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1. 发明申请

WO2023028441A1 ENGINEERED ITR SEQUENCES AND METHODS OF USE 审中-公开
公开(公告)号：WO2023028441A1
公开(公告)日：2023-03-02
申请号：PCT/US2022/075187
申请日：2022-08-19
申请人： BIOVERATIV THERAPEUTICS INC. , MAGHODIA, Ajay , ZAKAS, Philip
发明人： MAGHODIA, Ajay , ZAKAS, Philip
IPC分类号： C07K14/755 , C12N9/64 , C12N15/86 , C07K14/75
摘要： The present disclosure provides nucleic acid molecules comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette encoding a target sequence. In some embodiments, the first ITR and/or the second ITR is an ITR of a non-adeno-associated virus (AAV). Also disclosed are methods of using the nucleic acid molecules in gene therapy applications.

2. 发明申请

WO2020198509A2 MODIFIED OLIGONUCLEOTIDES WITH INCREASED STABILITY 审中-公开
公开(公告)号：WO2020198509A2
公开(公告)日：2020-10-01
申请号：PCT/US2020/025017
申请日：2020-03-26
申请人： UNIVERSITY OF MASSACHUSETTS
发明人： KHVOROVA, Anastasia , ROUX, Loïc Maurice René Jean , YAMADA, Ken
IPC分类号： C12N15/11
摘要： This disclosure relates to novel modified oligonucleotides. Novel modified siRNA are also provided.

3. 发明申请

WO2019209956A1 ARTIFICIAL EXOSOME COMPOSITION AND RELATED METHODS 审中-公开
公开(公告)号：WO2019209956A1
公开(公告)日：2019-10-31
申请号：PCT/US2019/028919
申请日：2019-04-24
申请人： UNIVERSITY OF MASSACHUSETTS
发明人： HARASZTI, Reka, Agnes , KHVOROVA, Anastasia , ARONIN, Neil
IPC分类号： A61K35/28 , C12N15/88 , C12N15/861
摘要： Novel artificial exosomes and methods for producing novel artificial exosomes are provided. Methods of delivering cargo molecules to a cell using artificial exosomes are also provided.

4. 发明申请

WO2019140452A1 MODULATORS OF DNM2 EXPRESSION 审中-公开
公开(公告)号：WO2019140452A1
公开(公告)日：2019-07-18
申请号：PCT/US2019/013689
申请日：2019-01-15
申请人： IONIS PHARMACEUTICALS, INC.
发明人： FREIER, Susan, M. , BUI, Huynh-Hoa , MURRAY, Susan, F. , MONIA, Brett, P. , GUO, Shuling
IPC分类号： C12N15/113 , A61K31/7115 , A61K31/712 , A61K31/7125
摘要： The present embodiments provide methods, compounds, and compositions useful for inhibiting DNM2 expression, which may be useful for treating, preventing, or ameliorating a disease associated with DNM2.

5. 发明申请

WO2018119299A1 HUMANIZED CXCR3 ANTIBODIES WITH DEPLETING ACTIVITY AND METHODS OF USE THEREOF 审中-公开
公开(公告)号：WO2018119299A1
公开(公告)日：2018-06-28
申请号：PCT/US2017/068003
申请日：2017-12-21
申请人： SANOFI , BRONDYK, William, H.
发明人： BRONDYK, William, H. , CHU, Ruiyin , CONNORS, Timothy, D. , PARK, Sunghae , QIU, Huawei , YOUD, Michele
IPC分类号： C07K16/28 , A61P37/06
CPC分类号： A61K39/395 , A61K38/195 , A61K2039/505 , A61P3/10 , A61P17/06 , A61P37/06 , C07K16/2866 , C07K2317/14 , C07K2317/24 , C07K2317/41 , C07K2317/52 , C07K2317/524 , C07K2317/56 , C07K2317/565 , C07K2317/72 , C07K2317/73 , C07K2317/76 , C07K2317/90 , C07K2317/92 , C07K2317/94
摘要： Provided are humanized CXCR3 antibodies and methods of using the antibodies to treat CXCR3-associated disorders such as type 1 diabetes mellitus (TID), particularly new-onset TID, and psoriasis. In certain embodiments, the anti-CXCR3 antibodies are humanized anti-human CXCR3 antibodies with enhanced effector function against cells expressing CXCR3 on their surface. Also provided are nucleic acid sequences encoding the antibodies, and pharmaceutical compositions comprising the antibodies.

6. 发明申请

WO2018013640A1 PROCESS OF DELIVERING SMALL RNAS TO SPERM 审中-公开
标题翻译：向精子提供小RNA的过程
公开(公告)号：WO2018013640A1
公开(公告)日：2018-01-18
申请号：PCT/US2017/041647
申请日：2017-07-12
申请人： UNIVERSITY OF MASSACHUSETTS
发明人： RANDO, Oliver , SHARMA, Upasna , CONINE, Colin
IPC分类号： C12N5/076 , C12N15/11 , C12Q1/68
摘要： Methods and compositions directed to altering a population of sRNAs in a sperm using vesicles isolated from an epididymosome are provided. Methods and compositions directed to altering a population of sRNAs in an oocyte using vesicles isolated from an epididymosome are also provided. Methods for altering an sRNA population in a sperm or an oocyte can be used to prevent, or reduce the severity of, a disease, disorder, or condition that would otherwise be inherited by progeny. For example, certain epigenetic inherited conditions due to paternal effects, such as certain metabolic and stress disorders and conditions, can be ameliorated in progeny using sperm or oocytes having an altered sRNA population.
摘要翻译：提供了使用从附睾体分离的囊泡来改变精子中的sRNA群体的方法和组合物。还提供了使用从附睾体分离的囊泡来改变卵母细胞中的sRNA群体的方法和组合物。用于改变精子或卵母细胞中sRNA群体的方法可以用于预防或降低否则会由子代遗传的疾病，病症或病症的严重程度。例如，由于父系效应（例如某些代谢和应激障碍和病症）的某些表观遗传条件可以在使用具有改变的sRNA群体的精子或卵母细胞的后代中得到改善。

7. 发明申请

WO2017062724A1 EARLY POST-TRANSFECTION ISOLATION OF CELLS (EPIC) FOR BIOLOGICS PRODUCTION 审中-公开
标题翻译：用于生物生产的细胞的早期转移分离（EPIC）
公开(公告)号：WO2017062724A1
公开(公告)日：2017-04-13
申请号：PCT/US2016/055918
申请日：2016-10-07
申请人： GENZYME CORPORATION
发明人： CAIRNS, Victor, R. , DEMARIA, Christine , VITKO, Jason
IPC分类号： C12N15/10 , G01N33/537
CPC分类号： C12N15/85 , C07K16/00 , C07K2317/10 , C12N15/1086 , C12N2510/02 , G01N33/5044 , G01N2333/70592
摘要： Provided herein are methods for selecting a population of cells expressing a target polypeptide. In some aspects, the disclosure provides methods for sorting and selecting populations of transfected host cells based on their early expression of a selectable polypeptide. In certain embodiments, the sorting is performed using fluorescence-activated cell sorting or magnetic-activated cell sorting based on the selectable polypeptide. Such selection methods can be further utilized to generate clonal populations of producer cells, e.g. for large-scale manufacturing of a target polypeptide of interest.
摘要翻译：本文提供了选择表达目标多肽的细胞群的方法。在一些方面，本公开提供了基于其可选择多肽的早期表达来分选和选择转染的宿主细胞群体的方法。在某些实施方案中，使用基于可选择多肽的荧光激活细胞分选或磁激活细胞分选进行分选。这样的选择方法可以进一步用于产生生产细胞的克隆群体，例如用于大规模制造感兴趣的靶多肽。

8. 发明申请

WO2016161388A1 FULLY STABILIZED ASYMMETRIC SIRNA 审中-公开
标题翻译：完全稳定的不对称SIRNA
公开(公告)号：WO2016161388A1
公开(公告)日：2016-10-06
申请号：PCT/US2016/025753
申请日：2016-04-01
申请人： UNIVERSITY OF MASSACHUSETTS
发明人： KHVOROVA, Anastasia , ARONIN, Neil , HASSLER, Matthew , ALTERMAN, Julia
IPC分类号： C12N15/11 , C12N15/113 , A61P43/00 , A61P25/14
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/343 , C12N2310/344 , C12N2310/346 , C12N2310/3515 , C12N2320/51 , C12N2320/53 , C12Y207/10001 , C12N2310/3521 , C12N2310/322 , C12N2310/3533
摘要： Provided herein are self-delivering oligonucleotides that are characterized by efficient RISC entry, minimum immune response and off-target effects, efficient cellular uptake without formulation, and efficient and specific tissue distribution.
摘要翻译：本文提供的是自身递送的寡核苷酸，其特征在于有效的RISC进入，最小的免疫应答和脱靶效应，有效的细胞摄取而不需要配制，以及有效和特定的组织分布。

9. 发明申请

WO2016141245A1 MODIFIED-IGG ANTIBODIES THAT BIND TRANSFORMING GROWTH FACTOR-BETA1 WITH HIGH AFFINITY, AVIDITY AND SPECIFICITY 审中-公开
标题翻译：具有高亲和力，高度和特异性的BIND转化生长因子-ATA的修饰型IGG抗体
公开(公告)号：WO2016141245A1
公开(公告)日：2016-09-09
申请号：PCT/US2016/020780
申请日：2016-03-03
申请人： GENZYME CORPORATION
发明人： QIU, Huawei , BIRD, Julie
IPC分类号： C07K16/22
CPC分类号： C07K16/22 , C07K2317/14 , C07K2317/21 , C07K2317/34 , C07K2317/52 , C07K2317/522 , C07K2317/53 , C07K2317/55 , C07K2317/56 , C07K2317/567 , C07K2317/622 , C07K2317/76 , C07K2317/92 , C07K2317/94
摘要： A modified IgG antibody binds and neutralizes TGFß1 selectively and with high affinity and avidity. The modified IgG antibody comprises four polypeptide chains and may comprise modifications to the elbow regions of the polypeptide chains. The modified IgG antibody may comprise the same VH and VL domains or CDR regions as metelimumab. The modified IgG antibody is useful in therapeutic and diagnostic applications.
摘要翻译：修饰的IgG抗体以高亲和力和亲和力选择性地结合并中和TGFβ1。修饰的IgG抗体包含四条多肽链，并且可以包括对多肽链的肘部区域的修饰。修饰的IgG抗体可以包含与metelimumab相同的VH和VL结构域或CDR区。经修饰的IgG抗体可用于治疗和诊断应用。

10. 发明申请

WO2016077763A1 BIO-ENGINEERED HYPER-FUNCTIONAL "SUPER" HELICASES 审中-公开
标题翻译：生物工程超级功能“超级”直升机
公开(公告)号：WO2016077763A1
公开(公告)日：2016-05-19
申请号：PCT/US2015/060693
申请日：2015-11-13
申请人： THE BOARD OF TRUSTEES OF THE UNIVERSITY OF ILLINOIS
发明人： HA, Taekjip , ARSLAN, Sinan
IPC分类号： C12M1/00
CPC分类号： C12N9/14 , C12N9/90 , C12P19/34 , C12Y306/04012
摘要： Conformationally-constrained helicases having improved activity and strength are provided. Methods of making conformationally-constrained helicases having improved activity and strength are provided. Methods of using conformationally-constrained helicases having improved activity and strength are provided. The present invention is based on the discovery of novel modified helicases that show dramatically enhanced helicase activity and increased strength as compared to unmodified helicases. As described further herein, it has been surprisingly discovered that, by controlling the conformation of certain subdomains such that the helicase remains in a closed form (e.g., by covalently crosslinking the 2B domain to the 1A domain or the 1B domain in a Rep helicase), a highly active and strong form of the helicase is achieved
摘要翻译：提供具有改善的活动和强度的构象约束解旋酶。提供了具有改进的活性和强度的构象约束解旋酶的方法。提供了具有改善活性和强度的构象约束解旋酶的方法。本发明基于与未修饰的解旋酶相比显示显着增强的解旋酶活性和增加的强度的新型改性解旋酶的发现。如本文进一步描述的，已经惊奇地发现，通过控制某些亚结构域的构象，使得解旋酶保持为封闭形式（例如，通过共价交联2B结构域到Rep解旋酶中的1A结构域或1B结构域），达到高度活跃和强力的解旋酶的形式

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