Patents by Inventor James M. Wilson

James M. Wilson 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).

  • Gene therapy for treating hemophilia B
    Patent number: 11191847
    Abstract: Compositions for the treatment of hemophilia B are provided. In certain embodiments, the composition is a recombinant adeno-associated virus (rAAV) comprising an AAVrh10 capsid and a vector genome packaged therein, wherein the vector genome comprises an AAV 5? inverted terminal repeat (ITR), a coding sequence for a human Factor IX (F9) having coagulation function operably linked to regulatory elements which direct expression of the human Factor IX in liver cells, and an AAV 3? ITR.
    Type: Grant
    Filed: April 13, 2017
    Date of Patent: December 7, 2021
    Assignee: The Trustees of the University of Pennsylvania
    Inventors: Lili Wang, James M. Wilson
  • ADENO-ASSOCIATED VIRUS (AAV) SEROTYPE 8 SEQUENCES, VECTORS CONTAINING SAME, AND USES THEREFOR
    Publication number: 20210348190
    Abstract: Sequences of a serotype 8 adeno-associated virus and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles.
    Type: Application
    Filed: January 11, 2021
    Publication date: November 11, 2021
    Inventors: Guangping Gao, James M. Wilson, Mauricio R. Alvira
  • NOVEL AAV8 MUTANT CAPSIDS AND COMPOSITIONS CONTAINING SAME
    Publication number: 20210340569
    Abstract: Provided herein are AAV8 mutant capsids and rAAV comprising the same. In one embodiment, vectors employing the AAV8 mutant capsid show increased transduction in a selected tissue as compared to AAV8.
    Type: Application
    Filed: July 12, 2021
    Publication date: November 4, 2021
    Inventors: James M. Wilson, Qiang Wang
  • Intrathecal administration of adeno-associated-viral vectors for gene therapy
    Patent number: 11135313
    Abstract: A composition comprising at least one AAV vector formulated for intrathecal delivery to the central nervous system is described. The composition comprises at least one expression cassette which contains sequences encoding an immunoglobulin construct linked to expression control sequences therefor and a pharmaceutically acceptable carrier. The immunoglobulin construct may be an immunoglobulin modified to have decreased or no measurable affinity for neonatal Fc receptor (FcRn).
    Type: Grant
    Filed: October 27, 2016
    Date of Patent: October 5, 2021
    Assignee: The Trustees of the University of Pennsylvania
    Inventors: James M. Wilson, Christian Hinderer, William Thomas Rothwell
  • ADENO-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME, AND USES THEREFOR
    Publication number: 20210299274
    Abstract: Sequences of novel adeno-associated virus capsids and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles. AAV-mediated delivery of therapeutic and immunogenic genes using the vectors of the invention is also provided.
    Type: Application
    Filed: April 7, 2021
    Publication date: September 30, 2021
    Inventors: James M. Wilson, Guangping Gao, Mauricio R. Alvira, Luc H. Vandenberghe
  • METHOD OF DETECTING AND/OR IDENTIFYING ADENO-ASSOCIATED VIRUS (AAV) SEQUENCES AND ISOLATING NOVEL SEQUENCES IDENTIFIED THEREBY
    Publication number: 20210285012
    Abstract: Adeno-associated virus rh.10 sequences, vectors containing same, and methods of use are provided.
    Type: Application
    Filed: May 13, 2021
    Publication date: September 16, 2021
    Inventors: Guangping Gao, James M. Wilson, Mauricio R. Alvira
  • GENE THERAPY FOR TREATING FAMILIAL HYPERCHOLESTEROLEMIA
    Publication number: 20210285013
    Abstract: Compositions and regimens useful in reducing one or more of: LDL-cholesterol, total cholesterol, and/or fasting triglycerides in a subject, and/or modifying fractional catabolic rate (FCR) of LDL apolipoprotein B (apoB) from baseline to a selected time point after rAAV administration are provided. The method involves administering to the human subject via a peripheral vein by infusion of a suspension of replication deficient recombinant adeno-associated virus (rAAV).
    Type: Application
    Filed: November 17, 2020
    Publication date: September 16, 2021
    Inventors: James M. Wilson, Daniel J. Rader
  • AAV-EPO for treating companion animals
    Patent number: 11117942
    Abstract: Compositions and methods are provided for treating companion animals are provided. An adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding erythropoietin (EPO). In desired embodiments, the subject is a cat or dog.
    Type: Grant
    Filed: August 30, 2016
    Date of Patent: September 14, 2021
    Assignee: The Trustees of the University of Pennsylvania
    Inventors: Christian Hinderer, James M. Wilson, Matthew Wilson
  • AAV8 mutant capsids and compositions containing same
    Patent number: 11091776
    Abstract: Provided herein are AAV8 mutant capsids and rAAV comprising the same. In one embodiment, vectors employing the AAV8 mutant capsid show increased transduction in a selected tissue as compared to AAV8.
    Type: Grant
    Filed: April 13, 2017
    Date of Patent: August 17, 2021
    Assignee: The Trustees of the University of Pennsylvania
    Inventors: James M. Wilson, Qiang Wang
  • REGULATABLE GENE EDITING COMPOSITIONS AND METHODS
    Publication number: 20210246466
    Abstract: Provided herein is a regulatable gene editing system. The system includes at least one regulatable promoter which controls expression of a gene editing nuclease. The system may optionally contain more than one regulatable promoter, e.g., one promoter for the guide RNA where the system is a CRISPR system and another promoter for a selected gene. The system involves delivering to a subject: (a) at least one nucleic acid encoding one or more DNA binding domains, (b) a nucleic acid sequence comprising a donor gene for insertion into a selected gene locus, (c) at least one nucleic acid sequence comprising a coding sequence of an activation domain for the regulatable promoter, and (d) at least one coding sequence encoding a nuclease; wherein expression of the nuclease is under the control of at least one regulatable promoter, and the promoter is activated and/or regulated by a pharmaceutical agent.
    Type: Application
    Filed: May 3, 2018
    Publication date: August 12, 2021
    Inventors: James M. Wilson, Lili Wang
  • GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I
    Publication number: 20210228740
    Abstract: A suspension useful for AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDUA gene is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating MPSI and the symptoms associated with Hurler, Hurler-Scheie and Scheie syndromes.
    Type: Application
    Filed: March 15, 2021
    Publication date: July 29, 2021
    Inventors: Christian Hinderer, James M. Wilson
  • Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
    Patent number: 11041171
    Abstract: Adeno-associated virus rh.20 sequences, vectors containing same, and methods of use are provided.
    Type: Grant
    Filed: November 13, 2019
    Date of Patent: June 22, 2021
    Assignee: The Trustees of the University of Pennsylvania
    Inventors: Guangping Gao, James M. Wilson, Mauricio R. Alvira
  • Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
    Patent number: 11034976
    Abstract: Adeno-associated virus rh.10 sequences, vectors containing same, and methods of use are provided.
    Type: Grant
    Filed: November 21, 2019
    Date of Patent: June 15, 2021
    Assignee: The Trustees of the University of Pennsylvania
    Inventors: Guangping Gao, James M. Wilson, Mauricio R. Alvira
  • Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
    Patent number: 11034977
    Abstract: Adeno-associated virus rh.20 sequences, vectors containing same, and methods of use are provided.
    Type: Grant
    Filed: November 27, 2019
    Date of Patent: June 15, 2021
    Assignee: The Trustees of the University of Pennsylvania
    Inventors: Guangping Gao, James M. Wilson, Mauricio R. Alvira
  • ADENO-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME, AND USES THEREFOR
    Publication number: 20210170050
    Abstract: Sequences of novel adeno-associated virus capsids and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles. AAV-mediated delivery of therapeutic and immunogenic genes using the vectors of the invention is also provided.
    Type: Application
    Filed: January 18, 2021
    Publication date: June 10, 2021
    Inventors: James M. Wilson, Guangping Gao, Mauricio R. Alvira, Luc H. Vandenberghe
  • Constructs and methods for delivering molecules via viral vectors with blunted innate immune responses
    Patent number: 11015210
    Abstract: A CpG-modified recombinant adeno-associated viral (AAV) vector is described. The vector carries a nucleic acid molecule comprising AAV inverted terminal repeat (ITR) sequences and an exogenous gene sequence under the control of regulatory sequences which control expression of the gene product, in which the nucleic acid sequences carried by the vector are modified to significantly reduce CpG di-nucleotides such that an immune response to the vector is reduced as compared to the unmodified AAV vector. Also provided are methods and regimens for delivering transgenes using these AAV viral vectors, in which the innate immune response to the vector and/or transgene is significantly modulated.
    Type: Grant
    Filed: April 15, 2016
    Date of Patent: May 25, 2021
    Inventors: Susan M. Faust, Joseph E. Rabinowitz, James M. Wilson
  • COMPOSITIONS FOR TARGETING CONDUCTING AIRWAY CELLS COMPRISING ADENO-ASSOCIATED VIRUS CONSTRUCTS
    Publication number: 20210147875
    Abstract: An artificial AAV capsid comprising a heterologous conducting airway targeting sequence is provided. The artificial AAV is useful as a targeting moiety, for delivery of heterologous molecules which are associated therewith. The artificial AAV is also useful in the generation of AAV vectors having the artificial capsid. Also described are methods of modifying the native tropism and transduction efficiency of vectors by improving and/or ablating their ability to transduce conducting airway cells. Methods of targeting conducting airway cells and delivering therapeutic and other molecules thereto are also provided.
    Type: Application
    Filed: January 14, 2021
    Publication date: May 20, 2021
    Inventors: Christie L. Bell, James M. Wilson
  • Compositions and Methods for Altering Tissue Specificity and Improving AAV9-Mediated Gene Transfer
    Publication number: 20210145857
    Abstract: A method of altering the targeting and/or cellular uptake efficiency of an adeno-associated virus (AAV) viral vector having a capsid containing an AAV9 cell surface binding domain is described. The method involves modifying a clade F cell surface receptor which comprises a glycan having a terminal sialic acid residue and a penultimate ?-galactose residue. The modification may involve retargeting the vector by temporarily functionally ablate AAV9 binding in a subset of cells, thereby redirecting the vector to another subset of cells. Alternatively, the modification may involve increasing cellular update efficiency by treating the cells with a neuraminidase to expose cell surface ?-galactose. Also provided are compositions containing the AAV9 vector and a neuraminidase. Also provided is a method for purifying AAV9 using ?-galactose linked to solid support.
    Type: Application
    Filed: January 27, 2021
    Publication date: May 20, 2021
    Inventors: James M. Wilson, Christie L. Bell, Luc H. Vandenberghe
  • NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS HAVING REDUCED CAPSID DEAMIDATION AND USES THEREFOR
    Publication number: 20210123073
    Abstract: A recombinant adeno-associated virus (rAAV) vector comprising an AAV capsid having a heterogeneous population of vp1 proteins, a heterogeneous population of vp2 protein and a heterogeneous population of vp3 proteins. The capsid contains modified amino acids as compared to the encoded VP1 amino acid sequence, the capsid containing highly deamidated asparagine residues at asparagine-glycine pair, and further comprising multiple other, less deamidated asparagine and optionally glutamine residues.
    Type: Application
    Filed: February 27, 2019
    Publication date: April 29, 2021
    Inventors: James M. Wilson, April Tepe, Kevin Turner, Joshua Joyner Sims, Qiang Wang
  • Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
    Patent number: 10973928
    Abstract: Sequences of novel adeno-associated virus capsids and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles. AAV-mediated delivery of therapeutic and immunogenic genes using the vectors of the invention is also provided.
    Type: Grant
    Filed: July 25, 2018
    Date of Patent: April 13, 2021
    Assignee: The Trustees of the University of Pennsylvania
    Inventors: James M. Wilson, Guangping Gao, Mauricio R. Alvira, Luc H. Vandenberghe