公开(公告)号：US20240316217A1

公开(公告)日：2024-09-26

申请号：US18264364

申请日：2022-02-04

Applicant: Regents of the University of Minnesota ,  REGENXBIO Inc.

Inventor： R. Scott McIvor ,  Nicholas Alexander Piers Sascha Buss ,  Troy C. Lund ,  Elizabeth Braunlin ,  Lalitha R. Belur ,  Carolyn Fairbanks ,  Marie-Laure Nevoret

IPC: A61K48/00 ,  A61K9/00 ,  A61K38/47 ,  A61P3/06 ,  A61P9/00 ,  A61P19/00 ,  C12N15/86

CPC classification number: A61K48/005 ,  A61K9/0019 ,  A61K9/0085 ,  A61K38/47 ,  A61K48/0075 ,  A61K48/0083 ,  A61P3/06 ,  A61P9/00 ,  A61P19/00 ,  C12N15/86 ,  C12Y302/01076 ,  C12N2750/14143

Abstract: A method to prevent, inhibit the progression of, reduce the severity of, or treat cardiac, vascular or skeletal dysfunction or defect(s) in a human having lysosomal storage disorder, is provided.

公开(公告)号：US20240307511A1

公开(公告)日：2024-09-19

申请号：US18538866

申请日：2023-12-13

Applicant: The Trustees of the University of Pennsylvania

Inventor： Christian Hinderer ,  James M. Wilson

IPC: A61K38/47 ,  A61K9/00 ,  A61K31/343 ,  A61K31/439 ,  A61K35/76 ,  A61K45/06 ,  A61K48/00 ,  A61P3/00 ,  C12N7/00 ,  C12N9/24 ,  C12N15/86

CPC classification number: A61K38/47 ,  A61K9/0019 ,  A61K31/343 ,  A61K31/439 ,  A61K35/76 ,  A61K45/06 ,  A61P3/00 ,  C12N7/00 ,  C12N9/2402 ,  C12N15/86 ,  C12Y302/01076 ,  A61K48/00 ,  A61K48/0083 ,  C12N2750/14121 ,  C12N2750/14132 ,  C12N2750/14143

Abstract: A co-therapeutic regimen comprising AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDUA gene and two or more immunosuppressants is provided herein. Also provided are methods useful for treating hIDUA deficiency (MPSI) and the symptoms associated with Hurler, Hurler-Scheie and Scheie syndromes.

公开(公告)号：US12036190B2

公开(公告)日：2024-07-16

申请号：US17408397

申请日：2021-08-21

Applicant: Duke University

Inventor： Priya Kishnani ,  Baodong Sun ,  Dwight D. Koeberl

IPC: A61K38/47 ,  A61K31/05 ,  A61K31/12 ,  A61K31/137 ,  A61K31/155 ,  A61K31/167 ,  A61K31/192 ,  A61K31/198 ,  A61K31/216 ,  A61K31/277 ,  A61K31/352 ,  A61K31/355 ,  A61K31/385 ,  A61K31/436 ,  A61K31/519 ,  A61K31/522 ,  A61K31/5415 ,  A61K31/55 ,  A61K31/575 ,  A61K31/7016 ,  A61K33/00 ,  A61K38/46 ,  A61P3/00

CPC classification number: A61K31/137 ,  A61K31/05 ,  A61K31/12 ,  A61K31/155 ,  A61K31/167 ,  A61K31/192 ,  A61K31/198 ,  A61K31/216 ,  A61K31/277 ,  A61K31/352 ,  A61K31/355 ,  A61K31/385 ,  A61K31/436 ,  A61K31/519 ,  A61K31/522 ,  A61K31/5415 ,  A61K31/55 ,  A61K31/575 ,  A61K31/7016 ,  A61K33/00 ,  A61K38/465 ,  A61K38/47 ,  A61P3/00 ,  C12Y301/04012 ,  C12Y302/0102 ,  C12Y302/01022 ,  C12Y302/01045 ,  C12Y302/01049 ,  C12Y302/01076 ,  A61K38/465 ,  A61K2300/00 ,  A61K38/47 ,  A61K2300/00

Abstract: The present disclosure is directed to methods of treating a steatosis-associated disorder and methods of treating a cytoplasmic glycogen storage disorder, including glycogen storage disease I, glycogen storage disease III, glycogen storage disease IV, and/or conditions associated with a PRKAG2 mutation, by administering a therapeutic agent selected from a lysosomal enzyme, an autophagy-inducing agent, or a combination thereof. Steatosis-associated disorders discussed herein include GSD Ia, GSD Ib, GSD Ic, NAFLD, and NASH. Other embodiments are directed to methods of reversing steatosis, modulating autophagy, inducing autophagy, and reversing glycogen storage. Methods of treating a cytoplasmic glycogen storage disorder by administering a lysosomal enzyme and a second therapeutic agent are also described. Other embodiments are directed to methods of treating a cytoplasmic glycogen storage disorder by administering a therapeutic agent as an adjunctive therapy to lysosomal enzyme replacement therapy.

公开(公告)号：US11857641B2

公开(公告)日：2024-01-02

申请号：US16534302

申请日：2019-08-07

Applicant: Sangamo Therapeutics, Inc. ,  Roselia C. Santana

Inventor： Dale Ando ,  Cheryl Wong Po Foo ,  Sagar A. Vaidya ,  Shelley Q. Wang

IPC: A61K48/00 ,  A61P3/00 ,  G01N33/66 ,  C12Q1/34 ,  A61K38/47

CPC classification number: A61K48/005 ,  A61K38/47 ,  A61P3/00 ,  C12Q1/34 ,  C12Y301/06013 ,  C12Y302/01076 ,  G01N33/66

Abstract: Described herein are methods and compositions for treating MPS I (Hurler/Hurler-Scheie/Scheie Syndrome) by administering to the subject a composition comprising a iduronidase (IDUA) polynucleotide. In one aspect, provided is a method of reducing, delaying and/or eliminating one or more of the need for additional treatment procedures, the onset, progression and/or severity of symptoms in a subject with MPS I, by administering to the subject a composition comprising a iduronidase (IDUA) polynucleotide.

公开(公告)号：US20230285329A1

公开(公告)日：2023-09-14

申请号：US18200557

申请日：2023-05-22

Applicant: Duke University ,  National University of Singapore

Inventor： Dwight D. Koeberl ,  Paul M. Yen ,  Benjamin L. Farah

IPC: A61K31/137 ,  A61K31/436 ,  A61K31/05 ,  A61K31/155 ,  A61K31/192 ,  A61K38/47 ,  A61K31/522 ,  A61K38/46 ,  A61K31/198 ,  A61K31/12 ,  A61K31/216 ,  A61K31/352 ,  A61K31/167 ,  A61P3/00 ,  A61K31/277 ,  A61K31/355 ,  A61K31/385 ,  A61K31/5415 ,  A61K31/55 ,  A61K31/575 ,  A61K31/7016 ,  A61K33/00

CPC classification number: A61K31/137 ,  A61K31/436 ,  A61K31/05 ,  A61K31/155 ,  A61K31/192 ,  A61K38/47 ,  C12Y302/0102 ,  A61K31/522 ,  A61K38/465 ,  A61K31/198 ,  A61K31/12 ,  A61K31/216 ,  C12Y301/04012 ,  C12Y302/01049 ,  A61K31/352 ,  A61K31/167 ,  C12Y302/01022 ,  C12Y302/01045 ,  C12Y302/01076 ,  A61P3/00 ,  A61K31/277 ,  A61K31/355 ,  A61K31/385 ,  A61K31/5415 ,  A61K31/55 ,  A61K31/575 ,  A61K31/7016 ,  A61K33/00

Abstract: The present disclosure is directed to methods of treating a steatosis-associated disorder by administering a therapeutic agent selected from a lysosomal enzyme, an autophagy-inducing agent, or a combination thereof. Steatosis-associated disorders discussed herein include GSD Ia, GSD Ib, GSD Ic, NAFLD, and NASH. Other embodiments are directed to methods of reversing steatosis, modulating autophagy, inducing autophagy, and reversing glycogen storage.

公开(公告)号：US20180135050A1

公开(公告)日：2018-05-17

申请号：US15847283

申请日：2017-12-19

Applicant: CuRNA, Inc.

Inventor： Joseph COLLARD ,  Olga KHORKOVA SHERMAN ,  Carlos COITO ,  Gang SHEN

IPC: C12N15/113 ,  C12N15/10 ,  G01N33/53 ,  A61K31/7088

CPC classification number: C12N15/113 ,  A61K31/7088 ,  C12N15/1034 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2320/50 ,  C12Y302/01076 ,  G01N33/5308 ,  C12N2310/111 ,  C12N2330/10

Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Alpha-L-Iduronidase (IDUA), in particular, by targeting natural antisense polynucleotides of Alpha-L-Iduronidase (IDUA). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of IDUA.

公开(公告)号：US20180036388A1

公开(公告)日：2018-02-08

申请号：US15717450

申请日：2017-09-27

Applicant: Regenxbio Inc.

Inventor： R. Scott McIvor ,  Lalitha R. Belur ,  Walter Low ,  Carolyn Fairbanks

IPC: A61K38/47 ,  C12N15/86 ,  C12N7/00 ,  A61K48/00

CPC classification number: A61K38/47 ,  A61K48/00 ,  C12N7/00 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2830/007 ,  C12Y302/01076 ,  H05K999/99

Abstract: A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.

公开(公告)号：US09469850B2

公开(公告)日：2016-10-18

申请号：US14272960

申请日：2014-05-08

Applicant: Genzyme Corporation

Inventor： Yunxiang Zhu ,  Seng H. Cheng ,  Canwen Jiang ,  Luis Z. Avila

IPC: C07H15/04 ,  A61K38/47 ,  A61K47/48 ,  C12N9/96 ,  C12N9/16 ,  C12N9/26

CPC classification number: A61K47/48092 ,  A61K38/47 ,  A61K47/549 ,  C07H15/04 ,  C12N9/16 ,  C12N9/2408 ,  C12N9/96 ,  C12Y301/04012 ,  C12Y302/0102 ,  C12Y302/01022 ,  C12Y302/01076

Abstract: The invention provides methods for the synthesis of oligosaccharides comprising an aminooxy group. The invention further provides oligosaccharides comprising an aminooxy group, methods for coupling oligosaccharides comprising an aminooxy group to glycoproteins, and oligosaccharide-protein conjugates. Also provided are methods of treating a lysosomal storage disorder in a mammal by administration of an oligosaccharide-protein conjugate.

Abstract translation: 本发明提供了合成包含氨基氧基的寡糖的方法。 本发明还提供了包含氨基氧基的寡糖，将包含氨基氧基的寡糖与糖蛋白偶联的方法和寡糖 - 蛋白质缀合物。 还提供了通过施用寡糖 - 蛋白质缀合物来治疗哺乳动物中的溶酶体储存障碍的方法。

公开(公告)号：US20160060656A1

公开(公告)日：2016-03-03

申请号：US14872537

申请日：2015-10-01

Applicant: Sangamo BioSciences, Inc.

Inventor： Edward J. Rebar

IPC: C12N15/90 ,  A61K38/46 ,  C12N9/22

CPC classification number: C12N15/907 ,  A61K35/407 ,  A61K38/465 ,  A61K38/47 ,  A61K48/00 ,  A61K48/005 ,  C07K2319/81 ,  C12N9/22 ,  C12Y301/06013 ,  C12Y302/0102 ,  C12Y302/01021 ,  C12Y302/01022 ,  C12Y302/01076

Abstract: Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of a lysosomal storage disease.

Abstract translation: 使用这些核酸酶以将编码治疗性蛋白质例如酶的序列插入细胞的核酸酶和方法，从而提供用于治疗和/或预防溶酶体贮积病的蛋白质或细胞治疗剂。

公开(公告)号：US09222088B2

公开(公告)日：2015-12-29

申请号：US13880409

申请日：2011-10-20

Applicant: Joseph Collard ,  Olga Khorkova Sherman ,  Carlos Coito ,  Gang Shen

Inventor： Joseph Collard ,  Olga Khorkova Sherman ,  Carlos Coito ,  Gang Shen

IPC: C12N15/11 ,  C12N15/113 ,  A61K31/7088 ,  C12N15/10 ,  G01N33/53

CPC classification number: C12N15/113 ,  A61K31/7088 ,  C12N15/1034 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2320/50 ,  C12Y302/01076 ,  G01N33/5308 ,  C12N2310/111 ,  C12N2330/10

Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Alpha-L-Iduronidase (IDUA), in particular, by targeting natural antisense polynucleotides of Alpha-L-Iduronidase (IDUA). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of IDUA.

Abstract translation: 本发明涉及通过靶向α-L-吡哆醛糖苷酶（IDUA）的天然反义多核苷酸来调节α-L-吡哆醛糖苷酶（IDUA）的表达和/或功能的反义寡核苷酸。 本发明还涉及这些反义寡核苷酸的鉴定及其在治疗与IDUA表达相关的疾病和病症中的用途。