公开(公告)号：US12129466B2

公开(公告)日：2024-10-29

申请号：US17576245

申请日：2022-01-14

申请人： Ionis Pharmaceuticals, Inc.

发明人： Paymaan Jafar-nejad

IPC分类号： C07H21/04 ,  C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/111 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/323 ,  C12N2310/3341 ,  C12N2310/341

摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A) in a cell or subject, and in certain instances increasing the expression of paternal UBE3A and the amount of UBE3A protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurogenetic disorder. Such symptoms and hallmarks include developmental delays, ataxia, speech impairment, sleep problems, seizures, and EEG abnormalities. Such neurogenetic disorders include Angelman Syndrome.

公开(公告)号：US12016884B2

公开(公告)日：2024-06-25

申请号：US16329448

申请日：2017-08-30

申请人： UNIVERSITY OF SOUTH FLORIDA ,  THE UNITED STATES DEPARTMENT OF VETERANS AFFAIRS

发明人： Denise R. Cooper ,  Lisa Gould ,  Niketa Patel ,  Mack Wu

IPC分类号： A61K35/35 ,  A61K9/00 ,  A61P17/02 ,  C12N5/077 ,  C12N5/0775 ,  C12N15/113

CPC分类号： A61K35/35 ,  A61K9/0014 ,  A61P17/02 ,  C12N5/0653 ,  C12N5/0667 ,  C12N15/113 ,  C12N2310/111 ,  C12N2310/20

摘要： Provided herein are exosomal compositions and exsosomal lncRNA compositions and formulations thereof. Also provided herein are methods of treating a wound in a subject in need thereof that can contain the step of administering an exosomal composition and/or exsosomal lncRNA compositions or formulation thereof to a wound in a subject in need thereof.

公开(公告)号：US20230265429A1

公开(公告)日：2023-08-24

申请号：US18178179

申请日：2023-03-03

申请人： Arrowhead Pharmaceuticals, Inc.

发明人： Xiaokai Li ,  Tao Pei ,  Teng Ai ,  Susan Phan ,  Susan Ramos-Hunter

IPC分类号： C12N15/113 ,  A61P21/00

CPC分类号： C12N15/113 ,  A61P21/00 ,  C12N2310/111

摘要： The present disclosure relates to delivery vehicles that specifically and efficiently direct payloads to skeletal muscle cells in a subject, in vivo. The delivery vehicles disclosed herein include targeting ligands (such as compounds that have affinity for integrins, including alpha-v-beta-6) and pharmacokinetic/pharmacodynamic (PK/PD) modulators, to facilitate the delivery of payloads to cells, including to skeletal muscle cells. Suitable payloads for use in the delivery vehicles disclosed herein include RNAi agents, which can be linked or conjugated to the delivery vehicles, and when delivered in vivo, provide for the inhibition of gene expression in skeletal muscle cells. Pharmaceutical compositions that include the skeletal muscle cell delivery vehicle are also described, as well as methods of use for the treatment of various diseases and disorders where delivery of a therapeutic payload to a skeletal muscle cell is desirable.

公开(公告)号：US11642364B2

公开(公告)日：2023-05-09

申请号：US17736148

申请日：2022-05-04

申请人： Sarepta Therapeutics, Inc.

发明人： Marco A. Passini ,  Gunnar J. Hanson

IPC分类号： A61K31/712 ,  A61K31/7125 ,  A61P21/00 ,  C12N15/113 ,  A61K47/64

CPC分类号： A61K31/712 ,  A61K31/7125 ,  A61K47/645 ,  A61P21/00 ,  C12N15/113 ,  C12N2310/111

摘要： Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 51 skipping are described.

公开(公告)号：US20190192576A1

公开(公告)日：2019-06-27

申请号：US16329448

申请日：2017-08-30

申请人： UNIVERSITY OF SOUTH FLORIDA ,  THE UNITED STATES DEPARTMENT OF VETERANS AFFAIRS

发明人： DENISE R. COOPER ,  LISA GOULD ,  NIKETA PATEL ,  MACK WU

IPC分类号： A61K35/35 ,  C12N15/113 ,  C12N5/0775 ,  A61P17/02 ,  A61K9/00

CPC分类号： A61K35/35 ,  A61K9/0014 ,  A61P17/02 ,  C12N5/0653 ,  C12N5/0667 ,  C12N15/113 ,  C12N2310/111 ,  C12N2310/20

摘要： Provided herein are exosomal compositions and exsosomal IncRNA compositions and formulations thereof. Also provided herein are methods of treating a wound in a subject in need thereof that can contain the step of administering an exosomal composition and/or exsosomal IncRNA compositions or formulation thereof to a wound in a subject in need thereof.

公开(公告)号：US20180355352A1

公开(公告)日：2018-12-13

申请号：US15660556

申请日：2017-07-26

申请人： Cancer Research Technology Limited

发明人： Magdalena Zernicka-Goetz ,  Florence Wianny ,  Martin John Evans ,  David Moore Glover

IPC分类号： C12N15/113 ,  C12N15/63 ,  C12N15/85 ,  C12N15/11 ,  A01K67/027 ,  A61K38/00

CPC分类号： C12N15/113 ,  A01K67/0275 ,  A01K2217/05 ,  A01K2217/058 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/03 ,  A61K38/00 ,  C12N15/111 ,  C12N15/1137 ,  C12N15/1138 ,  C12N15/63 ,  C12N15/8509 ,  C12N2015/8527 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/53 ,  C12N2330/30 ,  C12N2330/50 ,  C12Y207/11001 ,  C12Y302/01031

摘要： The present invention relates to the specific inhibition of gene expression in mammals by bringing the target gene into contact with double stranded RNA (dsRNA).

公开(公告)号：US20180327786A1

公开(公告)日：2018-11-15

申请号：US15844530

申请日：2017-12-16

申请人： The Broad Institute Inc. ,  Massachusetts Institute of Technology ,  President and Fellows of Harvard College ,  Rutgers, The State University of New Jersey ,  The United States Of America, As Represented By, The Secretary Department of Health And Human Servi ,  Skolkovo Institute of Science and Technology

发明人： Konstantin Severinov ,  Feng Zhang ,  Yuri I. Wolf ,  Sergey Shmakov ,  Ekaterina Semenova ,  Leonid Minakhin ,  Kira S. Makarova ,  Eugene Koonin ,  Silvana Konermann ,  Julia Joung ,  Jonathan S. Gootenberg ,  Omar O. Abudayyeh ,  Eric S. Lander

IPC分类号： C12N15/90 ,  C12N9/22 ,  C12N15/11 ,  C12N15/10 ,  C12N15/113

CPC分类号： C12N15/907 ,  C12N9/22 ,  C12N15/102 ,  C12N15/111 ,  C12N15/113 ,  C12N15/63 ,  C12N15/8201 ,  C12N15/85 ,  C12N2310/111 ,  C12N2310/20 ,  C12N2800/22

摘要： The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered RNA-targeting systems comprising a novel RNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA.

公开(公告)号：US20180265874A1

公开(公告)日：2018-09-20

申请号：US15518088

申请日：2015-10-09

申请人： GLOBAL BIOPHARMA, INC. ,  TAIPEI MEDICAL UNIVERSITY

发明人： HUIE LEE ,  YU-JU HUANG

IPC分类号： C12N15/113 ,  A61K45/06 ,  A61K31/713 ,  C07K16/28 ,  A61K39/395

CPC分类号： C12N15/1138 ,  A61K31/713 ,  A61K39/39558 ,  A61K45/06 ,  C07K16/2827 ,  C07K2317/21 ,  C07K2317/24 ,  C07K2317/31 ,  C07K2317/76 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/122 ,  C12N2310/14 ,  C12N2310/531

摘要： The invention relates to a method for treating and/or preventing a tumor growth, invasion and/or metastasis by inhibiting an overexpression of PD-L1 in the subject via an autocrine loop. The invention found that PD-L1-modulated p21 and VEGF-C expression via the TGFβ1/SMAD4 pathway is responsible for the cancer growth, invasion and metastasis.

公开(公告)号：US20180230491A1

公开(公告)日：2018-08-16

申请号：US15944562

申请日：2018-04-03

申请人： California Institute of Technology

发明人： Carlos Lois-Caballe ,  David Baltimore ,  Xiao-Feng Qin

IPC分类号： C12N15/86 ,  C12N15/113 ,  C12N7/00

CPC分类号： C12N15/86 ,  A61K38/00 ,  A61K48/00 ,  C12N7/00 ,  C12N15/113 ,  C12N15/1132 ,  C12N15/1138 ,  C12N15/867 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/53 ,  C12N2310/531 ,  C12N2330/30 ,  C12N2740/15043 ,  C12N2740/16043 ,  C12N2830/003 ,  C12N2830/006 ,  C12N2830/008 ,  C12N2830/30 ,  C12N2830/48 ,  C12N2830/60 ,  C12N2830/85 ,  C12N2840/20 ,  C12N2840/203 ,  C12Q1/6897 ,  Y02A50/465

摘要： The invention provides methods and compositions for the expression of small RNA molecules within a cell using a lentiviral vector. The methods can be used to express doubles stranded RNA complexes. Small interfering RNA (siRNA) can be expressed using the methods of the invention within a cell, which is capable of down regulating the expression of a target gene through RNA interference. A variety of cells can be treated according to the methods of the invention including embryos, embryogenic stem cells, allowing for the generation of transgenic animals or animals constituted partly by the transduced cells that have a specific gene or a group of genes down regulated.

公开(公告)号：US09976144B2

公开(公告)日：2018-05-22

申请号：US15255002

申请日：2016-09-01

申请人： Mubin I. Syed

发明人： Mubin I. Syed

IPC分类号： C12N15/113 ,  A61K9/14 ,  A61K33/42 ,  A61K38/45 ,  A61M25/01 ,  C12N7/00 ,  A61B34/20

CPC分类号： C12N15/1137 ,  A61B34/20 ,  A61B2034/2051 ,  A61B2034/2063 ,  A61K9/14 ,  A61K33/42 ,  A61K38/45 ,  A61M25/01 ,  C12N7/00 ,  C12N15/113 ,  C12N15/1136 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/122 ,  C12N2310/14 ,  C12N2310/531 ,  C12N2320/32 ,  C12Y203/01

摘要： Methods for controlling obesity using minimally-invasive procedures including introducing embolic crystal particles that are naturally occurring and mostly non-toxic salts into the arterial capillaries feeding the sections of the stomach where the appetite inducing hormone, ghrelin, is produced to limit the blood flow to the region reducing appetite; introducing a virus vector or antisense oligonucleotide to inhibit the production of ghrelin and reduce the appetite; and introducing a soluble embolic particle with a virus vector or antisense oligonucleotide which will inhibit the flow of blood initially and then dissolve and release the inhibit vector to the region, generating ghrelin to control the appetite.