CN107249646B - 用于青少年巴滕病的基因疗法 - Google Patents

用于青少年巴滕病的基因疗法 Download PDF

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CN107249646B
CN107249646B CN201580069717.4A CN201580069717A CN107249646B CN 107249646 B CN107249646 B CN 107249646B CN 201580069717 A CN201580069717 A CN 201580069717A CN 107249646 B CN107249646 B CN 107249646B
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cln3
vector
seq
aav
scaav9
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CN107249646A (zh
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塔米·基尔良
凯文·福斯特
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University of Nebraska System
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    • 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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    • 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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
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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
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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
    • C12N2750/14171—Demonstrated in vivo effect
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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CN201580069717.4A 2014-12-16 2015-12-16 用于青少年巴滕病的基因疗法 Expired - Fee Related CN107249646B (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201462092501P 2014-12-16 2014-12-16
US62/092,501 2014-12-16
US201562146793P 2015-04-13 2015-04-13
US62/146,793 2015-04-13
PCT/US2015/066206 WO2016100575A1 (en) 2014-12-16 2015-12-16 Gene therapy for juvenile batten disease

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CN107249646A CN107249646A (zh) 2017-10-13
CN107249646B true CN107249646B (zh) 2021-06-29

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US (2) US10876134B2 (enExample)
EP (1) EP3233131A1 (enExample)
JP (2) JP6754361B2 (enExample)
CN (1) CN107249646B (enExample)
AU (2) AU2015364636B9 (enExample)
CA (1) CA2967468A1 (enExample)
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Families Citing this family (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2013296425B2 (en) 2012-08-01 2018-06-07 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
CN115120746A (zh) 2013-05-15 2022-09-30 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
SG10201912761UA (en) 2016-04-15 2020-02-27 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
AU2017306558B2 (en) * 2016-08-03 2024-09-19 University Of South Florida Reelin compositions for treatment of neurological disorders
JP2020500928A (ja) * 2016-11-15 2020-01-16 リジェネクスバイオ インコーポレイテッド Ｍｐｓｉおよびｍｐｓｉｉならびに他の神経障害において神経機能を改善するための方法
JP7106534B2 (ja) * 2016-11-17 2022-07-26 ネイションワイド チルドレンズ ホスピタル， インコーポレイテッド メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達
CN118374493A (zh) * 2017-05-17 2024-07-23 西雅图儿童医院（Dba西雅图儿童研究所） 生成哺乳动物t细胞活化诱导型合成启动子(syn+pro)以改善t细胞疗法
KR102883778B1 (ko) * 2017-09-22 2025-11-12 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
WO2019094253A1 (en) 2017-11-08 2019-05-16 Avexis Inc. Means and method for preparing viral vectors and uses of same
SI3740222T1 (sl) * 2018-01-17 2023-11-30 Meiragtx Uk Ii Limited Modificiran kapsidni protein raav za gensko terapijo
US20190256867A1 (en) 2018-02-01 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
EP3801638A1 (en) 2018-06-08 2021-04-14 Novartis AG Cell-based assay for measuring drug product potency
CA3129077A1 (en) * 2019-02-04 2020-08-13 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery of cln3 polynucleotide
JP2022519597A (ja) * 2019-02-04 2022-03-24 リサーチ・インスティチュート・アット・ネーションワイド・チルドレンズ・ホスピタル Ｃｌｎ６ポリヌクレオチドのアデノ随伴ウイルス送達
CA3132379A1 (en) * 2019-03-22 2020-10-01 Rush University Medical Center Combination of nasal gene delivery and oral cinnamic acid, oleamide or gemfibrozil for lysosomal storage disorders
CA3134501A1 (en) * 2019-03-22 2020-10-01 Encoded Therapeutics, Inc. Multiplexing regulatory elements to identify cell-type specific regulatory elements
MX2021013275A (es) * 2019-04-29 2022-03-17 Abeona Therapeutics Inc Terapia genica intratecal e intravenosa para el tratamiento de la enfermedad de batten infantil.
SG11202111375RA (en) 2019-04-29 2021-11-29 Univ Pennsylvania Novel aav capsids and compositions containing same
US20220233655A1 (en) * 2019-05-17 2022-07-28 Research Institute At Nationwide Children's Hospital Improved delivery of gene therapy vectors to retinal cells using a glycoside hydrolase enzyme
CN114207135A (zh) 2019-06-11 2022-03-18 夏尔人类遗传性治疗公司 用于治疗由失调的血浆激肽释放酶介导的疾病的抗体的腺相关病毒载体递送
CA3158131A1 (en) * 2019-10-18 2021-04-22 Nicolas Sebastien Wein Materials and methods for the treatment of disorders associated mutations in the irf2bpl gene
WO2021081280A1 (en) 2019-10-23 2021-04-29 Shire Human Genetic Therapies, Inc. Adeno-associated virus vectors based gene therapy for hereditary angioedema
KR20220093337A (ko) * 2019-10-31 2022-07-05 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Cd38 녹아웃 일차 및 확장된 인간 nk 세포의 생성
TWI908746B (zh) * 2019-11-28 2025-12-21 美商銳進科斯生物股份有限公司 微小肌縮蛋白基因療法之構築體及其用途
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
BR112023002374A2 (pt) * 2020-08-12 2023-03-21 UCB Biopharma SRL Terapia genética usando construções de ácido nucléico compreendendo sequências promotoras de proteína 2 de ligação de metil cpg (mecp2)
CA3197316A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Neurod1 and dlx2 vector
WO2022072325A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Neurod1 combination vector
JP2023543360A (ja) * 2020-09-29 2023-10-13 ニューエクセル・セラピューティクス・インコーポレイテッド Ｄｌｘ２ベクター
US20220098616A1 (en) * 2020-09-29 2022-03-31 NeuExcell Therapeutics Inc. ISL1 and LHX3 VECTOR
EP4222270A4 (en) * 2020-09-29 2025-02-26 Neuexcell Therapeutics Inc. NEUROD1-VECTOR
US12019081B2 (en) 2020-11-06 2024-06-25 University Of Kentucky Research Foundation Method for detection and quantification of CLN3 protein
WO2022130014A1 (en) 2020-12-16 2022-06-23 Takeda Pharmaceutical Company Limited Adeno associated viral vector delivery of antibodies for the treatment of disease mediated by dysregulated plasma kallikrein
CN115487297A (zh) * 2021-06-17 2022-12-20 清华大学 治疗神经元蜡样质脂褐质沉积症的方法和药物
AU2022299552A1 (en) 2021-06-25 2024-01-04 Oxford Biomedica (Us) Llc Adeno-associated virus packaging systems
US20230090654A1 (en) 2021-08-24 2023-03-23 Homology Medicines, Inc. Adeno-associated virus formulations
WO2023077086A1 (en) 2021-10-29 2023-05-04 Homology Medicines, Inc. Methods and compositions for the purification of adeno-associated virus
WO2023114897A2 (en) 2021-12-15 2023-06-22 Homology Medicines, Inc. Methods and compositions for the production of adeno-associated virus
CA3263388A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. VIRAL PARTICLES RETRIBUTED TO TRANSFERRIN RECEPTOR 1
EP4651869A1 (en) * 2023-01-18 2025-11-26 Rush University Medical Center Oral gemfibrozil for the treatment of cln3-related juvenile neuronal ceroid lipofuscinosis
AU2024360964A1 (en) * 2023-10-11 2026-04-16 Emugen Therapeutics Llc Methods and compositions for altering gene expression
WO2025226836A1 (en) 2024-04-24 2025-10-30 Sarepta Therapeutics, Inc. Treatment of genetic neurological conditions with genomic editing
CN119265322B (zh) * 2024-12-10 2025-02-28 南京农业大学 一种与鸡蛋重性状相关的cspg4基因分子标记引物及其应用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997008308A1 (en) * 1995-08-31 1997-03-06 The General Hospital Corporation Batten disease gene
US20130225666A1 (en) * 2008-12-19 2013-08-29 Nationwide Children's Hospital, Inc. Delivery of polynucleotides using recombinant aav9

Family Cites Families (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
US5252334A (en) 1989-09-08 1993-10-12 Cygnus Therapeutic Systems Solid matrix system for transdermal drug delivery
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5697899A (en) 1995-02-07 1997-12-16 Gensia Feedback controlled drug delivery system
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US5656016A (en) 1996-03-18 1997-08-12 Abbott Laboratories Sonophoretic drug delivery system
US5797898A (en) 1996-07-02 1998-08-25 Massachusetts Institute Of Technology Microchip drug delivery devices
US5783208A (en) 1996-07-19 1998-07-21 Theratech, Inc. Transdermal drug delivery matrix for coadministering estradiol and another steroid
US5779708A (en) 1996-08-15 1998-07-14 Cyberdent, Inc. Intraosseous drug delivery device and method
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
CA2364417C (en) * 1999-02-19 2013-11-05 Matthew John During Peroral gene therapy of diabetes and obesity
US6582692B1 (en) 1999-11-17 2003-06-24 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
US20030083299A1 (en) 2000-11-04 2003-05-01 Ferguson Ian A. Non-invasive delivery of polypeptides through the blood-brain barrier
US6841357B1 (en) 2001-03-30 2005-01-11 Stratagene California Method and kits for titering adeno-associated virus vectors
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
DK2573170T3 (en) 2001-12-17 2018-04-09 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 9, vectors containing them, and their use
EP1620133B1 (en) 2003-05-01 2015-12-09 Genzyme Corporation Gene therapy for neurometabolic disorders
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
CN103849629B (zh) 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
EP2176283B1 (en) 2007-07-14 2016-11-02 University of Iowa Research Foundation Methods and compositions for treating brain diseases
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
WO2009137006A2 (en) 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
WO2010071832A1 (en) 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
HRP20250897T1 (hr) 2009-05-02 2025-09-26 Genzyme Corporation Genska terapija za neurodegenerativne poremećaje
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
WO2012057363A1 (ja) 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
AU2010366066B2 (en) 2010-12-22 2016-01-14 Fondazione Telethon Therapeutic strategies to treat CNS pathology in mucopolysaccharidoses
US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
KR102220688B1 (ko) 2012-07-04 2021-03-03 필립모리스 프로덕츠 에스.에이. 개선된 결합제를 갖는 가연성 열원
EP2900686B1 (en) 2012-09-28 2020-06-10 The University of North Carolina At Chapel Hill Aav vectors targeted to oligodendrocytes
ES2739288T3 (es) * 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
EP4344741A3 (en) 2014-11-21 2024-08-28 The University of North Carolina at Chapel Hill Aav vectors targeted to the central nervous system
KR102423442B1 (ko) * 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997008308A1 (en) * 1995-08-31 1997-03-06 The General Hospital Corporation Batten disease gene
US20130225666A1 (en) * 2008-12-19 2013-08-29 Nationwide Children's Hospital, Inc. Delivery of polynucleotides using recombinant aav9

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
Correction of Neurological Disease of Mucopolysaccharidosis IIIB in Adult Mice by rAAV9 Trans-Blood–Brain Barrier Gene Delivery;FU H.Y. et al.;《Molecular Therapy》;20110630;1025-1033 *
Partial Correction of the CNS Lysosomal Storage Defect in a Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis by Neonatal CNS Administration of an Adeno-Associated Virus Serotype rh.10 Vector Expressing the Human CLN3 Gene;SONDHI D. et al.;《HUMAN GENE THERAPY》;20140331;223-239 *
SONDHI D. et al..Partial Correction of the CNS Lysosomal Storage Defect in a Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis by Neonatal CNS Administration of an Adeno-Associated Virus Serotype rh.10 Vector Expressing the Human CLN3 Gene.《HUMAN GENE THERAPY》.2014,223-239. *

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EP3233131A1 (en) 2017-10-25
US20220049272A1 (en) 2022-02-17
AU2015364636B9 (en) 2021-12-02
US10876134B2 (en) 2020-12-29
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US20190032078A1 (en) 2019-01-31
US12297447B2 (en) 2025-05-13
JP6754361B2 (ja) 2020-09-09
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