Patents Examined by Brian Whiteman
  • Treatment of primary ciliary dyskinesia with synthetic messenger RNA
    Patent number: 11510997
    Abstract: Polynucleotides encoding peptides, proteins, enzymes, and functional fragments thereof are disclosed. The polynucleotides of the disclosure can be effectively delivered to an organ, such as the lung, and expressed within cells of the organ. The polyribonucleotides of the disclosure can be used to treat a disease or condition associated with cilia maintenance and function, impaired function of the axoneme, such as DNAI1 or DNAH5.
    Type: Grant
    Filed: February 9, 2022
    Date of Patent: November 29, 2022
    Assignee: TranscripTx, Inc.
    Inventors: David J. Lockhart, Brandon Wustman, Mirko Hennig, Daniella Ishimaru
  • Treatment of ophthalmic conditions with angiopoietin-like 7 (ANGPTL7) inhibitors
    Patent number: 11512309
    Abstract: The present disclosure provides methods of treating patients having an ophthalmic condition, methods of identifying subjects having an increased risk of developing an ophthalmic condition, methods of detecting human angiopoietin like 7 (ANGPTL7) variant nucleic acid molecules and variant polypeptides, and ANGPTL7 variant nucleic acid molecules and variant polypeptides.
    Type: Grant
    Filed: May 12, 2021
    Date of Patent: November 29, 2022
    Assignee: Regeneron Pharmaceuticals, Inc.
    Inventors: Kavita Praveen, Claudia Schurmann, Lauren Gurski, Tanya Teslovich Dostal, Goncalo Abecasis, Aris Baras, Giovanni Coppola
  • Exosomal loading using hydrophobically modified oligonucleotides
    Patent number: 11505807
    Abstract: In one aspect, the invention relates to a method of loading exosomes with oligonucleotide cargo, by incubating an oligonucleotide comprising one or more hydrophobic modifications with a population of exosomes for a period of time sufficient to allow loading of the exosomes with the oligonucleotide. Exosomes loaded with hydrophobically modified oligonucleotide cargo, and uses thereof, are also provided.
    Type: Grant
    Filed: December 13, 2019
    Date of Patent: November 22, 2022
    Assignee: University of Massachusetts
    Inventors: Anastasia Khvorova, Neil Aronin, Marie Cecile Didiot, Reka Haraszti
  • Senile dementia treatment formulation and application thereof
    Patent number: 11497817
    Abstract: The invention relates to a diagnosis and therapeutic preparation for senile dementia and an application thereof, in particular to an application of ARHGAP11A gene, SPAG7 gene and C16ORF7 gene in preparing a diagnosis and therapeutic preparation for senile dementia. In order to solve the problem that molecular markers of senile dementia are scarce at present, the inventors carried out the high-throughput sequencing on peripheral blood samples of patients with senile dementia and healthy people, selected candidate genes, and confirmed that there was a good correlation between the candidate genes and senile dementia through molecular cell experiments, which laid a foundation for clinical gene diagnosis of senile dementia.
    Type: Grant
    Filed: December 5, 2018
    Date of Patent: November 15, 2022
    Assignee: MEDINTELL BIOMED
    Inventors: Chenggang Yang, Feng Xiao, Changjuan Xiang
  • Dynamic pharmacokinetic-modifying anchors
    Patent number: 11492619
    Abstract: Therapeutic oligonucleotides comprising pharmacokinetic (PK)-modifying anchors are provided. Methods for treating diseases or disorders comprising administering to a subject a therapeutic oligonucleotide comprising one or more PK-modifying anchors are provided.
    Type: Grant
    Filed: January 17, 2020
    Date of Patent: November 8, 2022
    Assignee: UNIVERSITY OF MASSACHUSETTS
    Inventors: Anastasia Khvorova, Bruno Miguel Da Cruz Godinho, Matthew Hassler
  • MiRNAs for treatment and in vitro diagnosis of drug resistant tumors
    Patent number: 11492671
    Abstract: miRNAs for in vitro diagnosis of resistance of tumors to BRAF/MEK pathway (also named as MAPK 5 pathway) inhibiting drugs and for treatment of tumors which are treated with said drugs, such as melanoma, by stimulating or inhibiting the expression of down-regulated or up-regulated miRNAs, respectively.
    Type: Grant
    Filed: April 10, 2019
    Date of Patent: November 8, 2022
    Assignees: ISTITUTI FISIOTERAPICI OSPITALIERI, ISTITUTO NAZIONALE TUMORI I.R.C.C.S. “FONDAZIONE G. PASCALE”, UNIVERSITA' DEGLI STUDI DI ROMA “LA SAPIENZA”
    Inventors: Gennaro Ciliberto, Paolo Antonio Ascierto, Luigi Fattore, Gerardo Botti, Rita Mancini
  • MicroRNA-based logic gates and uses thereof
    Patent number: 11492622
    Abstract: The present disclosure, at least in part, relates to a miRNA based logic gate that comprises an engineered RNA carrier that comprises an nuclear export signal, a target site for a first miRNA and a pre-miRNA sequence for a second miRNA. Also provided by the disclosure are recombinant viruses (e.g., recombinant adeno-associated viruses (rAAV)) for delivery of the miRNA based logic gates.
    Type: Grant
    Filed: September 25, 2020
    Date of Patent: November 8, 2022
    Assignee: Massachusetts Institute of Technology
    Inventors: Ron Weiss, Giulio Alighieri
  • Nucleic acid molecule for reduction of PAPD5 and PAPD7 mRNA for treating hepatitis B infection
    Patent number: 11484546
    Abstract: The present invention relates to nucleic acid molecules that are complementary to both PAP associated domain containing 5 (PAPD5) and PAP associated domain containing 7 (PAPD7), leading to inhibition of the expression of both PAPD5 and PAPD7 when using a single nucleic acid molecule. The invention also provides for PAPD5 and PAPD7 specific nucleic acid molecules for use in treating and/or preventing a HBV infection, in particular a chronic HBV infection. Also comprised in the present invention is a pharmaceutical composition for use in the treatment and/or prevention of a HBV infection.
    Type: Grant
    Filed: October 23, 2019
    Date of Patent: November 1, 2022
    Assignee: HOFFMAN-LA ROCHE INC.
    Inventors: Susanne Kammler, Anaïs Lopez, Henrik Mueller, Søren Ottosen, Lykke Pedersen
  • In vitro nephrotoxicity screening assay
    Patent number: 11479818
    Abstract: The invention relates to methods for predicting the in vivo nephrotoxicity of a nucleic acid molecule, in particular a nucleic acid molecule such as a siRNA or an antisense oligonucleotide using an in vitro cell based assay measuring the levels of EGFR as toxicity biomarker, potentially in combination with other biomarkers like ATP and KIM-1.
    Type: Grant
    Filed: June 16, 2017
    Date of Patent: October 25, 2022
    Assignee: Hoffmann-La Roche Inc.
    Inventors: Marcel Gubler, Annie Moisan, Adrian B. Roth
  • Thiomorpholino oligonucleotides for the treatment of muscular dystrophy
    Patent number: 11479772
    Abstract: Improved compositions and methods for treating a disease or disorder through target exon skipping, and preferably muscular dystrophy by administering antisense thiomorpholino molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping to produce a functional Dystrophin protein.
    Type: Grant
    Filed: March 20, 2020
    Date of Patent: October 25, 2022
    Assignee: THE REGENTS OF THE UNIVERSITY OF COLORADO
    Inventors: Marvin Caruthers, Sibasish Paul, Rakesh N. Veedu, Katarzyna Jastrzebska, Heera Krishna
  • Methods of treating spinal muscular atrophy
    Patent number: 11474113
    Abstract: Featured are biomarkers for, e.g., diagnosis and prognosis of spinal muscular atrophy (SMA) as well as identification of responders to treatment of SMA. Also provided are methods of treating subjects with SMA.
    Type: Grant
    Filed: January 25, 2019
    Date of Patent: October 18, 2022
    Assignee: Biosen MA Inc.
    Inventors: Wildon Farwell, John Staropoli, Guolin Zhao, Alexander McCampbell, Christopher Cody Stebbins
  • Aptamer for ADAMTS5 and use for aptamer for ADAMTS5
    Patent number: 11473089
    Abstract: The present invention provides an aptamer containing a sequence shown by the following formula (1) or formula (2): (1) GGGGCCUCC-N1-GGACYAAACC (2) GGGGCCUCC-N1-GGACWYAAACC wherein N1 shows 3 to 24 bases in length, Y is C or U, and W is A or U (uracil is optionally thymine), wherein the aptamer binds to a disintegrin and metalloproteinase with thrombospondin motifs-5 (ADAMTS5).
    Type: Grant
    Filed: November 9, 2018
    Date of Patent: October 18, 2022
    Assignee: RIBOMIC INC.
    Inventor: Kazuteru Aoki
  • FMRP and cancer treatment
    Patent number: 11473087
    Abstract: The present invention provides compositions and methods for down-modulating the expression and/or the immuno-suppressive activity of i) the FMRP protein, ii) an mRNA encoding the FMRP protein, and/or iii) the FMR1 gene for the treatment and/or prevention of primary cancer and/or cancer metastasis in a subject in need thereof.
    Type: Grant
    Filed: May 6, 2020
    Date of Patent: October 18, 2022
    Assignee: ECOLE POLYTECHNIQUE FEDERALE DE LAUSANNE (EPFL)
    Inventors: Qiqun Zeng, Douglas Hanahan
  • Means and methods to treat inflammatory diseases
    Patent number: 11466270
    Abstract: The present application relates to the field of inflammatory diseases, particularly to inflammatory diseases characterized by an M1 macrophage response, even more particularly to sepsis and to Trypanosoma infection. The invention provides substances modulating miR210 expression and/or activity, in particular RNA molecules inhibiting miR210 expression and/or activity and medical uses of these miR210 inhibitors. Methods are disclosed to screen for medicaments for treating sepsis.
    Type: Grant
    Filed: October 3, 2017
    Date of Patent: October 11, 2022
    Assignees: VIB VZW, KATHOLIEKE UNIVERSITEIT LEUVEN, K.U. LEUVEN R & D
    Inventors: Massimiliano Mazzone, Anne-Theres Henze
  • IncRNAS for therapy and diagnosis of angiogenesis
    Patent number: 11459561
    Abstract: The present invention relates to a pharmaceutical composition comprising (i) a compound promoting the expression and/or the activity of one or more long non-coding RNAs (lncRNAs) selected from SEQ ID NOs 1 to 22, preferably selected from SEQ ID NOs 1 to 3; and/or (ii) a compound inhibiting the expression and/or the activity of one or more lncRNAs selected from SEQ ID NOs 23 to 42. The present invention also relates to a pharmaceutical composition comprising (i) a compound promoting the expression and/or the activity of one or more lncRNAs selected from SEQ ID NOs 23 to 42; and/or (ii) a compound inhibiting the expression and/or the activity of one or more long non-coding RNAs (lncRNAs) selected from SEQ ID NOs 1 to 22, preferably selected from SEQ ID NOs 1 to 3.
    Type: Grant
    Filed: January 15, 2019
    Date of Patent: October 4, 2022
    Assignee: MEDIZINISCHE HOCHSCHULE HANNOVER
    Inventors: Thomas Thum, Jan Fiedler
  • Treatment for NEAT1 associated disease
    Patent number: 11459563
    Abstract: An isolated or purified antisense oligomer for modifying RNA cleavage and processing in the NEAT1 gene transcript or part thereof.
    Type: Grant
    Filed: February 5, 2018
    Date of Patent: October 4, 2022
    Assignees: The University of Western Australia, MURDOCH UNIVERSITY
    Inventors: Stephen Donald Wilton, Sue Fletcher, Archa Fox, Ruohan Li
  • miRNA as biomarker for Parkinson's disease and diagnostic kit using same
    Patent number: 11447772
    Abstract: The present invention relates to a method for providing information on the diagnosis of Parkinson's disease. The present invention also relates to a composition for preventing, ameliorating or treating Parkinson's disease. The present invention uses at least one miRNA whose expression is specifically down- or up-regulated in a Parkinson's disease model. Therefore, the use of the miRNA is effective in diagnosing and treating Parkinson's disease.
    Type: Grant
    Filed: July 17, 2018
    Date of Patent: September 20, 2022
    Assignee: KOOKMIN UNIVERSITY INDUSTRY ACADEMY COOPERATION FOUNDATION
    Inventor: Hyunjeong Liew
  • Methods and compositions for the specific inhibition of KRAS by asymmetric double-stranded RNA
    Patent number: 11447777
    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.
    Type: Grant
    Filed: June 22, 2020
    Date of Patent: September 20, 2022
    Assignee: Dicerna Pharmaceuticals, Inc.
    Inventor: Bob D. Brown
  • AAV-based treatment of cholesterol-related disorders
    Patent number: 11421230
    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
  • Methods relating to circulating tumor cell clusters and the treatment of cancer
    Patent number: 11421233
    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