Patents by Inventor Phillip D. Zamore

Phillip D. Zamore 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).

  • Patent number: 11060088
    Abstract: The disclosure relates, in some aspects, to compositions and methods for treating corneal disease (e.g., corneal neovascularization. In some embodiments, the disclosure relates to rAAV-mediated delivery of an cornea-associated transgene to a subject. In some embodiments, the rAAV transduces the corneal tissue of a subject.
    Type: Grant
    Filed: February 10, 2017
    Date of Patent: July 13, 2021
    Assignee: University of Massachusetts
    Inventors: Guangping Gao, Yi Lu, Qiang Zheng, Xu Xun, Phillip D. Zamore, Phillip Tai
  • Publication number: 20210205476
    Abstract: The invention in some aspects relates to isolated nucleic acids, compositions, and kits useful for identifying adeno-associated viruses in cells. In some aspects, the invention provides kits and methods for producing somatic transgenic animal models using recombinant AAV (rAAV) to an animal having at least one transgene that expresses a small interfering nucleic acid or at least one binding site for a miRNA.
    Type: Application
    Filed: December 22, 2020
    Publication date: July 8, 2021
    Applicant: University of Massachusetts
    Inventors: Guangping Gao, Phillip D. Zamore
  • Patent number: 11046955
    Abstract: The present disclosure relates to the field of rAAV delivery of transgenes. In some aspects, the disclosure relates to RNAi. Provided herein are recombinant adeno-associated virus (rAAV) vectors comprising modified ITRs. In some embodiments, the modified ITRs comprise a sequence encoding a shRNA, miRNA, or AmiRNA.
    Type: Grant
    Filed: April 15, 2016
    Date of Patent: June 29, 2021
    Assignee: University of Massachusetts
    Inventors: Guangping Gao, Jun Xie, Phillip D. Zamore
  • Patent number: 10975391
    Abstract: The disclosure in some aspects, relates to nucleic acids, compositions and kits useful for gene therapy with reduced immune response to transgene products.
    Type: Grant
    Filed: April 24, 2015
    Date of Patent: April 13, 2021
    Assignee: University of Massachusetts
    Inventors: Guangping Gao, Phillip D. Zamore, Shaoyong Li
  • Patent number: 10905776
    Abstract: The invention in some aspects relates to isolated nucleic acids, compositions, and kits useful for identifying adeno-associated viruses in cells. In some aspects, the invention provides kits and methods for producing somatic transgenic animal models using recombinant AAV (rAAV) to an animal having at least one transgene that expresses a small interfering nucleic acid or at least one binding site for a miRNA.
    Type: Grant
    Filed: April 3, 2019
    Date of Patent: February 2, 2021
    Assignee: University of Massachusetts
    Inventors: Guangping Gao, Phillip D. Zamore
  • Publication number: 20200392500
    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: Application
    Filed: June 23, 2020
    Publication date: December 17, 2020
    Applicant: University of Massachusetts
    Inventors: Guangping Gao, Phillip D. Zamore, Jun Xie
  • Publication number: 20200339988
    Abstract: The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).
    Type: Application
    Filed: February 20, 2020
    Publication date: October 29, 2020
    Applicant: University of Massachusetts
    Inventors: Terence Flotte, Christian Mueller, Phillip D. Zamore
  • Publication number: 20200291396
    Abstract: The present invention provides methods of enhancing the efficacy and specificity of RNA silencing. The invention also provides compositions for mediating RNA silencing. In particular, the invention provides siRNAs, siRNA-like molecules, shRNAs, vectors and transgenes having improved specificity and efficacy in mediating silencing of a target gene. Therapeutic methods are also featured.
    Type: Application
    Filed: February 24, 2020
    Publication date: September 17, 2020
    Inventors: Phillip D. ZAMORE, Gyorgy HUTVAGNER, Dianne SCHWARZ, Martin SIMARD
  • Publication number: 20200270602
    Abstract: The present invention relates to a Drosophila in vitro system which was used to demonstrate that dsRNA is processed to RNA segments 21-23 nucleotides (nt) in length. Furthermore, when these 21-23 nt fragments are purified and added back to Drosophila extracts, they mediate RNA interference in the absence of long dsRNA, Thus, these 21-23 nt fragments are the sequence-specific mediators of RNA degradation. A molecular signal, which may be their specific length, must be present in these 21-23 nt fragments to recruit cellular factors involved in RNAi. This present invention encompasses these 21-23 nt fragments and their use for specifically inactivating gene function. The use of these fragments (or chemically synthesized oligonucleotides of the same or similar nature) enables the targeting of specific mRNAs for degradation in mammalian cells, where the use of long dsRNAs to elicit RNAi is usually not practical, presumably because of the deleterious effects of the interferon response.
    Type: Application
    Filed: September 24, 2019
    Publication date: August 27, 2020
    Inventors: Thomas Tuschl, Phillip D. Zamore, Phillip A. Sharp, David P. Bartel
  • Publication number: 20200268905
    Abstract: The instant disclosure provides RNA-modulating agents that function to recruit one or more small regulatory RNA molecules (e.g., miRNA molecules, Y RNAs, and siRNAs) to a target mRNA thereby modulating (e.g., inhibiting) the translation of the target mRNA or destabilizing the mRNA. Also provided are miRNA inhibitors and diagnostic agents that have improved binding affinity for their target miRNAs. Methods for using the RNA-modulating agents, miRNA inhibitors and diagnostic agents are also provided.
    Type: Application
    Filed: December 20, 2019
    Publication date: August 27, 2020
    Inventors: Phillip D. Zamore, Jennifer Broderick
  • Patent number: 10731158
    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: December 18, 2018
    Date of Patent: August 4, 2020
    Assignee: University of Massachusetts
    Inventors: Guangping Gao, Phillip D. Zamore, Jun Xie
  • Patent number: 10731155
    Abstract: The invention provides engineered RNA precursors that when expressed in a cell are processed by the cell to produce targeted small interfering RNAs (siRNAs) that selectively silence targeted genes (by cleaving specific mRNAs) using the cell's own RNA interference (RNAi) pathway. By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vivo with appropriate regulatory sequences, expression of the engineered RNA precursors can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells.
    Type: Grant
    Filed: November 15, 2017
    Date of Patent: August 4, 2020
    Assignee: UNIVERSITY OF MASSACHUSETTS
    Inventors: Phillip D. Zamore, Juanita McLachlan, Gyorgy Hutvagner, Alla Grishok, Craig C. Mello
  • Publication number: 20200231953
    Abstract: The disclosure in some aspects relates to recombinant adeno-associated viruses having nuclease grafted to one or more capsid proteins. In some aspects, the disclosure relates to isolated AA V capsid proteins having terminally grafted nucleases and isolated nucleic acids encoding the same. Recent approaches to delivering nucleases to cells for gene editing have focused on delivering of expression vectors engineered to express the nucleases in target cells. However, these approaches have proved to be problematic in many instances due to genotoxicity resulting from to prolonged expression of gene editing system in vivo. To prevent such off-target genotoxicity due to prolonged presence of a gene editing system, several studies explored delivery of mRNA or protein instead of delivering the gene coding for the nucleases in cell culture.
    Type: Application
    Filed: January 29, 2020
    Publication date: July 23, 2020
    Applicant: University of Massachusetts
    Inventors: Guangping Gao, Phillip D. Zamore, Dan Wang
  • Patent number: 10604754
    Abstract: The present invention provides methods of enhancing the efficacy and specificity of RNA silencing. The invention also provides compositions for mediating RNA silencing. In particular, the invention provides siRNAs, siRNA-like molecules, shRNAs, vectors and transgenes having improved specificity and efficacy in mediating silencing of a target gene. Therapeutic methods are also featured.
    Type: Grant
    Filed: June 3, 2014
    Date of Patent: March 31, 2020
    Assignee: UNIVERSITY OF MASSACHUSETTS
    Inventors: Phillip D. Zamore, Gyorgy Hutvagner, Dianne Schwarz, Martin Simard
  • Patent number: 10597656
    Abstract: The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).
    Type: Grant
    Filed: August 9, 2018
    Date of Patent: March 24, 2020
    Assignee: University of Massachusetts
    Inventors: Terence Flotte, Christian Mueller, Phillip D. Zamore
  • Patent number: 10584321
    Abstract: The disclosure in some aspects relates to recombinant adeno-associated viruses having nuclease grafted to one or more capsid proteins. In some aspects, the disclosure relates to isolated AAV capsid proteins having terminally grafted nucleases and isolated nucleic acids encoding the same. Recent approaches to delivering nucleases to cells for gene editing have focused on delivering of expression vectors engineered to express the nucleases in target cells. However, these approaches have proved to be problematic in many instances due to genotoxicity resulting from to prolonged expression of gene editing system in vivo. To prevent such off-target genotoxicity due to prolonged presence of a gene editing system, several studies explored delivery of mRNA or protein instead of delivering the gene coding for the nucleases in cell culture.
    Type: Grant
    Filed: February 12, 2016
    Date of Patent: March 10, 2020
    Assignee: University of Massachusetts
    Inventors: Guangping Gao, Phillip D. Zamore, Dan Wang
  • Patent number: 10556020
    Abstract: The instant disclosure provides RNA-modulating agents that function to recruit one or more small regulatory RNA molecules (e.g., miRNA molecules, Y RNAs, and siRNAs) to a target mRNA thereby modulating (e.g., inhibiting) the translation of the target mRNA or destabilizing the mRNA. Also provided are miRNA inhibitors and diagnostic agents that have improved binding affinity for their target miRNAs. Methods for using the RNA-modulating agents, miRNA inhibitors and diagnostic agents are also provided.
    Type: Grant
    Filed: September 25, 2015
    Date of Patent: February 11, 2020
    Assignee: UNIVERSITY OF MASSACHUSETTS
    Inventors: Phillip D. Zamore, Jennifer Broderick
  • Publication number: 20200032261
    Abstract: Based at least in part on an understanding of the mechanisms by which small RNAs (e.g., naturally-occurring miRNAs) mediate RNA silencing in plants, rules have been established for determining, for example, the degree of complementarity required between an RNAi-mediating agent and its target, i.e., whether mismatches are tolerated, the number of mismatches tolerated, the effect of the position of the mismatches, etc. Such rules are useful, in particular, in the design of improved RNAi-mediating agents which allow for more exact control of the efficacy of RNA silencing.
    Type: Application
    Filed: June 13, 2019
    Publication date: January 30, 2020
    Inventors: Phillip D. ZAMORE, Guiliang TANG
  • Publication number: 20200024602
    Abstract: The present invention provides methods of enhancing the efficacy and specificity of RNAi using single or double blunt-ended siRNA. The invention also provides single and double-blunt ended siRNA compositions, vectors, and transgenes containing the same for mediating silencing of a target gene. Therapeutic methods are also featured.
    Type: Application
    Filed: June 28, 2019
    Publication date: January 23, 2020
    Inventors: Phillip D. ZAMORE, Dianne SCHWARZ
  • Publication number: 20200024599
    Abstract: The present invention relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington's disease (HD). The present invention utilizes RNA Interference technology (RNAi) against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease.
    Type: Application
    Filed: May 17, 2019
    Publication date: January 23, 2020
    Inventors: Neil Aronin, Phillip D. Zamore