Patents by Inventor Anna Tretiakova
Anna Tretiakova 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).
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Publication number: 20240009326Abstract: The present disclosure generally relates to polynucleotides and AAV vectors that provide for the expression of ALD protein in target (e.g., neurons or glial) cells when administered to subjects in need thereof. The present disclosure further relates to compositions comprising such a polynucleotide or vector. These polynucleotides, vectors, and compositions may be used for the treatment and prevention of ALD or AMN in subjects in need thereof.Type: ApplicationFiled: August 30, 2023Publication date: January 11, 2024Applicant: SwanBio Therapeutics LimitedInventors: Sean Clark, Karen Kozarsky, Tugba Guven-Ozkan, Anna Tretiakova
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Publication number: 20230374542Abstract: Disclosed herein are various optimized nucleic acids encoding the fukutin-related protein (FKRP). Recombinant vectors comprising the optimized nucleic acid (e.g. operatively linked to a muscle specific promoter), such as recombinant adeno-associated virus vectors, for expressing the protein (e.g. in skeletal and cardiac muscle), and therapeutic compositions contains the vectors are also disclosed. Therapeutic methods of administration of the vectors to a subject for the treatment of a subject with a dystroglycanopathy disorder (e.g., limb-girdle muscular dystrophy 2I) are also disclosed.Type: ApplicationFiled: October 6, 2021Publication date: November 23, 2023Applicants: Asklepios BioPharmaceutical, Inc., The University of North Carolina at Chapel HillInventors: Martin K. Childers, Jorge Omar Yanez-Cuna, Juan Manuel Iglesias Gonzalez, Sinclair Cooper, Antonia Evripioti, Michael L. Roberts, Anna Tretiakova, Lester Suarez, Anh Nguyen, SiewHui Low, Xiao Xiao
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Publication number: 20230365963Abstract: Aspects of the disclosure relate to compositions and methods useful for treating neurological diseases and disorders. In some embodiments, the disclosure provides a method for treating a neurological disease or disorder comprising administration of both a viral vector comprising interfering nucleic acids (e.g., artificial miRNAs) and a viral vector comprising a CYP46A1 protein. In some embodiments, the disclosure provides a method for treating Huntington's disease comprising administration of both a viral vector comprising interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and a viral vector comprising a CYP46A1 protein. In some embodiments, the viral vector comprises a modified viral capsid, such as for preferentially targeting cells in the CNS or PNS.Type: ApplicationFiled: September 21, 2021Publication date: November 16, 2023Applicant: ASKLEPIOS BIOPHARMACEUTICAL, INC.Inventors: Anna TRETIAKOVA, Lester Suarez, Anne Braae, Michael L. Roberts, Philippe Moullier
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Publication number: 20230340528Abstract: The present invention relates to methods of administration of rAAV vectors in a single administration method comprising a series of sub-administrations of sub-doses of rAAV vectors. The present invention also relates to rAAV vectors comprising cardiac-specific promoters, cardiac-cell specific promoters, multi-cell cardiac specific promoters, and elements thereof. The invention also relates to rAAV vectors, pharmaceutical compositions and uses thereof in methods for treating cardiovascular disease, heart diseases and heart failure in subjects in need thereof.Type: ApplicationFiled: August 5, 2021Publication date: October 26, 2023Applicant: ASKLEPIOS BIOPHARMACEUTICAL INC.Inventors: Michael W. O’CALLAGHAN, Michael L. ROBERTS, Jorg Omar YANEZ-CUNA, Juan Manuel IGLESIAS GONZALEZ, Antonia EVRIPIOTI, Sinclair COOPER, Ferzin SETHNA, Roger HAJJAR, Anna TRETIAKOVA
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Patent number: 11779655Abstract: The present disclosure generally relates to polynucleotides and AAV vectors that provide for the expression of ALD protein in target (e.g., neurons or glial) cells when administered to subjects in need thereof. The present disclosure further relates to compositions comprising such a polynucleotide or vector. These polynucleotides, vectors, and compositions may be used for the treatment and prevention of ALD or AMN in subjects in need thereof.Type: GrantFiled: December 21, 2021Date of Patent: October 10, 2023Assignee: SwanBio Therapeutics LimitedInventors: Sean Clark, Karen Kozarsky, Tugba Guven-Ozkan, Anna Tretiakova
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Publication number: 20230038520Abstract: Recombinant AAV (rAAV) vectors comprising a rAVV genome comprising a heterologous nucleic acid encoding a lysosomal protein, e.g., acid alpha-glucosidase (GAA) polypeptide, and optionally a signal peptide and/or optionally a targeting sequence, e.g., IGF2 targeting peptide, operatively linked to a liver-specific promoter (LSP), enabling the GAA polypeptide to be secreted from the liver and targeted to the lysosomes. Particular embodiments relate to a recombinant AAV (rAAV) vector encoding an alpha-glucosidase (GAA) polypeptide, having a liver secretory signal peptide and a IGF2 targeting peptide that binds human cation-independent mannose-6-phosphate receptor (CI-MPR) or to the IGF2 receptor, permitting proper subcellular localization of the GAA polypeptide to lysosomes. Also encompassed are cells, and methods to treat a lysosomal disease, for example, a glycogen storage disease type II (GSD II) disease and/or Pompe Disease with the rAAV vector.Type: ApplicationFiled: November 19, 2020Publication date: February 9, 2023Applicant: ASKLEPIOS BIOPHARMACEUTICAL, INC.Inventors: Michael ROBERTS, Juan Manuel IGLESIAS, Anna TRETIAKOVA, Michael W. O'CALLAGHAN, Achille FRANCOIS
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Publication number: 20220362403Abstract: The present disclosure generally relates to polynucleotides and AAV vectors that provide for the expression of ALD protein in target (e.g., neurons or glial) cells when administered to subjects in need thereof. The present disclosure further relates to compositions comprising such a polynucleotide or vector. These polynucleotides, vectors, and compositions may be used for the treatment and prevention of ALD or AMN in subjects in need thereof.Type: ApplicationFiled: December 21, 2021Publication date: November 17, 2022Applicant: SwanBio Therapeutics LimitedInventors: Sean Clark, Karen Kozarsky, Tugba Guven-Ozkan, Anna Tretiakova
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Publication number: 20220175965Abstract: The present disclosure generally relates to polynucleotides and AAV vectors that provide for the expression of ALD protein in target (e.g., neurons or glial) cells when administered to subjects in need thereof. The present disclosure further relates to compositions comprising such a polynucleotide or vector. These polynucleotides, vectors, and compositions may be used for the treatment and prevention of ALD or AMN in subjects in need thereof.Type: ApplicationFiled: December 21, 2021Publication date: June 9, 2022Applicant: SwanBio Therapeutics LimitedInventors: Sean Clark, Karen Kozarksy, Tugba Guven-Ozkan, Anna Tretiakova
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Patent number: 10975140Abstract: A recombinant adeno-associated virus (AAV) having an AAV capsid and packaged therein a heterologous nucleic acid which expresses two functional antibody constructs in a cell is described. Also described are antibodies comprising a heavy chain and a light chain from a heterologous antibody. In one embodiment, the antibodies are co-expressed from a vector containing: a first expression cassette which encodes at least a first open reading frame (ORF) for a first immunoglobulin under the control of regulatory control sequences which direct expression thereof; and a second expression cassette which comprises a second ORF, a linker, and a third ORF under the control of regulatory control sequences which direct expression thereof, wherein the second and third ORF for a second and third immunoglobulin construct. The vector co-expressing these two antibody constructs is in one embodiment an AAV, in which the 5? and 3? ITRs flank the expression cassettes and regulatory sequences.Type: GrantFiled: March 17, 2020Date of Patent: April 13, 2021Assignee: The Trustees of the University of PennsylvaniaInventors: James M. Wilson, Anna Tretiakova
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Publication number: 20200216520Abstract: A recombinant adeno-associated virus (AAV) having an AAV capsid and packaged therein a heterologous nucleic acid which expresses two functional antibody constructs in a cell is described. Also described are antibodies comprising a heavy chain and a light chain from a heterologous antibody. In one embodiment, the antibodies are co-expressed from a vector containing: a first expression cassette which encodes at least a first open reading frame (ORF) for a first immunoglobulin under the control of regulatory control sequences which direct expression thereof; and a second expression cassette which comprises a second ORF, a linker, and a third ORF under the control of regulatory control sequences which direct expression thereof, wherein the second and third ORF for a second and third immunoglobulin construct. The vector co-expressing these two antibody constructs is in one embodiment an AAV, in which the 5? and 3? ITRs flank the expression cassettes and regulatory sequences.Type: ApplicationFiled: March 17, 2020Publication date: July 9, 2020Inventors: James M. Wilson, Anna Tretiakova
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Patent number: 10647758Abstract: A recombinant adeno-associated virus (AAV) having an AAV capsid and packaged therein a heterologous nucleic acid which expresses two functional antibody constructs in a cell is described. Also described are antibodies comprising a heavy chain and a light chain from a heterologous antibody. In one embodiment, the antibodies are co-expressed from a vector containing: a first expression cassette which encodes at least a first open reading frame (ORF) for a first immunoglobulin under the control of regulatory control sequences which direct expression thereof; and a second expression cassette which comprises a second ORF, a linker, and a third ORF under the control of regulatory control sequences which direct expression thereof, wherein the second and third ORF for a second and third immunoglobulin construct. The vector co-expressing these two antibody constructs is in one embodiment an AAV, in which the 5? and 3? ITRs flank the expression cassettes and regulatory sequences.Type: GrantFiled: July 2, 2019Date of Patent: May 12, 2020Assignee: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIAInventors: James M. Wilson, Anna Tretiakova
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Patent number: 10647998Abstract: Described herein are synonymously altered gene sequences which express protein in differing levels within secretory as compared to non-secretory target tissue. An expression cassette comprising an open reading frame (ORF) for a protein under the control of regulatory sequences which direct expression of the product in cell, which ORF has been modified to preferentially increase expression levels in a selected tissue, wherein the modified ORF is characterized by a triplet frequency of any one of Tables 3-12, 16 or 17.Type: GrantFiled: June 20, 2017Date of Patent: May 12, 2020Assignee: The Trustees of the University of PennsylvaniaInventors: James M. Wilson, Anna Tretiakova
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Publication number: 20190322725Abstract: A recombinant adeno-associated virus (AAV) having an AAV capsid and packaged therein a heterologous nucleic acid which expresses two functional antibody constructs in a cell is described. Also described are antibodies comprising a heavy chain and a light chain from a heterologous antibody. In one embodiment, the antibodies are co-expressed from a vector containing: a first expression cassette which encodes at least a first open reading frame (ORF) for a first immunoglobulin under the control of regulatory control sequences which direct expression thereof; and a second expression cassette which comprises a second ORF, a linker, and a third ORF under the control of regulatory control sequences which direct expression thereof, wherein the second and third ORF for a second and third immunoglobulin construct. The vector co-expressing these two antibody constructs is in one embodiment an AAV, in which the 5? and 3? ITRs flank the expression cassettes and regulatory sequences.Type: ApplicationFiled: July 2, 2019Publication date: October 24, 2019Inventors: James M. Wilson, Anna Tretiakova
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Patent number: 10385119Abstract: A recombinant adeno-associated virus (AAV) having an AAV capsid and packaged therein a heterologous nucleic acid which expresses two functional antibody constructs in a cell is described. Also described are antibodies comprising a heavy chain and a light chain from a heterologous antibody. In one embodiment, the antibodies are co-expressed from a vector containing: a first expression cassette which encodes at least a first open reading frame (ORF) for a first immunoglobulin under the control of regulatory control sequences which direct expression thereof; and a second expression cassette which comprises a second ORF, a linker, and a third ORF under the control of regulatory control sequences which direct expression thereof, wherein the second and third ORF for a second and third immunoglobulin construct. The vector co-expressing these two antibody constructs is in one embodiment an AAV, in which the 5? and 3? ITRs flank the expression cassettes and regulatory sequences.Type: GrantFiled: October 15, 2018Date of Patent: August 20, 2019Assignee: Trustees of the University of PennsylvaniaInventors: James M. Wilson, Anna Tretiakova
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Publication number: 20190031740Abstract: A recombinant adeno-associated virus (AAV) having an AAV capsid and packaged therein a heterologous nucleic acid which expresses two functional antibody constructs in a cell is described. Also described are antibodies comprising a heavy chain and a light chain from a heterologous antibody. In one embodiment, the antibodies are co-expressed from a vector containing: a first expression cassette which encodes at least a first open reading frame (ORF) for a first immunoglobulin under the control of regulatory control sequences which direct expression thereof; and a second expression cassette which comprises a second ORF, a linker, and a third ORF under the control of regulatory control sequences which direct expression thereof, wherein the second and third ORF for a second and third immunoglobulin construct. The vector co-expressing these two antibody constructs is in one embodiment an AAV, in which the 5? and 3? ITRs flank the expression cassettes and regulatory sequences.Type: ApplicationFiled: October 15, 2018Publication date: January 31, 2019Inventors: James M. Wilson, Anna Tretiakova
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Patent number: 10138295Abstract: A recombinant adeno-associated virus (AAV) having an AAV capsid and packaged therein a heterologous nucleic acid which expresses two functional antibody constructs in a cell is described. Also described are antibodies comprising a heavy chain and a light chain from a heterologous antibody. In one embodiment, the antibodies are co-expressed from a vector containing: a first expression cassette which encodes at least a first open reading frame (ORF) for a first immunoglobulin under the control of regulatory control sequences which direct expression thereof; and a second expression cassette which comprises a second ORF, a linker, and a third ORF under the control of regulatory control sequences which direct expression thereof, wherein the second and third ORF encode for a second and third immunoglobulin construct. The vector co-expressing these two antibody constructs is in one embodiment an AAV, in which the 5? and 3? ITRs flank the expression cassettes and regulatory sequences.Type: GrantFiled: May 13, 2015Date of Patent: November 27, 2018Assignee: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIAInventors: James M. Wilson, Anna Tretiakova
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Publication number: 20170292132Abstract: Described herein are synonymously altered gene sequences which express protein in differing levels within secretory as compared to non-secretory target tissue. An expression cassette comprising an open reading frame (ORF) for a protein under the control of regulatory sequences which direct expression of the product in cell, which ORF has been modified to preferentially increase expression levels in a selected tissue, wherein the modified ORF is characterized by a triplet frequency of any one of Tables 3-12, 16 or 17.Type: ApplicationFiled: June 20, 2017Publication date: October 12, 2017Inventors: James M. Wilson, Anna Tretiakova
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Patent number: 9719106Abstract: Described herein are synonymously altered gene sequences which express protein in differing levels within secretory as compared to non-secretory target tissue. An expression cassette comprising an open reading frame (ORF) for a protein under the control of regulatory sequences which direct expression of the product in cell, which ORF has been modified to preferentially increase expression levels in a selected tissue, wherein the modified ORF is characterized by a triplet frequency of any one of Tables 3-12, 16 or 17.Type: GrantFiled: April 29, 2014Date of Patent: August 1, 2017Assignee: The Trustees of the University of PennsylvaniaInventors: James M. Wilson, Anna Tretiakova
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Publication number: 20170081392Abstract: A recombinant adeno-associated virus (AAV) having an AAV capsid and packaged therein a heterologous nucleic acid which expresses two functional antibody constructs in a cell is described. Also described are antibodies comprising a heavy chain and alight chain from a heterologous antibody. In one embodiment, the antibodies are co-expressed from a vector containing: a first expression cassette which encodes at least a first open reading frame (ORF) for a first immunoglobulin under the control of regulatory control sequences which direct expression thereof; and a second expression cassette which comprises a second ORF, a linker, and a third ORF under the control of regulatory control sequences which direct expression thereof, wherein the second and third ORF encode a second and third immunoglobulin construct. The vector co-expressing these two antibody constructs is in one embodiment an AAV, in which the 5? and 3? ITRs flank the expression cassettes and regulatory sequences.Type: ApplicationFiled: May 13, 2015Publication date: March 23, 2017Inventors: James M. Wilson, Anna Tretiakova
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Publication number: 20160083748Abstract: Described herein are synonymously altered gene sequences which express protein in differing levels within secretory as compared to non-secretory target tissue. An expression cassette comprising an open reading frame (ORF) for a protein under the control of regulatory sequences which direct expression of the product in cell, which ORF has been modified to preferentially increase expression levels in a selected tissue, wherein the modified ORF is characterized by a triplet frequency of any one of Tables 3-12, 16 or 17.Type: ApplicationFiled: April 29, 2014Publication date: March 24, 2016Inventors: James M. Wilson, Anna Tretiakova