JP2021522842A - 肝臓を標的とするアデノ随伴ウイルスベクター - Google Patents

肝臓を標的とするアデノ随伴ウイルスベクター Download PDF

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JP2021522842A
JP2021522842A JP2020564217A JP2020564217A JP2021522842A JP 2021522842 A JP2021522842 A JP 2021522842A JP 2020564217 A JP2020564217 A JP 2020564217A JP 2020564217 A JP2020564217 A JP 2020564217A JP 2021522842 A JP2021522842 A JP 2021522842A
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aav
seq
protein
capsid
sequence
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JP2021522842A5 (enExample
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キャメロン コロシ ピーター
キャメロン コロシ ピーター
ラミレズ シルビア
ラミレズ シルビア
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ビオマリン プハルマセウトイカル インコーポレイテッド
ビオマリン プハルマセウトイカル インコーポレイテッド
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
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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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    • A61K48/0025—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 wherein the non-active part clearly interacts with the delivered nucleic acid
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2020564217A 2018-05-14 2019-05-14 肝臓を標的とするアデノ随伴ウイルスベクター Ceased JP2021522842A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862671265P 2018-05-14 2018-05-14
US62/671,265 2018-05-14
PCT/US2019/032097 WO2019222136A2 (en) 2018-05-14 2019-05-14 Novel liver targeting adeno-associated viral vectors

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JP2021522842A true JP2021522842A (ja) 2021-09-02
JP2021522842A5 JP2021522842A5 (enExample) 2022-05-24

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US (1) US11821008B2 (enExample)
EP (1) EP3794016A2 (enExample)
JP (1) JP2021522842A (enExample)
KR (1) KR20210010496A (enExample)
CN (1) CN112424217A (enExample)
AR (1) AR115097A1 (enExample)
AU (1) AU2019268302A1 (enExample)
BR (1) BR112020023292A2 (enExample)
CA (1) CA3100001A1 (enExample)
CL (2) CL2020002960A1 (enExample)
MX (1) MX2020012170A (enExample)
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PH (1) PH12020551938A1 (enExample)
SG (1) SG11202011145UA (enExample)
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WO (1) WO2019222136A2 (enExample)

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JP6994018B2 (ja) 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド 新規アデノ随伴ウイルスキャプシドタンパク質
KR20210008501A (ko) 2018-05-09 2021-01-22 바이오마린 파머수티컬 인크. 페닐케톤뇨증을 치료하는 방법
WO2020232044A1 (en) 2019-05-14 2020-11-19 Biomarin Pharmaceutical Inc. Methods of redosing gene therapy vectors
WO2021236908A2 (en) 2020-05-20 2021-11-25 Biomarin Pharmaceutical Inc. Use of regulatory proteins for the production of adeno-associated virus
CN115925818B (zh) * 2020-05-22 2026-02-06 中国医学科学院血液病医院(中国医学科学院血液学研究所) 腺相关病毒突变体及其应用
CN116121274B (zh) * 2020-07-29 2024-11-19 北京三诺佳邑生物技术有限责任公司 一组肝靶向新型腺相关病毒的获得及其应用
KR20230078805A (ko) 2020-11-02 2023-06-02 바이오마린 파머수티컬 인크. 아데노-연관 바이러스의 농축 공정
WO2023034980A1 (en) 2021-09-03 2023-03-09 Bomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034990A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
US20240384242A1 (en) 2021-09-03 2024-11-21 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
JP2024534894A (ja) 2021-09-03 2024-09-26 ビオマリン プハルマセウトイカル インコーポレイテッド Ａａｖカプシド組成物及び送達方法
EP4396201A1 (en) 2021-09-03 2024-07-10 BioMarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034989A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
CA3248631A1 (en) * 2022-01-21 2023-07-27 AstraZeneca Ireland Limited GENE THERAPY FOR GAUCHER'S DISEASE
UY40443A (es) 2022-09-22 2024-02-15 Biomarin Pharm Inc Tratamiento de la miocardiopatía con vectores de genoterapia con aav
UY40442A (es) 2022-09-22 2024-02-15 Biomarin Pharm Inc Tratamiento de la miocardiopatía arritmogénica con vectores de genoterapia con aav
WO2025051970A2 (en) 2023-09-06 2025-03-13 Dinaqor Ag Methods of treatment of hypertrophic cardiomyopathy with aav gene therapy vectors and therapeutic formulations
WO2025214477A1 (en) 2024-04-12 2025-10-16 Skyline Therapeutics (Shanghai) Co., Ltd. Treatment of genetic cardiomyopathies with aav gene therapy vectors
WO2026024045A1 (ko) * 2024-07-22 2026-01-29 한국생명공학연구원 변이체 캡시드를 포함하는 아데노-연관 바이러스 및 이의 용도

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