CN115989234A - 用于眼部基因疗法的经修饰的腺相关病毒衣壳蛋白及其使用方法 - Google Patents

用于眼部基因疗法的经修饰的腺相关病毒衣壳蛋白及其使用方法 Download PDF

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CN115989234A
CN115989234A CN202180011349.3A CN202180011349A CN115989234A CN 115989234 A CN115989234 A CN 115989234A CN 202180011349 A CN202180011349 A CN 202180011349A CN 115989234 A CN115989234 A CN 115989234A
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amino acid
raav
capsid protein
numbering
nucleic acid
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CN202180011349.3A
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English (en)
Chinese (zh)
Inventor
A·弗雷德里克
X·金
L·刘
C·奥瑞奥丹
J·沙利文
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Genzyme Corp
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Genzyme Corp
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    • C07—ORGANIC CHEMISTRY
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    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
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    • A61K48/0041—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 the non-active part being polymeric
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    • C12N2750/14011—Parvoviridae
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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
    • C12N2750/14145—Special targeting system for viral vectors

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  • Ophthalmology & Optometry (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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CN202180011349.3A 2020-01-29 2021-01-29 用于眼部基因疗法的经修饰的腺相关病毒衣壳蛋白及其使用方法 Pending CN115989234A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062967416P 2020-01-29 2020-01-29
US62/967,416 2020-01-29
PCT/US2021/015695 WO2021155137A1 (en) 2020-01-29 2021-01-29 Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof

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Publication Number Publication Date
CN115989234A true CN115989234A (zh) 2023-04-18

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CN202180011349.3A Pending CN115989234A (zh) 2020-01-29 2021-01-29 用于眼部基因疗法的经修饰的腺相关病毒衣壳蛋白及其使用方法

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US (1) US20210261625A1 (https=)
EP (1) EP4097121A1 (https=)
JP (1) JP2023513004A (https=)
KR (1) KR20220133941A (https=)
CN (1) CN115989234A (https=)
AR (1) AR121228A1 (https=)
AU (1) AU2021213786A1 (https=)
BR (1) BR112022014852A2 (https=)
CA (1) CA3165019A1 (https=)
CO (1) CO2022010227A2 (https=)
IL (1) IL294868A (https=)
MX (1) MX2022009252A (https=)
PH (1) PH12022551878A1 (https=)
TW (1) TW202142552A (https=)
WO (1) WO2021155137A1 (https=)

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Publication number Priority date Publication date Assignee Title
MX2024004217A (es) 2021-10-08 2024-06-26 Dyno Therapeutics Inc Variantes de cápside y métodos de uso de estas.
EP4551253A1 (en) 2022-07-08 2025-05-14 Ospedale San Raffaele S.r.l. Transgene cassettes
EP4303226A1 (en) 2022-07-08 2024-01-10 Ospedale San Raffaele S.r.l. Transgene cassettes and epigenetic silencers for the treatment of disorders
KR102901502B1 (ko) 2024-06-27 2025-12-18 이엔셀 주식회사 안구 세포 또는 조직을 표적하는 펩타이드 및 이를 포함하는 바이러스 캡시드 단백질 및 바이러스 벡터

Family Cites Families (18)

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AU722624B2 (en) 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
US6225291B1 (en) 1997-04-21 2001-05-01 University Of Florida Rod opsin mRNA-specific ribozyme compositions and methods for the treatment of retinal diseases
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
DE602004030327D1 (de) 2003-05-21 2011-01-13 Genzyme Corp Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind
DK2191001T3 (en) 2007-04-09 2016-09-19 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
US20100081707A1 (en) 2008-02-21 2010-04-01 Ali Robin R Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
HUE028341T2 (en) 2009-06-16 2016-12-28 Genzyme Corp Improved methods for purification of recombinant aav vectors
EP2826860B1 (en) 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
WO2012109570A1 (en) * 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
FI3628334T3 (fi) * 2014-03-21 2023-09-15 Genzyme Corp Geenihoito verkkokalvon pigmenttirappeuma
IL248102B (en) * 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
AU2015335923B2 (en) * 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
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US20190262373A1 (en) * 2016-08-16 2019-08-29 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
US12338267B2 (en) * 2018-05-15 2025-06-24 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties

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Publication number Publication date
IL294868A (en) 2022-09-01
MX2022009252A (es) 2022-11-09
US20210261625A1 (en) 2021-08-26
CA3165019A1 (en) 2021-08-05
EP4097121A1 (en) 2022-12-07
AR121228A1 (es) 2022-04-27
BR112022014852A2 (pt) 2022-10-11
WO2021155137A1 (en) 2021-08-05
AU2021213786A1 (en) 2022-09-22
TW202142552A (zh) 2021-11-16
CO2022010227A2 (es) 2022-08-09
JP2023513004A (ja) 2023-03-30
KR20220133941A (ko) 2022-10-05
PH12022551878A1 (en) 2024-02-12

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