JP2022552254A - バリアントｉｇｆ２構築体 - Google Patents

バリアントｉｇｆ２構築体 Download PDF

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JP2022552254A
JP2022552254A JP2022521217A JP2022521217A JP2022552254A JP 2022552254 A JP2022552254 A JP 2022552254A JP 2022521217 A JP2022521217 A JP 2022521217A JP 2022521217 A JP2022521217 A JP 2022521217A JP 2022552254 A JP2022552254 A JP 2022552254A
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disease
nucleic acid
seq
vigf2
peptide
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JP2022552254A5 (https=
Inventor
ハング ドゥ，
スティーヴン トゥスケ，
ラッセル ゴッシャル，
セ フェン リュウ，
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アミカス セラピューティックス インコーポレイテッド
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Publication of JP2022552254A publication Critical patent/JP2022552254A/ja
Publication of JP2022552254A5 publication Critical patent/JP2022552254A5/ja
Priority to JP2025167212A priority Critical patent/JP2026021331A/ja
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/30—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405—Glucanases
    • C12N9/2451—Glucanases acting on alpha-1,6-glucosidic bonds
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/485—Exopeptidases (3.4.11-3.4.19)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
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 (2)

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

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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

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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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112020021962A2 (pt) * 2018-04-30 2021-01-26 Amicus Therapeutics, Inc. construtos para terapia gênica e métodos de uso
CN115379850A (zh) 2019-12-24 2022-11-22 裘美娜治疗公司 再生性多肽及其用途
IL303239A (en) 2020-12-01 2023-07-01 Univ Pennsylvania Compositions and their uses for the treatment of Engelmann syndrome
WO2022271466A1 (en) 2021-06-21 2022-12-29 Juvena Therapeutics, Inc. Regenerative polypeptides and uses thereof
WO2022271981A2 (en) 2021-06-23 2022-12-29 Lycia Therapeutics, Inc. Bifunctional compounds containing igf-2 polypeptides
JP2025524569A (ja) * 2022-07-06 2025-07-30 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Ｃｌｎ１ポリヌクレオチドのアデノ随伴ウイルス送達
WO2024151982A1 (en) * 2023-01-13 2024-07-18 Amicus Therapeutics, Inc. Gene therapy constructs for the treatment of pompe disease
KR20250161626A (ko) * 2023-03-20 2025-11-17 스파크 테라퓨틱스, 인코포레이티드 Ppt1 유전자 치료
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
WO2025108375A1 (zh) * 2023-11-21 2025-05-30 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

Citations (6)

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JP2004535791A (ja) * 2001-04-30 2004-12-02 シンビオンティクス， インコーポレイテッド 治療的タンパク質の亜細胞標的化
JP2007521818A (ja) * 2004-02-10 2007-08-09 ザイストール セラピューティクス， インコーポレイテッド 酸性αグルコシダーゼおよびそのフラグメント
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
JP2014518063A (ja) * 2011-05-27 2014-07-28 カリダス・バイオファーマ，インコーポレーテッド リソソーム蓄積症の改善された治療のための組換えリソソーム酵素にターゲティングペプチドをカップリングするための方法
JP2016505539A (ja) * 2012-11-27 2016-02-25 バイオマリン ファーマシューティカル インコーポレイテッド 標的治療用ライソゾーム酵素融合タンパク質およびその使用
JP2016515510A (ja) * 2013-03-15 2016-05-30 アミカス セラピューティックス インコーポレイテッド 化学架橋剤

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* Cited by examiner, † Cited by third party
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US7560424B2 (en) * 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
JP4988599B2 (ja) * 2005-01-07 2012-08-01 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｉｇｆ−１融合ポリペプチドおよびその治療的使用
MA40460A (fr) * 2014-08-11 2016-02-18 Shire Human Genetic Therapies Ciblage lysosomial et utilisation correspondante
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
BR112020021962A2 (pt) * 2018-04-30 2021-01-26 Amicus Therapeutics, Inc. construtos para terapia gênica e métodos de uso
JP7585233B2 (ja) * 2019-04-30 2024-11-18 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ポンペ病の治療のために有用な組成物

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004535791A (ja) * 2001-04-30 2004-12-02 シンビオンティクス， インコーポレイテッド 治療的タンパク質の亜細胞標的化
JP2007521818A (ja) * 2004-02-10 2007-08-09 ザイストール セラピューティクス， インコーポレイテッド 酸性αグルコシダーゼおよびそのフラグメント
JP2014518063A (ja) * 2011-05-27 2014-07-28 カリダス・バイオファーマ，インコーポレーテッド リソソーム蓄積症の改善された治療のための組換えリソソーム酵素にターゲティングペプチドをカップリングするための方法
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
JP2016505539A (ja) * 2012-11-27 2016-02-25 バイオマリン ファーマシューティカル インコーポレイテッド 標的治療用ライソゾーム酵素融合タンパク質およびその使用
JP2016515510A (ja) * 2013-03-15 2016-05-30 アミカス セラピューティックス インコーポレイテッド 化学架橋剤

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Publication number Publication date
JP2026021331A (ja) 2026-02-10
MX2022004345A (es) 2022-07-27
IL292065A (en) 2022-06-01
AU2020361703A1 (en) 2022-04-28
AU2025200698A1 (en) 2025-02-27
EP4041284A1 (en) 2022-08-17
BR112022006842A2 (pt) 2022-07-05
KR20220077921A (ko) 2022-06-09
US20240091321A1 (en) 2024-03-21
EP4041284A4 (en) 2023-10-18
TW202128740A (zh) 2021-08-01
CN115087458A (zh) 2022-09-20
CA3154189A1 (en) 2021-04-15
WO2021072372A1 (en) 2021-04-15

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