JP2020514305A5 - - Google Patents

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Publication number
JP2020514305A5
JP2020514305A5 JP2019538173A JP2019538173A JP2020514305A5 JP 2020514305 A5 JP2020514305 A5 JP 2020514305A5 JP 2019538173 A JP2019538173 A JP 2019538173A JP 2019538173 A JP2019538173 A JP 2019538173A JP 2020514305 A5 JP2020514305 A5 JP 2020514305A5
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JP
Japan
Prior art keywords
pharmaceutical composition
composition according
subject
effective amount
disease
Prior art date
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JP2019538173A
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English (en)
Japanese (ja)
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JP7576293B2 (ja
JP2020514305A (ja
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Priority claimed from PCT/US2018/013509 external-priority patent/WO2018132667A1/en
Publication of JP2020514305A publication Critical patent/JP2020514305A/ja
Publication of JP2020514305A5 publication Critical patent/JP2020514305A5/ja
Priority to JP2022181240A priority Critical patent/JP2023011938A/ja
Priority to JP2024159770A priority patent/JP2024169509A/ja
Application granted granted Critical
Publication of JP7576293B2 publication Critical patent/JP7576293B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2019538173A 2017-01-13 2018-01-12 ファーバー病を治療するための組成物及び方法 Active JP7576293B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022181240A JP2023011938A (ja) 2017-01-13 2022-11-11 ファーバー病を治療するための組成物及び方法
JP2024159770A JP2024169509A (ja) 2017-01-13 2024-09-17 ファーバー病を治療するための組成物及び方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762446166P 2017-01-13 2017-01-13
US62/446,166 2017-01-13
PCT/US2018/013509 WO2018132667A1 (en) 2017-01-13 2018-01-12 Compositions and methods for treating farber disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022181240A Division JP2023011938A (ja) 2017-01-13 2022-11-11 ファーバー病を治療するための組成物及び方法

Publications (3)

Publication Number Publication Date
JP2020514305A JP2020514305A (ja) 2020-05-21
JP2020514305A5 true JP2020514305A5 (https=) 2021-02-12
JP7576293B2 JP7576293B2 (ja) 2024-10-31

Family

ID=62839661

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2019538173A Active JP7576293B2 (ja) 2017-01-13 2018-01-12 ファーバー病を治療するための組成物及び方法
JP2022181240A Pending JP2023011938A (ja) 2017-01-13 2022-11-11 ファーバー病を治療するための組成物及び方法
JP2024159770A Pending JP2024169509A (ja) 2017-01-13 2024-09-17 ファーバー病を治療するための組成物及び方法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2022181240A Pending JP2023011938A (ja) 2017-01-13 2022-11-11 ファーバー病を治療するための組成物及び方法
JP2024159770A Pending JP2024169509A (ja) 2017-01-13 2024-09-17 ファーバー病を治療するための組成物及び方法

Country Status (8)

Country Link
US (4) US20190343936A1 (https=)
EP (2) EP3568154B1 (https=)
JP (3) JP7576293B2 (https=)
AU (2) AU2018207564A1 (https=)
CA (1) CA3049771A1 (https=)
JO (1) JOP20190164B1 (https=)
MX (1) MX2019008038A (https=)
WO (1) WO2018132667A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3568154B1 (en) * 2017-01-13 2023-07-12 Icahn School of Medicine at Mount Sinai Compositions and methods for treating farber disease
US11597917B2 (en) * 2017-07-06 2023-03-07 The Medical College Of Wisconsin, Inc. In vitro and in vivo enrichment strategy targeting lymphocytes derived from vector transduced HSCs for therapy of disorders
WO2019150192A1 (en) * 2018-02-02 2019-08-08 Enzyvant Therapeutics Gmbh Methods for treating farber disease
EP3784695B1 (en) 2018-04-27 2023-08-09 The Medical College of Wisconsin, Inc. Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disorders
US20220088158A1 (en) * 2019-01-23 2022-03-24 Aceragen, Inc. Method of ameliorating a pro-inflammatory immunophenotype in farber disease subjects by repeated administration of a recombinant human acid ceramidase
WO2021055801A1 (en) 2019-09-18 2021-03-25 The Medical College Of Wisconsin, Inc. Improved alpha-galactosidase protein for enzyme replacement therapy (ert) and methods of use

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2897825T3 (es) 2012-06-01 2022-03-02 Icahn School Med Mount Sinai Niveles de ceramida en el tratamiento y prevención de infecciones
PT2968479T (pt) 2013-03-14 2019-08-07 Icahn School Med Mount Sinai Composições terapêuticas de ceramidase ácida e métodos de fabrico e utilização
EP3568154B1 (en) * 2017-01-13 2023-07-12 Icahn School of Medicine at Mount Sinai Compositions and methods for treating farber disease
WO2019060837A1 (en) * 2017-09-25 2019-03-28 Enzyvant Farber Gmbh PROCESS FOR PRODUCING RECOMBINANT HUMAN ACID CERAMIDASE
WO2019150192A1 (en) * 2018-02-02 2019-08-08 Enzyvant Therapeutics Gmbh Methods for treating farber disease

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