JP2023513004A - 眼科遺伝子療法のための改変されたアデノ随伴ウイルスカプシドタンパク質およびその使用方法 - Google Patents

眼科遺伝子療法のための改変されたアデノ随伴ウイルスカプシドタンパク質およびその使用方法 Download PDF

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JP2023513004A
JP2023513004A JP2022545976A JP2022545976A JP2023513004A JP 2023513004 A JP2023513004 A JP 2023513004A JP 2022545976 A JP2022545976 A JP 2022545976A JP 2022545976 A JP2022545976 A JP 2022545976A JP 2023513004 A JP2023513004 A JP 2023513004A
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amino acid
raav
capsid protein
modified
nucleic acid
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JP2023513004A5 (https=
JPWO2021155137A5 (https=
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エイミー・フレドリック
シャオイン・ジン
リン・リウ
キャサリン・オリオーダン
ジェニファー・サリバン
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Genzyme Corp
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Genzyme Corp
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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
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • 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
    • 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
    • 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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    • 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
    • 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
    • A—HUMAN NECESSITIES
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    • 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
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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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    • C12N2750/14011—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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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Ophthalmology & Optometry (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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JP2022545976A 2020-01-29 2021-01-29 眼科遺伝子療法のための改変されたアデノ随伴ウイルスカプシドタンパク質およびその使用方法 Pending JP2023513004A (ja)

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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JP2023513004A true JP2023513004A (ja) 2023-03-30
JP2023513004A5 JP2023513004A5 (https=) 2024-02-02
JPWO2021155137A5 JPWO2021155137A5 (https=) 2024-02-02

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JP2022545976A Pending JP2023513004A (ja) 2020-01-29 2021-01-29 眼科遺伝子療法のための改変されたアデノ随伴ウイルスカプシドタンパク質およびその使用方法

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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=)

Families Citing this family (4)

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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

Citations (3)

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Publication number Priority date Publication date Assignee Title
JP2017509632A (ja) * 2014-03-21 2017-04-06 ジェンザイム・コーポレーション 網膜色素変性症のための遺伝子治療
JP2017518271A (ja) * 2014-05-02 2017-07-06 ジェンザイム・コーポレーション 網膜およびｃｎｓ遺伝子治療用ａａｖベクター
JP2017532966A (ja) * 2014-10-21 2017-11-09 ユニバーシティ オブ マサチューセッツ 組み換えａａｖバリアントおよびその使用

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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
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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
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JP2017509632A (ja) * 2014-03-21 2017-04-06 ジェンザイム・コーポレーション 網膜色素変性症のための遺伝子治療
JP2017518271A (ja) * 2014-05-02 2017-07-06 ジェンザイム・コーポレーション 網膜およびｃｎｓ遺伝子治療用ａａｖベクター
JP2017532966A (ja) * 2014-10-21 2017-11-09 ユニバーシティ オブ マサチューセッツ 組み換えａａｖバリアントおよびその使用

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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
CN115989234A (zh) 2023-04-18
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
KR20220133941A (ko) 2022-10-05
PH12022551878A1 (en) 2024-02-12

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