Patents Examined by Brian Whiteman
  • Patent number: 12031132
    Abstract: Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the HBG1 and HBG2 loci, portions of the erythroid specific enhancer of the BCL11A gene, or a combination thereof, in cells and increasing expression of fetal hemoglobin.
    Type: Grant
    Filed: September 11, 2020
    Date of Patent: July 9, 2024
    Assignee: EDITAS MEDICINE, INC.
    Inventors: Jennifer Leah Gori, Edouard Aupepin De Lamothe-Dreuzy, Jack Heath, John Anthony Zuris, KaiHsin Chang
  • Patent number: 12024706
    Abstract: Novel oligonucleotides that enhance silencing of the expression of a gene containing a single nucleotide polymorphism (SNP) relative to the expression of the corresponding wild-type gene are provided. Methods of using novel oligonucleotides that enhance silencing of the expression of a gene containing a SNP relative to the expression of the corresponding wild-type gene are provided.
    Type: Grant
    Filed: August 7, 2020
    Date of Patent: July 2, 2024
    Assignee: UNIVERSITY OF MASSACHUSETTS
    Inventors: Anastasia Khvorova, Julia Alterman, Faith Conroy, Edith Pfister, Neil Aronin, Ken Yamada
  • Patent number: 12016875
    Abstract: The present invention relates to methods for treating and preventing ophthalmological disease and disorders, comprising administering Antagonist A or another pharmaceutically acceptable salt thereof, optionally in combination with another treatment, to a subject in need thereof. The present invention also relates to methods for treating and preventing ophthalmological disease and disorders, comprising administering an anti-C5 agent (e.g., ARC1905), optionally in combination with another treatment, to a subject in need thereof.
    Type: Grant
    Filed: April 7, 2023
    Date of Patent: June 25, 2024
    Assignee: IVERIC bio, Inc.
    Inventors: Samir Patel, Richard Everett, Douglas Brooks, Shane Xinxin Tian
  • Patent number: 12018260
    Abstract: The present invention relates, in general to agents that modulate the pharmacological activity of siRNAs. In addition, the invention relates generally to methods and systems for use in assessing the efficacy and safety of a pharmaceutical composition for use in the treatment or prophylaxis of a disease.
    Type: Grant
    Filed: January 25, 2022
    Date of Patent: June 25, 2024
    Assignee: ALNYLAM PHARMACEUTICALS, INC.
    Inventors: Ivan Zlatev, Adam Castoreno, Martin Maier, Vasant Jadhav, Jae Kim, Pushkal Garg
  • Patent number: 12016339
    Abstract: Disclosed are double stranded RNA (dsRNA) molecules that are toxic to insect pests. In particular, interfering RNA molecules capable of modulating expression of a pest insect target gene and that are toxic to the insect pest are provided. Further, methods of making and using the interfering RNA, for example as the active ingredient in an insecticidal composition or in a transgenic plant, to confer protection from insect damage are disclosed.
    Type: Grant
    Filed: November 6, 2018
    Date of Patent: June 25, 2024
    Assignee: SYNGENTA PARTICIPATIONS AG
    Inventors: Yann Naudet, Myriam Beghyn, Lien De Schrijver, Annelies Philips, Isabelle Maillet
  • Patent number: 12006500
    Abstract: Compositions and methods for development of potent siRNA therapeutics for prevention and treatment of Corona Virus (2019-nCoV; COVID-19) infections are provided. The compositions include a pharmaceutical composition comprising siRNA cocktails that target critical viral genes and pharmaceutically acceptable polymeric nanoparticle carriers and liposomal nanoparticle carriers. Administration methods for prevention and treatment are provided, including airway instillation, subcutaneous injections and nebulizer aerosolization.
    Type: Grant
    Filed: January 25, 2021
    Date of Patent: June 11, 2024
    Assignee: Sirnaomics, Inc.
    Inventors: Danny Tang, Xueping Chen, Patrick Y. Lu, Vera Simonenko, David Evans, John Xu, Deling Wang, Alan Lu
  • Patent number: 11999775
    Abstract: Provided are chimeric VEGF-binding proteins and nucleic acids (e.g., a vector) encoding chimeric VEGF-binding proteins, methods and host cells for producing these proteins and nucleic acids, and pharmaceutical compositions containing these proteins and nucleic acids. Also provided are methods of treating an angiogenic disease or disorder that include administering at least one of the chimeric VEGF-binding proteins or at least one of the nucleic acids (e.g., a vector) encoding a chimeric VEGF-binding protein.
    Type: Grant
    Filed: March 4, 2019
    Date of Patent: June 4, 2024
    Assignees: Children's Medical Center Corporation, The General Hospital Corporation, President and Fellows of Harvard College
    Inventors: Bob Carter, Jeng-Shin Lee, Szofia S. Bullain, Richard C. Mulligan
  • Patent number: 11981897
    Abstract: Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.
    Type: Grant
    Filed: February 10, 2021
    Date of Patent: May 14, 2024
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventor: Susan M. Freier
  • Patent number: 11970700
    Abstract: The present invention relates to an intracellular expression enhancing genetic element, which enhances the intracellular expression of target protein encoded in RNA therapeutics such as mRNA vaccines and RNA medicines. In more detail, the present invention relates to the RNA molecule which has the genetic element capable of enhancing the intracellular expression of target protein encoded in RNA therapeutics delivered into cells; the plasmid used to synthesize the RNA molecule having the genetic element “E3”; the method for synthesizing RNA molecule having the genetic element “E3” using the plasmid and utilizing the synthesized RNA molecule as RNA therapeutics.
    Type: Grant
    Filed: December 13, 2022
    Date of Patent: April 30, 2024
    Assignee: Intron Biotechnology, Inc.
    Inventors: Seong Jun Yoon, An Sung Kwon, Se Hwan Kim, Sang Hyeon Kang
  • Patent number: 11963982
    Abstract: Genome editing systems, guide RNAs, dead guide RNAs, and CRISPR-mediated methods are provided for altering portions of a target nucleic acid.
    Type: Grant
    Filed: November 8, 2019
    Date of Patent: April 23, 2024
    Assignee: EDITAS MEDICINE, INC.
    Inventors: Edouard Aupepin De Lamothe-Dreuzy, Jack Heath, Jennifer Leah Gori, Luis Barrera
  • Patent number: 11963975
    Abstract: The disclosure relates to methods and compositions for regulating expression of DUX4. In some aspects, methods described by the disclosure are useful for treating a disease associated with aberrant DUX4 expression (e.g., facioscapulohumeral muscular dystrophy).
    Type: Grant
    Filed: September 30, 2021
    Date of Patent: April 23, 2024
    Assignee: University of Massachusetts
    Inventors: Peter L. Jones, Michael R. Green
  • Patent number: 11965162
    Abstract: In one aspect, compositions are provided for the early diagnosis and treatment of pancreatic ductal adenocarcinoma and include microRNAs, e.g. miR-21 and inhibitors thereof. The treatment compositions can be useful for early detection, and for intercepting developing premalignant pancreatic lesions and other KRAS-driven premalignancies.
    Type: Grant
    Filed: April 16, 2021
    Date of Patent: April 23, 2024
    Assignee: The Johns Hopkins University
    Inventors: Elizabeth A. Jaffee, Nina Chu, Jacquelyn Winifred Zimmerman
  • Patent number: 11957788
    Abstract: Liposomal spherical nucleic acids that function as multivalent immune modulators are provided according to the invention. The liposomal spherical nucleic acids of the invention are useful prophylactic and therapeutic applications as well as research and diagnostic indications.
    Type: Grant
    Filed: August 18, 2021
    Date of Patent: April 16, 2024
    Assignee: Exicure Operating Company
    Inventors: Aleksandar Filip Radovic-Moreno, Richard Kang, Subbarao Nallagatla, Christopher C. Mader, Sergei Gryaznov
  • Patent number: 11958878
    Abstract: The present invention establishes a molecular therapy for glycogen storage disease type Ia. The present invention provides an oligonucleotide of 15-30 bases comprising a nucleotide sequence complementary to die cDNA of G6PC gene with c.648G>T mutation, wherein the oligonucleotide comprises a sequence complementary to a region comprising any site between the 82nd to the 92nd nucleotide from the 5? end of exon 5 of the G6PC gene with c.648C>T mutation, a pharmacologically acceptable salt or solvate thereof. Also provided is a pharmaceutical drug comprising the oligonucleotide, a pharmacologically acceptable salt or solvate thereof (e.g., therapeutic drug for glycogen storage disease type Ia).
    Type: Grant
    Filed: March 5, 2019
    Date of Patent: April 16, 2024
    Assignees: Daiichi Sankyo Company, Limited, Kobe Gakuin Educational Foundation
    Inventors: Makoto Koizumi, Yoshiyuki Onishi, Takeshi Masuda, Mitsuhiro Iwamoto, Yukiko Sekiguchi, Kentaro Ito, Shinnosuke Tsuji, Masafumi Matsuo
  • Patent number: 11952573
    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: April 1, 2022
    Date of Patent: April 9, 2024
    Assignee: Sirna Therapeutics, Inc.
    Inventors: Brandon Ason, Duncan Brown, Walter R. Strapps
  • Patent number: 11939633
    Abstract: A COTL1 gene or protein maintains and regulates the homeostasis of hematopoietic stem cells. A method of diagnosis and treatment of blood-related disease caused either by abnormalities in the homeostasis of hematopoietic stem cells, which result from a mutation in the COTL1 gene or a decrease in the expression of the COTL1 protein, or by an imbalance between the differentiation or proliferation and damage or death of hematopoietic stem cells, or by abnormalities in mitochondrial homeostasis are disclosed. The COTL1 gene or protein plays an important role in regulating mitochondrial morphology, and when it is knocked down, the number of hematopoietic stem cells decreases.
    Type: Grant
    Filed: May 10, 2018
    Date of Patent: March 26, 2024
    Assignee: AJOU UNIVERSITY INDUSTRY-ACADEMIC COOPERATION FOUNDATION
    Inventors: Seon-Yong Jeong, Eunkuk Park, Gijeong Kim
  • Patent number: 11939581
    Abstract: The present disclosure relates to small interfering RNA (siRNA) molecules directed to mRNA transcripts of CD274 to cause downregulation of programmed death-ligand 1 (PD-L1) expression in humans. The siRNA can be constructed of unmodified nucleotides or modified nucleotides that exhibit modified sugars, nucleobases, linkages, or covalently bound targeting moieties. Also disclosed herein are pharmaceutical compositions of siRNAs and uses of or methods of using the siRNAs for the treatment of PD-L1 related diseases including but not limited to liver diseases, cancer, hepatocellular carcinoma, viral diseases, or hepatitis B.
    Type: Grant
    Filed: February 26, 2021
    Date of Patent: March 26, 2024
    Assignee: Aligos Therapeutics, Inc.
    Inventors: Leonid Beigelman, Megan Elizabeth Fitzgerald, Saul Martinez Montero, Aneerban Bhattacharya
  • Patent number: 11932852
    Abstract: The present disclosure relates to RNA interference (RNAi) reagents, such as short hairpin microRNA (shmiR) and short hairpin RNA (shRNA), for treatment of oculopharyngeal muscular dystrophy (OPMD), compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto. The present disclosure also relates to the use of the RNAi reagents in combination with PABPN1 replacment reagents, such as constructs which encode functional PABPN1 protein, for treatment of OPMD, compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto.
    Type: Grant
    Filed: September 1, 2021
    Date of Patent: March 19, 2024
    Assignee: Benitec IP Holdings Inc.
    Inventors: Vanessa Strings-Ufombah, David Suhy
  • Patent number: 11926830
    Abstract: The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.
    Type: Grant
    Filed: September 2, 2021
    Date of Patent: March 12, 2024
    Assignee: Ionis Pharmaceuticals, Inc.
    Inventors: Tamar R. Grossman, Michael L McCaleb, Andrew T. Watt, Susan M. Freier
  • Patent number: 11926827
    Abstract: Provided herein are MAPT RNAi agents and compositions comprising a MAPT RNAi agent. Also provided herein are methods of using the MAPT RNAi agents or compositions comprising a MAPT RNAi agent for reducing MAPT expression and/or treating tauopathy in a subject.
    Type: Grant
    Filed: May 3, 2023
    Date of Patent: March 12, 2024
    Assignee: ELI LILLY AND COMPANY
    Inventors: Barbara Calamini, Sarah Katharina Fritschi, Rebecca Ruth Miles, Andrew Peter McCarthy, Douglas Raymond Perkins, Keith Geoffrey Phillips, Kaushambi Roy, Isabel Cristina Gonzalez Valcarcel, Jibo Wang, Shih-Ying Wu, Jeremy S. York