patent, its abstract states, claims “methods and means ... for reducing the phenotypic expression of a nucleic acid of interest in eukaryotic cells by providing aberrant, preferably unpolyadenylated, target-specific RNA to the nucleus of the host cell. Preferably, the unpolyadenylated, target-specific RNA is provided by transcription of a chimeric gene comprising a promoter and a DNA region encoding the target-specific RNA.”


Title: Therapeutic Compositions Comprising an RNAi Agent and a Neurotrophic Factor and Methods of Use Thereof

Patent Number: 8,183,219

Filed: Jan. 3, 2008

Inventor: Eric Burright, Medtronic

“The invention provides novel combination therapies for treating neurodegenerative disease, which comprise neurotrophic factors or suitable fragments thereof and agents capable of causing inhibition of a gene responsible for the neurodegenerative disease,” the patent's abstract states. “The invention provides novel nucleic acid sequences, methods, and systems suitable for applications of these combination therapies.”


Title: Suppression of SCN9A Gene Expression and/or Function for the Treatment of Pain

Patent Number: 8,183,221

Filed: Sept. 5, 2008

Lead Inventor: Deepak Thakker, Medtronic

The invention, the patent's abstract states, comprises “methods, sequences, and nucleic acid molecules used to treat pain. Specifically, the methods and sequences include locally administering molecules that suppress the expression of amino acid sequences that encode for [voltage gated sodium] channels or that otherwise inhibit the function of [voltage gated sodium] channels. Local suppression of [voltage gated sodium] channel levels and/or function will occur in the peripheral sensory neurons of the dorsal root ganglia.”


Title: Method and Medicament for Inhibiting the Expression of a Given Gene

Patent Number: 8,183,362

Filed: Oct. 31, 2007

Lead Inventor: Roland Kreutzer, Alnylam Pharmaceuticals

“The invention relates to an isolated RNA that mediates RNA interference of an mRNA to which it corresponds and a method of mediating RNA interference of mRNA of a gene in a cell or organism using the isolated RNA,” the patent's abstract states.


Title: Compositions and Methods for Immunostimulatory RNA Oligonucleotides

Application Number: 20120121551

Filed: Nov. 10, 2011

Lead Inventor: Gunther Hartmann, University Hospital Bonn

The invention, the patent application's abstract states, provides “4-nucleotide RNA motifs that confer immunostimulatory activity, in particular, IFN-alpha-inducing activity to an RNA oligonucleotide. The ... invention also provides RNA oligonucleotides, including siRNA, with high or low immunostimulatory activity … [as well as] the use of the RNA oligonucleotides of the invention for therapeutic purposes.”


Title: Small Interfering RNA Delivery

Application Number: 20120121689

Filed: Nov. 9, 2009

Lead Inventor: Andrew Bacon, Lipoxen Technologies

The invention, the patent application's abstract states, comprises siRNA-containing “liposomes ... formed of neutral liposome forming components and ... additionally sugar. The composition provides reduced expression of target gene, without causing systemic toxicity. The composition is produced by a dehydration-rehydration technique to provide high yields and good control of liposome size.”


Title: Compositions and Methods for the Diagnosis and Treatment of Inflammatory Disorders and Fibrotic Disease

Application Number: 20120121697

Filed: June 24, 2010

Lead Inventor: Stephen Rennard, University of Nebraska

The invention comprises “compositions and methods ... for the treatment and diagnosis of inflammatory diseases and disorders, including pulmonary diseases and fibrotic disorders,” including chronic obstructive pulmonary disease, the patent application's abstract states.

The application specifically claims the use of siRNAs and microRNAs for such applications.


Title: RNA Sequence-Specific Mediators of RNA Interference

Application Number: 20120122111

Filed: March 9, 2011

Lead Inventor: Thomas Tuschl, Max Planck

The invention relates to a “Drosophila in vitro system which was used to demonstrate that dsRNA is processed to RNA segments 21-23 nucleotides in length,” the patent application's abstract states. “Furthermore, when these ... fragments are purified and added back to Drosophila extracts, they mediate RNA interference in the absence of long dsRNA. Thus, these … fragments are the sequence-specific mediators of RNA degradation.

“A molecular signal, which may be their specific length, must be present in these 21-23 [nucleotide] fragments to recruit cellular factors involved in RNAi,” the abstract adds. “This ... invention encompasses these ... fragments and their use for specifically inactivating gene function. The use of these fragments enables the targeting of specific mRNAs for degradation in mammalian cells, where the use of long dsRNAs to elicit RNAi is usually not practical, presumably because of the deleterious effects of the interferon response. This specific targeting of a particular gene function is useful in functional genomic and therapeutic applications.”


Title: Methods and Compositions for Reducing Target Gene Expression Using Cocktails of siRNAs or Constructs Expressing siRNAs

Application Number: 20120122217

Filed: Jan. 27, 2012

Lead Inventor: David Brown, Applied Biosystems

The invention, the patent application's abstract states, “concerns methods and compositions involving the production or generation of siRNA mixtures or pools capable of triggering RNA-mediated interference in a cell. Compositions of the invention include kits that include reagents for producing or generating siRNA pools. The ... invention further concerns methods using polypeptides with RNase III activity for generating siRNA mixtures or pools that effect RNAi, including the generation of a number of RNA molecules to the same target gene.”


Title: Oligomeric Compounds and Compositions for Use in Modulation of Small Non-Coding RNAs

Application Number: 20120122216

Filed: Jan. 26, 2012

Lead Inventor: Christine Esau, Regulus Therapeutics

The invention comprises “compounds, compositions, and methods … for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds, targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs, as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs, are also provided.”


Title: Novel Lipids for Transfection of Nucleic Acids

Application Number: 20120122760

Filed: Oct. 11, 2011

Lead Inventor: Gulilat Gebeyehu, Molecular Transfer

“Cationic lipid compositions are provided that are useful for efficient delivery of macromolecules, such as nucleic acids, into a wide variety of eukaryotic cell types,” the patent application's abstract states. “Methods for using the compositions ... are provided.”


Title: Methods of Enhancing Utrophin Production Via Inhibition of microRNA

Application Number: 20120122953

Filed: April 27, 2009

Lead Inventor: Catherine Moorwood, University of Pennsylvania

“This invention provides a method for enhancing utrophin protein production in a cell by inhibiting an utrophin microRNA molecule,” the patent application's abstract states. “Moreover, the invention provides that methods for enhancing utrophin protein production in a muscle cell are used for treating a muscular dystrophy and/or other myopathies.”


Title: RNAi Inhibition of Serum Amyloid A for Treatment of Glaucoma

Application Number: 20120122961

Filed: Jan. 31, 2012

Lead Inventor: Abbot Clark, Novartis

“RNA interference is provided for inhibition of serum amyloid A mRNA expression in glaucomas involving SAA expression,” the patent application's abstract states.


Title: Modified Small Interfering RNA Molecules and Methods of Use

Application Number: 20120122962

Filed: March 30, 2011

Lead Inventor: Jan Han, Novartis

The invention relates to “double-stranded RNA molecules that mediate RNA interference in target cells, preferably hepatic cells” according to the patent application's abstract. “The invention also provides double-stranded RNA molecules that are modified to be resistant to nuclease degradation, which inactivates a virus, and more specifically, hepatitis C virus. The invention also provides a method of using these modified RNA molecules to inactivate virus in mammalian cells and a method of making modified small interfering RNAs using human Dicer.”


Title: Delivery Method

Application Number: 20120124683

Filed: June 29, 2011

Lead Inventor: Bruce Sullenger, Duke University

“The ... invention relates, in general, to siRNA and, in particular, to a method of effecting targeted delivery of siRNAs and to compounds suitable for use in such a method,” the patent application's abstract states.

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