JP2023540094A - Kh902(コンベルセプト)の送達のためのアデノ随伴ウイルスおよびその使用 - Google Patents

Kh902(コンベルセプト)の送達のためのアデノ随伴ウイルスおよびその使用 Download PDF

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JP2023540094A
JP2023540094A JP2023514415A JP2023514415A JP2023540094A JP 2023540094 A JP2023540094 A JP 2023540094A JP 2023514415 A JP2023514415 A JP 2023514415A JP 2023514415 A JP2023514415 A JP 2023514415A JP 2023540094 A JP2023540094 A JP 2023540094A
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raav
vegf
capsid protein
mir
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JPWO2022051537A5 (https=
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グアンピン ガオ,
フィリップ タイ,
クラウディオ プンゾ,
ハイジアン リン,
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ユニバーシティ オブ マサチューセッツ
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    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
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    • C12N2310/141MicroRNAs, miRNAs
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14171Demonstrated in vivo effect

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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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JP2023514415A 2020-09-03 2021-09-02 Kh902(コンベルセプト)の送達のためのアデノ随伴ウイルスおよびその使用 Pending JP2023540094A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063074361P 2020-09-03 2020-09-03
US63/074,361 2020-09-03
US202163179700P 2021-04-26 2021-04-26
US63/179,700 2021-04-26
PCT/US2021/048917 WO2022051537A1 (en) 2020-09-03 2021-09-02 Adeno-associated virus for delivery of kh902 (conbercept) and uses thereof

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JP2023540094A true JP2023540094A (ja) 2023-09-21
JPWO2022051537A5 JPWO2022051537A5 (https=) 2024-09-06
JP2023540094A5 JP2023540094A5 (https=) 2024-09-06

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US (1) US20230340529A1 (https=)
EP (1) EP4208478A4 (https=)
JP (1) JP2023540094A (https=)
KR (1) KR20230061441A (https=)
AU (1) AU2021336425A1 (https=)
BR (1) BR112023003548A2 (https=)
CA (1) CA3192736A1 (https=)
IL (1) IL300864A (https=)
MX (1) MX2023002695A (https=)
WO (1) WO2022051537A1 (https=)

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MX2020006435A (es) 2017-12-19 2021-02-09 Akouos Inc Administracion de anticuerpos terapeuticos mediada por vector de virus adenoasociado (aav) en el oido interno.
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
AU2024247959A1 (en) * 2023-03-28 2025-10-09 General Medicines Llc Nucleic acid off-switches and methods and uses thereof
WO2024220389A2 (en) * 2023-04-17 2024-10-24 University Of Massachusetts Aav2 variants and uses thereof
CN119656336A (zh) * 2023-09-21 2025-03-21 成都弘基生物科技有限公司 一种重组腺相关病毒在制备治疗与血管新生相关眼病的药物中的用途

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170166925A1 (en) * 2014-04-25 2017-06-15 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
JP2019521990A (ja) * 2016-06-16 2019-08-08 アドヴェラム バイオテクノロジーズ, インコーポレイテッド アフリベルセプトを含むaav2バリアントを使用したamdの処置
WO2019217927A1 (en) * 2018-05-10 2019-11-14 Regeneron Pharmaceuticals, Inc. High concentration vegf receptor fusion protein containing formulations
EP3701972A2 (en) * 2017-10-26 2020-09-02 Curogene Life Sciences Co., Ltd Composition comprising raav containing soluble vegfr-1 derivative cdna for treatment of macular degeneration

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR100897379B1 (ko) * 2004-06-08 2009-05-14 쳉두 캉홍 바이오테크놀로지스 코. 리미티드 혈관신생-저해 키메릭 단백질 및 그 사용
AU2014286996A1 (en) * 2013-07-12 2016-01-07 Iveric Bio, Inc. Methods for treating or preventing ophthalmological conditions
US11046955B2 (en) * 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
AU2018227483A1 (en) * 2017-02-28 2019-09-12 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
AU2018350990A1 (en) * 2017-10-18 2020-05-21 Regenxbio Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified VEGF-Trap

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170166925A1 (en) * 2014-04-25 2017-06-15 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
JP2019521990A (ja) * 2016-06-16 2019-08-08 アドヴェラム バイオテクノロジーズ, インコーポレイテッド アフリベルセプトを含むaav2バリアントを使用したamdの処置
EP3701972A2 (en) * 2017-10-26 2020-09-02 Curogene Life Sciences Co., Ltd Composition comprising raav containing soluble vegfr-1 derivative cdna for treatment of macular degeneration
WO2019217927A1 (en) * 2018-05-10 2019-11-14 Regeneron Pharmaceuticals, Inc. High concentration vegf receptor fusion protein containing formulations

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MX2023002695A (es) 2023-05-19
WO2022051537A1 (en) 2022-03-10
EP4208478A4 (en) 2025-04-02
CA3192736A1 (en) 2022-03-10
EP4208478A1 (en) 2023-07-12
IL300864A (en) 2023-04-01
US20230340529A1 (en) 2023-10-26
BR112023003548A2 (pt) 2023-04-04
AU2021336425A1 (en) 2023-03-16
KR20230061441A (ko) 2023-05-08

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