-
1.
公开(公告)号:US20190030038A1
公开(公告)日:2019-01-31
申请号:US16147153
申请日:2018-09-28
IPC分类号: A61K31/522 , A61K31/00
摘要: The present invention relates generally to the field of using udenafil or a pharmaceutically acceptable salt thereof in patients who have undergone the Fontan operation.
-
公开(公告)号:US11633485B2
公开(公告)日:2023-04-25
申请号:US16494101
申请日:2018-03-13
IPC分类号: A61K31/16 , A61K47/55 , A61P35/00 , A61K31/165 , A61K31/355 , A61K31/454
摘要: A co-drug according to Formula (I) or Formula (II) is provided, R—X—NH—CO—CO—OR1 (I) R—X—CO—O—CH2—CO—OR1 (II) wherein R is a tocol moiety, a tocol analog moiety, or a capsaicinoid moiety; X is a direct bond or a linking group; and OR1 is the residue of an anticancer or antirestenotic agent bearing at least one hydroxyl group by which the CO—OR1 ester linkage is formed. Nanoparticles that include the abovementioned co-drug are also provided, as well as a method of treating a cancer patient that includes administering an effective amount of the co-drug or nanoparticles.
-
公开(公告)号:US20230045199A1
公开(公告)日:2023-02-09
申请号:US17786147
申请日:2020-12-23
发明人: Gerd BLOBEL , Scott PESLAK , Junwei SHI
IPC分类号: C12N15/113 , A61P7/06 , A61K31/454 , A61K31/713
摘要: Methods and compositions for producing fetal hemoglobin and treating a hemoglobinopathy or thalassemia are disclosed.
-
公开(公告)号:US11491213B2
公开(公告)日:2022-11-08
申请号:US17305649
申请日:2021-07-12
发明人: Katherine A. High , Xavier Anguela
摘要: The invention relates to modified Factor IX coding sequence, expression cassette, vectors such as viral (e.g., lenti- or adeno-associated viral) vectors, and gene transfer methods and uses. In particular, to target Factor IX nucleic acid to cells, tissues or organs for expression (transcription) of Factor IX.
-
公开(公告)号:US11473084B2
公开(公告)日:2022-10-18
申请号:US15766549
申请日:2016-10-11
IPC分类号: C12N15/113 , C12N15/861 , C12N15/88 , C12N15/66 , C12N15/10 , A61K48/00 , A61P25/00 , C12N9/22
摘要: Compositions and methods are provided for the inhibition, treatment and/or prevention of Huntington's disease and related disorders.
-
公开(公告)号:US20220275402A1
公开(公告)日:2022-09-01
申请号:US17601581
申请日:2020-04-06
IPC分类号: C12N15/90 , C07K14/705 , C07K14/81 , C12N9/22 , A61K9/00
摘要: Compositions and methods for in utero gene editing in mammalian lung cells are disclosed.
-
公开(公告)号:US20220257653A1
公开(公告)日:2022-08-18
申请号:US17597580
申请日:2020-07-17
申请人: The Children's Hospital of Philadelphia , THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVIC , THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY
发明人: Kristopher BOSSE , John MARIS , David BARRETT , Jessica FOSTER , Dimiter DIMITROV , Crystal MACKALL , Robbie MAJZNER , ZHONGYU ZHU , Sabine HEITZENEDER , Dontcho V. JELEV
IPC分类号: A61K35/17 , C07K14/725 , C07K16/30 , C07K14/705 , A61P35/00
摘要: The present disclosure is directed to chimeric antigen receptors binding to Glypican 2, nucleic acids encoding the same, and cells expressing the same, and methods of using such cells to treat cancers that express or overexpress the Glypican 2 antigen.
-
公开(公告)号:US20220227824A1
公开(公告)日:2022-07-21
申请号:US17596622
申请日:2020-06-23
发明人: Ophir H. SHALEM , Katelyn SWEENEY
摘要: The present disclosure is directed to the use of SRR1 domain-containing protein (SRRD) or active fragment thereof to reduce protein aggregation, such as that occurring in neurodegenerative diseases.
-
公开(公告)号:US20220177878A1
公开(公告)日:2022-06-09
申请号:US17594651
申请日:2020-04-24
IPC分类号: C12N15/11 , C12N15/113 , C12N15/90 , C12N15/86 , C12N9/22
摘要: CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic diseases, such as spinocerebellar ataxia type 2, which is caused by mutations in the ATXN2 gene. Here, compositions and methods for the treatment of spinocerebellar taxia type 2 are provided. In some embodiments, a composition providing a vector encoding a guide RNA of the disclosure and a vector encoding a Cas9 protein or nuclease domain thereof of the disclosure are provided for use in the treatment of spinocerebellar ataxia type 2.
-
公开(公告)号:US20220151534A1
公开(公告)日:2022-05-19
申请号:US17438483
申请日:2020-03-13
摘要: Compositions and methods are provided for the detection and/or diagnosis of traumatic brain injury.
-
-
-
-
-
-
-
-
-