Patents Examined by Terra C. Gibbs
  • Patent number: 12259380
    Abstract: The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RNA downstream of SNORD1091B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.
    Type: Grant
    Filed: February 22, 2023
    Date of Patent: March 25, 2025
    Assignee: Hoffmann-La Roche Inc.
    Inventors: Veronica Costa, Maj Hedtjärn, Marius Hoener, Ravi Jagasia, Mads Aaboe Jensen, Christoph Patsch, Lykke Pedersen, Søren Vestergaard Rasmussen
  • Patent number: 12253515
    Abstract: Provided are an expression inhibitor of a cancer promoting factor based on the discovery of a new factor influencing the expression amount/level of a cancer promoting factor, and a development tool therefor. Provided are also a diagnostic agent and a diagnosis method for cancer. More specifically provided are: an expression inhibitor of a cancer promoting factor containing at least one kind of inhibitor selected from the group consisting of RBMS expression inhibitor and RBMS function inhibitor; a screening method using as an indicator the expression or the function of RBMS; an expression cassette useful for the method; as well as a diagnostic agent containing a product detection agent for RBMS gene expression and cancer detection method using as an indicator RBMS gene expression amount/level.
    Type: Grant
    Filed: November 9, 2018
    Date of Patent: March 18, 2025
    Assignees: National University Corporation Tokyo Medical and Dental University, Nippon Zoki Pharmaceutical Co., Ltd.
    Inventors: Hiroshi Asahara, Tomoki Chiba, Kentaro Abe
  • Patent number: 12246031
    Abstract: Provided are compositions and methods for treating cancer using administration of certain volumes of CpG oligonucleotides (CpG ODN) and, optionally, administration of a checkpoint inhibitor such as an anti-PD-1 antibody, an anti-PD-L1 antibody, and/or an anti-CTLA-4 antibody. In preferred embodiments, the CpG ODN are selected based on their propensity to induce high amounts of interferon alpha (IFN-a) and T-cell activation relative to interleukin-10 (IL-10) and B-cell activation. In certain embodiments, the methods further include pretreatment with radiotherapy, to potentiate the combination immunotherapy.
    Type: Grant
    Filed: February 12, 2019
    Date of Patent: March 11, 2025
    Assignee: CHECKMATE PHARMACEUTICALS, INC.
    Inventors: Arthur Krieg, Aaron Morris, David Mauro, George Weiner, Mohammed Milhem
  • Patent number: 12241064
    Abstract: The present invention relates, in general to agents that modulate the pharmacological activity of conjugated siRNAs.
    Type: Grant
    Filed: August 10, 2021
    Date of Patent: March 4, 2025
    Assignee: ALNYLAM PHARMACEUTICALS, INC.
    Inventors: Vasant Jadhav, John Maraganore, Martin Maier, Kallanthottathil G. Rajeev, Muthiah Manoharan, Akin Akinc, Ivan Zlatev
  • Patent number: 12201645
    Abstract: Methods of monitoring therapeutic efficacy in a subject with MDS are provided. Also provided is a method of identifying a subject with myelodysplastic syndrome (MDS) for treatment with a telomerase inhibitor, and methods of treating MDS. The subject methods can include administering to the subject an effective amount of a telomerase inhibitor and assessing the hTERT expression levels in a biological sample obtained from the subject. In some cases, a 50% or greater reduction in hTERT expression level identifies a subject who has an increased likelihood of benefiting from treatment with the telomerase inhibitor. The subject can be naive to treatment with a HMA, lenalidomide, or both. In some cases, the subject is classified as having low or intermediate-1 IPSS risk MDS and/or MDS relapsed/refractory to Erythropoiesis-Stimulating Agent (ESA). In some instances, the telomerase inhibitor is imetelstat sodium.
    Type: Grant
    Filed: November 26, 2019
    Date of Patent: January 21, 2025
    Assignee: Geron Corporation
    Inventors: Jacqueline Cirillo Bussolari, Fei Huang, Aleksandra Rizo
  • Patent number: 12173289
    Abstract: The present disclosure relates to double stranded RNA agents targeting the hepatitis B virus (HBV) genome, and methods of using such agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HBV infection or HBV-associated disorder, e.g., chronic hepatitis B infection.
    Type: Grant
    Filed: September 28, 2022
    Date of Patent: December 24, 2024
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Vasant R. Jadhav, Martin A. Maier, Stuart Milstein, Mark K. Schlegel
  • Patent number: 12173286
    Abstract: Provided herein are self-delivering oligonucleotides that are characterized by efficient RISC entry, minimum immune response and off-target effects, efficient cellular uptake without formulation, and efficient and specific tissue distribution.
    Type: Grant
    Filed: July 13, 2020
    Date of Patent: December 24, 2024
    Assignee: UNIVERSITY OF MASSACHUSETTS
    Inventors: Anastasia Khvorova, Neil Aronin, Julia Alterman, Matthew Hassler
  • Patent number: 12163128
    Abstract: The invention is directed generally to oligonucleotide compositions for the treatment of DNA repeat expansion diseases. The invention also relates to oligonucleotides directed to subunits of the DNA mismatch repair system.
    Type: Grant
    Filed: June 1, 2020
    Date of Patent: December 10, 2024
    Assignee: Board of Supervisors of Louisiana State University and Agricultural and Mechanical College
    Inventors: Edward L. Grabczyk, Kayla T. Fuselier
  • Patent number: 12157889
    Abstract: The present invention relates to compositions and methods for detecting motor neuron-specific miRNAs in a population of cells or subject. More particularly, the invention relates to detecting motor neuron-specific miRNAs to detect and treat motor neuron diseases associated with dysregulation of motor neuron-specific miRNAs, such as Amyotrophic Lateral Sclerosis (ALS).
    Type: Grant
    Filed: January 13, 2022
    Date of Patent: December 3, 2024
    Assignee: Washington University
    Inventors: Timothy M. Miller, Mariah Lawler Hoye, Ted Hyman, Erica Koval, Joseph Dougherty
  • Patent number: 12152241
    Abstract: Methods for treating cancer, e.g., cancer of epithelial origin, by specifically targeting human satellite II (HSATII) using sequence specific agents such as oligonucleotides.
    Type: Grant
    Filed: April 24, 2019
    Date of Patent: November 26, 2024
    Assignees: The General Hospital Corporation, Aalborg University
    Inventors: David T. Ting, Daniel A. Haber, Shyamala Maheswaran, Francesca Bersani, Anders M. Naar, Mihir Shivadatta Rajurkar, Sakari Kauppinen, Andreas Petri
  • Patent number: 12130285
    Abstract: Provided herein are hydrogel beads that mimic live, dead, and apoptotic cells. The present disclosure also provides kits and compositions of hydrogel beads. The present disclosure further comprises methods of using the kits, compositions, and hydrogel beads to determine if a target cell sample includes one or more live, dead, or apoptotic cells.
    Type: Grant
    Filed: March 18, 2024
    Date of Patent: October 29, 2024
    Assignee: Slingshot Biosciences, Inc.
    Inventors: Keunho Ahn, Zhenyu Deng, Martina De Geus, Anh Tuan Nguyen
  • Patent number: 12129522
    Abstract: The current invention pertains to miRNAs that are differentially expressed in samples of an individual having pancreatic cancer, or having a high risk of developing pancreatic cancer, as compared to the corresponding sample of an individual not having pancreatic cancer, or having low risk of developing pancreatic cancer, respectively. In certain embodiments, the miRNAs are differentially expressed in a tissue sample or blood plasma sample of an individual having a pancreatic lesion and having a high risk of developing pancreatic cancer as compared to the corresponding tissue sample or blood sample of an individual having the pancreatic lesion and having no risk or low risk of developing pancreatic cancer. These differentially expressed miRNAs can be used as biomarkers for diagnosis, treatment, and/or prevention of pancreatic cancer, particularly, in a subject having a pancreatic lesion.
    Type: Grant
    Filed: July 29, 2022
    Date of Patent: October 29, 2024
    Assignee: H. Lee Moffitt Cancer Center and Research Institute, Inc.
    Inventors: Mokenge P. Malafa, Jennifer Permuth, Dung-Tsa Chen
  • Patent number: 12123000
    Abstract: Ischemic conditions are a leading cause of death for both men and women. Ischemia, a condition characterized by reduced blood flow and oxygen to an organ. Re-establishment of blood flow, or reperfusion, and re-oxygenation of the affected area following an ischemic episode is critical to limit irreversible damage. However, reperfusion also associates potentially damaging consequences. For instance, increased vascular permeability is an important contributor to edema and tissue damage following ischemic events. Here the inventors shows that genetic inhibition of PI3K-C2? reduces cerebral infarction in two ischemia/reperfusion (I/R) models and improves neurological outcome. The genetic inhibition stabilizes the blood-brain barrier (BBB) after ischemic stroke and reduces inflammation.
    Type: Grant
    Filed: June 14, 2019
    Date of Patent: October 22, 2024
    Assignees: INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE), UNIVERSITE PAUL SABATIER TOULOUSE III, UNIVERSITY COLLEGE LONDON, CENTRE HOSPITALIER UNIVERSITAIRE DE TOULOUSE, UNIVERSITE DE CAEN NORMANDIE, CENTRE HOSPITALIER REGIONAL UNIVERSITAIRE DE CAEN
    Inventors: Marie-Pierre Gratacap, Jean Darcourt, Bart Vanhaesebroeck, Gaëtan Chicanne, Bernard Payrastre, Vincent Larrue, Romain Solinhac, Aude Jaffre, Denis Vivien, Typhaine Anquetil
  • Patent number: 12091659
    Abstract: Aspects of the disclosure relate to barcoded chimeric adeno-associated virus (AAV) capsid libraries, chimeric capsids and related recombinant AAVs (rAAVs) identified using the libraries. Specifically, the chimeric AAV capsid libraries comprise a plurality of nucleic adds encoding AAV capsid proteins, wherein each nucleic acid (i) encodes a unique AAV capsid protein having distinct polypeptide regions of greater than six amino acids in length that are derived from at least two different AAV serotypes, and (ii) comprises a unique barcode sequence. Further disclosed are methods of preparing an AAV library and identifying AAV capsids tropic for a target tissue.
    Type: Grant
    Filed: May 21, 2020
    Date of Patent: September 17, 2024
    Assignee: University of Massachusetts
    Inventors: Miguel Sena Esteves, Sourav Roy Choudhury
  • Patent number: 12083142
    Abstract: Provided are a siRNA for inhibiting the expression of hepatitis B virus gene, and a pharmaceutical composition and conjugate containing the siRNA. Each nucleotide in the siRNA is independently a modified nucleotide. The siRNA comprises a sense strand and an antisense strand. The sense strand of the siRNA comprises a nucleotide sequence 1 having the same length and no more than three nucleotides different from the nucleotide sequence shown in SEQ ID NO: 155, and the antisense strand of the siRNA comprises a nucleotide sequence 2 having the same length and no more than three nucleotides different from the nucleotide sequence shown in SEQ ID NO: 156.
    Type: Grant
    Filed: November 29, 2018
    Date of Patent: September 10, 2024
    Assignee: SUZHOU RIBO LIFE SCIENCE CO., LTD.
    Inventors: Hongyan Zhang, Shan Gao, Daiwu Kang
  • Patent number: 12084693
    Abstract: The invention provides nucleic acids encoding acid ?-glucosidase (GAA). In certain embodiments, nucleic acids have greater than about 86% sequence identity to a sequence selected from the group consisting of any of the sequences set forth as SEQ ID NOs:1-5. In certain embodiments, nucleic acids encoding acid ?-glucosidase (GAA) contain less than 127 CpG dinucleotides. Expression cassettes, vectors, cells and cell lines and methods of using such nucleic acids encoding acid ?-glucosidase (GAA) are also provided.
    Type: Grant
    Filed: May 15, 2019
    Date of Patent: September 10, 2024
    Assignee: SPARK THERAPEUTICS, INC.
    Inventors: Xavier Anguela, Sean Armour, Jayme Nordin
  • Patent number: 12065667
    Abstract: The present disclosure generally relates to systems, methods and compositions for use in Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cpf1 genome editing systems. Disclosed herein are modified Cpf1 mRNAs, modified guide RNAs, and combinations thereof, that confer increased levels of genome editing.
    Type: Grant
    Filed: April 14, 2017
    Date of Patent: August 20, 2024
    Assignee: OHIO STATE INNOVATION FOUNDATION
    Inventors: Yizhou Dong, Bin Li
  • Patent number: 12060556
    Abstract: Provided is a drug that allows highly-efficient skipping of exon. The present invention provides an antisense oligomer wherein two or more unit oligomers targeting sequences that are neither consecutive nor overlap with each other in the same exon are connected.
    Type: Grant
    Filed: November 2, 2021
    Date of Patent: August 13, 2024
    Assignees: NIPPON SHINYAKU CO., LTD., NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY
    Inventors: Naoki Watanabe, Yuuichirou Tone, Shin'ichi Takeda, Tetsuya Nagata
  • Patent number: 12049628
    Abstract: The present invention provides iRNA agents, e.g., double stranded iRNA agents, that target the transthyretin (TTR) gene and methods of using such iRNA agents for treating or preventing TTR-associated diseases.
    Type: Grant
    Filed: January 26, 2022
    Date of Patent: July 30, 2024
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Tracy Zimmermann, Amy Chan, Vasant R. Jadhav, Martin A Maier, Kallanthottathil G. Rajeev
  • Patent number: 12049630
    Abstract: The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Factor XII (F12) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of an F12 gene and to methods of preventing and treating an F12-associated disorder, e.g., heredity angioedema (HAE), prekallikrein deficiency, malignant essential hypertension, hypertension, end stage renal disease, Fletcher Factor Deficiency, thromboembolic disease, inflammatory disease, or Alzheimer's Disease.
    Type: Grant
    Filed: November 13, 2023
    Date of Patent: July 30, 2024
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Karyn Schmidt, Mark K. Schlegel, Adam Castoreno