Patents by Inventor Anastasia Khvorova

Anastasia Khvorova has filed for patents to protect the following inventions. This listing includes patent applications that are pending as well as patents that have already been granted by the United States Patent and Trademark Office (USPTO).

  • Publication number: 20210363518
    Abstract: Chemically modified crRNAs and tracrRNAs are provided. crRNAs and tracrRNAs with 5? and/or 3? conjugated moieties are provided. crRNAs and tracrRNAs with modifications in the repeat region of the crRNA or the anti-repeat region of the tracrRNA are provided. Methods of using the crRNAs and tracrRNAs for genome editing with a CRISPR nuclease and kits for performing the same are also provided.
    Type: Application
    Filed: March 18, 2019
    Publication date: November 25, 2021
    Inventors: Erik Joseph SONTHEIMER, Anastasia KHVOROVA, Jonathan Kenneth WATTS, Aamir MIR, Julia Frances ALTERMAN, Matthew Ryan HASSLER, Michael Harry BRODSKY, Alexandre Jean-Marie DEBACKER
  • Publication number: 20210363524
    Abstract: This disclosure relates to novel SNCA targeting sequences. Novel SNCA targeting oligonucleotides for the treatment of neurodegenerative diseases are also provided.
    Type: Application
    Filed: March 17, 2021
    Publication date: November 25, 2021
    Inventors: Anastasia Khvorova, Chantal Ferguson, Sarah Davis, Kathryn Monopoli
  • Publication number: 20210355491
    Abstract: This disclosure relates to novel MSH3 targeting sequences. Novel MSH3 targeting oligonucleotides for the treatment of neurodegenerative diseases are also provided.
    Type: Application
    Filed: April 20, 2021
    Publication date: November 18, 2021
    Inventors: Anastasia Khvorova, Chantal Ferguson
  • Publication number: 20210340533
    Abstract: This disclosure relates to a therapeutic combination of drugs for the treatment or management of a neurodegenerative disease, the combination comprising: a first conjugate comprising an RNA silencing agent and a first targeting agent that targets the first conjugate to the central nervous system, and a second conjugate comprising an antagonist of the RNA silencing agent and a second targeting agent that targets the second conjugate to a off-target tissue.
    Type: Application
    Filed: December 23, 2020
    Publication date: November 4, 2021
    Inventors: Anastasia Khvorova, Chantal Ferguson
  • Publication number: 20210340535
    Abstract: This disclosure relates to novel C9ORF72 targeting sequences. Novel sense and antisense dual-targeting oligonucleotides for the treatment of neurodegenerative diseases are also provided.
    Type: Application
    Filed: March 26, 2021
    Publication date: November 4, 2021
    Inventors: Anastasia Khvorova, Bruno Miguel da Cruz Godinho, James W. Gilbert
  • Publication number: 20210317460
    Abstract: This disclosure relates to novel PRNP targeting sequences. Novel PRNP targeting oligonucleotides for the treatment of neurodegenerative diseases are also provided.
    Type: Application
    Filed: February 26, 2021
    Publication date: October 14, 2021
    Inventors: Anastasia Khvorova, Zachary Kennedy
  • Patent number: 11118178
    Abstract: The present invention relates to methods for in vivo administration of sd-rxRNA molecules.
    Type: Grant
    Filed: February 7, 2019
    Date of Patent: September 14, 2021
    Assignee: Phio Pharmaceuticals Corp.
    Inventors: Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, James Cardia
  • Publication number: 20210267893
    Abstract: Novel artificial exosomes and methods for producing novel artificial exosomes are provided. Methods of delivering cargo molecules to a cell using artificial exosomes are also provided.
    Type: Application
    Filed: January 21, 2021
    Publication date: September 2, 2021
    Inventors: Reka Agnes Haraszti, Anastasia Khvorova, Neil Aronin
  • Publication number: 20210261968
    Abstract: The present invention relates to RNAi constructs with improved tissue and cellular uptake characteristics and methods of use of these compounds in dermal and fibrotic applications.
    Type: Application
    Filed: January 15, 2021
    Publication date: August 26, 2021
    Applicant: Phio Pharmaceuticals Corp.
    Inventors: Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, Pamela A. Pavco, Lyn Libertine, James Cardia, Karen G. Bulock
  • Publication number: 20210147849
    Abstract: The present invention relates to RNAi constructs with minimal double-stranded regions, and their use in gene silencing. RNAi constructs associated with the invention include a double stranded region of 8-14 nucleotides and a variety of chemical modifications, and are highly effective in gene silencing.
    Type: Application
    Filed: July 21, 2020
    Publication date: May 20, 2021
    Applicant: Phio Pharmaceuticals Corp.
    Inventors: Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, James Cardia
  • Publication number: 20210139901
    Abstract: Provided are compositions and methods comprising two-tailed siRNAs (tt-siRNAs) that exhibit unprecedented cellular uptake and silencing. Also provided are methods of treating neurological and other diseases with the two-tailed siRNAs of the invention.
    Type: Application
    Filed: October 15, 2020
    Publication date: May 13, 2021
    Inventors: Anastasia Khvorova, Julia Alterman, Matthew Hassler
  • Publication number: 20210115442
    Abstract: Novel oligonucleotides that are fully chemically stabilized are provided. Methods of using oligonucleotides that are fully chemically stabilized are also provided.
    Type: Application
    Filed: August 21, 2020
    Publication date: April 22, 2021
    Inventors: Anastasia Khvorova, Julia Alterman, Sarah Davis, Anton Turanov
  • Publication number: 20210108200
    Abstract: This disclosure relates to an siRNA-lipid conjugate of formula Y-L-(H)n. Y is an siRNA molecule, L is a linker covalently bonded to Y and H, each H is independently a hydrophobic chain comprising 5 to 50 carbon atoms, n is 1, 2, or 3, and linker L is bonded to the 3? end of the sense strand of the siRNA.
    Type: Application
    Filed: September 16, 2020
    Publication date: April 15, 2021
    Inventors: Anastasia Khvorova, Annabelle Biscans
  • Publication number: 20210085793
    Abstract: Provided herein are branched oligonucleotides exhibiting efficient and specific tissue distribution, cellular uptake, minimum immune response and off-target effects, without formulation.
    Type: Application
    Filed: September 4, 2020
    Publication date: March 25, 2021
    Inventors: Anastasia Khvorova, Matthew Hassler, Julia Alterman, Bruno Miguel da Cruz Godinho
  • Patent number: 10945955
    Abstract: Novel artificial exosomes and methods for producing novel artificial exosomes are provided. Methods of delivering cargo molecules to a cell using artificial exosomes are also provided.
    Type: Grant
    Filed: April 24, 2019
    Date of Patent: March 16, 2021
    Assignee: UNIVERSITY OF MASSACHUSETTS
    Inventors: Reka Agnes Haraszti, Anastasia Khvorova, Neil Aronin
  • Publication number: 20210071177
    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: Application
    Filed: August 7, 2020
    Publication date: March 11, 2021
    Inventors: Anastasia Khvorova, Julia Alterman, Faith Conroy, Edith Pfister, Neil Aronin, Ken Yamada
  • Publication number: 20210062195
    Abstract: The present invention relates to ocular administration of sd-rxRNA and rxRNAori molecules.
    Type: Application
    Filed: May 12, 2020
    Publication date: March 4, 2021
    Applicant: Phio Pharmaceuticals Corp.
    Inventors: Lyn Libertine, Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, James Cardia, Pamela A. Pavco
  • Patent number: 10920226
    Abstract: Efficient sequence specific gene silencing is possible through the use of siRNA technology. Be 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 the nucleotide sequences for LDHA.
    Type: Grant
    Filed: October 25, 2018
    Date of Patent: February 16, 2021
    Assignee: Thermo Fisher Scientific Inc.
    Inventors: Anastasia Khvorova, Angela Reynolds, Devin Leake, William Marshall, Steven Read, Stephen Scaringe
  • Patent number: 10913948
    Abstract: The present invention relates to RNAi constructs with improved tissue and cellular uptake characteristics and methods of use of these compounds in dermal and fibrotic applications.
    Type: Grant
    Filed: March 12, 2018
    Date of Patent: February 9, 2021
    Assignee: Phio Pharmaceuticals Corp.
    Inventors: Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, Pamela A. Pavco, Lyn Libertine, James Cardia, Karen G. Bulock
  • Publication number: 20210024926
    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: Application
    Filed: July 13, 2020
    Publication date: January 28, 2021
    Inventors: Anastasia Khvorova, Neil Aronin, Matthew Hassler, Julia Alterman