JP2023523622A - 遺伝性黄斑変性症の治療のための組成物及び方法 - Google Patents

遺伝性黄斑変性症の治療のための組成物及び方法 Download PDF

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JP2023523622A
JP2023523622A JP2022565707A JP2022565707A JP2023523622A JP 2023523622 A JP2023523622 A JP 2023523622A JP 2022565707 A JP2022565707 A JP 2022565707A JP 2022565707 A JP2022565707 A JP 2022565707A JP 2023523622 A JP2023523622 A JP 2023523622A
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transposase
nucleic acid
composition
abca4
cells
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ヒギンズ、ジョセフ、ジェイ．
マクミラン、スコット
タビビアザール、レイ
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サリオジェン セラピューティクス インコーポレイテッド
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/007—Vector systems having a special element relevant for transcription cell cycle specific enhancer/promoter combination

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2022565707A 2020-04-29 2021-04-29 遺伝性黄斑変性症の治療のための組成物及び方法 Pending JP2023523622A (ja)

Applications Claiming Priority (3)

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US202063017442P 2020-04-29 2020-04-29
US63/017,442 2020-04-29
PCT/US2021/030007 WO2021222654A1 (en) 2020-04-29 2021-04-29 Compositions and methods for treatment of inherited macular degeneration

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JP2023523622A true JP2023523622A (ja) 2023-06-06
JP2023523622A5 JP2023523622A5 (https=) 2024-05-15

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US (1) US20230193319A1 (https=)
EP (1) EP4142796A4 (https=)
JP (1) JP2023523622A (https=)
KR (1) KR20230004573A (https=)
CN (1) CN115461082A (https=)
AU (1) AU2021265838A1 (https=)
CA (1) CA3175197A1 (https=)
IL (1) IL297718A (https=)
WO (1) WO2021222654A1 (https=)

Families Citing this family (6)

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Publication number Priority date Publication date Assignee Title
EP4146798A4 (en) 2020-05-04 2025-02-12 Saliogen Therapeutics, Inc. TRANSPOSITION-BASED THERAPIES
WO2023160454A1 (zh) * 2022-02-25 2023-08-31 北京中因科技有限公司 表达盒组合及其应用
EP4511496A2 (en) * 2022-04-18 2025-02-26 Abeona Therapeutics Inc. Compositions and methods for treating dominant optic atrophy and x-linked retinoschisis
CN120230798A (zh) * 2023-12-29 2025-07-01 上海朗昇生物科技有限公司 用于视网膜基因递送的重组腺相关病毒载体及其应用
WO2025147466A1 (en) * 2024-01-02 2025-07-10 Alkeus Pharmaceuticals, Inc. Treatment for asymptomatic stargardt disease patients
WO2025188836A1 (en) * 2024-03-05 2025-09-12 Saliogen Therapeutics, Inc. Compositions and methods of using gene coding for treating ocular disease

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2010540541A (ja) * 2007-09-27 2010-12-24 リヴィジョン セラピューティクス，インク． レチノール関連疾患を治療するための方法及び化合物
US20110311506A1 (en) * 2009-02-25 2011-12-22 The Johns Hopkins University Piggybac transposon variants and methods of use
JP2016516424A (ja) * 2013-04-18 2016-06-09 フォンダッツィオーネ・テレソン デュアルａａｖベクターによる大型遺伝子の効果的送達
JP2016522674A (ja) * 2012-05-16 2016-08-04 ラナ セラピューティクス インコーポレイテッド 遺伝子発現を調節するための組成物及び方法
JP2019500348A (ja) * 2015-12-14 2019-01-10 コールド スプリング ハーバー ラボラトリー 眼疾患の処置のための組成物および方法
WO2019067766A1 (en) * 2017-09-27 2019-04-04 Sigilon Therapeutics, Inc. METHODS, COMPOSITIONS AND IMPLANTABLE ELEMENTS COMPRISING ACTIVE CELLS

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7309487B2 (en) * 2004-02-09 2007-12-18 George Inana Methods and compositions for detecting and treating retinal diseases
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
WO2008137066A1 (en) * 2007-05-02 2008-11-13 The Board Of Regents Of The University Of Oklahoma Use of compacted nucleic acid nanoparticles in non-viral treatments of ocular diseases
US20090214478A1 (en) * 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
AU2015234242B2 (en) * 2014-03-21 2020-09-24 Children's Medical Research Institute Stable gene transfer to proliferating cells
WO2020077360A1 (en) * 2018-10-12 2020-04-16 Poseida Therapeutics, Inc. Hematopoietic stem cell compositions, methods of making and method of use

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2010540541A (ja) * 2007-09-27 2010-12-24 リヴィジョン セラピューティクス，インク． レチノール関連疾患を治療するための方法及び化合物
US20110311506A1 (en) * 2009-02-25 2011-12-22 The Johns Hopkins University Piggybac transposon variants and methods of use
JP2016522674A (ja) * 2012-05-16 2016-08-04 ラナ セラピューティクス インコーポレイテッド 遺伝子発現を調節するための組成物及び方法
JP2016516424A (ja) * 2013-04-18 2016-06-09 フォンダッツィオーネ・テレソン デュアルａａｖベクターによる大型遺伝子の効果的送達
JP2019500348A (ja) * 2015-12-14 2019-01-10 コールド スプリング ハーバー ラボラトリー 眼疾患の処置のための組成物および方法
WO2019067766A1 (en) * 2017-09-27 2019-04-04 Sigilon Therapeutics, Inc. METHODS, COMPOSITIONS AND IMPLANTABLE ELEMENTS COMPRISING ACTIVE CELLS

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Publication number Publication date
EP4142796A4 (en) 2024-06-26
AU2021265838A1 (en) 2022-10-20
IL297718A (en) 2022-12-01
US20230193319A1 (en) 2023-06-22
CA3175197A1 (en) 2021-11-04
CN115461082A (zh) 2022-12-09
KR20230004573A (ko) 2023-01-06
WO2021222654A1 (en) 2021-11-04
EP4142796A1 (en) 2023-03-08

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