公开(公告)号：US20180280497A1

公开(公告)日：2018-10-04

申请号：US15758505

申请日：2016-09-09

申请人： The United States of America, as represented by the Secretary, Department of Health and Human Serv ,  The Chancellor, Masters and Scholars of the University of Oxford

发明人： Robert SEDER ,  Geoffrey LYNN ,  Leonard SEYMOUR

IPC分类号： A61K39/39 ,  A61K47/64 ,  A61K48/00 ,  A61K47/59 ,  A61K47/65

CPC分类号： A61K39/39 ,  A61K47/59 ,  A61K47/6455 ,  A61K47/65 ,  A61K48/0041 ,  A61K2039/53 ,  A61K2039/55511 ,  A61K2039/55555 ,  A61K2039/6093

摘要： Embodiments of a novel system for delivering an expression vector encoding an antigen to a subject that allows for spatiotemporal control over stimulation of the subject's immune response to the antigen are provided. In some embodiments, the expression vector delivery system includes a polymer linked to an adjuvant in prodrug form that can form polymer nanoparticles and enter a cell (such as an immune cell) under physiological conditions. In some embodiments, the adjuvant is linked to the polymer by an enzyme degradable labile bond, the cleavage of which activates the adjuvant to stimulate an immune response.

公开(公告)号：US20180256740A1

公开(公告)日：2018-09-13

申请号：US15790866

申请日：2017-10-23

申请人： The Board of Regents of the University of Texas System

发明人： Renata PASQUALINI ,  Wadih ARAP ,  Juri GELOVANI ,  Frank C. MARINI, III ,  Amin HAJITOU ,  Mian ALAUDDIN ,  Martin TREPEL

IPC分类号： A61K47/64 ,  A61K47/69 ,  A61K48/00 ,  C12N15/86

CPC分类号： A61K47/6455 ,  A61K47/62 ,  A61K47/6901 ,  A61K48/00 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2795/14143 ,  C12N2810/405 ,  C12N2810/85

摘要： Embodiments of the invention are generally directed to compositions and methods of delivering one or more transgene to a target cell, such as a tumor cell, in a site-specific manner to achieve enhanced expression and to constructs and compositions useful in such applications. In certain aspects, expression from a therapeutic nucleic acid may be assessed prior to administration of a treatment or diagnostic procedure to or on a subject.

公开(公告)号：US20180223282A1

公开(公告)日：2018-08-09

申请号：US15787876

申请日：2017-10-19

申请人： RaNA Therapeutics, Inc. ,  The General Hospital Corporation d/b/a Massachusetts General Hospital

发明人： Arthur M. Krieg ,  Romesh Subramanian ,  James McSwiggen ,  Jeannie T. Lee

IPC分类号： C12N15/113 ,  C07H21/00 ,  A61K31/7125 ,  A61K31/712 ,  A61K31/7115 ,  C07H21/04 ,  C07H21/02

CPC分类号： C12N15/113 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61K47/555 ,  A61K47/61 ,  A61K47/64 ,  A61K47/6455 ,  C07H21/00 ,  C07H21/02 ,  C07H21/04 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3521

摘要： Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of MECP2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of MECP2. Methods for modulating expression of MECP2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of MECP2.

公开(公告)号：US20180169267A1

公开(公告)日：2018-06-21

申请号：US15885783

申请日：2018-01-31

申请人： CureVac AG

发明人： Mariola FOTIN-MLECZEK ,  Patrick BAUMHOF

IPC分类号： A61K48/00 ,  C12N15/87 ,  C12N9/02 ,  C07K19/00 ,  A61K47/64 ,  A61K39/00 ,  A61K38/45

CPC分类号： A61K48/005 ,  A61K9/0019 ,  A61K38/45 ,  A61K38/4846 ,  A61K39/00 ,  A61K47/61 ,  A61K47/6455 ,  A61K48/0033 ,  A61K48/0041 ,  A61K48/0066 ,  A61K48/0075 ,  A61K2039/53 ,  C07K19/00 ,  C12N9/0069 ,  C12N15/87 ,  C12Y113/12007 ,  C12Y204/01007 ,  C12Y304/21022

摘要： The present invention relates to a complexed RNA, comprising at least one RNA complexed with one or more oligopeptides, wherein the oligopeptide, which has the function of cell-penetrating peptide (CPP), has a length of 8 to 15 amino acids and has the empirical formula (Arg)l;(Lys)m;(His)n;(Om)o;(Xaa)x with the majority of residues being selected from Arg, Lys, His, Om. The invention further relates to a method for transfecting a cell or an organism, thereby applying the inventive complexed RNA. Additionally, pharmaceutical compositions and kits comprising the inventive complexed RNA, as well as the use of the inventive complexed RNA for transfecting a cell, tissue or an organism and/or for modulating, preferably inducing or enhancing, an immune response are disclosed herein.

公开(公告)号：US09968686B2

公开(公告)日：2018-05-15

申请号：US14911264

申请日：2014-09-09

申请人： University of Vienna

发明人： Christian R. Noe ,  Mehrdad Dirin ,  Johannes Winkler ,  Bodo Lachmann ,  Ernst Urban

IPC分类号： C07H21/04 ,  A61K47/48 ,  A61K31/7088 ,  A61K33/24 ,  A61K45/06 ,  A61K31/713 ,  C12N15/113

CPC分类号： A61K47/6455 ,  A61K31/7088 ,  A61K31/713 ,  A61K33/24 ,  A61K45/06 ,  A61K47/60 ,  A61K47/645 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/351 ,  C12N2320/30 ,  A61K2300/00

摘要： The invention relates to an antisense or siRNA oligonucleotide or conjugate with improved pharmacokinetic properties, methods of producing the same as well as the use of such compounds and conjugates, e.g. as pharmaceutical composition, a pharmaceutical kit, a medicament or a tool in biomedical research.The conjugates of the invention have the formula I being P-(L-S—S—Y—X)n, wherein P represents a natural, artificial and/or modified oligonucleotide, L represents a linker group attached to one or more nucleosides at any position within the oligonucleotides sequence; S represents sulfur; X represents a ligand; Y represents a spacer and n is an integer ranging from 1 to the oligonucleotide length of P.The oligonucleotides of the invention have the formula II being P-(L-S—R)n, wherein P represents a natural, artificial and/or modified oligonucleotide, L represents a linker group attached to one or more nucleosides at any position within the oligonucleotides sequence, S represents sulfur, R represents either hydrogen or a thiol protecting group, preferably trityl or tertiary butyl, and n is an integer ranging from 1 to the oligonucleotide length of P.In addition, the pharmaceutical composition or pharmaceutical kit or conjugate can be used for the treatment of a disease or disorder that can be at least in part alleviated by therapy, wherein the disease or disorder is selected from the group consisting of bacterial infections, viral infections, cancer, metabolic diseases and immunological disorders and preferably cancer.

公开(公告)号：US09968685B2

公开(公告)日：2018-05-15

申请号：US12338480

申请日：2008-12-18

申请人： Brad N. Taylor ,  Rajeshwari Mehta ,  Tohru Yamada ,  Craig Beattie ,  Tapas Das Gupta

发明人： Brad N. Taylor ,  Rajeshwari Mehta ,  Tohru Yamada ,  Craig Beattie ,  Tapas Das Gupta

IPC分类号： A61K38/03 ,  C07K14/195 ,  C07K14/21 ,  C07K14/35 ,  A61K47/48 ,  A61K49/00 ,  C07K14/415 ,  C12N9/02 ,  G01N33/50 ,  G01N33/569 ,  A61K38/00

CPC分类号： A61K47/6455 ,  A61K38/00 ,  A61K38/03 ,  A61K47/64 ,  A61K49/0002 ,  A61K49/0032 ,  A61K49/0056 ,  C07K14/195 ,  C07K14/21 ,  C07K14/35 ,  C07K14/415 ,  C12N9/0004 ,  G01N33/5011 ,  G01N33/569 ,  G01N2510/00

摘要： The present invention discloses methods and materials for killing and/or inhibiting the growth of a cancer cell via preferential entry of a cytotoxic compound. Preferential entry of the cytotoxic compound is accomplished by the use of protein transduction domains derived from cupredoxins, including the p18 and p28 truncations of azurin.

公开(公告)号：US09950073B2

公开(公告)日：2018-04-24

申请号：US15407802

申请日：2017-01-17

申请人： Positec Power Tools (Suzhou) Co Ltd ,  Pu Chen

发明人： Pu Chen ,  Mousa Jafari ,  Wen Xu ,  Baoling Chen ,  Ran Pan ,  Nedra Karunaratne

IPC分类号： A61K47/48 ,  C12N15/113

CPC分类号： A61K47/64 ,  A61K9/0019 ,  A61K9/08 ,  A61K31/713 ,  A61K38/00 ,  A61K47/6455 ,  C07K14/4723 ,  C07K19/00 ,  C12N15/111 ,  C12N15/113 ,  C12N15/1135 ,  C12N15/87 ,  C12N2310/14 ,  C12N2320/32 ,  C12N2320/51

摘要： Peptides that have been found to facilitate the delivery of siRNA molecules into cells and to function in siRNA mediated silencing of cellular targets are disclosed. Complexes that include one of the peptides and a cargo molecule are disclosed, wherein the peptide and the cargo molecule are coupled non-covalently. Also disclosed are methods of producing and using the peptides/complexes.

公开(公告)号：US20180092987A1

公开(公告)日：2018-04-05

申请号：US15722436

申请日：2017-10-02

申请人： The Regents of the University of California

发明人： William C. Trogler ,  Sadik C. Esener ,  Davorka Messmer ,  Johan Ulrik Lind ,  Kristina K.P. Mitchell ,  Jian Yang

IPC分类号： C12N15/87 ,  A61K9/00 ,  A61K9/14 ,  C01B33/18 ,  C01B33/12 ,  B82Y5/00 ,  A61K49/18 ,  A61K49/04 ,  A61K48/00 ,  A61K39/00 ,  A61K9/51 ,  A61K9/16 ,  A61K9/20

CPC分类号： A61K47/6923 ,  A61K9/0019 ,  A61K9/14 ,  A61K9/1611 ,  A61K9/2009 ,  A61K9/5115 ,  A61K9/5192 ,  A61K39/0011 ,  A61K47/59 ,  A61K47/6455 ,  A61K47/6925 ,  A61K48/0041 ,  A61K49/04 ,  A61K49/183 ,  A61K2039/53 ,  A61K2039/6093 ,  B82Y5/00 ,  C01B33/12 ,  C01B33/18 ,  C12N15/87 ,  Y10S977/773 ,  Y10T428/2982

摘要： The disclosure provide hollow nanospheres and methods of making and using the same. The methods and compositions of the disclosure are useful for drug delivery and gene transfer.

公开(公告)号：US09907862B2

公开(公告)日：2018-03-06

申请号：US15065769

申请日：2016-03-09

申请人： CureVac AG

发明人： Patrick Baumhof ,  Thomas Schlake

IPC分类号： A61K38/00 ,  C07K2/00 ,  C07K4/00 ,  C07K5/00 ,  C07K7/00 ,  C07K14/00 ,  C07K16/00 ,  C07K17/00 ,  A61K48/00 ,  A61K47/60 ,  A61K47/64

CPC分类号： A61K48/0041 ,  A61K47/60 ,  A61K47/6455

摘要： The present invention is directed to an inventive polymeric carrier molecule according to generic formula (I) and variations thereof, which allows for efficient transfection of nucleic acids into cells in vivo and in vitro, a polymeric carrier cargo complex formed by a nucleic acid and the inventive polymeric carrier molecule, but also to methods of preparation of this inventive polymeric carrier molecule and of the inventive polymeric carrier cargo complex. The present invention also provides methods of application and use of this inventive polymeric carrier molecule and the inventive polymeric carrier cargo complex as a medicament, for the treatment of various diseases, and in the preparation of a pharmaceutical composition for the treatment of such diseases.

公开(公告)号：US20180057813A1

公开(公告)日：2018-03-01

申请号：US15690526

申请日：2017-08-30

申请人： Children's Hospital Medical Center

发明人： Assem G. Ziady ,  Matthew Siefert ,  Songbai Lin ,  Harrison Brown

IPC分类号： C12N15/113 ,  A61K38/17 ,  A61K35/28 ,  A61K47/60 ,  A61K47/64 ,  A61K31/713 ,  A61K31/395 ,  A61K31/4709 ,  A61K31/565 ,  A61K31/132 ,  A61K31/573 ,  A61K31/352 ,  A61K31/265 ,  A61K31/05 ,  A61K31/704 ,  A61K31/519 ,  A61K31/52 ,  A61K31/7048 ,  A61K48/00 ,  A61K31/7088 ,  C12N15/85

CPC分类号： C12N15/113 ,  A61K31/05 ,  A61K31/132 ,  A61K31/265 ,  A61K31/352 ,  A61K31/395 ,  A61K31/4709 ,  A61K31/519 ,  A61K31/52 ,  A61K31/565 ,  A61K31/573 ,  A61K31/704 ,  A61K31/7048 ,  A61K31/7088 ,  A61K31/713 ,  A61K35/28 ,  A61K38/1709 ,  A61K45/06 ,  A61K47/60 ,  A61K47/6455 ,  A61K48/0008 ,  A61K48/0066 ,  C12N15/111 ,  C12N15/85 ,  C12N2310/14 ,  C12N2320/31 ,  C12N2320/32

摘要： Disclosed are methods for the enhancement of nucleic acid delivery systems. The methods may employ treatment with a compound and/or an RNAi molecule in combination with a nucleic acid to improve nucleic acid uptake into a cell. In particular, the disclosed methods may be useful for improved gene therapy techniques.