KR20220133941A - 안과 유전자 치료를 위한 변형 아데노-관련 바이러스 캡시드 단백질 및 이의 사용 방법 - Google Patents

안과 유전자 치료를 위한 변형 아데노-관련 바이러스 캡시드 단백질 및 이의 사용 방법 Download PDF

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KR20220133941A
KR20220133941A KR1020227029428A KR20227029428A KR20220133941A KR 20220133941 A KR20220133941 A KR 20220133941A KR 1020227029428 A KR1020227029428 A KR 1020227029428A KR 20227029428 A KR20227029428 A KR 20227029428A KR 20220133941 A KR20220133941 A KR 20220133941A
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amino acid
raav
capsid protein
modified
numbering
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Korean (ko)
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아미 프레더릭
샤오잉 진
린 리우
캐서린 오리오던
제니퍼 설리번
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젠자임 코포레이션
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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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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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    • 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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    • A—HUMAN NECESSITIES
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KR1020227029428A 2020-01-29 2021-01-29 안과 유전자 치료를 위한 변형 아데노-관련 바이러스 캡시드 단백질 및 이의 사용 방법 Pending KR20220133941A (ko)

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
KR20220133941A true KR20220133941A (ko) 2022-10-05

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KR1020227029428A Pending KR20220133941A (ko) 2020-01-29 2021-01-29 안과 유전자 치료를 위한 변형 아데노-관련 바이러스 캡시드 단백질 및 이의 사용 방법

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Country Link
US (1) US20210261625A1 (enExample)
EP (1) EP4097121A1 (enExample)
JP (1) JP7866941B2 (enExample)
KR (1) KR20220133941A (enExample)
CN (1) CN115989234A (enExample)
AR (1) AR121228A1 (enExample)
AU (2) AU2021213786A1 (enExample)
BR (1) BR112022014852A2 (enExample)
CA (1) CA3165019A1 (enExample)
CO (1) CO2022010227A2 (enExample)
IL (1) IL294868A (enExample)
MX (1) MX2022009252A (enExample)
PH (1) PH12022551878A1 (enExample)
WO (1) WO2021155137A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
US20260000790A1 (en) 2022-07-08 2026-01-01 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

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CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
NZ500427A (en) 1997-04-21 2002-12-20 Univ Florida The use of ribozyme treatment for retinal diseases
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6995006B2 (en) 1997-09-05 2006-02-07 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
JP4559429B2 (ja) 2003-05-21 2010-10-06 ジェンザイム・コーポレーション 空キャプシドを実質的に含まない組換えａａｖビリオン調製物を生成するための方法
DK2623605T3 (en) 2007-04-09 2019-03-25 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
JP5956331B2 (ja) 2009-06-16 2016-07-27 ジェンザイム・コーポレーション 組換えａａｖベクターの改良された精製方法
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
CA2826273C (en) * 2011-02-10 2021-11-02 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
PT3628334T (pt) * 2014-03-21 2023-09-26 Genzyme Corp Terapia genética para retinopatia pigmentar
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
WO2016065001A1 (en) * 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
HUE053747T2 (hu) 2016-08-15 2021-07-28 Genzyme Corp Eljárások AAV kimutatására
CA3033125A1 (en) * 2016-08-16 2018-02-22 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
WO2019221992A1 (en) * 2018-05-15 2019-11-21 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

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Publication number Publication date
PH12022551878A1 (en) 2024-02-12
TW202142552A (zh) 2021-11-16
IL294868A (en) 2022-09-01
AU2026203237A1 (en) 2026-05-14
US20210261625A1 (en) 2021-08-26
JP7866941B2 (ja) 2026-05-28
JP2023513004A (ja) 2023-03-30
AU2021213786A1 (en) 2022-09-22
EP4097121A1 (en) 2022-12-07
CA3165019A1 (en) 2021-08-05
CO2022010227A2 (es) 2022-08-09
MX2022009252A (es) 2022-11-09
BR112022014852A2 (pt) 2022-10-11
CN115989234A (zh) 2023-04-18
AR121228A1 (es) 2022-04-27
WO2021155137A1 (en) 2021-08-05

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