JPWO2021072372A5 - - Google Patents

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Publication number
JPWO2021072372A5
JPWO2021072372A5 JP2022521217A JP2022521217A JPWO2021072372A5 JP WO2021072372 A5 JPWO2021072372 A5 JP WO2021072372A5 JP 2022521217 A JP2022521217 A JP 2022521217A JP 2022521217 A JP2022521217 A JP 2022521217A JP WO2021072372 A5 JPWO2021072372 A5 JP WO2021072372A5
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JP
Japan
Prior art keywords
disease
nucleic acid
acid construct
type
alpha
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2022521217A
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English (en)
Japanese (ja)
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JP2022552254A5 (https=
JP2022552254A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/055251 external-priority patent/WO2021072372A1/en
Publication of JP2022552254A publication Critical patent/JP2022552254A/ja
Publication of JP2022552254A5 publication Critical patent/JP2022552254A5/ja
Publication of JPWO2021072372A5 publication Critical patent/JPWO2021072372A5/ja
Priority to JP2025167212A priority Critical patent/JP2026021331A/ja
Pending legal-status Critical Current

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JP2022521217A 2019-10-10 2020-10-12 バリアントigf2構築体 Pending JP2022552254A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025167212A JP2026021331A (ja) 2019-10-10 2025-10-03 バリアントigf2構築体

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962913677P 2019-10-10 2019-10-10
US62/913,677 2019-10-10
US201962929054P 2019-10-31 2019-10-31
US62/929,054 2019-10-31
PCT/US2020/055251 WO2021072372A1 (en) 2019-10-10 2020-10-12 Variant igf2 constructs

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025167212A Division JP2026021331A (ja) 2019-10-10 2025-10-03 バリアントigf2構築体

Publications (3)

Publication Number Publication Date
JP2022552254A JP2022552254A (ja) 2022-12-15
JP2022552254A5 JP2022552254A5 (https=) 2023-10-20
JPWO2021072372A5 true JPWO2021072372A5 (https=) 2023-10-20

Family

ID=75438187

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022521217A Pending JP2022552254A (ja) 2019-10-10 2020-10-12 バリアントigf2構築体
JP2025167212A Pending JP2026021331A (ja) 2019-10-10 2025-10-03 バリアントigf2構築体

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025167212A Pending JP2026021331A (ja) 2019-10-10 2025-10-03 バリアントigf2構築体

Country Status (12)

Country Link
US (1) US20240091321A1 (https=)
EP (1) EP4041284A4 (https=)
JP (2) JP2022552254A (https=)
KR (1) KR20220077921A (https=)
CN (1) CN115087458A (https=)
AU (2) AU2020361703A1 (https=)
BR (1) BR112022006842A2 (https=)
CA (1) CA3154189A1 (https=)
IL (1) IL292065A (https=)
MX (1) MX2022004345A (https=)
TW (1) TW202128740A (https=)
WO (1) WO2021072372A1 (https=)

Families Citing this family (12)

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Publication number Priority date Publication date Assignee Title
KR20210005154A (ko) * 2018-04-30 2021-01-13 아미쿠스 세라퓨틱스, 인코포레이티드 유전자 요법 작제물 및 사용 방법
JP2023512423A (ja) 2019-12-24 2023-03-27 ジュベナ・セラピューティクス・インコーポレイテッド 再生性ポリペプチドおよびその使用
KR20230128001A (ko) 2020-12-01 2023-09-01 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 엔젤만 증후군의 치료를 위한 조성물 및 이의 용도
KR20240029031A (ko) 2021-06-21 2024-03-05 쥬베나 테라퓨틱스, 인크. 재생 폴리펩티드 및 이의 용도
US20240279360A1 (en) 2021-06-23 2024-08-22 Lycia Therapeutics, Inc. Bifunctional compounds containing igf-2 polypeptides
AU2023303015A1 (en) * 2022-07-06 2025-01-23 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery of cln1 polynucleotide
WO2024151982A1 (en) * 2023-01-13 2024-07-18 Amicus Therapeutics, Inc. Gene therapy constructs for the treatment of pompe disease
WO2024196947A2 (en) * 2023-03-20 2024-09-26 Spark Therapeutics, Inc. Ppt1 gene therapy
US20240360431A1 (en) * 2023-04-30 2024-10-31 Sichuan Real&Best Biotech Co., Ltd. Engineered alpha-galactosidase a (a-gal a) peptides and functional variants thereof and associated methods of treating fabry disease
PL445164A1 (pl) * 2023-06-07 2024-12-09 Recepton Spółka Z Ograniczoną Odpowiedzialnością Cząsteczki indukujące internalizację receptorów i białek zewnątrzkomórkowych
CN120025420A (zh) * 2023-11-21 2025-05-23 深圳湾实验室 用于蛋白降解的构建体及其应用
WO2025259950A1 (en) * 2024-06-14 2025-12-18 Cz Biohub Sf, Llc Genetically encoded lysosome targeting chimeras for cell-mediated delivery

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE384736T1 (de) * 2001-04-30 2008-02-15 Zystor Therapeutics Inc Subzelluläres targeting von therapeutischen proteinen
US7560424B2 (en) * 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
CN1922313B (zh) * 2004-02-10 2011-10-26 生物马林医药公司 酸性α-糖苷酶及其片段
DK2241575T3 (en) * 2005-01-07 2015-08-24 Regeneron Pharma IGF-1 fusion polypeptides and therapeutic uses thereof
LT2714752T (lt) * 2011-05-27 2018-03-12 Amicus Therapeutics, Inc. Nukreipiančių peptidų kopuliavimo ant rekombinantinių lizosominių fermentų būdai, skirti lizosominio kaupimo ligų gydymo pagerinimui
WO2014082080A2 (en) * 2012-11-26 2014-05-30 Callidus Biopharma, Inc. Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases
HUE039334T2 (hu) * 2012-11-27 2018-12-28 Biomarin Pharm Inc Célzott terápiás lizoszómális enzim fúziós fehérjék és azok alkalmazásai
CN105189742A (zh) * 2013-03-15 2015-12-23 阿米库斯治疗学公司 化学交联剂
WO2016025523A1 (en) * 2014-08-11 2016-02-18 Shire Human Genetic Therapies, Inc. Lysosomal targeting and uses thereof
EP3374504B1 (en) * 2015-11-09 2025-03-19 CureVac SE Optimized nucleic acid molecules
AU2017315328A1 (en) * 2016-08-24 2019-03-21 Immunexpress Pty Ltd Systemic inflammatory and pathogen biomarkers and uses therefor
KR20210005154A (ko) * 2018-04-30 2021-01-13 아미쿠스 세라퓨틱스, 인코포레이티드 유전자 요법 작제물 및 사용 방법
CN114127275A (zh) * 2019-04-30 2022-03-01 宾夕法尼亚州大学信托人 可用于治疗庞贝病的组合物

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