公开(公告)号：US20240315980A1

公开(公告)日：2024-09-26

申请号：US18672495

申请日：2024-05-23

申请人： OMNISPIRANT LIMITED

发明人： Gerard Bernard MCCAULEY

IPC分类号： A61K9/50 ,  A61K9/00 ,  A61K48/00 ,  A61M11/00 ,  A61P11/00

CPC分类号： A61K9/5068 ,  A61K9/0078 ,  A61K48/0091 ,  A61M11/005 ,  A61P11/00

摘要： Vesicles, including exosomes, having a coating of a hydrophilic, neutral polymer such as PEG have an increased ability to form a suspension or colloid compared to uncoated vesicles. This enables the coated vesicles to be used to form aerosol droplets such that a liquid formulation containing vesicles can be used in a nebulizer for inhaled administration thereof. Such coated vesicles are also able to pass through mucus and can deliver their cargo into lung cells. Exosomes from mesenchymal stem cells can deliver additional proteins, miRs, mRNAs and other nucleic acid sequences to lung cells providing a regenerative gene therapy for CF, COPD lung cancer and other lung diseases.

公开(公告)号：US12059478B2

公开(公告)日：2024-08-13

申请号：US16302120

申请日：2017-05-19

申请人： Ospedale San Raffaele S.r.l. ,  Fondazione Telethon ETS

发明人： Anna Christina Kajaste-Rudnitski ,  Francesco Piras

IPC分类号： A61K48/00 ,  A61P7/06 ,  A61P43/00 ,  C07K14/47 ,  C12N15/00 ,  C12N15/86

CPC分类号： A61K48/0058 ,  A61K48/0008 ,  A61K48/0066 ,  A61K48/0091 ,  A61P7/06 ,  A61P43/00 ,  C07K14/4746 ,  C12N15/86 ,  C12N2750/14132 ,  C12N2750/14141

摘要： An inhibitor of p53 activation for use in haematopoietic stem and/or progenitor cell gene therapy, preferably wherein the inhibitor is an inhibitor of p53 phosphorylation, more preferably an inhibitor of p53 Serine 15 phosphorylation.

公开(公告)号：US20240229068A9

公开(公告)日：2024-07-11

申请号：US18366495

申请日：2023-08-07

申请人： NXGEN VECTOR SOLUTIONS, LLC

发明人： Joseph E. Rabinowitz

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

CPC分类号： C12N15/86 ,  A61K48/0058 ,  A61K48/0075 ,  A61K48/0091 ,  C12N7/00 ,  C07H21/04 ,  C12N2750/14141

摘要： 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.

公开(公告)号：US20240156989A1

公开(公告)日：2024-05-16

申请号：US18305863

申请日：2023-04-24

申请人： The Regents of the University of California

发明人： April D. Pyle ,  Courtney S. Young ,  Melissa J. Spencer

IPC分类号： A61K48/00 ,  A61P21/00 ,  C07K14/47 ,  C12N5/074 ,  C12N15/113 ,  C12N15/90

CPC分类号： A61K48/0066 ,  A61K48/0091 ,  A61P21/00 ,  C07K14/4708 ,  C12N5/0696 ,  C12N15/113 ,  C12N15/907 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/0306 ,  C12N15/861 ,  C12N2310/20

摘要： The present disclosure provides a method of modifying a mutant dystrophin gene in the genome of a cell. The present disclosure further provides compositions and kits for modifying a mutant dystrophin gene in the genome of a cell.

公开(公告)号：US20240139344A1

公开(公告)日：2024-05-02

申请号：US18269307

申请日：2021-12-22

申请人： Akouos, Inc.

发明人： Emmanuel John Simons ,  Robert Ng

IPC分类号： A61K48/00 ,  C07K14/705 ,  C12N15/86

CPC分类号： A61K48/0058 ,  A61K48/0075 ,  A61K48/0091 ,  C07K14/705 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14152 ,  C12N2830/008 ,  C12N2830/42 ,  C12N2830/50 ,  C12N2840/445

摘要： The present disclosure provides constructs comprising a coding sequence operably linked to a promoter, wherein the coding sequence encodes a clarin 1 protein. Exemplary constructs include AAV constructs. Also provided are methods of using disclosed constructs for the treatment of hearing loss and/or deafness. Also provided are methods of using disclosed constructs for the treatment of vision loss.

公开(公告)号：US11965165B2

公开(公告)日：2024-04-23

申请号：US18063972

申请日：2022-12-09

申请人： Intellia Therapeutics, Inc.

发明人： Arti Mahendra Prakash Kanjolia ,  Shobu Odate ,  Jessica Lynn Seitzer ,  Reynald Michael Lescarbeau ,  Walter Strapps

IPC分类号： A61P25/28 ,  C12N9/22 ,  C12N15/10 ,  C12N15/113 ,  A61K48/00 ,  C12N15/11

CPC分类号： C12N15/113 ,  A61P25/28 ,  C12N9/22 ,  C12N15/102 ,  A61K48/00 ,  A61K48/0091 ,  C12N15/111 ,  C12N2310/20 ,  C12N2310/321 ,  C12N2800/80

摘要： Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), are provided.

公开(公告)号：US11951184B2

公开(公告)日：2024-04-09

申请号：US17346639

申请日：2021-06-14

申请人： WISCONSIN ALUMNI RESEARCH FOUNDATION

发明人： Matthew Michael Millette ,  Erik Wolfe Dent

IPC分类号： C07H21/04 ,  A61K48/00 ,  A61P25/28

CPC分类号： A61K48/0066 ,  A61K48/0008 ,  A61K48/0058 ,  A61K48/0091 ,  A61P25/28

摘要： An expression cassette for gene silencing and replacement, including, in operable communication, a promoter, an expression attenuator, a nucleotide sequence encoding a gene for a replacement target protein, and an shRNA sequence for knockdown of an endogenous variant of the target protein, wherein the promoter, the expression attenuator, the nucleotide sequence encoding the gene for the replacement target protein, and the shRNA are expressed as a single transcript. Also included are expression vectors and cells. Also included are methods of silencing and replacement of a target gene in a cell in culture by transforming the cells with the expression vectors described herein. Also included are minimal expression cassettes suitable for therapeutic methods.

公开(公告)号：US11951180B2

公开(公告)日：2024-04-09

申请号：US18295152

申请日：2023-04-03

申请人： Translate Bio, Inc.

发明人： Frank DeRosa ,  Braydon Charles Guild ,  Michael Heartlein

IPC分类号： C12Q1/00 ,  A61K9/00 ,  A61K9/127 ,  A61K9/51 ,  A61K31/7105 ,  A61K31/713 ,  A61K38/18 ,  A61K38/47 ,  A61K38/48 ,  A61K38/57 ,  A61K48/00 ,  C07K14/505 ,  C07K14/81 ,  C12N9/40 ,  C12N9/64 ,  C12N15/52 ,  C12N15/85

CPC分类号： A61K48/0008 ,  A61K9/0019 ,  A61K9/0073 ,  A61K9/1271 ,  A61K9/1272 ,  A61K9/5123 ,  A61K31/713 ,  A61K38/1816 ,  A61K38/47 ,  A61K38/4846 ,  A61K38/57 ,  A61K48/0075 ,  A61K48/0091 ,  C07K14/505 ,  C07K14/8125 ,  C12N9/2465 ,  C12N9/644 ,  C12N15/52 ,  C12N15/85 ,  C12Y302/01022 ,  C12Y304/21022 ,  A61K48/00

摘要： Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.

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：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.