-
公开(公告)号:US20230089654A1
公开(公告)日:2023-03-23
申请号:US17821703
申请日:2022-08-23
申请人: The United States of America, as represented by the Secretary, Dept. of Health and Human Services
摘要: Modified natural killer (NK) or T cells expressing hematopoietic growth factor receptors are provided. In some embodiments, the NK cells or T cells express a thrombopoietin receptor or an erythropoietin receptor. Methods of treating a subject with cancer are also provided, including administering the modified NK cells or T cells to the subject in combination with a thrombopoietin receptor agonist or erythropoietin receptor agonist, and in some example, interleukin-2, particularly reduced or low-dose amounts of IL-2.
-
公开(公告)号:US20220372525A1
公开(公告)日:2022-11-24
申请号:US17848563
申请日:2022-06-24
申请人: The Broad Institute, Inc. , Massachusetts Institute of Technology , President and Fellows of Harvard College , Rutgers, The State University of New Jersey , Skolkovo Institute of Science and Technology , The United States of America, as represented by the Secretary, Dept. of Health and Human Services
发明人: Konstantin Severinov , Feng Zhang , Yuri I. Wolf , Sergey Shmakov , Ekaterina Semenova , Leonid Minakhin , Kira S. Makarova , Eugene Koonin , Silvana Konermann , Julia Joung , Jonathan S. Gootenberg , Omar O. Abudayyeh , Eric S. Lander
IPC分类号: C12N15/90 , C12N15/10 , C12N15/63 , C12N9/22 , C12N15/11 , C12N15/82 , C12N15/85 , C12N15/113
摘要: The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered RNA-targeting systems comprising a novel RNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA.
-
公开(公告)号:US20220313680A1
公开(公告)日:2022-10-06
申请号:US17706461
申请日:2022-03-28
申请人: Duke University , The United States of America, as Represented by the Secretary, Dept. of Health and Human Services
发明人: Nicole Calakos , Zachary F. Caffall , Joseph Rittiner , Min Shen , Jennifer T. Fox , Zhuyin Li
IPC分类号: A61K31/47 , C12Q1/6883 , G01N33/68 , C12Q1/68 , A61K31/17 , A61K31/4706 , A61P25/14 , A61K31/4725 , A61K38/05 , A61K31/427 , A61K31/472 , A61K31/497 , A61K31/513
摘要: The present disclosure provides methods and compositions for the treatment, identification, diagnosis, and prognosis of dystonia, or dystonia related disorders.
-
公开(公告)号:US11434268B2
公开(公告)日:2022-09-06
申请号:US16051062
申请日:2018-07-31
申请人: The United States of America, as represented by the Secretary, Dept. of Health and Human Services
发明人: Donald P. Bottaro , Fabiola Cecchi
IPC分类号: A61K38/18 , C07K14/475 , C07K14/52 , C07K14/71
摘要: The present invention provides variant VEGF polypeptides which have been altered in their C-terminal heparin binding region to lower their heparin binding affinity. These variants have been found to act as receptor antagonists for VEGF receptors and antagonize angiogenesis. These variants are useful to treat diseases characterized by pathological angiogenesis.
-
公开(公告)号:US20220184111A1
公开(公告)日:2022-06-16
申请号:US17682655
申请日:2022-02-28
申请人: The United States of America, as represented by the Secretary, Dept. of Health and Human Services
IPC分类号: A61K31/713 , A61K31/704 , A61K31/52 , A61K31/137 , A61K31/365 , A61K31/65 , A61K31/706 , A61K39/395 , A61K31/711 , A61K31/7064 , A61K31/437 , A61K31/505 , A61K31/712 , C07K16/28 , A61P39/00 , A61K31/351 , A61K31/787 , A61P35/00 , A61K31/513 , A61K45/06
摘要: Disclosed herein are methods of reducing cytotoxicity of a chemotherapeutic agent to non-cancer cells by administering to a subject with cancer an effective amount of an agent that inhibits CD47 signaling and a chemotherapeutic agent. Example disclosed methods reduce cardiotoxicity of a chemotherapeutic agent. Also disclosed are methods of increasing cytotoxicity of a chemotherapeutic agent in cancer cells by administering to a subject with a tumor an effective amount of an agent that inhibits CD47 signaling and a chemotherapeutic agent. In some embodiments, the inhibitor of CD47 signaling is administered to the subject before, during, or after the administration of the chemotherapeutic agent.
-
公开(公告)号:US20220168379A1
公开(公告)日:2022-06-02
申请号:US17673361
申请日:2022-02-16
申请人: The United States of America, as represented by the Secretary, Dept. of Health and Human Services
发明人: Alan T. Remaley , Scott M. Gordon
IPC分类号: A61K38/00 , A61K38/07 , A61K38/10 , A61K38/08 , A61K38/58 , A61K38/17 , A61K47/54 , A61K47/60
摘要: Described herein is the design and construction of a class of lipoprotein targeting protease inhibitors. Small peptides with protease inhibitor activity are conjugated to hydrophobic, lipoprotein targeting molecules using, for instance, amine reactive chemistry. Methods of use of the resultant lipoprotein targeting protease inhibitor (antiprotease) molecules are also described. Also described is the production and use of protease inhibitor enriched HDL particles, as well as A1AT-peptide-enriched HDL particles, and their use in various therapeutic contexts.
-
公开(公告)号:US11254735B2
公开(公告)日:2022-02-22
申请号:US16443415
申请日:2019-06-17
申请人: The United States of America, as represented by the Secretary, Dept. of Health and Human Services , Washington University
IPC分类号: C07K16/18 , A61K48/00 , G01N33/68 , C07K7/06 , C07K9/00 , C07K14/475 , C07K14/78 , A61K39/395 , A61K45/06 , B01J21/06 , B01J21/12 , B01J23/00 , B01J23/30 , B01J27/188 , B01J37/02 , C07C45/00 , C07C45/52 , C07C51/235 , C07C51/25 , C07C253/26 , A61P7/04 , A61K39/00
摘要: Provided herein are compositions for preventing, ameliorating, and/or reducing tissue ischemia and/or tissue damage due to ischemia, increasing blood vessel diameter, blood flow and tissue perfusion in the presence of vascular disease including peripheral vascular disease, atherosclerotic vascular disease, coronary artery disease, stroke and influencing other conditions, by suppressing CD47 and/or blocking TSP1 and/or CD47 activity or interaction. Influencing the interaction of CD47-TSP1 in blood vessels allows for control of blood vessel diameter and blood flow, and permits modification of blood pressure and cardiac function. Under conditions of decreased blood flow, for instance through injury or atherosclerosis, blocking TSP1-CD47 interaction allows blood vessels to dilate and increases blood flow, tissue perfusion and tissue survival.
-
公开(公告)号:US11078239B2
公开(公告)日:2021-08-03
申请号:US16344772
申请日:2017-10-25
申请人: The United States of America, as represented by the Secretary, Dept. of Health and Human Services , Institute for Research in Biomedicine
发明人: Baoshan Zhang , Guillaume Stewart-Jones , Tongqing Zhou , John Mascola , Kai Xu , Yongping Yang , Paul Thomas , Gwo-Yu Chuang , Li Ou , Peter Kwong , Yaroslav Tsybovsky , Wing-Pui Kong , Aliaksandr Druz , Davide Corti , Antonio Lanzavecchia
IPC分类号: A61K39/155 , C07K14/005 , A61P31/14 , C12N7/00
摘要: Embodiments of a recombinant human Parainfluenza Virus (hPIV) F ectodomain trimer stabilized in a prefusion conformation are provided. Also disclosed are nucleic acids encoding the hPIV F ectodomain trimer and methods of producing the hPIV F ectodomain trimer. Methods for inducing an immune response in a subject are also disclosed. In some embodiments, the method can be a method for treating or inhibiting a hPIV infection in a subject by administering a effective amount of the recombinant hPIV F ectodomain trimer to the subject.
-
9.
公开(公告)号:US10988778B2
公开(公告)日:2021-04-27
申请号:US15769294
申请日:2016-10-21
申请人: The United States of America, as represented by the Secretary, Dept. of Health and Human Services
发明人: Stephen G. Kaler
摘要: Disclosed herein are codon-optimized nucleic acids encoding a reduced-size ATP7A protein. Also disclosed are vectors and recombinant viruses (such as recombinant adeno-associated viruses) including the codon-optimized nucleic acids encoding the reduced-size ATP7A protein and compositions including the disclosed vectors and viruses. Further disclosed herein are methods of treating copper transport disorders, for example by administering a disclosed nucleic acid, vector, or recombinant virus to a subject with a copper transport disorder, such as Menkes disease, occipital horn syndrome, or ATP7A-related distal motor neuropathy.
-
公开(公告)号:US20210024523A1
公开(公告)日:2021-01-28
申请号:US16762998
申请日:2018-11-12
申请人: THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPT. OF HEALTH AND HUMAN SERVICES
IPC分类号: C07D471/10 , C07D211/58 , C07D241/04
摘要: Disclosed herein are a series of modafinil analogue compounds that bind with moderate to high affinity to the dopamine (DA) transporter (DAT) and several analogues also having affinity for the serotonin (5-HT) transporter (SERT) and/or sigma-1 receptor. Employing aminopiperidine, piperidineamino, spirobicyclodiaza, or substituted piperazine functional groups, desired dopamine transporter affinity has been retained along with improved metabolic stability over unsubstituted piperazine ring analogues. Importantly, these compounds have no predicted addictive liability. Also disclosed are methods for treating substance use disorders as well as other neuropsychiatric disorders such as ADHD, depression, narcolepsy, and cognitive impairment.
-
-
-
-
-
-
-
-
-