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：US20240317814A1

公开(公告)日：2024-09-26

申请号：US18735636

申请日：2024-06-06

申请人： Genethon ,  INSERM (Institut National de la Santé et de la Recherche Médicale) ,  Universite d'Evry Val d'Essonne ,  Sorbonne Universite ,  Association Institut de Myologie

发明人： Isabelle Richard ,  Evelyne Gicquel ,  Federico Mingozzi

IPC分类号： C07K14/005 ,  A61K48/00 ,  A61P25/28 ,  C07K14/075 ,  C12N7/00 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

CPC分类号： C07K14/005 ,  A61P25/28 ,  C07K14/075 ,  C12N7/00 ,  C12N9/22 ,  C12N15/111 ,  C12N15/86 ,  A61K48/00 ,  C12N2310/20 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14152 ,  C12N2750/14171

摘要： The invention relates to a recombinant adeno-associated virus (AAV) capsid protein, which is a hybrid between AAV serotype 9 (AAV9) and AAV serotype 74 (AAVrh74) capsid proteins, wherein said recombinant hybrid AAV capsid protein has a reduced liver tropism compared to the parent AAV9 and AAVrh74 capsid proteins. The invention relates also to the derived hybrid AAV serotype vector particles packaging a gene of interest and their use in gene therapy, in particular for treating neuromuscular genetic diseases.

公开(公告)号：US20240307511A1

公开(公告)日：2024-09-19

申请号：US18538866

申请日：2023-12-13

申请人： The Trustees of the University of Pennsylvania

发明人： Christian Hinderer ,  James M. Wilson

IPC分类号： A61K38/47 ,  A61K9/00 ,  A61K31/343 ,  A61K31/439 ,  A61K35/76 ,  A61K45/06 ,  A61K48/00 ,  A61P3/00 ,  C12N7/00 ,  C12N9/24 ,  C12N15/86

CPC分类号： A61K38/47 ,  A61K9/0019 ,  A61K31/343 ,  A61K31/439 ,  A61K35/76 ,  A61K45/06 ,  A61P3/00 ,  C12N7/00 ,  C12N9/2402 ,  C12N15/86 ,  C12Y302/01076 ,  A61K48/00 ,  A61K48/0083 ,  C12N2750/14121 ,  C12N2750/14132 ,  C12N2750/14143

摘要： A co-therapeutic regimen comprising AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDUA gene and two or more immunosuppressants is provided herein. Also provided are methods useful for treating hIDUA deficiency (MPSI) and the symptoms associated with Hurler, Hurler-Scheie and Scheie syndromes.

公开(公告)号：US12064460B2

公开(公告)日：2024-08-20

申请号：US16964142

申请日：2019-01-25

申请人： TOKUSHIMA UNIVERSITY ,  JICHI MEDICAL UNIVERSITY ,  GENE THERAPY RESEARCH INSTITUTION CO., LTD.

发明人： Kohji Itoh ,  Daisuke Tsuji ,  Shinichi Muramatsu ,  Katsuhito Asai

IPC分类号： A61K35/76 ,  A61P25/28 ,  C12N7/00

CPC分类号： A61K35/76 ,  A61P25/28 ,  C12N7/00 ,  C12N2750/14121 ,  C12N2750/14143 ,  C12N2750/14171

摘要： According to a conventional method for treatment of Sandhoff disease and Tay-Sachs disease comprising administering a modified β-subunit to a patient in the form of a protein, it is necessary that administration be performed frequently. This invention relates to a recombinant adeno-associated virus virion comprising: capsomere comprising a protein capable of forming a virus virion; and a polynucleotide packaged in the capsomere comprising a promoter sequence and nucleotide sequences operably linked to the promoter sequence encoding a first amino acid sequence derived from the amino acid sequence of the β-subunit of wild-type human β-hexosaminidase composed of amino acids 55 to 556 in the sequence as shown in SEQ ID NO: 28 by substitution of amino acids 312 to 318 with glycine, serine, glutamic acid, proline, serine, glycine, and threonine in that order and a second amino acid sequence, which is an amino acid sequence of a signal peptide linked to the N terminus of the first amino acid.

公开(公告)号：US20240150410A1

公开(公告)日：2024-05-09

申请号：US18284408

申请日：2022-04-13

申请人： Capsida, Inc.

发明人： Nicholas C. Flytzanis ,  Nicholas S. Goeden ,  Troy E. Sandberg ,  Brandon G. Wheeler

IPC分类号： C07K14/005 ,  A61K38/00 ,  C12N7/00 ,  C12N15/86

CPC分类号： C07K14/005 ,  C12N7/00 ,  C12N15/86 ,  A61K38/00 ,  C12N2750/14121 ,  C12N2750/14132 ,  C12N2750/14143 ,  C12N2750/14151

摘要： Described herein are compositions and kits comprising recombinant adeno-associated viruses (rAAVs) with increased viral transduction in the CNS. The rAAV compositions described herein encapsidate a transgene, such as a therapeutic nucleic acid. Gene therapy using the rAAVs is described. Also described are methods of treating CNS-related diseases and conditions.

公开(公告)号：US20240117322A1

公开(公告)日：2024-04-11

申请号：US18484856

申请日：2023-10-11

申请人： The Trustees of the University of Pennsylvania

发明人： James M. Wilson ,  Qiang Wang ,  April Tepe ,  Kevin Turner

IPC分类号： C12N7/00 ,  A61K35/761 ,  C07K14/005 ,  C07K16/32

CPC分类号： C12N7/00 ,  A61K35/761 ,  C07K14/005 ,  C07K16/32 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14143

摘要： A recombinant adeno-associated virus (rAAV) vector comprising an AAVhu68 capsid produced in a production system comprising a nucleotide sequence of SEQ ID NO: 1, or a sequence at least 75% identical thereto which encodes SEQ ID NO:2. The AAVhu68 capsid comprises subpopulations of highly deamidated asparagine residues in asparagine-glycine pairs in the amino acid sequence of SEQ ID NO: 2. Also provided are compositions containing the rAAV and uses thereof. Additionally, rAAV having an engineered AAV capsid comprising at least one subpopulation of vp1 or vp2 proteins having a Val at amino acid position 157 with reference to the AAVhu68 vp1 numbering are provided.

公开(公告)号：US20240101969A9

公开(公告)日：2024-03-28

申请号：US17503972

申请日：2021-10-18

申请人： California Institute of Technology

发明人： Viviana Gradinaru ,  Nicholas C. Flytzanis ,  Nicholas S. Goeden

IPC分类号： C12N7/00

CPC分类号： C12N7/00 ,  C12N2750/14071 ,  C12N2750/14121 ,  C12N2750/14122

摘要： Described herein are compositions and kits comprising recombinant adeno-associated viruses (rAAVs) with tropisms showing increased specificity and efficiency of viral transduction in targeted cell types such as the brain and lung. The rAAV compositions described herein also have tropisms showing decreased specificity and decreased efficiency of viral transduction in an off-target cell type such as the liver. The rAAV compositions described herein encapsidate a transgene, such as a therapeutic nucleic acid. Upon systemic delivery to a subject, the rAAV is capable of increased specificity and increased transduction of the transgene in a target cell-type, as compared to a parental or reference AAV.

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：US11821009B2

公开(公告)日：2023-11-21

申请号：US16411906

申请日：2019-05-14

申请人： Cornell University

发明人： Ronald G. Crystal ,  Katie Stiles

IPC分类号： C12N7/00 ,  A61K39/23 ,  A61K35/761

CPC分类号： C12N7/00 ,  A61K35/761 ,  C12N2750/14121 ,  C12N2750/14132 ,  C12N2750/14141

摘要： Modified AAV vectors and uses thereof are provided.

公开(公告)号：US20230321282A1

公开(公告)日：2023-10-12

申请号：US18066151

申请日：2022-12-14

申请人： The Regents of the University of California ,  The Trustees of the University of Pennsylvania

发明人： David V. Schaffer ,  John G. Flannery ,  William A. Beltran ,  Leah C. Byrne ,  Meike Visel

IPC分类号： A61K9/00 ,  C12N7/00 ,  A61K48/00

CPC分类号： A61K48/0091 ,  A61K9/0048 ,  A61K48/0075 ,  C12N7/00 ,  C12N2750/14121 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145

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 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.