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公开(公告)号：US20130295077A1
公开(公告)日：2013-11-07
申请号：US13892076
申请日：2013-05-10
Applicant: Shire Human Genetic Therapies, Inc.
Inventor： Michael F. Concino , Pericles Calias , Jing Pan , Kevin Holmes , Paolo Martini , Alla Romashko , Muthuraman Meiyappan , Bohong Zhang , Andrea Iskenderian , Dianna Lundberg , Angela Norton , Bettina Strack-Logue , Huang Yan , Mary Alessandrini , Richard Pfeifer
CPC classification number: A61K38/47 , A61K9/0019 , A61K9/0085 , A61K9/08 , A61K9/19 , A61K35/76 , A61K35/761 , A61K38/46 , A61K38/465 , A61K47/02 , A61K47/26 , C07K14/65 , C12N9/2402 , C12N9/2437 , C12Y301/06008 , C12Y301/06013 , C12Y302/01045 , C12Y302/01046 , C12Y302/0105 , C12Y310/01001
Abstract: Among other things, the present invention provides methods and compositions of treating Sanfilippo syndrome type B (Sanfilippo B) by, e.g., intrathecal (IT) administration of a Naglu protein. A suitable Naglu protein can be a recombinant, gene-activated or natural protein. In some embodiments, a suitable Naglu protein is a recombinant Naglu protein. In some embodiments, a recombinant Naglu protein is a fusion protein containing a Naglu domain and a lysosomal targeting moiety. In some embodiments, the lysosomal targeting domain is an IGF-II moiety.
Abstract translation: 除此之外，本发明提供了通过例如鞘内（IT）施用Naglu蛋白来治疗Sanfilippo综合征B型（Sanfilippo B）的方法和组合物。 合适的Naglu蛋白可以是重组的，基因激活的或天然的蛋白质。 在一些实施方案中，合适的Naglu蛋白是重组Naglu蛋白。 在一些实施方案中，重组Naglu蛋白是含有Naglu结构域和溶酶体靶向部分的融合蛋白。 在一些实施方案中，溶酶体靶向结构域是IGF-II部分。
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公开(公告)号：US20120076767A1
公开(公告)日：2012-03-29
申请号：US13094321
申请日：2011-04-26
Applicant: Stefan Nilsson
Inventor： Stefan Nilsson
CPC classification number: A61K38/45 , A61K38/465 , A61K38/47 , C07K1/34 , C07K1/36 , C12N9/1085 , C12Y205/01061 , C12Y301/06001 , C12Y301/06008 , C12Y302/01024 , C12Y302/01046
Abstract: The present invention comprises a method of concentrating a composition comprising a polypeptide of interest and the use of such a concentrated composition for the treatment of diseases in mammals, in particular by subcutaneous injection.
Abstract translation: 本发明包括浓缩包含感兴趣的多肽的组合物和使用这种浓缩组合物用于治疗哺乳动物疾病，特别是通过皮下注射的方法。
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公开(公告)号：US20090246187A1
公开(公告)日：2009-10-01
申请号：US12295848
申请日：2007-04-04
Applicant: Stefan Nilsson
Inventor： Stefan Nilsson
CPC classification number: A61K38/45 , A61K38/465 , A61K38/47 , C07K1/34 , C07K1/36 , C12N9/1085 , C12Y205/01061 , C12Y301/06001 , C12Y301/06008 , C12Y302/01024 , C12Y302/01046
Abstract: The present invention comprises a method of concentrating a composition comprising a polypeptide of interest and the use of such a concentrated composition for the treatment of diseases in mammals, in particular by subcutaneous injection.
Abstract translation: 本发明包括浓缩包含感兴趣的多肽的组合物和使用这种浓缩组合物用于治疗哺乳动物疾病，特别是通过皮下注射的方法。
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公开(公告)号：US20230310652A1
公开(公告)日：2023-10-05
申请号：US17926678
申请日：2021-05-19
Applicant: NEUWAY Pharma GmbH
Inventor： Victoria DEMINA , Oliver ERNST , Anke BONSE , Markus STAPF
CPC classification number: A61K48/005 , C12N9/2402 , C12N9/80 , C12N15/86 , C12Y302/01046 , C12Y305/01015 , C12N2710/22023 , C12N2710/22042
Abstract: The invention relates to virus like particles (VLP) associated with an enzyme abnormally expressed in particular leukodystrophies or an expression vector encoding the enzyme or an mRNA encoding the enzyme or a combination thereof which are used in a method for the treatment of the particular leukodystrophies in a subject in the need thereof, preferably a human. The invention also relates to a pharmaceutical composition for use in a method for the treatment of the particular leukodystrophies, to an expression vector encoding the abnormally expressed enzyme and to a method of associating a VLP with the enzyme, an expression vector encoding the enzyme or an mRNA encoding the enzyme or a combination thereof.
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公开(公告)号：US20230165942A1
公开(公告)日：2023-06-01
申请号：US17820155
申请日：2022-08-16
Applicant: Shire Human Genetic Therapies, Inc.
Inventor： Pericles Calias , Jing Pan , Jan Powell , Lawrence Charnas , Thomas McCauley , Teresa Leah Wright , Richard Pfeifer , Zahra Shahrokh
IPC: A61K38/47 , A61K35/76 , A61K35/761 , A61K9/19 , C12N9/24 , A61K9/00 , A61K38/46 , C07K14/65 , C12N9/42 , A61K9/08 , A61K47/02 , A61K47/26
CPC classification number: A61K38/47 , A61K35/76 , A61K35/761 , C12Y302/01045 , A61K9/19 , C12Y302/0105 , C12N9/2402 , A61K9/0085 , A61K38/465 , A61K9/0019 , C07K14/65 , C12N9/2437 , C12Y301/06013 , A61K9/08 , C12Y301/06008 , C12Y302/01046 , A61K38/46 , A61K47/02 , A61K47/26 , C12Y310/01001
Abstract: The present invention provides an effective and less invasive approach for direct delivery of therapeutic agents to the central nervous system (CNS). In some embodiments, the present invention provides methods including a step of administering intrathecally to a subject suffering from or susceptible to a lysosomal storage disease associated with reduced level or activity of a lysosomal enzyme, a composition comprising a replacement enzyme for the lysosomal enzyme.
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公开(公告)号：US20180085438A1
公开(公告)日：2018-03-29
申请号：US15715748
申请日：2017-09-26
Applicant: Shire Human Genetic Therapies, Inc.
Inventor： Michael F. Concino , Pericles Calias , Jing Pan , Kevin Holmes , Paolo Martini , Alla Romashko , Muthuraman Meiyappan , Bohong Zhang , Andrea Iskenderian , Dianna Lundberg , Angela Norton , Bettina Strack-Logue , Huang Yan , Mary Alessandrini , Richard Pfeifer
IPC: A61K38/47 , A61K9/00 , A61K9/19 , A61K38/46 , C12N9/24 , A61K35/76 , A61K35/761 , C07K14/65 , C12N9/42 , A61K9/08 , A61K47/02 , A61K47/26
CPC classification number: A61K38/47 , A61K9/0019 , A61K9/0085 , A61K9/08 , A61K9/19 , A61K35/76 , A61K35/761 , A61K38/46 , A61K38/465 , A61K47/02 , A61K47/26 , C07K14/65 , C12N9/2402 , C12N9/2437 , C12Y301/06008 , C12Y301/06013 , C12Y302/01045 , C12Y302/01046 , C12Y302/0105 , C12Y310/01001
Abstract: Among other things, the present invention provides methods and compositions of treating Sanfilippo syndrome type B (Sanfilippo B) by, e.g., intrathecal (IT) administration of a Naglu protein. A suitable Naglu protein can be a recombinant, gene-activated or natural protein. In some embodiments, a suitable Naglu protein is a recombinant Naglu protein. In some embodiments, a recombinant Naglu protein is a fusion protein containing a Naglu domain and a lysosomal targeting moiety. In some embodiments, the lysosomal targeting domain is an IGF-II moiety.
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公开(公告)号：US20180008543A1
公开(公告)日：2018-01-11
申请号：US15651832
申请日：2017-07-17
Applicant: Translate Bio, Inc.
Inventor： Braydon Charles Guild , Michael Heartlein , Frank DeRosa , Jerry Chi Zhang
IPC: A61K9/19 , A61K9/127 , A61K31/7088 , A61K38/45 , A61K38/46 , A61K38/47 , A61K38/50 , C07C323/25 , C07C323/27 , C07C323/44 , C12N15/88 , C07J43/00 , C07D233/64 , C07J41/00 , A61K9/51
CPC classification number: A61K9/19 , A61K9/1271 , A61K9/1272 , A61K9/5123 , A61K31/7088 , A61K38/45 , A61K38/465 , A61K38/47 , A61K38/50 , C07C323/25 , C07C323/27 , C07C323/44 , C07D233/64 , C07J41/0055 , C07J43/003 , C12N15/88 , C12Y201/03003 , C12Y203/01 , C12Y207/08015 , C12Y301/06 , C12Y302/01022 , C12Y302/01035 , C12Y302/01046 , C12Y302/01076 , C12Y305/03001
Abstract: Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent iransfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.
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公开(公告)号：US20170202927A1
公开(公告)日：2017-07-20
申请号：US15473460
申请日：2017-03-29
Applicant: Chiesi Farmaceutici S.p.A.
Inventor： Stefan Nilsson
CPC classification number: A61K38/45 , A61K38/465 , A61K38/47 , C07K1/34 , C07K1/36 , C12N9/1085 , C12Y205/01061 , C12Y301/06001 , C12Y301/06008 , C12Y302/01024 , C12Y302/01046
Abstract: The present invention comprises a method of concentrating a composition comprising a polypeptide of interest and the use of such a concentrated composition for the treatment of diseases in mammals, in particular by subcutaneous injection.
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公开(公告)号：US20170042978A1
公开(公告)日：2017-02-16
申请号：US15016141
申请日：2016-02-04
Applicant: Shire Human Genetic Therapies, Inc.
Inventor： Pericles Calias , Jing Pan , Jan Powell , Lawrence Charnas , Thomas McCauley , Teresa Leah Wright , Richard Pfeifer , Zahra Shahrokh
CPC classification number: A61K38/47 , A61K9/0019 , A61K9/0085 , A61K9/08 , A61K9/19 , A61K35/76 , A61K35/761 , A61K38/46 , A61K38/465 , A61K47/02 , A61K47/26 , C07K14/65 , C12N9/2402 , C12N9/2437 , C12Y301/06008 , C12Y301/06013 , C12Y302/01045 , C12Y302/01046 , C12Y302/0105 , C12Y310/01001
Abstract: The present invention provides an effective and less invasive approach for direct delivery of therapeutic agents to the central nervous system (CNS). In some embodiments, the present invention provides methods including a step of administering intrathecally to a subject suffering from or susceptible to a lysosomal storage disease associated with reduced level or activity of a lysosomal enzyme, a composition comprising a replacement enzyme for the lysosomal enzyme.
Abstract translation: 本发明提供了用于将治疗剂直接递送至中枢神经系统（CNS）的有效且较少侵入性的方法。 在一些实施方案中，本发明提供了包括以下步骤的方法：鞘内施用患有或易受溶酶体酶降低的水平或活性的溶酶体贮积病的受试者，包含溶酶体酶的替代酶的组合物。
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40.发明申请METHODS AND COMPOSITIONS FOR CNS DELIVERY OF HEPARAN N-SULFATASE 审中-公开用于中枢神经系统递送HEPARAN N-SULFATASE的方法和组合物
公开(公告)号：US20170042977A1
公开(公告)日：2017-02-16
申请号：US15077046
申请日：2016-03-22
Applicant: Shire Human Genetic Therapies, Inc.
Inventor： Farah Natoli , Gaozhong Zhu , Jennifer Terew , Yuan Jiang , Jamie Tsung , Zahra Shahrokh , Brian Vernaglia , Jing Pan , Richard Pfeifer , Pericles Calias
CPC classification number: A61K38/47 , A61K9/0019 , A61K9/0085 , A61K9/08 , A61K9/19 , A61K35/76 , A61K35/761 , A61K38/46 , A61K38/465 , A61K47/02 , A61K47/26 , C07K14/65 , C12N9/2402 , C12N9/2437 , C12Y301/06008 , C12Y301/06013 , C12Y302/01045 , C12Y302/01046 , C12Y302/0105 , C12Y310/01001
Abstract: The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising a heparan N-sulfatase (HNS) protein, salt, and a polysorbate surfactant for the treatment of Sanfilippo Syndrome Type A.
Abstract translation: 本发明尤其提供了用于CNS递送溶酶体酶以有效治疗溶酶体贮积病的组合物和方法。 在一些实施方案中，本发明包括用于直接CNS鞘内施用的稳定制剂，其包含用于治疗Sanfilippo综合征A型的肝素N-硫酸酯酶（HNS）蛋白质，盐和聚山梨醇酯表面活性剂。
