公开(公告)号：US20230210941A1

公开(公告)日：2023-07-06

申请号：US17998330

申请日：2021-05-11

Applicant: The Trustees of the University of Pennsylvania

Inventor： James M. Wilson ,  Juliette Hordeaux ,  Nathan Katz

IPC: A61K38/16 ,  C12N15/86 ,  C12N9/24 ,  A61K9/00

CPC classification number: A61K38/162 ,  A61K9/0085 ,  C12N9/2402 ,  C12N15/86 ,  C12Y302/01046 ,  C12N2750/14122 ,  C12N2750/14143

Abstract: A pharmaceutical composition formulated for delivery of a recombinant adeno-associated virus (rAAV) vector comprising an AAV capsid and a vector genome having human galactosylceramidase (GALC) coding sequence is provided. Also provided are 5 methods and uses of a pharmaceutical composition comprising a rAAV for the treatment of Krabbe disease.

公开(公告)号：US20140314735A1

公开(公告)日：2014-10-23

申请号：US14321642

申请日：2014-07-01

Applicant: ZYMENEX A/S

Inventor： Stefan NILSSON

IPC: A61K38/47 ,  A61K38/45 ,  A61K38/46

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。

公开(公告)号：US20130195835A1

公开(公告)日：2013-08-01

申请号：US13620259

申请日：2012-09-14

Applicant: Nico Luc Marc Callewaert ,  Wouter Vervecken ,  Karen Jacqueline Marcel De Pourcq ,  Steven Christian Jozef Geysens ,  Mouna Guerfal

Inventor： Nico Luc Marc Callewaert ,  Wouter Vervecken ,  Karen Jacqueline Marcel De Pourcq ,  Steven Christian Jozef Geysens ,  Mouna Guerfal

IPC: C12N9/24 ,  C12P21/06

CPC classification number: C12N9/2488 ,  A61K38/00 ,  C07K14/00 ,  C12N9/1051 ,  C12N9/24 ,  C12N9/2402 ,  C12N9/2465 ,  C12P21/005 ,  C12P21/06 ,  C12Y204/01232 ,  C12Y302/01024 ,  C12Y302/01045 ,  C12Y302/01046 ,  C12Y302/01084 ,  C12Y302/01113 ,  Y02P20/52

Abstract: Described herein are methods and genetically engineered cells useful for producing an altered N-glycosylation form of a target molecule. Also described are methods and molecules with altered N-glycosylation useful for treating a variety of disorders such as metabolic disorders.

公开(公告)号：US20130190253A1

公开(公告)日：2013-07-25

申请号：US13620306

申请日：2012-09-14

Applicant: Nico Luc Marc Callewaert ,  Wouter Vervecken ,  Karen Jacqueline Marcel De Pourcq ,  Steven Christian Jozef Geysens ,  Mouna Guerfal

Inventor： Nico Luc Marc Callewaert ,  Wouter Vervecken ,  Karen Jacqueline Marcel De Pourcq ,  Steven Christian Jozef Geysens ,  Mouna Guerfal

IPC: C07K14/00

CPC classification number: C12N9/2488 ,  A61K38/00 ,  C07K14/00 ,  C12N9/1051 ,  C12N9/24 ,  C12N9/2402 ,  C12N9/2465 ,  C12P21/005 ,  C12P21/06 ,  C12Y204/01232 ,  C12Y302/01024 ,  C12Y302/01045 ,  C12Y302/01046 ,  C12Y302/01084 ,  C12Y302/01113 ,  Y02P20/52

Abstract: Described herein are methods and genetically engineered cells useful for producing an altered N-glycosylation form of a target molecule. Also described are methods and molecules with altered N-glycosylation useful for treating a variety of disorders such as metabolic disorders.

Abstract translation: 本文描述了可用于产生改变的N-糖基化形式的靶分子的方法和遗传工程改造的细胞。 还描述了具有改变的N-糖基化的方法和分子，其可用于治疗各种疾病如代谢紊乱。

公开(公告)号：US20020127219A1

公开(公告)日：2002-09-12

申请号：US09753126

申请日：2000-12-29

Inventor： Jens Sigurd Okkels ,  Anne Dam Jensen ,  Torben Halkier ,  Rikke Bolding Jensen ,  Hans Thalsgard Schambye

IPC: A61K038/47 ,  C12N009/36

CPC classification number: C12Y302/01045 ,  A61K38/24 ,  C07K14/475 ,  C07K2319/00 ,  C12N9/2402 ,  C12N9/2465 ,  C12P21/005 ,  C12Y302/01018 ,  C12Y302/0102 ,  C12Y302/01022 ,  C12Y302/01046 ,  C12Y302/01052 ,  C12Y302/01076

Abstract: A polypeptide selected from the group of lysosomal enzymes and lysosomal enzyme activators, comprising at least one introduced glycosylation site as compared to a corresponding parent enzyme or activator. By introducing additional glycosylation sites the resulting glycosylated lysosomal enzyme or activator obtains improved in vivo activity and thereby provides for improved treatment of lysosomal storage diseases.

Abstract translation: 与相应的亲本酶或活化剂相比，选自溶酶体酶和溶酶体酶激活剂组的多肽包含至少一个引入的糖基化位点。 通过引入另外的糖基化位点，得到的糖基化溶酶体酶或活化剂获得改善的体内活性，从而提供改进的溶酶体储存疾病的治疗。

公开(公告)号：US09713634B2

公开(公告)日：2017-07-25

申请号：US15041841

申请日：2016-02-11

Applicant: Shire Pharmaceuticals Ireland Limited

Inventor： Stefan Nilsson

IPC: C12N9/16 ,  A61K38/46 ,  A61K38/47 ,  C07K1/34 ,  C07K1/36 ,  A61K38/45

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 concentrated composition for the treatment of diseases in mammals, in particular by subcutaneous injection.

公开(公告)号：US09222083B2

公开(公告)日：2015-12-29

申请号：US13095532

申请日：2011-04-27

Applicant: Nico Luc Marc Callewaert ,  Wouter Vervecken ,  Karen Jacqueline Marcel De Pourcq ,  Steven Christian Jozef Geysens ,  Mouna Guerfal

Inventor： Nico Luc Marc Callewaert ,  Wouter Vervecken ,  Karen Jacqueline Marcel De Pourcq ,  Steven Christian Jozef Geysens ,  Mouna Guerfal

IPC: A61K38/16 ,  C12N9/24 ,  C12N9/10 ,  C12P21/00 ,  C07K14/00 ,  C12N9/40 ,  C12P21/06 ,  C07K14/39 ,  A61K38/00

CPC classification number: C12N9/2488 ,  A61K38/00 ,  C07K14/00 ,  C12N9/1051 ,  C12N9/24 ,  C12N9/2402 ,  C12N9/2465 ,  C12P21/005 ,  C12P21/06 ,  C12Y204/01232 ,  C12Y302/01024 ,  C12Y302/01045 ,  C12Y302/01046 ,  C12Y302/01084 ,  C12Y302/01113 ,  Y02P20/52

Abstract: Described herein are methods and genetically engineered cells useful for producing an altered N-glycosylation form of a target molecule. Also described are methods and molecules with altered N-glycosylation useful for treating a variety of disorders such as metabolic disorders.

Abstract translation: 本文描述了可用于产生改变的N-糖基化形式的靶分子的方法和遗传工程改造的细胞。 还描述了具有改变的N-糖基化的方法和分子，其可用于治疗各种疾病如代谢紊乱。

公开(公告)号：US20150196014A1

公开(公告)日：2015-07-16

申请号：US14418038

申请日：2013-08-07

Applicant: Ace Biosciences A/S

Inventor： Jens Fogh ,  Claes Andersson

IPC: A01K67/027 ,  C12N15/85 ,  A61K49/00 ,  C12N9/24

CPC classification number: A01K67/0278 ,  A01K2217/072 ,  A01K2227/105 ,  A01K2267/0318 ,  A61K49/0008 ,  C12N9/2402 ,  C12N15/8509 ,  C12Y302/01046

Abstract: The present invention relates to an animal model for infantile globoid cell leucodystrophy, and use of said animal model for screening and/or validation of agents which may be useful as a medicament for treatment of globoid cell leukodystrophy.

Abstract translation: 本发明涉及一种婴幼儿球囊细胞白血病的动物模型，以及所述动物模型用于筛选和/或验证可用作治疗球形细胞白血病的药物的药剂。

公开(公告)号：US20140271598A1

公开(公告)日：2014-09-18

申请号：US13974457

申请日：2013-08-23

Applicant: Shire Human Genetic Therapies, Inc.

Inventor： Gaozhong Zhu ,  Kris Lowe ,  Zahra Shahrokh ,  James Christian ,  Rick Fahrner ,  Jing Pan ,  Teresa Leah Wright ,  Pericles Calias

IPC: A61K38/46

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 an iduronate-2-sulfatase (I2S) protein, salt, and a polysorbate surfactant for the treatment of Hunters Syndrome.

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，其包含用于治疗猎人综合征的艾杜糖醛酸-2-硫酸酯酶（I2S）蛋白质，盐和聚山梨醇酯表面活性剂。

公开(公告)号：US20130295071A1

公开(公告)日：2013-11-07

申请号：US13862187

申请日：2013-04-12

Applicant: Shire Human Genetic Therapies, Inc.

Inventor： Nazila Salamat-Miller ,  Katherine Taylor ,  Ken Manning ,  Gaozhong Zhu ,  Paul Campolieto ,  Zahra Shahrokh ,  Pericles Calias ,  Thomas McCauley

IPC: A61K9/00 ,  A61K38/47

CPC classification number: A61K9/0019 ,  A61K9/0085 ,  A61K9/19 ,  A61K38/465 ,  A61K38/47 ,  A61K47/02 ,  A61K47/26 ,  C07K14/65 ,  C12N9/2402 ,  C12N9/2437 ,  C12Y301/06013 ,  C12Y302/01046

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 an B-Galactocerebrosidase protein, salt, and a polysorbate surfactant for the treatment of GLD Disease.

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GLD疾病的B-半乳糖脑苷脂酶蛋白质，盐和聚山梨醇酯表面活性剂。