JP2023537903A - リソソーム障害に対する遺伝子療法 - Google Patents

リソソーム障害に対する遺伝子療法 Download PDF

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Publication number
JP2023537903A
JP2023537903A JP2023507760A JP2023507760A JP2023537903A JP 2023537903 A JP2023537903 A JP 2023537903A JP 2023507760 A JP2023507760 A JP 2023507760A JP 2023507760 A JP2023507760 A JP 2023507760A JP 2023537903 A JP2023537903 A JP 2023537903A
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raav
dose
day
sirolimus
subject
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JP2023537903A5 (https=
Inventor
アベリオビック，アーサ
セヴィグニー，ジェフリー
ルイス，トラヴィス
アスペンスカヤ，オルガ
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Prevail Therapeutics Inc
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    • A—HUMAN NECESSITIES
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/436—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
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    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
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    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • A—HUMAN NECESSITIES
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    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2023507760A 2020-08-10 2021-08-10 リソソーム障害に対する遺伝子療法 Pending JP2023537903A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063063851P 2020-08-10 2020-08-10
US63/063,851 2020-08-10
PCT/US2021/045447 WO2022035903A2 (en) 2020-08-10 2021-08-10 Gene therapies for lysosomal disorders

Publications (2)

Publication Number Publication Date
JP2023537903A true JP2023537903A (ja) 2023-09-06
JP2023537903A5 JP2023537903A5 (https=) 2024-08-20

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JP2023507760A Pending JP2023537903A (ja) 2020-08-10 2021-08-10 リソソーム障害に対する遺伝子療法

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US (1) US20230310654A1 (https=)
EP (1) EP4192516A2 (https=)
JP (1) JP2023537903A (https=)
KR (1) KR20230051529A (https=)
CN (1) CN116157527A (https=)
AU (1) AU2021324686A1 (https=)
CA (1) CA3190866A1 (https=)
IL (1) IL300407A (https=)
MX (1) MX2023001562A (https=)
WO (1) WO2022035903A2 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2018346104B2 (en) 2017-10-03 2023-07-27 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
US20250205365A1 (en) * 2022-03-18 2025-06-26 University Of Florida Research Foundation, Incorporated Methods and compositions for treating rbm20 related cardiomyopathy with a viral vector
WO2024229161A1 (en) * 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
US20250152747A1 (en) * 2023-11-15 2025-05-15 Genzyme Corporation Methods of treating gaucher disease and gba-parkinson’s disease
WO2025229340A1 (en) 2024-05-01 2025-11-06 Spur Therapeutics Limited Methods and compositions for treatment of a synucleinopathy and/or a disease associated with a gcase deficiency
KR20260021150A (ko) 2024-08-05 2026-02-13 충북대학교 산학협력단 Gba1 돌연변이 보인자의 dj-1 발현수준에 따른 파킨슨병의 위험도 예측 방법

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017531652A (ja) * 2014-10-06 2017-10-26 アルスロジェン ビー．ブイ．Ａｒｔｈｒｏｇｅｎ Ｂ．Ｖ． Ａａｖに基づく遺伝子治療
WO2019010335A1 (en) * 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania AAV9 MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I
WO2019070893A1 (en) * 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISORDERS

Family Cites Families (6)

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WO2008024998A2 (en) 2006-08-24 2008-02-28 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
HK1256341A1 (zh) * 2015-05-15 2019-09-20 明尼苏达大学董事会 用於治疗性递送到中枢神经系统的腺相关物
JP7496667B2 (ja) 2016-04-21 2024-06-07 ビロベク，インコーポレイテッド 昆虫細胞中でのａａｖ生成、方法およびその組成物
CN112501208A (zh) 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
BR112020006661A2 (pt) * 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
JP7664848B2 (ja) * 2019-04-10 2025-04-18 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害のための遺伝子療法

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017531652A (ja) * 2014-10-06 2017-10-26 アルスロジェン ビー．ブイ．Ａｒｔｈｒｏｇｅｎ Ｂ．Ｖ． Ａａｖに基づく遺伝子治療
WO2019010335A1 (en) * 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania AAV9 MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I
WO2019070893A1 (en) * 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISORDERS

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Publication number Publication date
WO2022035903A3 (en) 2022-09-29
US20230310654A1 (en) 2023-10-05
IL300407A (en) 2023-04-01
AU2021324686A1 (en) 2023-04-13
CN116157527A (zh) 2023-05-23
WO2022035903A2 (en) 2022-02-17
KR20230051529A (ko) 2023-04-18
MX2023001562A (es) 2023-05-03
EP4192516A2 (en) 2023-06-14
CA3190866A1 (en) 2022-02-17

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