公开(公告)号：US20240358640A1

公开(公告)日：2024-10-31

申请号：US18771481

申请日：2024-07-12

申请人： The Penn State Research Foundation ,  The United States of America, as represented by the Secretary, Department of Health and Human Serv

发明人： Joel P. Schneider ,  Scott H. Medina

IPC分类号： A61K9/127 ,  A61K9/00 ,  A61K47/65 ,  B82Y5/00 ,  C07K7/06

CPC分类号： A61K9/1273 ,  A61K9/0009 ,  A61K47/65 ,  C07K7/06 ,  B82Y5/00 ,  C07K2319/01

摘要： The present invention is directed to peptisomes, including nanopeptisomes, which have a perfluorocarbon liquid core containing a perfluorocarbon liquid and a cargo, such as a therapeutically active agent, dispersed in the perfluorocarbon liquid, and a plurality of amphiphilic peptide molecules surrounding the perfluorocarbon liquid core, wherein the amphiphilic peptide is represented by Formula (I) HB-CL-HP wherein HB is a fluorinated hydrophobic block, such as a fluorinated hydrophobic amino acid sequence, CL is a cross-linking motif, and HP is a hydrophilic amino acid sequence. The present invention is also directed to methods of use of the amphiphilic peptides and peptisomes, such as nanopeptisomes, to deliver a cargo, such as a therapeutically active agent, to a cell, wherein the cell may be in vitro, ex vivo, or in vivo.

公开(公告)号：US20240358634A1

公开(公告)日：2024-10-31

申请号：US18603065

申请日：2024-03-12

申请人： DOMPE FARMACEUTICI S.P.A ,  The Regents of the University of California

发明人： Jay LICHTER ,  Andrew M. TRAMMEL ,  Fabrice PIU ,  Qiang YE ,  Michael Christopher SCAIFE ,  Benedikt VOLLRATH ,  Sergio G. DURON ,  Luis A. DELLAMARY ,  Carl LEBEL ,  Jeffrey P. HARRIS

IPC分类号： A61K9/00 ,  A61K9/06 ,  A61K9/127 ,  A61K9/14 ,  A61K9/16 ,  A61K31/05 ,  A61K31/13 ,  A61K31/137 ,  A61K31/325 ,  A61K31/43 ,  A61K31/436 ,  A61K31/519 ,  A61K31/5513 ,  A61K31/5517 ,  A61K38/18 ,  A61K39/00 ,  A61K47/14 ,  A61K47/18 ,  A61K47/32 ,  A61K47/38 ,  C07K16/24

CPC分类号： A61K9/0046 ,  A61K9/06 ,  A61K9/127 ,  A61K9/14 ,  A61K9/16 ,  A61K31/05 ,  A61K31/13 ,  A61K31/137 ,  A61K31/325 ,  A61K31/43 ,  A61K31/436 ,  A61K31/519 ,  A61K31/5513 ,  A61K31/5517 ,  A61K38/18 ,  A61K38/1808 ,  A61K38/1825 ,  A61K38/185 ,  A61K38/1858 ,  A61K38/1883 ,  A61K47/14 ,  A61K47/18 ,  A61K47/32 ,  A61K47/38 ,  C07K16/241 ,  A61K2039/505 ,  C07K2317/21 ,  C07K2317/76

摘要： Disclosed herein are compositions and methods for the treatment of otic disorders with immunomodulating agents and auris pressure modulators. In these methods, the auris compositions and formulations are administered locally to an individual afflicted with an otic disorder, through direct application of the immunomodulating and/or auris pressure modulating compositions and formulations onto the auris media and/or Auris interna target areas, or via perfusion into the auris media and/or Auris interna structures.

公开(公告)号：US20240342307A1

公开(公告)日：2024-10-17

申请号：US18431597

申请日：2024-02-02

申请人： Translate Bio, Inc.

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

IPC分类号： A61K48/00 ,  A61K9/00 ,  A61K9/127 ,  A61K31/7105 ,  C07J43/00 ,  C12N15/67

CPC分类号： A61K48/0033 ,  A61K9/0019 ,  A61K9/1271 ,  A61K9/1272 ,  A61K31/7105 ,  A61K48/0008 ,  A61K48/005 ,  C07J43/003 ,  C12N15/67

摘要： Disclosed herein are compositions and methods of modulating the expression of gene or the production of a protein by transfecting target cells with nucleic acids. The compositions disclosed herein demonstrate a high transfection efficacy and are capable of ameliorating diseases associated with protein or enzyme deficiencies.

公开(公告)号：US20240342089A1

公开(公告)日：2024-10-17

申请号：US18600544

申请日：2024-03-08

申请人： Chung Shan Medical University

发明人： YUAN-YEN CHANG ,  HUI-WEN LIN ,  YI-FENG KAO

IPC分类号： A61K9/127 ,  A61K31/12 ,  A61K31/352

CPC分类号： A61K9/1277 ,  A61K31/12 ,  A61K31/352

摘要： An aquatic liposome encapsulating a natural compound is provided, wherein an average particle size (a median particle size) of the aquatic liposome encapsulating the natural compound ranges from 80 nm to 200 nm. A manufacturing method of an aquatic liposome encapsulating a natural compound is provided and includes performing an ultrasonic oscillation after mixing the aquatic liposome and the natural compound, so that the natural compound is encapsulated in the aquatic liposome. Experiments are conducted to prove that the aquatic liposome encapsulating the natural compound could effectively enter microglia and retinal pigment epithelium cells to relieve the inflammatory response and hinder the apoptosis.

公开(公告)号：US12115138B2

公开(公告)日：2024-10-15

申请号：US16811759

申请日：2020-03-06

申请人： RENIBUS THERAPEUTICS, INC.

发明人： Donald Jeffrey Keyser ,  Alvaro F. Guillem ,  Bhupinder Singh ,  Richard A. Zager

IPC分类号： A61K31/12 ,  A61K9/127 ,  A61K31/145 ,  A61K31/16 ,  A61K31/355 ,  A61K31/375 ,  A61K31/385 ,  A61K31/7048 ,  A61K47/10 ,  A61K47/24 ,  A61K47/34

CPC分类号： A61K31/12 ,  A61K9/127 ,  A61K31/145 ,  A61K31/16 ,  A61K31/355 ,  A61K31/375 ,  A61K31/385 ,  A61K31/7048 ,  A61K47/10 ,  A61K47/24 ,  A61K47/34

摘要： The present invention relates to novel tetrahydrocurcumin (THCu) compositions, novel methods of manufacturing, and methods of using these compositions for therapeutic applications. The novel synthetic pathway(s) result in THCu compositions that generally lack hexahydrocurcumin (HHC), and include an improved impurity profile with reduced additional species that are generally present in hydrogenated curcumin compositions.

公开(公告)号：US20240335383A1

公开(公告)日：2024-10-10

申请号：US18700066

申请日：2022-10-11

申请人： NewImmune II, LLC

发明人： Brian HORSBURGH ,  Mark David MOODY

IPC分类号： A61K9/127 ,  A61K31/4745 ,  A61K38/20 ,  A61K47/10 ,  A61K47/24 ,  A61K47/28 ,  A61K47/69 ,  A61P35/00

CPC分类号： A61K9/1271 ,  A61K31/4745 ,  A61K38/2013 ,  A61K47/10 ,  A61K47/24 ,  A61K47/28 ,  A61K47/6951 ,  A61P35/00

摘要： The present disclosure relates to nanoparticles and nanoparticulate compositions; methods of preparing such nanoparticles and nanoparticulate compositions; and associated methods of medical treatment and uses of such nanoparticles and nanoparticulate compositions for medical treatment, including the use of such nanoparticles for the manufacture of medicaments for medical treatment.

公开(公告)号：US12109309B2

公开(公告)日：2024-10-08

申请号：US16322014

申请日：2017-07-28

申请人： Academia Sinica

发明人： Che-Ming Jack Hu ,  Hui-Wen Chen ,  Yuan-I Chen ,  Chen-Ying Chien ,  Jung-Chen Lin

IPC分类号： A61K9/127 ,  C12N5/078 ,  G01N33/50 ,  G01N33/543 ,  G01N33/556

CPC分类号： A61K9/127 ,  A61K9/1275 ,  A61K9/1277 ,  C12N5/0634 ,  G01N33/5008 ,  G01N33/5432 ,  G01N33/556 ,  G01N2500/02 ,  G01N2500/10

摘要： A method of generating an internally fixed lipid vesicle, comprising: providing a precursor lipid vesicle that contains an aqueous interior enclosed by a lipid membrane, wherein the lipid membrane of the precursor lipid vesicle is non-permeable to a crosslinker; permeabilizing the lipid membrane transiently to generate a permeable vesicle; contacting the permeable vesicle with an inactive activatable crosslinker, whereby the inactive activatable crosslinker enters the permeable vesicle; allowing the permeable vesicle to return to a non-permeable vesicle; removing any extravesicular crosslinker; and activating the inactive activatable crosslinker to allow crosslinking to occur inside the non-permeable vesicle, whereby an internally fixed lipid vesicle is generated.

公开(公告)号：US20240325521A1

公开(公告)日：2024-10-03

申请号：US18576244

申请日：2022-05-10

申请人： JENKEM TECHNOLOGY CO., LTD.(TIANJIN) ,  CANSINO (SHANGHAI) BIOLOGICAL RESEARCH CO., LTD.

发明人： Jing HAO ,  Qingbin WANG ,  Shengyong YAN ,  Haomeng WANG ,  Zhihong YAN ,  Jian LIU ,  Xuefeng YU ,  Dongxu Qiu ,  Meina LIN ,  Jun GUO ,  Yanli XIONG ,  Tao ZHU ,  Xuan ZHAO

IPC分类号： A61K39/215 ,  A61K9/127 ,  A61K39/00 ,  C07C215/14 ,  C07C229/12 ,  C07C233/36

CPC分类号： A61K39/215 ,  A61K9/1272 ,  C07C215/14 ,  C07C229/12 ,  C07C233/36 ,  A61K2039/53

摘要： Provided are an ionizable lipid compound of formula I for nucleic acid delivery and an LNP composition thereof, which can efficiency and stably deliver a biologically active substance to a target cell or an organ. The mRNA LNP prepared by using the lipid compound as a cationic lipid has better stability and transfection efficiency, and can cause a higher specific antibody response in an experimental animal body.

公开(公告)号：US12103955B2

公开(公告)日：2024-10-01

申请号：US16944014

申请日：2020-07-30

申请人： MODERNATX, INC.

发明人： Barry Ticho ,  Nadege Briancon-Eris ,  Zhinan Xia ,  Athanasios Dousis ,  Seymour de Picciotto ,  Vladimir Presnyak ,  Stephen Hoge ,  Iain Mcfadyen ,  Kerry Benenato ,  Ellalahewage Sathyajith Kumarasinghe

IPC分类号： C12N15/62 ,  A61K31/7105 ,  A61K38/17 ,  A61K47/14 ,  A61K47/69 ,  A61K48/00 ,  A61P9/00 ,  C07K14/64 ,  C07K16/26 ,  C12N15/52 ,  A61K9/00 ,  A61K9/127 ,  A61K38/00

CPC分类号： C07K14/64 ,  A61K31/7105 ,  A61K38/1796 ,  A61K47/6929 ,  A61P9/00 ,  C07K16/26 ,  C12N15/52 ,  C12N15/62 ,  A61K9/0019 ,  A61K9/127 ,  A61K38/00 ,  C07K2319/00 ,  C07K2319/31

摘要： The invention relates to mRNA therapy for the treatment of fibrosis and/or cardiovascular disease. mRNAs for use in the invention, when administered in vivo, encode human relaxin, isoforms thereof, functional fragments thereof, and fusion proteins comprising relaxin. 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 relaxin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient relaxin activity in subjects.

公开(公告)号：US20240316212A1

公开(公告)日：2024-09-26

申请号：US18420112

申请日：2024-01-23

申请人： Verve Therapeutics, Inc.

发明人： Kallanthottathil G. Rajeev ,  Lisa N. Kasiewicz ,  Padma Malyala ,  Souvik Biswas ,  Ellen Rohde ,  Alexandra Chadwick ,  Caroline Reiss ,  Christopher Cheng ,  Andrew Bellinger

IPC分类号： A61K47/69 ,  A61K9/127 ,  A61K9/51 ,  A61K31/711

CPC分类号： A61K47/6929 ,  A61K9/1271 ,  A61K9/1277 ,  A61K9/5123 ,  A61K9/5192 ,  A61K31/711

摘要： Provided herein are compositions, methods of making the same, and methods for targeted delivery of therapeutic agents for modifying expression and function of target genes, e.g. proteins involved in lipid and cholesterol metabolism such as PCSK9.