KR20250024745A - FN3 도메인-siRNA 접합체와 효소 대체 요법(FN3 Domain-siRNA Conjugates with Enzyme Replacement Therapy) - Google Patents

FN3 도메인-siRNA 접합체와 효소 대체 요법(FN3 Domain-siRNA Conjugates with Enzyme Replacement Therapy) Download PDF

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KR20250024745A
KR20250024745A KR1020247036275A KR20247036275A KR20250024745A KR 20250024745 A KR20250024745 A KR 20250024745A KR 1020247036275 A KR1020247036275 A KR 1020247036275A KR 20247036275 A KR20247036275 A KR 20247036275A KR 20250024745 A KR20250024745 A KR 20250024745A
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seq
binds
amino acid
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스와프닐 쿨카르니
러셀 아디스
스티븐 나들러
야오 신
잔나 드루지나
카린 오'넬
로버트 코라코프스키
스테픈 앤더슨
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에이알오 바이오테라퓨틱스 컴패니
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    • A—HUMAN NECESSITIES
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    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/6435—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent the peptide or protein in the drug conjugate being a connective tissue peptide, e.g. collagen, fibronectin or gelatin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/46—Hydrolases (3)
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    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/30—Chemical structure
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    • C12N2310/3513—Protein; Peptide
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)

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KR1020247036275A 2022-04-14 2023-04-14 FN3 도메인-siRNA 접합체와 효소 대체 요법(FN3 Domain-siRNA Conjugates with Enzyme Replacement Therapy) Pending KR20250024745A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263330797P 2022-04-14 2022-04-14
US63/330,797 2022-04-14
PCT/US2023/065812 WO2023201362A2 (en) 2022-04-14 2023-04-14 Fn3 domain-sirna conjugates with enzyme replacement therapy

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KR20250024745A true KR20250024745A (ko) 2025-02-19

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US (1) US20240043844A1 (enExample)
EP (1) EP4507702A2 (enExample)
JP (1) JP2025515996A (enExample)
KR (1) KR20250024745A (enExample)
CN (1) CN119604311A (enExample)
AU (1) AU2023252976A1 (enExample)
CA (1) CA3248570A1 (enExample)
IL (1) IL316071A (enExample)
WO (1) WO2023201362A2 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN119119240A (zh) 2019-10-14 2024-12-13 Aro生物疗法公司 结合cd71的纤维粘连蛋白iii型结构域
CA3214552A1 (en) 2021-04-14 2022-10-20 Russell C. Addis Cd71 binding fibronectin type iii domains
EP4323008A4 (en) 2021-04-14 2026-02-25 Aro Biotherapeutics Company FN3-ARNSI DOMAIN CONJUGATES AND THEIR USES
WO2025231388A1 (en) * 2024-05-03 2025-11-06 Aro Biotherapeutics Company Methods and compositions for treating pompe disease
WO2025245474A1 (en) * 2024-05-23 2025-11-27 Aro Biotherapeutics Company Formulations comprising fn3-sirna conjugates and uses thereof

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005072057A2 (en) * 2004-01-30 2005-08-11 Quark Biotech, Inc. Oligoribonucleotides and methods of use thereof for treatment of fibrotic conditions and other diseases
EA201001734A1 (ru) * 2008-05-02 2011-10-31 Новартис Аг Улучшенные связывающие молекулы на основе фибронектина и их применение
US11781138B2 (en) * 2019-10-14 2023-10-10 Aro Biotherapeutics Company FN3 domain-siRNA conjugates and uses thereof
WO2021226107A1 (en) * 2020-05-05 2021-11-11 Avidity Biosciences, Inc. Compositions and methods of treating pompe disease
EP4323008A4 (en) * 2021-04-14 2026-02-25 Aro Biotherapeutics Company FN3-ARNSI DOMAIN CONJUGATES AND THEIR USES

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US20240043844A1 (en) 2024-02-08
WO2023201362A2 (en) 2023-10-19
EP4507702A2 (en) 2025-02-19
CA3248570A1 (en) 2023-10-19
JP2025515996A (ja) 2025-05-23
WO2023201362A9 (en) 2024-06-20
IL316071A (en) 2024-12-01
CN119604311A (zh) 2025-03-11
WO2023201362A3 (en) 2024-02-15
AU2023252976A1 (en) 2024-11-07

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