CN110290814A - 用于治疗威尔逊病的基因疗法 - Google Patents

用于治疗威尔逊病的基因疗法 Download PDF

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Publication number
CN110290814A
CN110290814A CN201780085376.9A CN201780085376A CN110290814A CN 110290814 A CN110290814 A CN 110290814A CN 201780085376 A CN201780085376 A CN 201780085376A CN 110290814 A CN110290814 A CN 110290814A
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raav
atp7b
copper
mice
aav8
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詹姆斯·M·威尔逊
詹妮·艾格尼丝·西德兰
拉克什曼安·戈文达萨米
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University of Pennsylvania Penn
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    • A—HUMAN NECESSITIES
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P3/00—Drugs for disorders of the metabolism
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
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    • C12N2750/14011—Parvoviridae
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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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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  • Wood Science & Technology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Gastroenterology & Hepatology (AREA)
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  • Neurosurgery (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Environmental Sciences (AREA)
CN201780085376.9A 2016-12-30 2017-12-29 用于治疗威尔逊病的基因疗法 Pending CN110290814A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662440659P 2016-12-30 2016-12-30
US62/440,659 2016-12-30
US201762473656P 2017-03-20 2017-03-20
US62/473,656 2017-03-20
PCT/US2017/068919 WO2018126116A1 (en) 2016-12-30 2017-12-29 Gene therapy for treating wilson's disease

Publications (1)

Publication Number Publication Date
CN110290814A true CN110290814A (zh) 2019-09-27

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CN201780085376.9A Pending CN110290814A (zh) 2016-12-30 2017-12-29 用于治疗威尔逊病的基因疗法

Country Status (9)

Country Link
US (2) US11473106B2 (https=)
EP (2) EP3562514B1 (https=)
JP (1) JP7128190B2 (https=)
KR (2) KR102863181B1 (https=)
CN (1) CN110290814A (https=)
BR (1) BR112019013245A2 (https=)
CA (1) CA3048044A1 (https=)
MX (1) MX2019007873A (https=)
WO (1) WO2018126116A1 (https=)

Cited By (4)

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CN115216533A (zh) * 2022-06-30 2022-10-21 湖南家辉生物技术有限公司 一种用于诊断威尔逊病的生物标志物、扩增引物组、检测试剂及应用
CN115216481A (zh) * 2021-04-21 2022-10-21 四川至善唯新生物科技有限公司 一种表达量提高的截短型atp7b基因及其应用
CN117947035A (zh) * 2024-03-25 2024-04-30 上海凌医生物科技有限公司 一种增强基因表达的启动子
CN120457199A (zh) * 2023-03-06 2025-08-08 凌意(杭州)生物科技有限公司 截短的atp7b及其用途

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BR112019013245A2 (pt) 2016-12-30 2020-02-11 The Trustees Of The University Of Pennsylvania Terapia gênica para tratamento da doença de wilson
EP3717505A4 (en) 2017-12-01 2021-12-01 Encoded Therapeutics, Inc. GENENICALLY MODIFIED DNA-BINDING PROTEINS
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
BR112021006052A2 (pt) 2018-10-01 2021-09-08 Ultragenyx Pharmaceutical Inc. Terapia gênica para tratar acidemia propiônica
EP3880809A4 (en) * 2018-11-16 2023-01-11 Encoded Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF WILSON'S DISEASE
DK3906066T5 (da) 2019-01-04 2024-08-05 Ultragenyx Pharmaceutical Inc Geneterapikonstruktioner til behandling af wilsons sygdom
PL3976798T3 (pl) 2019-05-29 2026-04-07 Encoded Therapeutics, Inc. Kompozycje i sposoby selektywnej regulacji genów
CN111088285B (zh) * 2019-08-15 2021-12-28 北京锦篮基因科技有限公司 携带atp7b基因表达框及变异体的aav载体及应用
KR102490457B1 (ko) * 2019-11-26 2023-01-19 한국생명공학연구원 단백질 발현용 mRNA 구조체 및 이의 용도
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
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
US20240307553A1 (en) * 2021-04-01 2024-09-19 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
TW202323528A (zh) * 2021-10-18 2023-06-16 美商邏輯生物療法公司 用於治療威爾森氏病之基因療法
US20250320509A1 (en) * 2022-05-23 2025-10-16 The Johns Hopkins University Compositions and methods for treating wilson's disease
WO2024193638A1 (en) * 2023-03-22 2024-09-26 Skyline Therapeutics (Shanghai) Co., Ltd. Recombinant aav for the gene therapy of wilson disease
US20240360431A1 (en) * 2023-04-30 2024-10-31 Sichuan Real&Best Biotech Co., Ltd. Engineered alpha-galactosidase a (a-gal a) peptides and functional variants thereof and associated methods of treating fabry disease
CN120882865A (zh) * 2023-07-28 2025-10-31 凌意(杭州)生物科技有限公司 用于治疗威尔逊病的多核苷酸
WO2025110984A1 (en) * 2023-11-21 2025-05-30 Hydrogene Therapeutics, Inc. Compositions and methods for treating wilson's disease

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WO2016097219A1 (en) * 2014-12-17 2016-06-23 Fundación Para La Investigación Mèdica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson disease
CN113302290A (zh) * 2018-11-16 2021-08-24 编码治疗公司 治疗威尔逊氏病的组合物和方法

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Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115216481A (zh) * 2021-04-21 2022-10-21 四川至善唯新生物科技有限公司 一种表达量提高的截短型atp7b基因及其应用
CN115216533A (zh) * 2022-06-30 2022-10-21 湖南家辉生物技术有限公司 一种用于诊断威尔逊病的生物标志物、扩增引物组、检测试剂及应用
CN120457199A (zh) * 2023-03-06 2025-08-08 凌意(杭州)生物科技有限公司 截短的atp7b及其用途
CN117947035A (zh) * 2024-03-25 2024-04-30 上海凌医生物科技有限公司 一种增强基因表达的启动子

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US20220389455A1 (en) 2022-12-08
BR112019013245A2 (pt) 2020-02-11
JP2020514286A (ja) 2020-05-21
EP3562514A1 (en) 2019-11-06
EP4361277A3 (en) 2024-07-31
EP4361277A2 (en) 2024-05-01
CA3048044A1 (en) 2018-07-05
US11473106B2 (en) 2022-10-18
KR102604096B1 (ko) 2023-11-23
KR20230160411A (ko) 2023-11-23
US12460228B2 (en) 2025-11-04
MX2019007873A (es) 2019-11-18
EP3562514B1 (en) 2026-04-29
JP7128190B2 (ja) 2022-08-30
WO2018126116A1 (en) 2018-07-05
EP3562514A4 (en) 2020-08-05
KR102863181B1 (ko) 2025-09-23
US20190338310A1 (en) 2019-11-07
KR20190101410A (ko) 2019-08-30

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