JP2022513034A - 神経セロイドリポフスチン症の遺伝子治療 - Google Patents

神経セロイドリポフスチン症の遺伝子治療 Download PDF

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JP2022513034A
JP2022513034A JP2021526294A JP2021526294A JP2022513034A JP 2022513034 A JP2022513034 A JP 2022513034A JP 2021526294 A JP2021526294 A JP 2021526294A JP 2021526294 A JP2021526294 A JP 2021526294A JP 2022513034 A JP2022513034 A JP 2022513034A
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raav
pharmaceutical composition
certain embodiments
tpp1
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JP2022513034A5 (https=
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コンドラガンタ バルガビ
カルムチル メレチル スブハ
ピエール ギベール マシュー
クリストファー マーサー アンドリュー
ジェームズ キャレル マイケル
トーマス シュターデルマン ロバート
マイケル ゲルナー フランツ
アレクサンダー ピアーズ サッシャ バス ニコラス
ビー ジャレド
ジェームズ マーシャル トリスタン
デパズ ロベルト
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レジェンクスバイオ インコーポレーテッド
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5176—Compounds of unknown constitution, e.g. material from plants or animals
    • A61K9/5184—Virus capsids or envelopes enclosing drugs
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    • A61P25/00—Drugs for disorders of the nervous system
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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JP2021526294A 2018-11-14 2019-11-13 神経セロイドリポフスチン症の遺伝子治療 Pending JP2022513034A (ja)

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JP2025076129A JP2025128087A (ja) 2018-11-14 2025-05-01 神経セロイドリポフスチン症の遺伝子治療

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US201862767410P 2018-11-14 2018-11-14
US62/767,410 2018-11-14
US201962924060P 2019-10-21 2019-10-21
US62/924,060 2019-10-21
PCT/US2019/061206 WO2020102369A1 (en) 2018-11-14 2019-11-13 Gene therapy for neuronal ceroid lipofuscinoses

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US (1) US20210393802A1 (https=)
EP (1) EP3880235A4 (https=)
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KR (1) KR20210092755A (https=)
AU (1) AU2019379141A1 (https=)
BR (1) BR112021009370A2 (https=)
CA (1) CA3117982A1 (https=)
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Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
PT4021575T (pt) * 2019-08-29 2024-07-30 Biomarin Pharm Inc Métodos para o tratamento da doença cln2 em sujeitos pediátricos
WO2021071835A1 (en) * 2019-10-07 2021-04-15 Regenxbio Inc. Adeno-associated virus vector pharmaceutical composition and methods
AU2021358781A1 (en) 2020-10-07 2023-05-18 Tern Therapeutics, Llc Gene therapy for ocular manifestations of cln2 disease
US20240066146A1 (en) * 2020-12-18 2024-02-29 Sangamo Therapeutics, Inc. Improved pharmaceutical compositions containing adeno-associated viral vector
WO2022165313A1 (en) * 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
EP4323531A1 (en) * 2021-04-12 2024-02-21 Affinia Therapeutics Inc. Recombinant aav for treatment of neural disease
WO2023113806A1 (en) * 2021-12-16 2023-06-22 Affinia Therapeutics, Inc. Recombinant aav for treatment of neural disease
AU2022279062A1 (en) * 2021-05-17 2024-01-04 Sarepta Therapeutics, Inc. Production of recombinant aav vectors for treating muscular dystrophy
WO2023177626A1 (en) * 2022-03-14 2023-09-21 Regeneron Pharmaceuticals, Inc. Lyophilized formulations of aav drug products
US20260034243A1 (en) * 2022-04-06 2026-02-05 Regenxbio Inc. Pharmaceutical composition comprising a recombinant adeno-associated virus vector with an expression cassette encoding a transgene for suprachoroidal administration
CN115869425B (zh) * 2022-12-12 2024-08-20 北京生物制品研究所有限责任公司 一种aav眼用注射液及其制备方法和应用
WO2025122725A1 (en) * 2023-12-06 2025-06-12 The Broad Institute, Inc. Methods and compositions for base editing of tpp1 in the treatment of batten disease

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160108099A1 (en) * 2011-10-27 2016-04-21 Wellstat Ophthalmics Corporation Vectors encoding rod-derived cone viability factor
JP2016535587A (ja) * 2013-10-24 2016-11-17 ユニキュアー アイピー ビー．ブイ． Ａａｖ５カプシドタンパク質を含むアデノ随伴ウイルス（ａａｖ）を用いた神経学的疾患の処置
WO2017181113A1 (en) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
WO2018045347A1 (en) * 2016-09-02 2018-03-08 Spark Therapeutics, Inc. Methods and vectors for treating cns disorders
JP2018506585A (ja) * 2015-02-10 2018-03-08 ジェンザイム・コーポレーション 線条体および皮質へのウイルス粒子の強化された送達
WO2018085688A1 (en) * 2016-11-04 2018-05-11 The Children's Hospital Of Philadelphia Gene transfer compositions, methods and uses for treating neurodegenerative diseases
WO2018160585A2 (en) * 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of spinal muscular atrophy
WO2018209205A1 (en) * 2017-05-11 2018-11-15 The Trustees Of The University Of Pennsylvania Gene therapy for neuronal ceroid lipofuscinoses

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120322861A1 (en) * 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
JP5332064B2 (ja) * 2007-03-01 2013-11-06 ウェルスタット イムノセラピューティクス， エルエルシー 炎症により特徴付けられる疾患の治療
WO2012135857A1 (en) * 2011-03-31 2012-10-04 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
CA2853482C (en) * 2011-10-28 2020-05-19 Joshua Grieger Cell line for production of adeno-associated virus
US20140314740A1 (en) * 2011-12-09 2014-10-23 The Johns Hopkins University Compositions and methods for the prevention or treatment of diabetic complications
US20140359799A1 (en) * 2011-12-23 2014-12-04 Case Western Reserve University Targeted gene modification using hybrid recombinant adeno-associated virus
TWI752907B (zh) * 2015-05-08 2022-01-21 美商拜奧馬林製藥公司 用於治療cln2疾病之tpp1調配物及方法

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160108099A1 (en) * 2011-10-27 2016-04-21 Wellstat Ophthalmics Corporation Vectors encoding rod-derived cone viability factor
JP2016535587A (ja) * 2013-10-24 2016-11-17 ユニキュアー アイピー ビー．ブイ． Ａａｖ５カプシドタンパク質を含むアデノ随伴ウイルス（ａａｖ）を用いた神経学的疾患の処置
JP2018506585A (ja) * 2015-02-10 2018-03-08 ジェンザイム・コーポレーション 線条体および皮質へのウイルス粒子の強化された送達
WO2017181113A1 (en) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
WO2018045347A1 (en) * 2016-09-02 2018-03-08 Spark Therapeutics, Inc. Methods and vectors for treating cns disorders
WO2018085688A1 (en) * 2016-11-04 2018-05-11 The Children's Hospital Of Philadelphia Gene transfer compositions, methods and uses for treating neurodegenerative diseases
WO2018160585A2 (en) * 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of spinal muscular atrophy
WO2018209205A1 (en) * 2017-05-11 2018-11-15 The Trustees Of The University Of Pennsylvania Gene therapy for neuronal ceroid lipofuscinoses

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
HOCQUEMILLER, M., ET AL.: "Adeno-Associated Virus-Based Gene Therapy for CNS Diseases", HUMAN GENE THERAPY, vol. 27, no. 7, JPN6023052670, 2016, pages 478 - 496, ISSN: 0005496479 *
KATZ, ML., ET AL.: "AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form o", SCIENCE TRANSLATIONAL MEDICINE, vol. Vol.7, No.313, Article No.313ra180, JPN6023052671, 2015, pages 1 - 10, ISSN: 0005496478 *

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SG11202104295UA (en) 2021-06-29
EP3880235A1 (en) 2021-09-22
IL282808A (en) 2021-06-30
MX2021005517A (es) 2021-06-18
JP2025128087A (ja) 2025-09-02
EP3880235A4 (en) 2022-08-10
US20210393802A1 (en) 2021-12-23
WO2020102369A1 (en) 2020-05-22
IL282808B1 (en) 2024-10-01
KR20210092755A (ko) 2021-07-26
AU2019379141A1 (en) 2021-06-03
IL282808B2 (en) 2025-02-01
CA3117982A1 (en) 2020-05-22

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