公开(公告)号：US20190247470A1

公开(公告)日：2019-08-15

申请号：US16397823

申请日：2019-04-29

申请人： Excision Bio Therapeutics, Inc. ,  Temple University of the Commonwealth System of Higher Education

发明人： Kamel Khalili ,  Thomas Malcolm ,  Kenneth I. Kohn

IPC分类号： A61K38/46 ,  A61K48/00 ,  C12N15/11 ,  C07K16/28 ,  A61K39/395 ,  C12Q1/70 ,  A61P31/20 ,  C12N15/10

CPC分类号： A61K38/465 ,  A61K39/39541 ,  A61K48/00 ,  A61K2039/505 ,  A61P31/20 ,  C07K16/2839 ,  C12N15/102 ,  C12N15/11 ,  C12N2310/20 ,  C12Q1/70 ,  Y02A50/467

摘要： A method of eliminating the risk of JCV activation in a subject undergoing immunosuppressive therapy, by administering an effective amount of a gene editing composition directed toward at least one target sequence in the JCV genome, cleaving the target sequence in the JCV genome, disrupting the JCV genome, eliminating the JCV infection, eliminating the risk of JCV activation, and treating the subject with an immunosuppressive therapy. A pharmaceutical composition including at least one isolated nucleic acid sequence encoding a CRISPR-associated endonuclease and at least one gRNA having a spacer sequence complementary to a target sequence in a JCV DNA, the isolated nucleic acid sequences being included in at least one expression vector. Pharmaceutical compositions including at least one isolated nucleic acid sequence encoding at least one TALEN, at least one ZFN, and gene editing composition of C2c1, C2c3, TevCas9, Archaea Cas9, CasY.1-CasY.6, CasX, or argonaute protein, which target at least one nucleotide sequence of the JCV genome.

公开(公告)号：US20190247443A1

公开(公告)日：2019-08-15

申请号：US16345622

申请日：2016-10-31

申请人： Seattle Children's Hospital (dba Seattle Children's Research Institute)

发明人： Andrew M. Scharenberg ,  David J. Rawlings ,  Troy Torgerson

IPC分类号： A61K35/545 ,  A61K38/20 ,  C12N5/0789 ,  C12N15/10 ,  C12N15/66 ,  C12N15/85 ,  A61P37/06 ,  C12N15/52

CPC分类号： A61K35/545 ,  A61K38/2013 ,  A61K48/00 ,  A61P37/06 ,  C12N5/0647 ,  C12N15/102 ,  C12N15/52 ,  C12N15/66 ,  C12N15/85

摘要： Disclosed are methods of making a genetically cell that expressed FOXP3 and methods of treatment. In some embodiments, the method can providing a first nucleotide sequence, wherein the first nucleotide sequence comprises a coding strand, the coding strand comprising one or more regulatory elements and a FOXP3 gene or portion thereof providing a nuclease and performing a gene editing process on the first nucleotide sequence, which edits said one or more regulatory elements, and optionally edits the FOXP3 gene or portion thereof. Methods of treating a subject suffering from an autoimmune disease and subjects suffering the effects of organ transplantation are also provided.

公开(公告)号：US20190247414A1

公开(公告)日：2019-08-15

申请号：US16335017

申请日：2017-09-25

申请人： NATIONAL UNIVERSITY CORPORATION TOKYO MEDICAL AND DENTAL UNIVERSITY

发明人： Takanori Yokota ,  Tetsuya Nagata

IPC分类号： A61K31/711 ,  A61K47/22 ,  A61K47/28 ,  C12N15/113 ,  A61K47/54 ,  A61P27/02 ,  A61P25/00 ,  A61K31/7125 ,  A61K9/00

CPC分类号： A61K31/711 ,  A61K9/0019 ,  A61K31/7088 ,  A61K31/7125 ,  A61K47/22 ,  A61K47/28 ,  A61K47/542 ,  A61K47/554 ,  A61K48/00 ,  A61P25/00 ,  A61P27/02 ,  C12N15/09 ,  C12N15/113

摘要： The present invention provides a composition and a method for efficiently delivering a nucleic acid agent to the central nervous system and/or the retina and bringing about antisense effects. Provided is a composition for reducing the expression level of a target transcription product in the central nervous system and/or retina of a subject, the composition including a nucleic acid complex that includes a first nucleic acid strand and a second nucleic acid strand, wherein: the first nucleic acid strand comprises a base sequence capable of hybridizing with at least part of the target transcription product and has an antisense effect on the target transcription product; the second nucleic acid strand comprises a base sequence complementary to the first nucleic acid strand and is conjugated to tocopherol, cholesterol, or an analog thereof; and the first nucleic acid strand is annealed to the second nucleic acid strand.

公开(公告)号：US20190241925A1

公开(公告)日：2019-08-08

申请号：US16336417

申请日：2017-09-26

申请人： SOLA Biosciences, LLC

发明人： Akinori HISHIYA

IPC分类号： C12P21/02 ,  C12N9/64 ,  C07K14/715 ,  C07K14/705 ,  C07K16/24 ,  C07K16/22 ,  C07K14/755

CPC分类号： C12P21/02 ,  A61K48/00 ,  C07K14/705 ,  C07K14/70575 ,  C07K14/7151 ,  C07K14/7155 ,  C07K14/745 ,  C07K14/755 ,  C07K16/00 ,  C07K16/22 ,  C07K16/241 ,  C07K16/244 ,  C07K2317/14 ,  C07K2317/52 ,  C07K2319/03 ,  C07K2319/30 ,  C07K2319/35 ,  C07K2319/43 ,  C07K2319/70 ,  C12N9/6437 ,  C12N9/644 ,  C12N2710/16622

摘要： Cell-associated secretion-enhancing fusion proteins are disclosed that comprise a target protein binding domain and a transmembrane retention domain. Co-expression in a host cell of a fusion protein and a target protein of interest that is temporarily bound by the fusion protein leads to an increased level of target protein secreted from the host cell. The fusion proteins are engineered to be retained with the producing host cell, thus eliminating a non-natural component from the extracellular media of the host cell and simplifying purification of the target protein. Nucleic acid molecules encoding such fusion proteins are also disclosed for use in expressing the fusion proteins in host cells, for use in restoring lost or diminished cell functions, and for use in treating diseases characterized by a lost or diminished cell function. Methods and compositions comprising fusion proteins of the invention are disclosed for use in enhancing the level of co-expressed target proteins secreted from host cells.

公开(公告)号：US20190241633A1

公开(公告)日：2019-08-08

申请号：US16098844

申请日：2017-05-04

申请人： CureVac AG

发明人： Mariola FOTIN-MLECZEK ,  Ingmar HOERR

IPC分类号： C07K14/505

CPC分类号： C07K14/505 ,  A61K31/7088 ,  A61K38/00 ,  A61K48/00 ,  A61K48/005

摘要： The present invention relates to an RNA encoding a therapeutic protein. In particular, the present invention relates to RNA suitable for use as a medicament. The present invention concerns such novel RNA as well as compositions and kits comprising the RNA. Furthermore, the present invention relates to the RNA, compositions or kits as disclosed herein for use as a medicament. The present invention also provides the use of the RNA, compositions or kits as disclosed herein far increasing the expression of said encoded protein, in particular in gene therapy.

公开(公告)号：US20190240352A1

公开(公告)日：2019-08-08

申请号：US16337808

申请日：2017-09-29

申请人： NATIONAL UNIVERSITY CORPORATION TOKYO MEDICAL AND DENTAL UNIVERSITY

发明人： Takanori Yokota ,  Kotaro Yoshioka

IPC分类号： A61K48/00 ,  A61K9/00 ,  A61K9/70 ,  A61K31/7125 ,  A61P35/00 ,  A61P25/28

CPC分类号： A61K48/0066 ,  A61K9/0019 ,  A61K9/7023 ,  A61K31/7088 ,  A61K31/7125 ,  A61K48/00 ,  A61P25/28 ,  A61P35/00 ,  C12N15/09 ,  C12N15/113

摘要： Provided is a nucleic acid strand that can efficiently deliver an antisense oligonucleotide into the body, particularly a nucleic acid complex comprising a first nucleic acid strand and a second nucleic acid strand, wherein the first nucleic acid strand includes a base sequence that is capable of hybridizing with at least a portion of a target transcription product, and exerts an antisense effect on the target transcription product; the second nucleic acid strand includes a complementary region having a base sequence complementary to the first nucleic acid strand and at least one overhang region located on the 5′ and/or 3′ side of the complementary region; and the first nucleic acid strand is annealed to the complementary region in the second nucleic acid strand.

公开(公告)号：US20190240343A1

公开(公告)日：2019-08-08

申请号：US16341412

申请日：2017-04-07

申请人： Baylor College of Medicine

发明人： Nabil M. Ahmed ,  Hebatalla S. Samaha ,  Antonella Pignata ,  Kristen Fousek

IPC分类号： A61K47/64 ,  C07K14/705 ,  A61K35/28 ,  A61K35/17 ,  A61P35/00

CPC分类号： A61K47/6425 ,  A61K35/17 ,  A61K35/28 ,  A61K39/38 ,  A61K39/39 ,  A61K48/00 ,  A61P5/00 ,  A61P31/10 ,  A61P35/00 ,  C07K14/70596 ,  C07K2319/02 ,  C07K2319/03 ,  C07K2319/33 ,  C07K2319/70

摘要： Embodiments of the disclosure concern methods and compositions for delivering therapeutic, diagnostic or interventional moieties, such as complex and simple entities such as biologies, including at least cells, for example. The methods employ targeted delivery by employing at least one ALCAM-binding moiety on the therapeutic, diagnostic or interventional moiety to be delivered. In specific cases, the ALCAM-binding moiety is present on or with the therapeutic moiety in multiple iterations. In certain embodiments, the ALCAM-binding moiety comprises at least one SRCR domain from CD6 and a stalk, such as from CD6, of the secretable or molecular form thereof.

公开(公告)号：US20190233516A1

公开(公告)日：2019-08-01

申请号：US16340196

申请日：2017-10-10

申请人： THE NATIONAL INSTITUTE FOR BIOTECHNOLOGY IN THE NEGEV LTD.

发明人： Alon MONSONEGO ,  Angel PORGADOR ,  Roee ATLAS

IPC分类号： C07K16/28 ,  A61P25/28 ,  A61K35/15 ,  A61P35/00 ,  A61P3/00 ,  A61P9/00 ,  C12N5/0783 ,  A61K35/17

CPC分类号： C07K16/2809 ,  A61K35/15 ,  A61K35/17 ,  A61K48/00 ,  A61P3/00 ,  A61P9/00 ,  A61P25/28 ,  A61P35/00 ,  C07K14/7051 ,  C07K14/70532 ,  C07K16/18 ,  C07K16/2818 ,  C07K2317/74 ,  C07K2317/75 ,  C07K2319/03 ,  C07K2319/41 ,  C07K2319/60 ,  C12N5/0636 ,  C12N5/0638 ,  C12N5/0646 ,  C12N2501/25 ,  C12N2501/335 ,  C12N2501/515 ,  C12N2502/30 ,  C12N2510/00

摘要： Modified cells comprising a transmembrane polypeptide comprising at least one extracellular target receptor-binding domain, a transmembrane domain and an intracellular domain, wherein said intracellular domain is not capable of transducing any signal are provided. Methods of inducing or inhibiting signaling by a target receptor in a target cell comprising contacting the target cell with a modified cell of the invention are also provided.

公开(公告)号：US20190218555A1

公开(公告)日：2019-07-18

申请号：US16371026

申请日：2019-03-31

申请人： BIONEER CORPORATION

发明人： Taewoo Lee ,  Sanghyung Shim ,  Ungsik Yu ,  Han Oh Park

IPC分类号： C12N15/113 ,  A61P35/00 ,  A61K48/00 ,  A61K31/713 ,  A61K31/7105

CPC分类号： C12N15/113 ,  A61K31/7105 ,  A61K31/713 ,  A61K48/00 ,  A61P35/00 ,  C12N15/1138 ,  C12N2310/141

摘要： The present invention relates to a pharmaceutical composition for treatment of cancer, which comprises, as an active ingredient, one or more miRNAs selected from the group consisting of miR-3670, miR-8078, and miR-4477a. The pharmaceutical composition for treatment of cancer according to the present invention exhibits excellent effects of inhibiting cancer cell proliferation and inducing cancer cell apoptosis. Thus, the pharmaceutical composition of the present invention can be effectively used as an anticancer therapeutic agent.

公开(公告)号：US20190194689A1

公开(公告)日：2019-06-27

申请号：US16234834

申请日：2018-12-28

申请人： University of Massachusetts

发明人： Guangping Gao ,  Hongwei Zhang ,  Hongyan Wang ,  Zuoshang Xu

IPC分类号： C12N15/86 ,  A61K31/713 ,  A61K38/50 ,  C12N7/00 ,  A61K48/00 ,  C12N9/80 ,  C12N15/864 ,  C12N15/113

CPC分类号： C12N15/86 ,  A61K31/713 ,  A61K38/50 ,  A61K48/00 ,  A61K48/0058 ,  A61K48/0075 ,  A61P25/00 ,  C12N7/00 ,  C12N9/80 ,  C12N15/1137 ,  C12N15/635 ,  C12N15/8645 ,  C12N2310/141 ,  C12N2750/14133 ,  C12N2750/14141 ,  C12N2750/14143 ,  C12N2750/14145 ,  C12N2750/14162 ,  C12N2810/10 ,  C12N2840/007 ,  C12Y305/01015

摘要： The invention in some aspects relates to recombinant adeno-associated viruses useful for targeting transgenes to CNS tissue, and compositions comprising the same, and methods of use thereof. In some aspects, the invention provides methods and compositions for treating CNS-related disorders.