专利快速检索-快速检索全球专利，免费商用专利数据库-IPRDB

1. 发明申请

WO2020023533A1 DRY POWER FORMULATIONS FOR MESSENGER RNA 审中-公开
公开(公告)号：WO2020023533A1
公开(公告)日：2020-01-30
申请号：PCT/US2019/043074
申请日：2019-07-23
申请人： TRANSLATE BIO, INC.
发明人： KARVE, Shrirang , DEROSA, Frank , HEARTLEIN, Michael , PATEL, Zarna , SARODE, Ashish
IPC分类号： A61K9/00 , A61K9/127 , A61K9/16
摘要： The present invention provides stable, dry powder messenger RNA formulations for therapeutic use, and methods of making and using the same.

2. 发明申请

WO2017210599A1 ENGINEERED INFLUENZA ANTIGENIC POLYPEPTIDES AND IMMUNOGENIC COMPOSITIONS THEREOF 审中-公开
标题翻译：工程流感抗原多肽及其免疫原性组合物
公开(公告)号：WO2017210599A1
公开(公告)日：2017-12-07
申请号：PCT/US2017/035747
申请日：2017-06-02
申请人： SANOFI PASTEUR INC.
发明人： STRUGNELL, Tod , OLOO, Eliud
IPC分类号： C07K14/005 , C07K14/11 , A61K39/145
摘要： The present invention provides, among other things, a novel and improved method for generating "mosaic" influenza antigenic polypeptides including hemagglutinin (HA) and neuraminidase (NA) polypeptides based on unique combination of epitope patterns that maximize exposure to epitopes present across multiple HA or NA sequences and therefore improved influenza strain coverage. In particular, the present invention provides engineered influenza B hemagglutinin (HA) polypeptides that are comprised of novel combinations of protective epitopes and antigenic regions from multiple influenza B viral strains. Such engineered HA polypeptides have improved properties over HA polypeptides developed through conventional approaches that rely on consensus alignments of viral sequences.
摘要翻译：除其他之外，本发明提供了一种新颖且改进的用于生成“马赛克”图像的方法。基于表位模式的独特组合的流感抗原性多肽，包括血凝素（HA）和神经氨酸酶（NA）多肽，所述表位模式最大限度地暴露于跨多个HA或NA序列存在的表位，并因此改善流感病毒株覆盖度。具体而言，本发明提供工程化的B型流感血球凝集素（HA）多肽，其由包含来自多个B型流感病毒株的保护性表位和抗原性区域的新组合组成。这种工程化的HA多肽相对于通过依赖于病毒序列的共有序列比对的常规方法开发的HA多肽具有改进的性质。

3. 发明申请

WO2017087882A1 RECOMBINANT HUMAN C1 ESTERASE INHIBITOR AND USES THEREOF 审中-公开
标题翻译：重组人C1类酯酶抑制剂及其用途
公开(公告)号：WO2017087882A1
公开(公告)日：2017-05-26
申请号：PCT/US2016/062906
申请日：2016-11-18
申请人： SHIRE HUMAN GENETIC THERAPIES, INC.
发明人： NORTON, Angela, W. , COPPOLA, Germano
IPC分类号： A61K38/57 , C07K14/81
CPC分类号： A61K38/57 , C07K14/8121
摘要： The present invention provides, among other things, methods and compositions for treating complement mediated disease. In some embodiments, recombinant human C1 esterase inhibitor proteins having similar or longer half-life than native plasma-derived human C1 esterase inhibitor, and methods of making the same are provided. In some embodiments, the invention provides a method for administering an effective amount of a recombinant human C1 esterase inhibitor protein to an individual who is suffering from or susceptible to a complement-mediated disease such that at least one symptom or feature of said complement-mediated disease is prevented and/or reduced in intensity, severity, or frequency.
摘要翻译：本发明尤其提供了用于治疗补体介导的疾病的方法和组合物。在一些实施方案中，提供了具有与天然血浆衍生的人C1酯酶抑制剂相似或更长的半衰期的重组人C1酯酶抑制剂蛋白及其制备方法。在一些实施方案中，本发明提供了向患有或易患补体介导的疾病的个体施用有效量的重组人C1酯酶抑制剂蛋白以使得所述补体介导的至少一种症状或特征预防和/或减少疾病的强度，严重程度或频率。

4. 发明申请

WO2017015145A2 DISSOCIABLE TRANSITION-METAL NANOPARTICLES 审中-公开
标题翻译：不可转移的金属纳米粒子
公开(公告)号：WO2017015145A2
公开(公告)日：2017-01-26
申请号：PCT/US2016/042589
申请日：2016-07-15
申请人： SELUX DIAGNOSTICS, INC.
发明人： STERN, Eric , VACIC, Aleksandar , FLYER, Alec, Nathanson , SPEARS, Benjamin , CLARDY, Susan
IPC分类号： B01J31/18 , B82Y15/00 , G01N33/543 , G01N21/76 , G01N33/541 , G01N33/537 , G01N33/577 , G01N33/68
CPC分类号： B01J31/165 , B01J31/184 , B01J35/0013 , B01J35/023 , B01J2531/0258 , B01J2531/16 , B01J2531/62 , B01J2531/72 , B01J2531/842 , B01J2531/845 , B01J2531/847 , B82Y15/00 , G01N33/586 , G01N33/587
摘要： Assay methods for detecting analytes in samples involving the use of nanoparticles that comprise a signal inducing agent, e.g., a transition- metal catalyst. After binding to an analyte, the nanoparticle is dissociated by a trigger to release the signal inducing agent, which releases a detectable signal via a physical or chemical reaction. Also described herein are nanoparticles, and compositions and uses thereof, where the nanoparticle comprises chemiluminophore, a chemiluminophore precursor, a soluble absorber, or a soluble absorber precursor, that effects chemical reactions that serve as signal amplifiers.
摘要翻译：用于检测涉及使用包含信号诱导剂例如过渡金属催化剂的纳米颗粒的样品中的分析物的测定方法。在结合分析物之后，纳米颗粒通过触发剂解离以释放信号诱导剂，其通过物理或化学反应释放可检测的信号。本文还描述了纳米颗粒及其组合物和用途，其中纳米颗粒包含化学发光基团，化学发光原子前体，可溶性吸收剂或可溶性吸收剂前体，其实现用作信号放大器的化学反应。

5. 发明申请

WO2016100603A9 ENZYMATIC ACTIVITY ASSAYS FOR I2S 审中-公开
公开(公告)号：WO2016100603A9
公开(公告)日：2016-06-23
申请号：PCT/US2015/066263
申请日：2015-12-17
申请人： SHIRE HUMAN GENETIC THERAPIES, INC.
发明人： BERNHARDT, Peter , TIEN, Claire , FIELDING, Elisha , CHHAJLANI, Vijay
IPC分类号： C12Q1/34
摘要： In certain embodiments of the present invention, kinetic parameters of I2S enzyme are determined. In some instances, a sample including I2S enzyme is incubated under defined conditions, with a series of determined amounts of I2S substrate including a detectable label. Following incubation, the reaction mixture can be analyzed, e.g., by a method including chromatography. A detection unit can be used to measure the presence of the detectable label. Data can be analyzed to determine kinetic parameters.

6. 发明申请

WO2016100556A1 ENZYMATIC ACTIVITY ASSAYS FOR GLUCOCEREBROSIDASE 审中-公开
标题翻译：葡萄糖苷酶的酶活性测定
公开(公告)号：WO2016100556A1
公开(公告)日：2016-06-23
申请号：PCT/US2015/066168
申请日：2015-12-16
申请人： SHIRE HUMAN GENETIC THERAPIES, INC.
发明人： BERNHARDT, Peter , YEN, Chen-Chung Willy , CHHAJLANI, Vijay
IPC分类号： C12Q1/34
CPC分类号： C12Q1/40 , C12Q1/34 , C12Y302/01045 , G01N2333/924 , G01N2500/20
摘要： The present invention provides, among other things, methods and compositions for determining enzyme kinetic parameters (e.g., Vmax, Km, and specific activity, etc.) indicative of clinically relevant properties of glucocerebrosidase using a physiologically relevant substrate, in particular, a substrate that is representative of substrates that typically accumulate in patients suffering from Gaucher disease such as glucosylceramide. Thus, the present invention is particularly useful to measure a kinetic parameter relating to the activity of glucocerebrosidase in a drug substance, drug product, and stability sample for enzyme replacement therapy.
摘要翻译：本发明尤其提供了用于使用生理学相关底物，特别是基质，其指示使用生理相关底物指示葡糖脑苷脂酶的临床相关性质的酶动力学参数（例如，Vmax，Km和比活性等）的方法和组合物，是典型地积聚在患有戈谢病例如葡萄糖神经酰胺的患者中的底物的代表。因此，本发明特别可用于测量与药物物质，药物产品中的葡萄糖脑苷脂酶的活性和酶替代疗法的稳定性样品有关的动力学参数。

7. 发明申请

WO2016077775A1 DETERMINATION OF GLYCOSAMINOGLYCAN LEVELS BY MASS SPECTROMETRY 审中-公开
标题翻译：通过质谱法测定谷氨酰胺糖苷含量
公开(公告)号：WO2016077775A1
公开(公告)日：2016-05-19
申请号：PCT/US2015/060714
申请日：2015-11-13
申请人： SHIRE HUMAN GENETIC THERAPIES, INC.
发明人： NAIMY, Hicham , QIU, Yongchang , HASLETT, Patrick, Anthony, John , BARBIER, Ann
IPC分类号： G01N33/68 , C12Q1/34
CPC分类号： G01N33/6848 , C12Q1/34 , G01N33/6893 , G01N2400/40 , G01N2560/00 , G01N2800/042 , G01N2800/52
摘要： Detecting glycosaminoglycans (GAGs) and/or determining the level of one or more glycosaminoglycans can be useful, e.g., in identifying or monitoring various medical conditions, the status of patients having various medical conditions, and/or the response to treatment of individuals having various medical conditions. The present invention provides methods for detecting glycosaminoglycans and/or determining the level of glycosaminoglycans through the use of, e.g., mass spectrometry.
摘要翻译：检测糖胺聚糖（GAG）和/或确定一种或多种糖胺聚糖的水平可以是有用的，例如在鉴定或监测各种医学状况，具有各种医学状况的患者的状态和/或对具有各种各样的患者的治疗的反应医疗条件。本发明提供了通过使用例如质谱法检测糖胺聚糖和/或测定糖胺聚糖水平的方法。

8. 发明申请

WO2016025523A1 LYSOSOMAL TARGETING AND USES THEREOF 审中-公开
标题翻译：物理指标及其用途
公开(公告)号：WO2016025523A1
公开(公告)日：2016-02-18
申请号：PCT/US2015/044718
申请日：2015-08-11
申请人： SHIRE HUMAN GENETIC THERAPIES, INC.
发明人： CONCINO, Michael F. , STRACK-LOGUE, Bettina , MEIYAPPAN, Muthuraman , NORTON, Angela W. , ZHANG, Bohong , ISKENDERIAN, Andrea , FENG, Jianwen , HOLMES, Kevin , PAN, Jing
IPC分类号： A61K47/48 , A61P43/00
CPC分类号： A61K38/47 , A61K47/64 , C07K14/475 , C07K14/70571 , C07K16/18 , C07K2319/06 , C12N9/2402 , C12Y302/0105
摘要： The invention provides compositions and methods for effective lysosomal targeting mediated by SORT1. In particular, the compositions and methods provided by the invention may be used to treat lysosomal storage diseases such as Sanfilippo syndrome type B.
摘要翻译：本发明提供了由SORT1介导的有效溶酶体靶向的组合物和方法。特别地，本发明提供的组合物和方法可用于治疗溶酶体贮积病，例如B型Sanfilippo综合征。

9. 发明申请

WO2015200675A1 GENERATION OF MANNOSE-6-PHOSPHATE CONTAINING RECOMBINANT ALPHA-N-ACETYL GLUCOSAMINIDASE 审中-公开
标题翻译：含有重组脂肪酸N-乙酰谷氨酰胺糖苷酶的曼诺-6-磷酸酶的产生
公开(公告)号：WO2015200675A1
公开(公告)日：2015-12-30
申请号：PCT/US2015/037756
申请日：2015-06-25
申请人： SHIRE HUMAN GENETIC THERAPIES, INC.
发明人： ZHANG, Bohong , CONCINO, Michael F. , NORTON, Angela W. , MEIYAPPAN, Muthuraman , LUNDBERG, Dianna
IPC分类号： A61K38/46 , C12P21/00 , C12N9/24
CPC分类号： C12N9/2402 , A61K38/47 , C12P21/005 , C12Y302/0105
摘要： A method of producing mannose-6-phosphate (M6P)-containing recombinant alpha-N-acetyl-glucosaminidase (Naglu), including the steps of providing a high mannose containing recombinant Naglu protein; and contacting the high mannose containing recombinant Naglu protein with N-acetyl-glucosamine-1-phosphotransferase (GNPT) under conditions that permit phosphorylation of one or more mannose residues on the recombinant Naglu protein, thereby generating M6P-containing recombinant Naglu. A mannose-6-phosphate (M6P)-containing recombinant alpha-N-acetyl-glucosaminidase (Naglu) protein produced by this method, a composition comprising such Naglu protein, and a method of treating Sanfilippo Syndrome Type B (MPS IIIB) including administering to a subject in need of treatment this composition.
摘要翻译：一种制备含有甘露糖-6-磷酸（M6P）的重组α-N-乙酰氨基葡糖苷酶（Naglu）的方法，包括以下步骤：提供含有高甘露糖的重组Naglu蛋白; 并在允许在重组Naglu蛋白上磷酸化一个或多个甘露糖残基的条件下使含有高甘露糖的重组Naglu蛋白与N-乙酰葡糖胺-1-磷酸转移酶（GNPT）接触，由此产生含M6P的重组Naglu。通过该方法制备的含有甘露糖-6-磷酸（M6P）的重组α-N-乙酰氨基葡糖苷酶（Naglu）蛋白，包含这种Naglu蛋白的组合物和治疗Sanfilippo B型综合征（MPS IIIB）的方法，包括施用到需要治疗的一个主题这个组成。

10. 发明申请

WO2015148247A1 MRNA THERAPY FOR THE TREATMENT OF OCULAR DISEASES 审中-公开
标题翻译：用于治疗眼部疾病的MRNA治疗
公开(公告)号：WO2015148247A1
公开(公告)日：2015-10-01
申请号：PCT/US2015/021403
申请日：2015-03-19
申请人： SHIRE HUMAN GENETIC THERAPIES, INC.
发明人： CALIAS, Pericles , DEROSA, Frank , HEARTLEIN, Michael
IPC分类号： C12N15/02 , C12P21/06 , A61K48/00
CPC分类号： A61K48/0066 , A61K9/0048 , A61K9/127 , A61K9/51 , A61K48/005 , A61K48/0075
摘要： The present invention provides, among other things, a method of ocular delivery of messenger RNA (mRNA), comprising administering into an eye of a subject in need of delivery a composition comprising an mRNA encoding a protein, such that the administration of the composition results in expression of the protein encoded by the mRNA in the eye.
摘要翻译：本发明尤其提供了一种信使RNA（mRNA）的眼部递送方法，包括向需要递送的受试者的眼睛施用包含编码蛋白质的mRNA的组合物，使得施用组合物结果表达由mRNA在mRNA中编码的蛋白质。

你已经成功收藏专利！

检索式保存成功!

IPRDB

热门服务

关于我们

友情链接

联系方式