-
公开(公告)号：WO2023034870A2
公开(公告)日：2023-03-09
申请号：PCT/US2022/075772
申请日：2022-08-31
发明人： RIGO, Frank , DWYER, Chrissa, A.
IPC分类号： C12N15/113 , A61K47/06 , A61K47/26 , A61K47/68 , A61K31/7088 , A61K47/549 , A61K47/6807 , A61K47/6849 , C12N15/1137 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/345 , C12N2310/346 , C12N2310/351
摘要： Provided are oligomeric compounds, methods, and pharmaceutical compositions for DMPK the amount or activity of DMPK RNA in a cell or animal, and in certain instances reducing the amount of DMPK protein in a cell or animal. Such oligomeric compounds, methods, and pharmaceutical compositions are useful to treat type 1 myotonic dystrophy.
-
2.发明申请
公开(公告)号：WO2021202511A2
公开(公告)日：2021-10-07
申请号：PCT/US2021/024858
申请日：2021-03-30
发明人： SOUNDARAPANDIAN, Mangala Meenakshi , MCININCH, James D. , FISHILEVICH, Elane , CASTORENO, Adam , KAITTANIS, Charalambos , SCHLEGEL, Mark K. , FARLEY, Jonathan Edward , ZUBER, Jeffrey
IPC分类号： A61P25/28 , C12N15/113 , A61P25/16 , C12N2310/14 , C12N2310/312 , C12N2310/315 , C12N2310/3183 , C12N2310/343 , C12N2310/345 , C12N2310/346 , C12N2320/11
摘要： The disclosure relates to double stranded ribonucleic acid interference (dsRNAi) agents and compositions targeting a microtubule-associated protein tau (MAPT) gene, as well as methods of inhibiting expression of a MAPT gene and methods of treating subjects having a MAPT-associated disease or disorder, e.g., Alzheimer's disease, frontotemporal dementia, progressive supranuclear palsy, or other tauopathies, using such dsRNAi agents and compositions.
-
公开(公告)号：WO2022271836A2
公开(公告)日：2022-12-29
申请号：PCT/US2022/034539
申请日：2022-06-22
申请人： ACURASTEM, INC.
IPC分类号： C12N15/113 , A61P25/00 , A61K31/7115 , A61K31/712 , A61K31/7125 , A61K31/713 , C12N15/1137 , C12N2310/11 , C12N2310/31 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/345 , C12N2310/346 , C12N2320/32 , C12Y207/0115
摘要： The present invention relates to PIKFYVE antisense oligonucleotides (ASOs), pharmaceutical compositions containing them, and methods for treating, inhibiting, suppressing, and preventing neurological diseases with them.
-
公开(公告)号：WO2021174031A2
公开(公告)日：2021-09-02
申请号：PCT/US2021/019954
申请日：2021-02-26
发明人： RIGO, Frank , JAFAR-NEJAD, Paymaan
IPC分类号： C12N15/113 , C07H21/04 , C07H19/00 , C12N2310/11 , C12N2310/315 , C12N2310/322 , C12N2310/3341 , C12N2310/345 , C12N2310/346
摘要： Provided are compounds, methods, and pharmaceutical compositions for modulating splicing of a pre-mRNA in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a disease or disorder.
-
公开(公告)号：WO2023057746A1
公开(公告)日：2023-04-13
申请号：PCT/GB2022/052509
申请日：2022-10-04
发明人： WU, Qianxin , TREHAN, Ashutosh
IPC分类号： C12N9/22 , C12N15/113 , C12Q1/6806 , C12N15/10 , C12N15/1093 , C12N15/11 , C12N2310/20 , C12N2310/345 , C12N2330/31 , C12Y301/21004
摘要： The invention relates to a method of generating a library of polynucleotide molecules encoding guide RNAs (gRNAs) from target polynucleotide(s). The invention also relates to a library of polynucleotide molecules encoding gRNAs obtainable by the aforementioned method, and a gRNA library generation kit thereof.
-
公开(公告)号：WO2021252799A2
公开(公告)日：2021-12-16
申请号：PCT/US2021/036873
申请日：2021-06-10
IPC分类号： A61K31/7088 , A61K31/711 , A61K31/7115 , A61K31/712 , C12N15/113 , C12N2310/315 , C12N2310/322 , C12N2310/3231 , C12N2310/341 , C12N2310/345 , C12N2310/346
摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of MSH3 RNA in a cell or subject, and in certain instances reducing the amount of MSH3 protein in a cell or subject. These compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a repeat expansion disease. Such symptoms and hallmarks include brain atrophy, muscle atrophy, nerve degeneration, uncontrolled movement, seizure, tremors, muscle weakness, muscle cramping, difficulty swallowing, difficulty speaking, decreased memory, decreased cognition, anxiety, and depression. Non-limiting examples of repeat expansion diseases that benefit from these compounds, methods, and pharmaceutical compositions are myotonic dystrophy (DM1 and DM2), amyotrophic lateral sclerosis, frontotemporal dementia, Huntington's disease, various polyglutamine disorders, Friedrich's ataxia, Fragile X syndrome, or spinocerebellar ataxia (e.g., SCA1, SCA2, SCA3, SCA6, SCA7, SCA8, SCA10, or SCA17).
-
公开(公告)号：WO2021231606A2
公开(公告)日：2021-11-18
申请号：PCT/US2021/032038
申请日：2021-05-12
发明人： SONTHEIMER, Erik Joseph , KHVOROVA, Anastasia , WATTS, Jonathan Kenneth , AMRANI, Nadia , CHEN, Zexiang , HASSLER, Matthew , MORENO, Dimas Echeverria , ALTERMAN, Julia Frances , WOLFE, Scot , YAMADA, Ken , DEVI, Gitali , ZHANG, Han
IPC分类号： C12N15/113 , C12N15/11 , C12N15/907 , C12N2310/20 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/345 , C12N2310/346 , C12N2310/3515 , C12N2800/80 , C12N9/22
摘要： Chemically modified crRNAs and tracrRNAs are provided. crRNAs and tracrRNAs with 5' and/or 3' conjugated moieties are provided. crRNAs and tracrRNAs with modifications in the repeat region of the crRNA or the anti-repeat region of the tracrRNA are provided. Methods of using the crRNAs and tracrRNAs for genome editing with a CRISPR nuclease and kits for performing the same are also provided.
-
8.发明申请
公开(公告)号：WO2021150969A8
公开(公告)日：2021-07-29
申请号：PCT/US2021/014729
申请日：2021-01-22
发明人： MCININCH, James D. , GILBERT, Jason , CASTORENO, Adam , DANG, Lan Thi Hoang , LEBLANC, Sarah , PENG, Haiyan , KAITTANIS, Charalambos , SOUNDARAPANDIAN, Mangala Meenakshi
IPC分类号： C12N15/113 , A61K31/712 , A61K31/7125 , A61K48/00 , A61K31/713 , A61P25/28 , C12N15/1137 , C12N2310/14 , C12N2310/312 , C12N2310/315 , C12N2310/343 , C12N2310/345 , C12N2310/346 , C12N2310/3515 , C12N2320/11 , C12N2320/32
摘要： The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a leucine-rich repeat kinase 2 (LRRK2) gene, as well as methods of inhibiting expression of a LRRK2 gene and methods of treating subjects having a LRRK2-associated disease or disorder, e.g., Parkinson's disease, using such dsRNAi agents and compositions.
-
9.发明申请
公开(公告)号：WO2023059629A1
公开(公告)日：2023-04-13
申请号：PCT/US2022/045664
申请日：2022-10-04
申请人： AMGEN INC.
发明人： LU, Jiamiao , OLLMANN, Michael , COLLINS, Patrick , LI, Chi-Ming , WANG, Songli
IPC分类号： C12N15/113 , C12N15/1137 , C12N2310/11 , C12N2310/14 , C12N2310/20 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/3341 , C12N2310/345 , C12N2310/346 , C12N2310/3515 , C12Y203/01051 , C12Y306/05002
摘要： The present invention relates to compositions and methods for enhancing the gene silencing activity of oligonucleotide compounds. In particular, the invention relates to inhibiting the expression or activity of suppressor proteins, such as RAB18, ZW10, STX18, SCFD2, NAPG, SAMD4B, or VPS37A, to increase the efficacy of ligand-conjugated oligonucleotide compounds in reducing the expression of target genes in a cell.
-
10.发明申请
公开(公告)号：WO2023015264A1
公开(公告)日：2023-02-09
申请号：PCT/US2022/074554
申请日：2022-08-04
IPC分类号： C12N15/113 , A61K31/712 , A61K31/7125 , A61K31/713 , A61P35/00 , A61K35/17 , A61K47/551 , C12N15/1138 , C12N2310/14 , C12N2310/3125 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3341 , C12N2310/335 , C12N2310/343 , C12N2310/344 , C12N2310/345 , C12N2310/346 , C12N2310/3515
摘要： The disclosure relates, in some aspects, to compositions comprising oligonucleotide agents capable of inhibiting suppression of an immune response by reducing expression of one or more genes involved with an immune suppression mechanism in natural killer (NK) cells, and methods of using the same. In some embodiments, compositions and methods described by the disclosure are useful as immunogenic modulators for treating cancer.
-
-
-
-
-
-
-
-
-
搜索结果
14 条记录 (耗时 0.012 秒)
