公开(公告)号：US12104188B2

公开(公告)日：2024-10-01

申请号：US17716253

申请日：2022-04-08

申请人： Allogene Therapeutics, Inc.

发明人： Meritxell Galindo Casas ,  Thomas John Van Blarcom

IPC分类号： C12N9/64 ,  A61K31/7088 ,  A61K35/17 ,  A61K38/48 ,  A61K38/52 ,  C12N5/0783 ,  C12N9/90 ,  C12N11/18

CPC分类号： C12N9/6472 ,  A61K31/7088 ,  A61K35/17 ,  A61K38/4873 ,  A61K38/52 ,  C12N5/0636 ,  C12N9/90 ,  C12N11/18 ,  C12Y304/22062 ,  C12Y502/01008 ,  C07K2319/50 ,  C07K2319/70 ,  C12N2501/734 ,  C12N2510/00 ,  C12N2740/16043

摘要： Provided herein are modified caspase-9 polypeptides, and chimeric caspase-9 proteins containing the modified caspase-9 polypeptides. The disclosure further provides polynucleotides encoding these proteins, engineered host cells containing these polynucleotides and proteins, including host cells that co-express a chimeric antigen receptor, and methods of making and using the same.

公开(公告)号：US20230407330A1

公开(公告)日：2023-12-21

申请号：US18037986

申请日：2021-11-18

申请人： Umoja Biopharma, Inc.

发明人： Andrew SCHARENBERG ,  Laurie BEITZ

IPC分类号： C12N15/86 ,  C12N9/90 ,  C07K14/715

CPC分类号： C12N15/86 ,  C12Y502/01008 ,  C12N9/90 ,  C07K14/7155 ,  C12N2740/15043

摘要： The present disclosure relates to a vector system comprising at least two polynucleotides, each polynucleotide may comprise a polynucleotide sequence encoding a polypeptide component of a macromolecular complex. Assembly of the macromolecular complex in a cell transduced with the polynucleotides may promote growth and/or survival of a cell.

公开(公告)号：US20230270831A1

公开(公告)日：2023-08-31

申请号：US17934501

申请日：2022-09-22

申请人： Baylor College of Medicine

发明人： Malcolm K. Brenner ,  Jack James

IPC分类号： A61K38/52 ,  A61K35/28 ,  A61K35/545 ,  A61K39/00 ,  C12N5/0783 ,  C12N5/0775 ,  A61K35/17 ,  A61K38/48

CPC分类号： A61K38/52 ,  A61K35/28 ,  A61K35/545 ,  A61K39/001 ,  C12N5/0636 ,  C12N5/0663 ,  A61K35/17 ,  A61K38/4873 ,  C07K2319/00 ,  A61K2039/5156 ,  A61K2039/5158 ,  C12N2501/48 ,  C12N2510/00 ,  C12Y304/22062 ,  C12Y502/01008

摘要： Provided herein are methods for cell therapy by modifying transfused cells to express an inducible caspase 9 protein, so that the cells may be selectively killed if the patient experiences dangerous side effects. Provided also within relates in part to methods for preventing or treating Graft versus Host Disease by modifying T cells before administration to a patient, so that they may be selectively killed if GvHD develops in the patient.

公开(公告)号：US20180320182A1

公开(公告)日：2018-11-08

申请号：US16038029

申请日：2018-07-17

申请人： Thermo Fisher Scientific Inc.

发明人： Anastasia Khvorova ,  Angela Reynolds ,  Devin Leake ,  William Marshall ,  Steven Read ,  Stephen Scaringe

IPC分类号： C12N15/113 ,  C12N15/10 ,  A61K31/713 ,  C12N15/11

CPC分类号： C12N15/113 ,  A61K31/713 ,  C12N15/1048 ,  C12N15/111 ,  C12N15/1135 ,  C12N15/1136 ,  C12N15/1137 ,  C12N15/1138 ,  C12N2310/14 ,  C12N2320/10 ,  C12N2320/11 ,  C12Y113/12007 ,  C12Y502/01008

摘要： Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for TTR.

公开(公告)号：US20180271961A1

公开(公告)日：2018-09-27

申请号：US15921530

申请日：2018-03-14

申请人： Baylor College of Medicine

发明人： Malcolm K. BRENNER

IPC分类号： A61K38/52 ,  A61K35/28 ,  A61K35/545 ,  A61K38/48 ,  A61K39/00 ,  C12N5/0783 ,  C12N5/0775 ,  A61K35/17

CPC分类号： A61K38/52 ,  A61K35/17 ,  A61K35/28 ,  A61K35/545 ,  A61K38/4873 ,  A61K39/001 ,  A61K2039/5156 ,  A61K2039/5158 ,  C07K2319/00 ,  C12N5/0636 ,  C12N5/0663 ,  C12N2501/48 ,  C12N2510/00 ,  C12Y304/22062 ,  C12Y502/01008

摘要： Provided herein are methods for cell therapy by modifying transfused cells to express an inducible caspase 9 protein, so that the cells may be selectively killed if the patient experiences dangerous side effects. Provided also within relates in part to methods for preventing or treating Graft versus Host Disease by modifying T cells before administration to a patient, so that they may be selectively killed if GvHD develops in the patient.

公开(公告)号：US20180201934A1

公开(公告)日：2018-07-19

申请号：US15862479

申请日：2018-01-04

申请人： Thermo Fisher Scientific Inc.

发明人： Anastasia Khvorova ,  Angela Reynolds ,  Devin Leake ,  William Marshall ,  Steven Read ,  Stephen Scaringe

IPC分类号： C12N15/113 ,  A61K31/713 ,  C12N15/10 ,  C12N15/11 ,  C12N15/82

CPC分类号： C12N15/1137 ,  A61K31/713 ,  C12N15/1048 ,  C12N15/111 ,  C12N15/113 ,  C12N15/1135 ,  C12N15/1136 ,  C12N15/1138 ,  C12N15/8218 ,  C12N2310/14 ,  C12N2310/31 ,  C12N2310/322 ,  C12N2320/10 ,  C12N2320/11 ,  C12N2320/13 ,  C12Y113/12007 ,  C12Y502/01008

摘要： Efficient sequence specific gene silencing is possible through the use of siRNA technology. Be selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods compositions, and kits generated through rational design of siRNAs are disclosed, including those directed to the nucleotide sequences for HAO1.

公开(公告)号：US10011836B2

公开(公告)日：2018-07-03

申请号：US14936403

申请日：2015-11-09

申请人： Thermo Fisher Scientific Inc.

发明人： Anastasia Khvorova ,  Angela Reynolds ,  Devin Leake ,  William Marshall ,  Steven Read ,  Stephen Scaringe

IPC分类号： C12N15/11 ,  C12N15/113 ,  A61K31/713 ,  C12N15/10 ,  C12N15/82

CPC分类号： C12N15/1138 ,  A61K31/713 ,  C12N15/1048 ,  C12N15/111 ,  C12N15/113 ,  C12N15/1135 ,  C12N15/1136 ,  C12N15/1137 ,  C12N15/8218 ,  C12N2310/14 ,  C12N2320/10 ,  C12N2320/11 ,  C12N2320/13 ,  C12N2330/31 ,  C12Y113/12007 ,  C12Y502/01008

摘要： Efficient sequence specific gene silencing is possible through the use of siRNA technology. Be selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods compositions, and kits generated through rational design of siRNAs are disclosed, including those directed to the nucleotide sequences for F12.

公开(公告)号：US09932572B2

公开(公告)日：2018-04-03

申请号：US15224441

申请日：2016-07-29

申请人： BELLICUM PHARMACEUTICALS, INC.

发明人： David Spencer ,  Wei-Chun Chang

IPC分类号： C07H21/04 ,  A61K48/00 ,  C12N9/64 ,  A61K31/4545 ,  A61K38/48 ,  A61K38/52 ,  C12N9/90 ,  A01K67/00

CPC分类号： C12N9/6472 ,  A61K31/4545 ,  A61K38/4873 ,  A61K38/52 ,  C12N9/90 ,  C12Y304/22062 ,  C12Y502/01008

摘要： The technology relates in part to compositions comprising modified Caspase-9 polypeptides, compositions comprising nucleic acids coding for modified Caspase-9 polypeptides, chimeric modified Caspase-9 polypeptides, and methods of use thereof, including methods for cell therapy. Methods for cell therapy include modifying transfused cells to express an inducible modified Caspase-9 protein, with reduced basal activity in the absence of the inducer.

公开(公告)号：US20180072667A1

公开(公告)日：2018-03-15

申请号：US15703056

申请日：2017-09-13

申请人： Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V.

发明人： Steffen Gaali ,  Felix Hausch ,  Alexander Kirschner ,  Xixi Feng ,  Andreas Bracher ,  Gerd Ruehter

IPC分类号： C07D207/16 ,  G06F19/16 ,  C12N9/90 ,  C07D211/78 ,  C07D401/14 ,  C07D279/12 ,  C07D405/12 ,  C07D401/12 ,  C07D211/60

CPC分类号： C07D207/16 ,  C07B2200/13 ,  C07D211/60 ,  C07D211/78 ,  C07D279/12 ,  C07D401/12 ,  C07D401/14 ,  C07D405/12 ,  C07K5/06139 ,  C12N9/90 ,  C12Y502/01008 ,  G06F19/16

摘要： The present invention relates to compounds having a selective FKBP51 ligand scaffold, pharmaceutically acceptable salts of these compounds and pharmaceutical compositions containing at least one of these compounds together with pharmaceutically acceptable carrier, excipient and/or diluents. Said selective FKBP51 ligand compounds can be used for prophylaxis and/or treatment of psychiatric disorders and neurodegenerative diseases, disorders and conditions.

公开(公告)号：US20180042963A1

公开(公告)日：2018-02-15

申请号：US15801133

申请日：2017-11-01

申请人： The Regents of the University of California

发明人： Chia-Yung Wu ,  James Onuffer ,  Wendell A. Lim

IPC分类号： C07K14/705 ,  C07K16/28 ,  C12N9/14 ,  C12N5/0783 ,  C07K16/18 ,  C07K14/725 ,  C12N9/90 ,  A61K39/00

CPC分类号： A61K35/17 ,  A61K47/6891 ,  A61K2039/505 ,  C07K14/705 ,  C07K14/7051 ,  C07K14/70521 ,  C07K14/70575 ,  C07K14/70578 ,  C07K16/18 ,  C07K16/2803 ,  C07K16/2866 ,  C07K2317/622 ,  C07K2319/00 ,  C07K2319/03 ,  C07K2319/20 ,  C07K2319/33 ,  C07K2319/74 ,  C12N5/0636 ,  C12N9/14 ,  C12N9/90 ,  C12N2510/00 ,  C12Y502/01008

摘要： The present disclosure provides a heterodimeric, conditionally active chimeric antigen receptor (CAR), and a nucleic acid comprising a nucleotide sequence encoding the CAR. The present disclosure provides cells genetically modified to produce the CAR. A CAR of the present disclosure can be used in various methods, which are also provided.