Patents by Inventor Richard Jude Samulski

Richard Jude Samulski 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: 20210340568
    Abstract: The present invention provides a gene editing system having reduced off target effects comprising (a) a vector comprising a nucleic acid sequence encoding a nuclease, wherein the nucleic acid encoding the nuclease contains within its sequence a regulatory nucleic acid sequence having a first and second set of splice elements defining a first and second intron, wherein the first and second intron flank a sequence encoding a non-naturally occurring exon sequence containing an in-frame stop codon sequence, and wherein the first and second intron are spliced from the mRNA message to produce an mRNA encoding a non-functional nuclease that contains an amino acid sequence encoded by the non-naturally occurring exon; and (b) an oligonucleotide that binds to the regulatory sequence. Further provided are methods of using the gene editing system of this invention to regulate transgene expression.
    Type: Application
    Filed: October 9, 2019
    Publication date: November 4, 2021
    Inventor: Richard Jude Samulski
  • Patent number: 11124546
    Abstract: This invention relates to modified parvovirus capsid proteins with enhanced transduction efficiency, viral vectors comprising the same, and methods of using the same for delivery of nucleic acids to a cell or a subject.
    Type: Grant
    Filed: December 14, 2016
    Date of Patent: September 21, 2021
    Assignee: The University of North Carolina at Chapel Hill
    Inventors: Richard Jude Samulski, Jayme Warischalk
  • Patent number: 11116850
    Abstract: This invention relates to viral vectors for delivery of alpha-L-iduronidase to the cornea of a subject and methods of using the same for treatment and prevention of corneal clouding and blindness in a subject due to mucopolysaccharidosis I.
    Type: Grant
    Filed: February 22, 2017
    Date of Patent: September 14, 2021
    Assignee: The University of North Carolina at Chapel Hill
    Inventors: Matthew Louis Hirsch, Richard Jude Samulski
  • Publication number: 20210269828
    Abstract: Disclosed herein are vectors for delivery of nucleic acid sequence into a target cell. The vectors are non-viral DNA constructs. The vectors have at least one DD-ITR, and complementary copies of the nucleic acid sequence operatively linked to regulatory elements that promote expression. The construct has covalently closed ends having a hairpin structure, and persists within the recipient cells as they divide. Delivery of the vector to the target cell results in sustained expression of the nucleic acid sequences in the target cell. Also disclosed are DNA vector constructs having at least one synthetic ITR, wherein the DNA construct forms linear DNA with hairpin covalently closed ends. Methods of generating the constructs and introducing target cells to thereby promote sustained expression of the nucleic acid sequences contained therein, are also disclosed. Further disclosed are cells and populations thereof, which contain the vectors.
    Type: Application
    Filed: June 21, 2019
    Publication date: September 2, 2021
    Applicant: ASKLEPIOS BIOPHARMACEUTICAL, INC.
    Inventor: Richard Jude SAMULSKI
  • Publication number: 20210147877
    Abstract: The present invention provides a polyploid adeno-associated virus (AAV) capsid, wherein the capsid comprises capsid protein VP1, wherein said capsid protein VP1 is from one or more than one first AAV serotype, wherein said capsid protein VP2 is from one or more than one first AAV serotype and capsid protein VP3, wherein said capsid protein VP3 is from one or more than one second AAV serotype and wherein at least one of said first AAV serotype is different from at least one of said second AAV serotype and is different from at least one of said third AAV serotype, in any combination.
    Type: Application
    Filed: November 18, 2020
    Publication date: May 20, 2021
    Inventors: Chengwen Li, Richard Jude Samulski
  • Publication number: 20210071148
    Abstract: This invention relates to modified parvovirus inverted terminal repeats (ITRs) that do not functionally interact with wild-type large Rep proteins, synthetic Rep proteins that functionally interact with the modified ITRs, and methods of using the same for delivery of nucleic acids to a cell or a subject. The modifications provide a novel Rep-ITR interaction that limits vector mobilization, increasing the safety of viral vectors.
    Type: Application
    Filed: November 19, 2020
    Publication date: March 11, 2021
    Inventors: Curtis Hewitt, Richard Jude Samulski
  • Patent number: 10934560
    Abstract: The present invention provides a polyploid adeno-associated virus (AAV) capsid, wherein the capsid comprises capsid protein VP1, wherein said capsid protein VP1 is from one or more than one first AAV serotype, wherein said capsid protein VP2 is from one or more than one first AAV serotype and capsid protein VP3, wherein said capsid protein VP3 is from one or more than one second AAV serotype and wherein at least one of said first AAV serotype is different from at least one of said second AAV serotype and is different from at least one of said third AAV serotype, in any combination.
    Type: Grant
    Filed: October 10, 2019
    Date of Patent: March 2, 2021
    Assignee: The University of North Carolina at Chapel Hill
    Inventors: Chengwen Li, Richard Jude Samulski
  • Patent number: 10889833
    Abstract: The present invention provides a polyploid adeno-associated virus (AAV) capsid, wherein the capsid comprises capsid protein VP1, wherein said capsid protein VP1 is from one or more than one first AAV serotype, wherein said capsid protein VP2 is from one or more than one first AAV serotype and capsid protein VP3, wherein said capsid protein VP3 is from one or more than one second AAV serotype and wherein at least one of said first AAV serotype is different from at least one of said second AAV serotype and is different from at least one of said third AAV serotype, in any combination.
    Type: Grant
    Filed: October 10, 2019
    Date of Patent: January 12, 2021
    Assignee: The University of North Carolina at Chapel Hill
    Inventors: Chengwen Li, Richard Jude Samulski
  • Patent number: 10858632
    Abstract: This invention relates to modified parvovirus inverted terminal repeats (ITRs) that do not functionally interact with wild-type large Rep proteins, synthetic Rep proteins that functionally interact with the modified ITRs, and methods of using the same for delivery of nucleic acids to a cell or a subject. The modifications provide a novel Rep-ITR interaction that limits vector mobilization, increasing the safety of viral vectors.
    Type: Grant
    Filed: February 8, 2019
    Date of Patent: December 8, 2020
    Assignee: The University of North Carolina at Chapel Hill
    Inventors: Curtis Hewitt, Richard Jude Samulski
  • Publication number: 20200340013
    Abstract: Disclosed herein are methods and compositions for inhibition of an innate immune response associated with AAV transduction.
    Type: Application
    Filed: January 18, 2019
    Publication date: October 29, 2020
    Inventors: Chengwen Li, Richard Jude Samulski
  • Publication number: 20200325456
    Abstract: Disclosed herein is an adeno-associated virus (AAV) particle comprising a surface-bound peptide that enhances transduction of cells across the blood-brain barrier (BBB). Also disclosed herein is a modified AAV capsid protein comprising an insertion of a polypeptide that enhances transduction of cells across the BBB, and an AAV particle comprising the modified AAV capsid protein. Specific peptides are provided. Pharmaceutical formulations and method of administering/delivering a nucleic acid to a cell of the brain and/or central nervous system are also disclosed.
    Type: Application
    Filed: December 19, 2018
    Publication date: October 15, 2020
    Inventors: Chengwen Li, Richard Jude Samulski
  • Publication number: 20200318080
    Abstract: Methods and compositions for treating symptoms of conditions such as but not limited to osteoarthritis in horses. The methods may feature direct intraarticular injection of a recombinant self-complementary adeno-associated virus (sc-rAAV) with a vector adapted to express a modified IL-1 Ra peptide. The methods of the present invention may express a therapeutically effective amount of the modified IL-1 Ra peptide so as to ameliorating symptoms associated with the condition being treated.
    Type: Application
    Filed: August 18, 2017
    Publication date: October 8, 2020
    Inventors: Laurie R. Goodrich, C. Wayne McILwraith, Jeffrey S. Bartlett, Richard Jude Samulski
  • Publication number: 20200109418
    Abstract: The present invention provides a polyploid adeno-associated virus (AAV) capsid, wherein the capsid comprises capsid protein VP1, wherein said capsid protein VP1 is from one or more than one first AAV serotype, wherein said capsid protein VP2 is from one or more than one first AAV serotype and capsid protein VP3, wherein said capsid protein VP3 is from one or more than one second AAV serotype and wherein at least one of said first AAV serotype is different from at least one of said second AAV serotype and is different from at least one of said third AAV serotype, in any combination.
    Type: Application
    Filed: October 10, 2019
    Publication date: April 9, 2020
    Inventors: Chengwen Li, Richard Jude Samulski
  • Patent number: 10550405
    Abstract: The present invention provides a polyploid adeno-associated virus (AAV) capsid, wherein the capsid comprises capsid protein VP1, wherein said capsid protein VP1 is from one or more than one first AAV serotype, wherein said capsid protein VP2 is from one or more than one first AAV serotype and capsid protein VP3, wherein said capsid protein VP3 is from one or more than one second AAV serotype and wherein at least one of said first AAV serotype is different from at least one of said second AAV serotype and is different from at least one of said third AAV serotype, in any combination.
    Type: Grant
    Filed: July 31, 2018
    Date of Patent: February 4, 2020
    Assignee: The University of North Carolina at Chapel Hill
    Inventors: Chengwen Li, Richard Jude Samulski
  • Publication number: 20190382452
    Abstract: This invention relates to modified parvovirus capsid proteins with enhanced transduction efficiency, viral vectors comprising the same, and methods of using the same for delivery of nucleic acids to a cell or a subject.
    Type: Application
    Filed: December 14, 2016
    Publication date: December 19, 2019
    Inventors: Richard Jude Samulski, Jayme Warischalk
  • Publication number: 20190262373
    Abstract: The present invention provides AAV capsid proteins comprising a modification in the amino acid sequence and virus capsids and virus vectors comprising the modified AAV capsid protein. The invention also provides methods of administering the virus vectors and virus capsids of the invention to a cell or to a subject in vivo.
    Type: Application
    Filed: August 16, 2017
    Publication date: August 29, 2019
    Inventors: Kenton Woodard, Richard Jude Samulski
  • Publication number: 20190185823
    Abstract: This invention relates to modified parvovirus inverted terminal repeats (ITRs) that do not functionally interact with wild-type large Rep proteins, synthetic Rep proteins that functionally interact with the modified ITRs, and methods of using the same for delivery of nucleic acids to a cell or a subject. The modifications provide a novel Rep-ITR interaction that limits vector mobilization, increasing the safety of viral vectors.
    Type: Application
    Filed: February 8, 2019
    Publication date: June 20, 2019
    Inventors: Curtis Hewitt, Richard Jude Samulski
  • Patent number: 10233428
    Abstract: This invention relates to modified parvovirus inverted terminal repeats (ITRs) that do not functionally interact with wild-type large Rep proteins, synthetic Rep proteins that functionally interact with the modified ITRs, and methods of using the same for delivery of nucleic acids to a cell or a subject. The modifications provide a novel Rep-ITR interaction that limits vector mobilization, increasing the safety of viral vectors.
    Type: Grant
    Filed: October 26, 2015
    Date of Patent: March 19, 2019
    Assignee: The University of North Carolina at Chapel Hill
    Inventors: Curtis Hewitt, Richard Jude Samulski
  • Publication number: 20190048363
    Abstract: This invention relates to viral vectors for delivery of alpha-L-iduronidase to the cornea of a subject and methods of using the same for treatment and prevention of corneal clouding and blindness in a subject due to mucopolysaccharidosis I.
    Type: Application
    Filed: February 22, 2017
    Publication date: February 14, 2019
    Inventors: Matthew Louis Hirsch, Richard Jude Samulski
  • Publication number: 20180371496
    Abstract: The present invention provides a polyploid adeno-associated virus (AAV) capsid, wherein the capsid comprises capsid protein VP1, wherein said capsid protein VP1 is from one or more than one first AAV serotype, wherein said capsid protein VP2 is from one or more than one first AAV serotype and capsid protein VP3, wherein said capsid protein VP3 is from one or more than one second AAV serotype and wherein at least one of said first AAV serotype is different from at least one of said second AAV serotype and is different from at least one of said third AAV serotype, in any combination.
    Type: Application
    Filed: July 31, 2018
    Publication date: December 27, 2018
    Inventors: Chengwen Li, Richard Jude Samulski