公开(公告)号：US20240327468A1

公开(公告)日：2024-10-03

申请号：US18743549

申请日：2024-06-14

申请人： The University of North Carolina at Chapel Hill

发明人： Aravind Asokan ,  Richard Samulski

IPC分类号： C07K14/005 ,  A61K48/00 ,  C07K14/075 ,  C12N7/00 ,  C12N15/86 ,  C12N15/861

CPC分类号： C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  A61K48/00 ,  C07K14/075 ,  C12N15/861 ,  C12N2750/14122 ,  C12N2750/14141 ,  C12N2750/14142 ,  C12N2750/14171

摘要： The present invention provides methods and compositions comprising an adeno-associated virus (AAV) capsid protein, comprising one or more amino acids substitutions, wherein the substitutions introduce a new glycan binding site into the AAV capsid protein.

公开(公告)号：US12104175B2

公开(公告)日：2024-10-01

申请号：US16687426

申请日：2019-11-18

申请人： Encoded Therapeutics, Inc.

发明人： Kartik Ramamoorthi ,  Stephanie Tagliatela ,  Anne Tanenhaus ,  Andrew Young ,  Szu-Ying Chen ,  Chi Zhang ,  Stephanie Martin ,  David Oberkofler

IPC分类号： C12N7/00 ,  A61K48/00 ,  A61P1/16 ,  A61P7/02 ,  C12N15/86

CPC分类号： C12N7/00 ,  A61K48/005 ,  A61P1/16 ,  A61P7/02 ,  C12N15/86 ,  C12N2750/14121 ,  C12N2750/14132 ,  C12N2750/14142 ,  C12N2750/14143

摘要： Provided herein are compositions and methods for driving high expression of a transgene. Compositions and methods for driving high expression of a transgene comprising one or more human-derived regulatory elements, which, when operably linked to a transgene, can result in high expression of the transgene in one or more cell types or tissues.

公开(公告)号：US20240067987A1

公开(公告)日：2024-02-29

申请号：US18468142

申请日：2023-09-15

申请人： 4D MOLECULAR THERAPEUTICS INC.

发明人： Melissa Kotterman ,  Peter Francis ,  Melissa Calton ,  Johnny Gonzales ,  Roxanne Croze ,  Christopher Schmitt

IPC分类号： C12N15/86 ,  A61K9/00 ,  A61K9/12 ,  A61P11/00 ,  C07K14/005 ,  C07K14/47

CPC分类号： C12N15/86 ,  A61K9/0078 ,  A61K9/12 ,  A61P11/00 ,  C07K14/005 ,  C07K14/4712 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14141 ,  C12N2750/14142 ,  C12N2750/14143 ,  C12N2750/14151

摘要： The present disclosure provides a variant AAV capsid protein that confers tropism to lung cells and recombinant adeno-associated viruses comprising the variant A.AV and pharmaceutical compositions comprising same and their use in the delivery of heterologous nucleic acids to lung cells for the treatment of pulmonary disorders.

公开(公告)号：US11866480B2

公开(公告)日：2024-01-09

申请号：US16318070

申请日：2017-07-26

申请人： The University of North Carolina at Chapel Hill ,  North Carolina State University

发明人： Matthew Louis Hirsch ,  Brian Christopher Gilger

IPC分类号： C07K14/705 ,  C07K14/74 ,  A61P27/02 ,  A61P37/06 ,  A61K9/00 ,  C12N15/86

CPC分类号： C07K14/70539 ,  A61K9/0048 ,  A61P27/02 ,  A61P37/06 ,  C12N15/86 ,  C12N2750/14142 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2830/50

摘要： This invention relates to vectors for delivery of human leukocyte antigen G to the eye and/or to cornea explants and methods of using the same for treatment and/or prevention of corneal transplant rejection and other disorders associated with an immune response and/or vascularization.

公开(公告)号：US20230346985A1

公开(公告)日：2023-11-02

申请号：US18313158

申请日：2023-05-05

申请人： The Children's Medical Center Corporation

发明人： Zhigang HE ,  Xue YAO ,  Zicong ZHANG ,  Benedikt BROMMER

IPC分类号： C12N15/86 ,  A61K49/00

CPC分类号： A61K49/0097 ,  C12N15/86 ,  C12N2750/14123 ,  C12N2750/14132 ,  C12N2750/14142 ,  C12N2750/14143

摘要： The present invention features adeno-associated virus (AAV) vectors, compositions thereof, and methods of use thereof for transducing neurons with injured axons.

公开(公告)号：US20230340532A1

公开(公告)日：2023-10-26

申请号：US18061000

申请日：2022-12-02

申请人： The Trustees of the University of Pennsylvania

发明人： Luk Vandenberghe ,  Guangping Gao ,  James M. Wilson

IPC分类号： C12N15/86 ,  A61K48/00 ,  C07K14/005 ,  C12N7/00

CPC分类号： C12N15/86 ,  A61K48/0091 ,  C07K14/005 ,  C12N7/00 ,  A61K48/005 ,  C12N2750/14122 ,  C12N2750/14143 ,  A61K48/00 ,  C12N2750/14142 ,  C12N2750/14152

摘要： A method of correcting singletons in a selected AAV sequence in order to increasing the packaging yield, transduction efficiency, and/or gene transfer efficiency of the selected AAV is provided. This method involves altering one or more singletons in the parental AAV capsid to conform the singleton to the amino acid in the corresponding position(s) of the aligned functional AAV capsid sequences.

公开(公告)号：US11781142B2

公开(公告)日：2023-10-10

申请号：US16347461

申请日：2017-11-03

申请人： University of Florida Research Foundation, Incorporated

发明人： Maria Zajac-Kaye ,  Kyungah Maeng

IPC分类号： C12N15/113 ,  C12N15/864 ,  A61P35/00 ,  C12N7/00 ,  A01K67/027

CPC分类号： C12N15/1137 ,  A01K67/0278 ,  A61P35/00 ,  C12N7/00 ,  A01K2217/072 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/0331 ,  C12N2310/11 ,  C12N2310/122 ,  C12N2310/14 ,  C12N2310/335 ,  C12N2310/531 ,  C12N2320/32 ,  C12N2330/51 ,  C12N2750/14142 ,  C12N2750/14143

摘要： Aspects of the disclosure relate to methods and compositions for treating pancreatic cancer (e.g., islet cell tumors). In some aspects, adeno-associated virus (AAV) may be used to deliver an interfering RNA that targets thymidylate synthase (TS).

公开(公告)号：US20190218627A1

公开(公告)日：2019-07-18

申请号：US16230080

申请日：2018-12-21

申请人： The Regents of the University of California

发明人： David V. Schaffer ,  Ryan R. Klimczak ,  James T. Koerber ,  John G. Flannery ,  Deniz Dalkara Mourot ,  Meike Visel ,  Leah C.T. Byrne

IPC分类号： C12Q1/70 ,  C12N15/113 ,  A61K48/00 ,  C07K14/005 ,  C12N15/86 ,  C12N7/00 ,  G01N33/50 ,  A61K9/00 ,  A61K38/17 ,  C12N15/115

CPC分类号： C12Q1/701 ,  A61K9/0019 ,  A61K9/0048 ,  A61K38/1709 ,  A61K48/0008 ,  A61K48/0025 ,  A61K48/0075 ,  C07K14/005 ,  C07K2319/33 ,  C12N7/00 ,  C12N15/113 ,  C12N15/115 ,  C12N15/86 ,  C12N2310/14 ,  C12N2310/16 ,  C12N2320/32 ,  C12N2750/14021 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14142 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14152 ,  C12N2810/40 ,  C12Q1/70 ,  C12Q2600/158 ,  C12Q2600/16 ,  G01N33/5008 ,  G01N33/5058

摘要： The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.

公开(公告)号：US20190202866A1

公开(公告)日：2019-07-04

申请号：US16217284

申请日：2018-12-12

申请人： WILLIAM MARSH RICE UNIVERSITY

发明人： Junghae SUH ,  Nicole THADANI ,  Christopher DEMPSEY

IPC分类号： C07K14/005

CPC分类号： C07K14/005 ,  C07K2319/00 ,  C12N2750/14122 ,  C12N2750/14123 ,  C12N2750/14133 ,  C12N2750/14142

摘要： Provided herein are recombinant viral nanoparticles (VNPs) which comprise truncated viral proteins. The VNPs may be mosaic VNPs which are activatable at desired levels. The VNPs may be used to administer therapeutic agents to target cells.

公开(公告)号：US09775918B2

公开(公告)日：2017-10-03

申请号：US14847528

申请日：2015-09-08

申请人： University of Florida Research Foundation, Inc.

发明人： Li Zhong ,  Sergei Zolotukhin ,  Lakshmanan Govindasamy ,  Mavis Agbandje-McKenna ,  Arun Srivastava

IPC分类号： A61K48/00 ,  C12N15/86 ,  C12N15/864 ,  A61K35/76 ,  C12N7/00

CPC分类号： C07K14/005 ,  A61K35/76 ,  A61K39/0011 ,  A61K48/0008 ,  A61K48/005 ,  A61K48/0091 ,  A61K2039/5158 ,  C12N7/00 ,  C12N15/86 ,  C12N15/8645 ,  C12N2750/14122 ,  C12N2750/14132 ,  C12N2750/14142 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14171 ,  C12N2810/6027

摘要： Disclosed are tyrosine-modified rAAV vectors, as well as infectious virions, compositions, and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and methods for using the disclosed tyrosine-phosphorylated capsid protein mutant rAAV vectors in a variety of diagnostic and therapeutic applications including in vivo and ex vivo gene therapy, and large-scale production of rAAV vectors.