KR20210092755A - 신경원성 세로이드 리포푸신증에 대한 유전자 요법 - Google Patents

신경원성 세로이드 리포푸신증에 대한 유전자 요법 Download PDF

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KR20210092755A
KR20210092755A KR1020217017295A KR20217017295A KR20210092755A KR 20210092755 A KR20210092755 A KR 20210092755A KR 1020217017295 A KR1020217017295 A KR 1020217017295A KR 20217017295 A KR20217017295 A KR 20217017295A KR 20210092755 A KR20210092755 A KR 20210092755A
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raav
pharmaceutical composition
certain embodiments
tpp1
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Korean (ko)
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바르가비 콘드라군타
수브하 카루머틸 메렛힐
마티유 피에르 구이베르트
앤드류 크리스토퍼 머서
마이클 제임스 카렐
로버트 토마스 스타델만
프란즈 마이클 게르너
니콜라스 알렉산더 피어스 사샤 버스
자레드 비
트리스탄 제임스 마샬
로베르토 데파즈
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리젠엑스바이오 인크.
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
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    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14001—Dipeptidyl-peptidase I (3.4.14.1), i.e. cathepsin-C
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    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
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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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Botany (AREA)
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KR1020217017295A 2018-11-14 2019-11-13 신경원성 세로이드 리포푸신증에 대한 유전자 요법 Ceased KR20210092755A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
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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Publication Number Publication Date
KR20210092755A true KR20210092755A (ko) 2021-07-26

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KR1020217017295A Ceased KR20210092755A (ko) 2018-11-14 2019-11-13 신경원성 세로이드 리포푸신증에 대한 유전자 요법

Country Status (11)

Country Link
US (1) US20210393802A1 (https=)
EP (1) EP3880235A4 (https=)
JP (2) JP2022513034A (https=)
KR (1) KR20210092755A (https=)
AU (1) AU2019379141A1 (https=)
BR (1) BR112021009370A2 (https=)
CA (1) CA3117982A1 (https=)
IL (2) IL315701B1 (https=)
MX (1) MX2021005517A (https=)
SG (1) SG11202104295UA (https=)
WO (1) WO2020102369A1 (https=)

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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

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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
CA2853379C (en) * 2011-10-27 2020-11-24 Wellstat Ophthalmics Corporation Vectors encoding rod-derived cone viability factor
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
CN105745326A (zh) * 2013-10-24 2016-07-06 优尼科Ip有限公司 用于基因治疗神经疾病的aav-5假型载体
CN107530447A (zh) * 2015-02-10 2018-01-02 建新公司 病毒颗粒至纹状体和皮质的增强递送
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
SG10201912761UA (en) * 2016-04-15 2020-02-27 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
JP7610923B2 (ja) * 2016-09-02 2025-01-09 スパーク セラピューティクス インコーポレイテッド Ｃｎｓ障害を治療するための方法及びベクター
CA3041548A1 (en) * 2016-11-04 2018-05-11 The Children's Hospital Of Philadelphia Gene transfer compositions, methods and uses for treating neurodegenerative diseases
JOP20190200A1 (ar) * 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
IL316926A (en) * 2017-05-11 2025-01-01 Univ Pennsylvania Gene therapy for neuronal ceroid lipofuscinosis

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BR112021009370A2 (pt) 2021-08-17
IL315701B1 (en) 2026-04-01
IL315701A (en) 2024-11-01
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
JP2022513034A (ja) 2022-02-07
IL282808B1 (en) 2024-10-01
AU2019379141A1 (en) 2021-06-03
IL282808B2 (en) 2025-02-01
CA3117982A1 (en) 2020-05-22

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