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IP Update: Recent Patents, Patent Applications Awarded to Sylentis, Quark, NIH, and More


Title: Inducible Small RNA Expression Constructs for Targeted Gene Silencing

Patent Number: 8,198,077

Filed: Jan. 15, 2004

Lead Inventor: Thomas Tuschl, Max Plank Institute

“The invention relates to vectors for the inducible expression of RNA molecules in eukaryotic, particularly mammalian, cells and transgenic animals,” the patent's abstract states.

Title: Methods and Compositions for the Treatment of Eye Disorders with Increased Intraocular Pressure

Patent Number: 8,198,250

Filed: July 9, 2008

Lead Inventor: Ana Jimenez, Sylentis

The invention, the patent's abstract states, “relates to methods and compositions that decrease intraocular pressure of the eye. The compositions of the invention comprise short interfering nucleic acid molecules including, but not limited to, siRNA that decrease expression of genes associated with production or drainage of intraocular fluid. The compositions of the invention can be used in the preparation of a medicament for the treatment of eye conditions displaying increased [intraocular pressure] such as glaucoma, infection, inflammation, uveitis, and diabetic retinopathy. The methods of the invention comprise the administration to a patient in need thereof an effective amount of one or more siNAs of the invention.”

Title: siRNA Inhibition of pI3K, p85, p110, and Akt2 and Methods of Use

Patent Number: 8,198,252

Filed: May 18, 2007

Lead Inventor: Mark Evans, University of Texas

The invention comprises “polynucleotides, compositions including polynucleotides, and the uses thereof for treating cancer in a subject,” the patent's abstract states. “The polynucleotides silence the expression of coding regions that encode polypeptides such as p85-alpha, p110-alpha, and Akt2. The cancers treatable using the methods described herein include colorectal cancer, breast cancer, lung cancer, and metastases thereof.”

Title: siRNA-Mediated Inhibition of Doublecortin and Ca2+/Calmodulin-Dependent Kinase-Like-1

Patent Number: 8,198,255

Filed: May 15, 2009

Lead Inventor: Courtney Houchen, University of Oklahoma

“DCAMKL-1 has been identified as a biomarker for stem cells, as well as cancer stem cells,” the patent's abstract states. “Methods of detecting the presence of at least one stem cell, methods of isolating stem cells, and methods of inhibiting growth of cancer cells utilizing DCAMKL-1 are disclosed herein.”

Title: Method of Treating Influenza

Patent Number: 8,198,256

Filed: July 2, 2008

Lead Inventor: Misako Nakazawa, Kyorin Pharmaceutical

The invention provides “a double-stranded RNA which inhibits replication of influenza B viruses by RNA interference, in which the double-stranded RNA comprises an RNA having 19 to 25 nucleotides homologous with a part of an mRNA transcribed from a genomic RNA of the influenza B viruses and an antisense RNA thereof,” according to the patent's abstract.

Title: Oligoribonucleotides and Methods of Use Thereof for Treatment of Fibrotic Conditions and Other Diseases

Patent Number: 8,198,258

Filed: April 22, 2011

Lead Inventor: Orna Mor, Quark Pharmaceuticals

“The invention relates to a double-stranded compound, preferably an oligoribonucleotide, [that] down-regulates the expression of a human TGaseII gene at the post-transcriptional level,” the patent's abstract states. “The invention also relates to a pharmaceutical composition comprising the compound, or a vector capable of expressing the oligoribonucleotide compound, and a pharmaceutically acceptable carrier. The ... invention also contemplates a method of treating a patient suffering from a fibrotic disease such as pulmonary, kidney, and liver fibrosis or ocular scarring comprising administering to the patient the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the patient. The invention also relates to treatment of fibrotic and other diseases by use of antibodies to TGaseII polypeptide.”

Title: Treatment of Neurological Disorders by dsRNA Administration

Patent Number: 8,198,259

Filed: Nov. 4, 2011

Lead Inventor: Gabriele Dorn, Novartis

The invention, the patent's abstract states, “relates to methods to treat neurological disorders [using] intrathecal injection of an effective amount of a double-stranded RNA into a subject in need, wherein the dsRNA inhibits the expression of a target gene and to pharmaceutical compositions useful for such treatment.”

Title: siRNA Targeting Catenin, Beta-1

Patent Number: 8,198,427

Filed: Oct. 30, 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 nucleotide sequences for CTNNB1.”

Title: Identification of Novel Genes Coding for Small Temporal RNAs

Patent Number: 8,198,428

Filed: May 7, 2010

Lead Inventor: Thomas Tuschl, Max Planck Institute

“In Caenorhabditis elegans, lin-4 and let-7 encode 22- and 21-nucleotide RNAs, respectively, that function as key regulators of developmental timing,” the patent's abstract states. “Because the appearance of these short RNAs is regulated during development, they are also referred to as small temporal RNAs. We show that many more 21- and 22-[nucleotide] expressed RNAs, termed microRNAs, exist in invertebrates and vertebrates, and that some of these novel RNAs, similar to let-7 stRNA, are also highly conserved. This suggests that sequence-specific post-transcriptional regulatory mechanisms mediated by small RNAs are more general than previously appreciated.”

Title: Modified siNA

Application Number: 20120142011

Filed: June 11, 2010

Lead Inventor: Peter Hahn, Qiagen

The invention, the patent application's abstract states, “pertains to the use of at least one abasic modification within the first 8 nucleotide positions of the 5' region of the antisense strand of a small interfering nucleic acid molecule for reducing off-target effects. Provided are suitable modified siNAs, compositions and methods for producing respective siNAs, as well as kits comprising respective siNAs.”

Title: Oligonucleotide End Caps

Application Number: 20120142101

Filed: July 7, 2010

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

The invention comprises “modified nucleic acids ... including pharmaceutical compositions comprising the modified nucleic acids, and methods of using the modified nucleic acids,” the patent application's abstract states.

Title: Diagnosis and Treatment of Adrenocortical Tumors Using Human microRNA-483

Application Number: 20120142753

Filed: Dec. 6, 2010

Lead Inventor: Electron Kebebew, National Cancer Institute

The patent application, its abstract states, claims “methods of diagnosing and treating a malignant adrenocortical tumor, including adrenocortical carcinoma. In some examples, methods of diagnosing a malignant adrenocortical tumor include detecting expression of at least one microRNA gene product, such as miR-100, miR-125b, miR-195, miR-483-3p, miR-483-5p, and IGF2 mRNA in a sample obtained from the subject with an adrenocortical tumor and comparing expression of at least one of these miR gene products and IGF2 mRNA in the sample obtained from the subject to a control. Altered expression of at least one of the miR gene products and IGF2 mRNA, such as a decrease in miR-100, miR-125b or miR-195 or an increase in miR-483-3p, miR-483-5p, and an increase in IGF2 mRNA, in the sample obtained from the subject compared to the control indicates a malignant adrenocortical tumor.”

Title: RNAi Modulation of Aha and Therapeutic Uses Thereof

Application Number: 20120142757

Filed: Jan. 4, 2012

Lead Inventor: Rainer Constieu, Alnylam Pharmaceuticals

“The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of an Aha gene, comprising an antisense strand having a nucleotide sequence which is less than 30 nucleotides in length, generally 19 [to] 25 nucleotides in length, and which is substantially complementary to at least a part of an Aha gene,” according to the patent application's abstract. “The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by Aha1 expression and the expression of an Aha gene using the pharmaceutical composition; and methods for inhibiting the expression of an Aha gene in a cell.”

Title: Treatment of Down Syndrome Gene-Related Diseases by Inhibition of Natural Antisense Transcript to a Down Syndrome Gene

Application Number: 20120142758

Filed: June 24, 2010

Lead Inventor: Joseph Collard, CuRNA (Opko Health)

The invention relates to “antisense oligonucleotides that modulate the expression of and/or function of a Down syndrome gene, in particular, by targeting natural antisense polynucleotides of a Down syndrome gene,” according to the patent application's abstract. “The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of Down syndrome genes.”

Title: Nucleic Acid-Delivery Compositions and Methods of Use Thereof

Application Number: 20120142763

Filed: June 1, 2010

Lead Inventor: Steven Dowdy, University of California

The invention, the patent application's abstract states, “relates to nucleic acid constructs modified to have a reduced net anionic charge. The constructs comprise phosphotriester and/or phosphothioate protecting groups. The disclosure also provides methods of making and using such constructs.”

Title: Sphingosine-Bound siRNA

Application Number: 20120142765

Filed: June 22, 2010

Lead Inventor: Ana Jimenez, Sylentis

“The invention relates to novel oligomer analogues and their use in oligonucleotide-based therapies,” according to the patent application's abstract. “More specifically, the invention concerns oligonucleotides carrying lipid molecules and their use as potential inhibitors of gene expression.”

Title: Gene Silencing

Application Number: 20120144527

Filed: Dec. 18, 2007

Inventor: Milo Aukerman, DuPont

“The invention provides methods and compositions useful in target sequence suppression and target sequence validation,” the patent application's abstract states. “The invention provides polynucleotide constructs useful for gene silencing, as well as cells, plants and seeds comprising the polynucleotides. The invention also provides a method for using microRNA to silence a target sequence.”

The Scan

Sick Newborns Selected for WGS With Automated Pipeline

Researchers successfully prioritized infants with potential Mendelian conditions for whole-genome sequencing or rapid whole-genome sequencing, as they report in Genome Medicine.

Acne-Linked Loci Found Through GWAS Meta-Analysis

Researchers in the European Journal of Human Genetics find new and known acne vulgaris risk loci with a genome-wide association study and meta-analysis, highlighting hair follicle- and metabolic disease-related genes.

Retina Cell Loss Reversed by Prime Editing in Mouse Model of Retinitis Pigmentosa

A team from China turns to prime editing to correct a retinitis pigmentosa-causing mutation in the PDE6b gene in a mouse model of the progressive photoreceptor loss condition in the Journal of Experimental Medicine.

CRISPR Screens Reveal Heart Attack-Linked Gene

Researchers in PLOS Genetics have used CRISPR screens to home in on variants associated with coronary artery disease that affect vascular endothelial function.