公开(公告)号：US20240335516A1

公开(公告)日：2024-10-10

申请号：US18744175

申请日：2024-06-14

申请人： Beam Therapeutics Inc.

发明人： Shaunna Berkovitch ,  Jason Michael Gehrke ,  Holly Rees ,  Conrad Rinaldi ,  Tanggis Bohnuud

IPC分类号： A61K38/50 ,  A61K31/7105 ,  A61K38/46 ,  C07K14/47 ,  C12N9/22 ,  C12N9/78 ,  C12N15/11 ,  C12N15/90

CPC分类号： A61K38/50 ,  A61K31/7105 ,  A61K38/465 ,  C07K14/4702 ,  C12N9/22 ,  C12N9/78 ,  C12N15/11 ,  C12N15/907 ,  C12Y305/04004 ,  C12Y305/04005 ,  C12N2310/20

摘要： The invention of the disclosure features compositions and methods for treating spinal muscular atrophy (SMA) by introducing alterations to a survival of motor neuron 2 (SMN2) polynucleotide(s) in a cell. In particular embodiments, the invention provides a base editor system (e.g., a fusion protein or complex comprising a programmable DNA binding protein, a nucleobase editor, and gRNA) for modifying an SMN2 polynucleotide(s), where the alteration is associated with increased expression of the SMN2 polypeptide(s) encoded by the polynucleotide(s).

公开(公告)号：US20240307470A1

公开(公告)日：2024-09-19

申请号：US18272986

申请日：2022-01-19

申请人： Seneca Therapeutics, Inc.

发明人： Paul L. HALLENBECK ,  Sunil CHADA

IPC分类号： A61K35/768 ,  A61K31/165 ,  A61K31/553 ,  A61K38/18 ,  A61K38/19 ,  A61K38/20 ,  A61K38/50 ,  A61K39/395 ,  A61K45/06 ,  C12N15/86

CPC分类号： A61K35/768 ,  A61K31/165 ,  A61K31/553 ,  A61K38/1841 ,  A61K38/195 ,  A61K38/2013 ,  A61K38/2086 ,  A61K38/50 ,  A61K39/3955 ,  A61K45/06 ,  C12N15/86 ,  C12Y305/04001 ,  C12N2770/32022 ,  C12N2770/32032 ,  C12N2770/32043

摘要： Provided herein in are armed Seneca Valley Viruses which have been altered to carry a therapeutic payload, i.e. to encode an agent for treating cancer. These armed Seneca Valley Viruses are oncolytic and express a cancer treating agent. Also provided herein are compositions and methods of using an armed Seneca Valley Virus to treat cancer in a subject.

公开(公告)号：US12065666B2

公开(公告)日：2024-08-20

申请号：US16475412

申请日：2018-01-04

申请人： Rutgers, the State University

发明人： Shengkan Jin ,  Juan-Carlos Collantes

IPC分类号： A61K31/7088 ,  A61K38/46 ,  A61K38/50 ,  A61K48/00 ,  C12N9/22 ,  C12N9/78 ,  C12N15/113 ,  C12N15/62 ,  C12N15/90

CPC分类号： C12N15/90 ,  A61K31/7088 ,  A61K38/465 ,  A61K38/50 ,  A61K48/00 ,  C12N9/22 ,  C12N9/78 ,  C12N15/113 ,  C12N15/62 ,  C07K2319/00 ,  C12N2310/20 ,  C12N2310/3519 ,  C12N2800/80

摘要： The present invention discloses systems for targeted gene editing and related uses.

公开(公告)号：US20240226243A9

公开(公告)日：2024-07-11

申请号：US18131560

申请日：2023-04-06

申请人： Applied Molecular Transport Inc.

发明人： Randall J. Mrsny ,  Tahir Mahmood

IPC分类号： A61K38/45 ,  A61K38/16 ,  A61K38/17 ,  A61K38/20 ,  A61K38/26 ,  A61K38/27 ,  A61K38/48 ,  A61K38/49 ,  A61K38/50 ,  A61K38/51 ,  C07K14/54 ,  C07K16/24

CPC分类号： A61K38/45 ,  A61K38/164 ,  A61K38/166 ,  A61K38/1793 ,  A61K38/20 ,  A61K38/2066 ,  A61K38/26 ,  A61K38/27 ,  A61K38/482 ,  A61K38/49 ,  A61K38/50 ,  A61K38/51 ,  C07K14/5428 ,  C07K16/241 ,  C12Y204/02036 ,  C12Y304/21068 ,  C12Y304/21073 ,  C12Y305/04004 ,  C12Y402/02001 ,  C07K2317/76 ,  C07K2319/00 ,  C07K2319/21 ,  C07K2319/30 ,  C07K2319/32 ,  C07K2319/50 ,  C07K2319/55 ,  C07K2319/60 ,  C07K2319/90

摘要： The present disclosure relates to pharmaceutical compositions comprising a non-naturally occurring fusion molecule and one or more pharmaceutically acceptable carriers, formulated for oral delivery to a subject, and designed to provide for improved, effective therapies for treatment of, e.g., inflammatory diseases, autoimmune diseases, cancer, metabolic disorders, and growth deficiency disorders.

公开(公告)号：US11980669B2

公开(公告)日：2024-05-14

申请号：US16807375

申请日：2020-03-03

申请人： PFIZER INC.

发明人： Xiaotian Zhong ,  Amarnauth Shastrie Prashad ,  Ronald William Kriz ,  Tao He ,  Will Somers ,  Wenge Wang ,  Leo Joseph Letendre

IPC分类号： A61K47/68 ,  A61K38/05 ,  A61K38/50 ,  C07K16/00 ,  C07K16/30

CPC分类号： A61K47/6889 ,  A61K38/05 ,  A61K47/68 ,  A61K47/6817 ,  A61K47/6851 ,  A61K47/6855 ,  C07K16/00 ,  C07K16/30 ,  C07K2317/14 ,  C07K2317/40 ,  C07K2317/526 ,  C07K2317/55 ,  C07K2317/94

摘要： An antibody production process in mammalian cells in which engineered unpaired cysteine residues are post-translationally modified and capped with particular chemical entities, which capped antibodies are well suited to further site-specific conjugation steps to form antibody-drug conjugates (ADCs) or protein drug conjugates; ADCs produced using these capped antibodies including in particular ADCs formed by the selective reduction of the capped antibodies' cysteine residues, and ADCs formed using chemical handles such as aldehyde/azide/alkyne biorthogonal groups, which permit additional drug conjugation chemistry; and uncapped antibodies produced by cells in low cysteine, cysteine and glutathione media, and ADCs produced via direct conjugation to these uncapped antibodies.

公开(公告)号：US20240131118A1

公开(公告)日：2024-04-25

申请号：US18131560

申请日：2023-04-05

申请人： Applied Molecular Transport Inc.

发明人： Randall J. Mrsny ,  Tahir Mahmood

IPC分类号： A61K38/45 ,  A61K38/16 ,  A61K38/17 ,  A61K38/20 ,  A61K38/26 ,  A61K38/27 ,  A61K38/48 ,  A61K38/49 ,  A61K38/50 ,  A61K38/51 ,  C07K14/54 ,  C07K16/24

CPC分类号： A61K38/45 ,  A61K38/164 ,  A61K38/166 ,  A61K38/1793 ,  A61K38/20 ,  A61K38/2066 ,  A61K38/26 ,  A61K38/27 ,  A61K38/482 ,  A61K38/49 ,  A61K38/50 ,  A61K38/51 ,  C07K14/5428 ,  C07K16/241 ,  C12Y204/02036 ,  C12Y304/21068 ,  C12Y304/21073 ,  C12Y305/04004 ,  C12Y402/02001 ,  C07K2317/76 ,  C07K2319/00 ,  C07K2319/21 ,  C07K2319/30 ,  C07K2319/32 ,  C07K2319/50 ,  C07K2319/55 ,  C07K2319/60 ,  C07K2319/90

摘要： The present disclosure relates to pharmaceutical compositions comprising a non-naturally occurring fusion molecule and one or more pharmaceutically acceptable carriers, formulated for oral delivery to a subject, and designed to provide for improved, effective therapies for treatment of, e.g., inflammatory diseases, autoimmune diseases, cancer, metabolic disorders, and growth deficiency disorders.

公开(公告)号：US20240108734A1

公开(公告)日：2024-04-04

申请号：US18273716

申请日：2022-01-21

申请人： The Regents of the University of California

发明人： Catriona JAMIESON ,  Wenxue MA

IPC分类号： A61K47/54 ,  A61K9/127 ,  A61K31/365 ,  A61K31/506 ,  A61K31/519 ,  A61K31/5383 ,  A61K31/635 ,  A61K31/7064 ,  A61K35/28 ,  A61K38/50 ,  A61K45/06 ,  A61M15/08 ,  A61P31/14 ,  A61P31/16 ,  A61P35/02 ,  C12N15/113 ,  C12N15/86

CPC分类号： A61K47/549 ,  A61K9/127 ,  A61K31/365 ,  A61K31/506 ,  A61K31/519 ,  A61K31/5383 ,  A61K31/635 ,  A61K31/7064 ,  A61K35/28 ,  A61K38/50 ,  A61K45/06 ,  A61M15/08 ,  A61P31/14 ,  A61P31/16 ,  A61P35/02 ,  C12N15/1137 ,  C12N15/86 ,  C12Y305/04 ,  C12N2310/14 ,  C12N2310/531 ,  C12N2740/15043

摘要： In alternative embodiments, provided are methods for treating and ameliorating a cancer such as a leukemia such as acute myeloid leukemia (AML) comprising administration to an individual in need thereof a pharmaceutical composition comprising imetelstat, or imetelstat and second drug such as dasatinib, or ruxolitinib, fedratinib, 8-aza-adenosine, raltegravir and/or dolutegravir or any combination thereof. In alternative embodiments, provided are methods for the in vivo inhibition of myeloproliferative neoplasm (MPN) or AML stem cell propagation comprising administration to an individual in need thereof a pharmaceutical composition comprising imetelstat, or imetelstat and second drug. In alternative embodiments, provided are methods for the in vivo inhibition pre-leukemia stem cell (pre-LSC) transformation into leukemia stem cells (LSCs) comprising administration to an individual in need thereof a pharmaceutical composition comprising imetelstat, or imetelstat and second drug such as dastinib, or ruxolitinib, fedratinib, 8-aza-adenosine, raltegravir and/or dolutegravir or any combination thereof.

公开(公告)号：US20240066107A1

公开(公告)日：2024-02-29

申请号：US16996846

申请日：2020-08-18

申请人： UNIVERSITY-INDUSTRY FOUNDATION(UIF), YONSEI UNIVER

发明人： Jae-Chul PYUN

IPC分类号： A61K38/50 ,  A61K38/44 ,  G01N33/68

CPC分类号： A61K38/50 ,  A61K38/44 ,  G01N33/6848

摘要： The present invention relates to a pharmaceutical composition for treating gout, and a gout treatment kit. According to an embodiment of the present invention, a pharmaceutical composition for treating gout comprising a probe containing at least one or more peptides specifically coupled to a monosodium urate MSU crystals; and a first enzyme which is coupled to the probe, and reacts with the target MSU crystal coupled to the probe or urea of surrounding joint fluid to remove the target MSU crystal is provided.

公开(公告)号：US11883474B2

公开(公告)日：2024-01-30

申请号：US18049791

申请日：2022-10-26

申请人： Theriva Biologics, Inc.

发明人： Michael Kaleko ,  Sheila Connelly

IPC分类号： A61K38/50 ,  A61K9/00 ,  A61K9/48 ,  A61K9/50 ,  A61K31/424 ,  A61K38/14

CPC分类号： A61K38/50 ,  A61K9/0053 ,  A61K9/4808 ,  A61K9/4858 ,  A61K9/4866 ,  A61K9/4891 ,  A61K9/5026 ,  A61K9/5084 ,  A61K31/424 ,  C12Y305/02006 ,  A61K38/14

摘要： This invention provides, in part, various compositions and methods for protecting the gastrointestinal microbiome from antibiotic disruption from orally administered antibiotics.

公开(公告)号：US20240009283A1

公开(公告)日：2024-01-11

申请号：US18160112

申请日：2023-01-26

申请人： Aerase, Inc.

发明人： George Georgiou ,  Everett Stone

IPC分类号： A61K38/50 ,  A61K47/60 ,  C12N9/78 ,  A61K33/24 ,  A61K47/64

CPC分类号： A61K38/50 ,  C12Y305/03001 ,  A61K47/60 ,  C12N9/78 ,  A61K33/24 ,  A61K47/64 ,  A61K38/00

摘要： Compositions and methods for the treatment of cancer are described, and, more preferably, to the treatment of cancers that do not express, or are otherwise deficient in, argininosuccinate synthetase, with enzymes that deplete L-Arginine in serum. In one embodiment, the present invention contemplates an arginase protein, such as a human Arginase I protein, comprising at least one amino acid substitution and a metal cofactor, said protein comprising an increased catalytic activity when compared with a native human Arginase I.