公开(公告)号：US20240350583A1

公开(公告)日：2024-10-24

申请号：US18294934

申请日：2022-08-02

申请人： Chung Ang University Industry Academic Cooperation Foundation ,  Haplnscience Inc.

发明人： Dae Kyong Kim ,  Yong Soon Kim ,  Moon Jung Back ,  Jung Hoon Pyo ,  David Kim ,  Yong Wei Piao ,  Min A Yeom

IPC分类号： A61K38/17 ,  A61K48/00 ,  A61P11/00 ,  A61P13/12 ,  C12N15/85

CPC分类号： A61K38/1709 ,  A61K48/005 ,  A61P11/00 ,  A61P13/12 ,  C12N15/85

摘要： Disclosed in the present invention are: a pharmaceutical composition for preventing or treating fibrotic diseases, comprising hyaluronan and proteoglycan link protein 1 (HAPLN1) or a gene encoding same as an active ingredient; and a method for preventing or treating fibrotic diseases using the composition. According to the present invention, fibrosis of cells or tissues is prevented and inhibited such that the occurrence or progression of various kinds of diseases caused by fibrosis is fundamentally suppressed, and thus the diseases can be prevented or treated.

公开(公告)号：US12123030B2

公开(公告)日：2024-10-22

申请号：US16302368

申请日：2017-05-18

申请人： ModernaTX, Inc.

发明人： Kerry Benenato ,  Stephen Hoge ,  Paolo Martini ,  Iain McFadyen ,  Vladimir Presnyak ,  Ellalahewage Sathyajith Kumarasinghe

IPC分类号： A61K48/00 ,  A61K9/127 ,  A61K9/51 ,  A61K47/14 ,  C12N9/20 ,  B82Y5/00 ,  B82Y30/00

CPC分类号： C12N9/20 ,  A61K9/1274 ,  A61K9/5123 ,  A61K47/14 ,  B82Y5/00 ,  B82Y30/00 ,  C12Y301/01034

摘要： The invention relates to mRNA therapy for the treatment of hyperlipidemia. mRNAs for use in the invention, when administered in vivo, encode human lipoprotein lipase (LPL), isoforms thereof, functional fragments thereof, and fusion proteins comprising LPL. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto, mRNA therapies of the invention increase and/or restore deficient levels of LPL expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of triglycerides associated with deficient LPL activity in subjects.

公开(公告)号：US12122844B2

公开(公告)日：2024-10-22

申请号：US16927675

申请日：2020-07-13

申请人： Cornell University ,  Memorial Sloan-Kettering Cancer Center

发明人： Ronald G. Crystal ,  Stephen M. Kaminsky ,  Martin J. Hicks ,  Viviane Tabar

IPC分类号： C07K16/30 ,  A61K39/00 ,  A61K48/00 ,  C07K16/22 ,  C07K16/24 ,  C07K16/28

CPC分类号： C07K16/3053 ,  A61K48/005 ,  C07K16/22 ,  C07K16/24 ,  C07K16/2863 ,  A61K2039/505 ,  A61K48/00 ,  C07K2317/21 ,  C07K2317/24 ,  C12N2750/14141 ,  C12N2799/025

摘要： The invention provides methods to inhibit or treat brain cancers by locally inhibiting expression or activity of growth factors or growth factor receptors.

公开(公告)号：US12121743B2

公开(公告)日：2024-10-22

申请号：US16605360

申请日：2018-04-02

申请人： Albert Einstein College of Medicine

发明人： Young-Hwan Jo ,  Jae Hoon Jeong ,  Streamson Chua, Jr.

IPC分类号： A61K48/00 ,  A61K38/17 ,  A61N5/06 ,  C12N7/00 ,  C12N15/63 ,  C12N15/86 ,  A01K67/00

CPC分类号： A61N5/062 ,  A61K38/177 ,  A61K48/0058 ,  A61N5/0622 ,  A61N5/0625 ,  C12N7/00 ,  C12N15/86 ,  A61N2005/0645 ,  A61N2005/0651 ,  A61N2005/0663 ,  C12N2750/14143 ,  C12N2750/14171

摘要： Methods and devices are disclosed for non-invasive optogenetic stimulation of autonomic efferent fibers to regulate glucose metabolism in the liver and brown adipose tissue and to treat metabolic disorders.

公开(公告)号：US12121595B2

公开(公告)日：2024-10-22

申请号：US17203232

申请日：2021-03-16

申请人： RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL

发明人： Paul Taylor Martin

IPC分类号： C12N15/86 ,  A61K38/45 ,  A61K48/00 ,  A61P21/00 ,  C12N9/10 ,  C12Q1/68 ,  C07H21/04 ,  C12N15/864

CPC分类号： A61K48/0058 ,  A61K38/45 ,  A61K48/0075 ,  A61P21/00 ,  C12N9/1048 ,  C12N9/1051 ,  C12N15/86 ,  C12Q1/68 ,  C12Y204/01165 ,  C07H21/04 ,  C12N15/8645 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/42

摘要： The present disclosure relates to recombinant adeno-associated virus (rAAV) delivery of a GALGT2 polynucleotide. The disclosure provides rAAV and methods of using the rAAV for GALGT2 gene therapy of neuromuscular disorders. Exemplary neuromuscular disorders include, but are not limited to, muscular dystrophies such as Duchenne muscular dystrophy, Congenital Muscular Dystrophy 1A and Limb Girdle Muscular Dystrophy 2D.

公开(公告)号：US12121594B2

公开(公告)日：2024-10-22

申请号：US17820243

申请日：2022-08-16

申请人： International Centre for Genetic Engineering and Biotechnology (ICGEB)

发明人： Mauro Giacca ,  Giulia Ruozi ,  Francesca Bortolotti

IPC分类号： A61K48/00 ,  A61K38/17 ,  C07K14/47

CPC分类号： A61K48/005 ,  A61K38/1709 ,  C07K14/47 ,  A01K2207/20 ,  A01K2217/052 ,  A01K2227/105 ,  A01K2267/0393 ,  C12N2750/14143

摘要： A protein selected from the group consisting of Chrdl1, Fam3c, Fam3b and a fragment thereof, or a polynucleotide encoding therefor, for use in treating or reducing the risk of heart disease.

公开(公告)号：US12121592B2

公开(公告)日：2024-10-22

申请号：US17832196

申请日：2022-06-03

申请人： Translate Bio, Inc.

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

IPC分类号： A61K48/00 ,  A61K9/00 ,  A61K9/127 ,  A61K9/51 ,  A61K31/713 ,  A61K38/18 ,  A61K38/47 ,  A61K38/48 ,  A61K38/57 ,  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：US20240343779A1

公开(公告)日：2024-10-17

申请号：US18294626

申请日：2022-08-04

申请人： ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES ,  UNIVERSITÉ PARIS-EST CRÉTEIL (UPEC) ,  INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)

发明人： Valentina TAGLIETTI ,  Frédéric RELAIX

IPC分类号： C07K14/72 ,  A61K31/352 ,  A61K48/00 ,  C12N15/86 ,  C12N15/88

CPC分类号： C07K14/723 ,  A61K31/352 ,  C12N15/86 ,  C12N15/88 ,  A61K48/00 ,  C12N2750/14143

摘要： An activator for preserving and regenerating muscle structure and function activates the thyroid-stimulating hormone receptor (TSHR) signaling pathway in muscle stem cells (satellite cells), thereby blocking senescence. A medical product includes a plurality of activators, such as a genetic activator, and an adenoviral vector comprising a thyroid-stimulating hormone receptor (TSHR) protein sequence of SEQ ID NO: 1, or a TSHR nucleotide sequence of SEQ ID NO: 2.

公开(公告)号：US20240342313A1

公开(公告)日：2024-10-17

申请号：US18622513

申请日：2024-03-29

申请人： Pharma Cinq, LLC

发明人： Tianci LUO ,  Daniel LIPUT ,  Fong Qi LIANG

IPC分类号： A61K48/00 ,  A61P27/02 ,  C07K14/47 ,  C12N9/02 ,  C12N15/86

CPC分类号： A61K48/0058 ,  A61K48/0083 ,  A61P27/02 ,  C07K14/47 ,  C12N9/0051 ,  C12N15/86 ,  C12Y108/01008 ,  C07K2319/02 ,  C07K2319/30 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14171

摘要： The present invention relates to nucleic acids coding for and capable of expressing a full-length rod-derived cone viability factor (RdCVF) and human IgK signal sequence and viral vectors containing these nucleic acids. The invention also relates to compositions and pharmaceutical preparations comprising these nucleic acids or vectors, methods of producing or secreting a full-length RdCVF and human IgK signal sequence, and methods of treatment.

公开(公告)号：US20240342309A1

公开(公告)日：2024-10-17

申请号：US18433053

申请日：2024-02-05

申请人： Janssen Pharmaceutica NV

发明人： Krista Spiller ,  Geena Skariah ,  Jonathan Haldeman

IPC分类号： A61K48/00 ,  A61K38/45 ,  A61P25/16 ,  C12N15/86

CPC分类号： A61K48/005 ,  A61K38/45 ,  A61P25/16 ,  C12N15/86 ,  C12Y207/11001 ,  C12N2750/14143

摘要： Disclosed herein are recombinant gene therapy vectors comprising a PTEN-induced kinase 1 (PINK1) encoding gene that is operatively linked to a promoter and methods of using the recombinant therapy vectors for inhibiting, reducing, or delaying degeneration or death of neurons of a subject.