Abstract: This invention relates to compounds, compositions, and methods useful for reducing KRAS target RNA and protein levels via use of Dicer substrate siRNA (DsiRNA) agents possessing asymmetric end structures.
Abstract: The invention in some aspects relates to methods and compositions for assessing the effectiveness of miRNA inhibitors. In other aspects of the invention, methods and compositions for treating cholesterol related disorders are provided. In one aspect of the invention, miRNA inhibitors against miR-122 and rAAV-based compositions comprising the same are provided.
Type:
Grant
Filed:
June 23, 2020
Date of Patent:
August 23, 2022
Assignee:
University of Massachusetts
Inventors:
Guangping Gao, Phillip D. Zamore, Jun Xie
Abstract: Described herein are methods and assays relating to the presence and/or level of circulating tumor cells (CTCs). These CTC-Cs represent a highly metastatic subpopulation of CTCs. In some embodiments, the methods and assays described herein relate to the treatment of cancer.
Type:
Grant
Filed:
June 16, 2020
Date of Patent:
August 23, 2022
Assignee:
THE GENERAL HOSPITAL CORPORATION
Inventors:
Nicola Aceto, Daniel Arie Haber, Shyamala Maheswaran
Abstract: Compositions and methods for treating a disease are described herein. Compositions having plant preparations, microRNAs, and one or more rate limiters are administered to a patient to promote DNA damage repair and modulate endothelial and mitochondrial function, thereby allowing for healing to occur.
Abstract: Provided are a siRNA for inhibiting the expression of an angiopoietin-like protein 3 (ANGPTL3) gene, and a pharmaceutical composition and a conjugate comprising the siRNA; wherein each nucleotide in the siRNA is independently a modified or unmodified nucleotide, and the siRNA comprises a sense strand and an antisense strand; the sense strand comprises a nucleotide sequence A, the nucleotide sequence A having the same length as the nucleotide sequence as represented by SEQ ID NO:1 with no more than 3 nucleotide differences; the antisense strand comprises a nucleotide sequence B, the nucleotide sequence B having the same length as the nucleotide sequence as represented by SEQ ID NO:2 with no more than 3 nucleotide differences.
Type:
Grant
Filed:
November 29, 2018
Date of Patent:
August 16, 2022
Assignee:
SUZHOU RIBO LIFE SCIENCE CO., LTD.
Inventors:
Hongyan Zhang, Shan Gao, Daiwu Kang, Lina Kong
Abstract: The present invention relates to a highly efficient aptamer complex comprising a branched DNA and an aptamer, and a pharmaceutical use thereof. More specifically, the aptamer complex of the present invention relates to a highly efficient aptamer complex including a Y-shaped DNA as the branched DNA and using vascular endothelial growth factor (VEGF) as a target molecule. The aptamer complex of the present invention and a pharmaceutical composition comprising the same as an active ingredient are expected to be widely used in the medical field since the binding efficiency with the target molecule is more remarkable than that of the conventional aptamer.
Abstract: One aspect of the present invention relates to double-stranded RNAi (dsRNA) duplex agent capable of inhibiting the expression of a target gene. The dsRNA duplex comprises one or more motifs of three identical modifications on three consecutive nucleotides in one or both strand, particularly at or near the cleavage site of the strand. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.
Type:
Grant
Filed:
April 16, 2020
Date of Patent:
August 9, 2022
Assignee:
ALNYLAM PHARMACEUTICALS, INC.
Inventors:
Kallanthottathil G. Rajeev, Tracy Zimmermann, Muthiah Manoharan, Martin Maier, Satyanarayana Kuchimanchi, Klaus Charisse
Abstract: The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the apolipoprotein C3 (APOC3) gene, and methods of using such RNAi agents to inhibit expression of APOC3 and methods of treating subjects having an APOC3 associated disorder, e.g., hypertriglyceridemia.
Type:
Grant
Filed:
June 15, 2021
Date of Patent:
August 9, 2022
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Kevin Fitzgerald, William Querbes, James Butler, Stephanie Williams, Abigail Liebow, Gregory Hinkle, Martin A. Maier, Stuart Milstein, Satyanarayana Kuchimanchi, Muthiah Manoharan
Abstract: The present invention provides, among other things, methods of formulating nucleic acid-containing nanoparticles with an enzyme to afford efficient delivery of payload to a cell or tissue of interest via subcutaneous administration. In some embodiments, the present invention provides a process in which mRNA-loaded lipid nanoparticles are co-mixed with various amounts of hyaluronidase and administered via subcutaneous administration. The resulting payload can be efficiently delivered to the liver and other organs or tissues of a treated subject.
Type:
Grant
Filed:
November 10, 2017
Date of Patent:
August 2, 2022
Assignee:
Translate Bio, Inc.
Inventors:
Shrirang Karve, Frank DeRosa, Zarna Bhavsar, Michael Heartlein
Abstract: The invention provides eukaryotic cell-based screening methods to identify an aptamer that specifically binds a ligand, or a ligand that specifically binds an aptamer, using a polynucleotide cassette for the regulation of the expression of a reporter gene where the polynucleotide cassette contains a riboswitch in the context of a 5? intron-alternative exon-3? intron. The riboswitch comprises an effector region and an aptamer such that when the aptamer binds a ligand, reporter gene expression occurs.
Type:
Grant
Filed:
August 3, 2017
Date of Patent:
June 28, 2022
Assignee:
MEIRAGTX UK II LIMITED
Inventors:
Xuecui Guo, Alexandria Forbes, Lei Feng
Abstract: Compositions for expressing a polypeptide within a target organ, the composition comprising a delivery particle, and at least a first mRNA sequence complexed with, encapsulated by, or otherwise associated with the delivery particle, wherein the mRNA sequence comprises at least one coding sequence which codes for at least one polypeptide, at least a first untranslated region (UTR) sequence, and at least one micro-RNA (miRNA) binding site sequence, wherein the miRNA binding site sequence is located within, immediately 5? to, or immediately 3? to, the first UTR sequence; and wherein the miRNA binding site sequence provides for differential expression of the coding sequence between first and second cell types comprised within the target organ.
Abstract: Provided herein are genetic circuits and cell state classifiers for detecting the microRNA profile of a cell. The cell state classifiers of the present disclosure are designed to incorporate multiple genetic circuits integrated together by transcriptional or translational control. Multiple inputs can be sensed simultaneously by coupling their detection to different portions of the genetic circuit such that the output molecule is produced only when the correct input profile of miRNAs is detected. The genetic circuits and cell state classifiers may be used in various applications (e.g., therapeutic or diagnostic applications).
Abstract: The present invention relates to a method for determining a decrease in the functions of the hippocampus by using the correlation between a micro RNA (miRNA) and an N-methyl-D-aspartate receptor (NMDAR), a method for inhibiting the decrease in the functions, and a method for screening for inhibitors of the decrease in the functions.
Type:
Grant
Filed:
June 1, 2020
Date of Patent:
June 14, 2022
Assignees:
DAEGU GYEONGBUK INSTITUTE OF SCIENCE AND TECHNOLOGY, INSTITUTE FOR BASIC SCIENCE
Inventors:
Keetae Kim, Hong Gil Nam, Chand Parvez Danka Mohammed
Abstract: The present disclosure concerns aptamers which specifically bind to Legionella pneumophila which can be used to detect the presence of Legionella pneumophila in a sample.
Type:
Grant
Filed:
April 16, 2020
Date of Patent:
May 31, 2022
Assignee:
THE ROYAL INSTITUTION FOR THE ADVANCEMENT OF LEARNING/MCGILL UNIVERSITY
Abstract: Provided herein are genetic circuits and cell state classifiers for detecting the microRNA profile of a cell. In some embodiments, the cell state classifiers described herein utilize an endoribonuclease and a self-amplifying RNA molecule for controlling the expression of an output molecule. In some embodiments, the cell state classifiers described herein are encoded on a single RNA transcript, which is then processed to produce individual genetic circuits that function independently. The genetic circuits and cell state classifiers described herein may be used in various applications (e.g., therapeutic or diagnostic applications).
Abstract: Methods and compositions are provided to identify oligonucleotides that bind targets of interest. The targets include tissues, cells, circulating biomarkers such as microvesicles, including those derived from various diseases. The oligonucleotides can be used in diagnostic and therapeutic applications.
Type:
Grant
Filed:
June 10, 2020
Date of Patent:
May 17, 2022
Assignee:
Caris Science, Inc.
Inventors:
Valeriy Domenyuk, Tassilo Hornung, Heather O'Neill, Mark Miglarese, David Spetzler
Abstract: The invention provides compositions and methods for the diagnosis, treatment, assessment, and characterization of hyperlipidemia-related diseases and disorders, including atherosclerosis, non-alcoholic fatty liver disease, obesity and diabetes mellitus in a subject in need thereof, based on the expression level of at least one miRNA that is associated with these diseases and disorders.
Type:
Grant
Filed:
May 21, 2019
Date of Patent:
May 17, 2022
Assignee:
Temple University-Of The Commonwealth System of Higher Education
Abstract: Bispecific aptamers having a first end that specifically binds to a first tumor specific marker, tumor antigen, or viral protein and a second end that specifically binds to a second tumor specific marker, tumor antigen, or viral protein are provide. The bispecific aptamers can be used to treat cancer or virally infected cells. Generally, the bispecific aptamers bind to two surface proteins, preferably different proteins, on the same cell. In a preferred embodiment the bispecific aptamers bind to two different tumor markers, tumor antigens, tumor specific proteins and combinations thereof.
Type:
Grant
Filed:
February 20, 2018
Date of Patent:
May 17, 2022
Assignee:
AUGUSTA UNIVERSITY RESEARCH INSTITUTE, INC.
Abstract: The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of PHD2 gene expression and/or activity, and/or modulate a beta-catenin gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against PHD2 gene expression.
Type:
Grant
Filed:
January 23, 2020
Date of Patent:
May 17, 2022
Assignee:
Sima Therapeutics, Inc.
Inventors:
Brandon Ason, Duncan Brown, Walter R. Strapps