KR20240031947A - Fn3 도메인-시르나 접합체 및 이의 용도 - Google Patents

Fn3 도메인-시르나 접합체 및 이의 용도 Download PDF

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KR20240031947A
KR20240031947A KR1020237039175A KR20237039175A KR20240031947A KR 20240031947 A KR20240031947 A KR 20240031947A KR 1020237039175 A KR1020237039175 A KR 1020237039175A KR 20237039175 A KR20237039175 A KR 20237039175A KR 20240031947 A KR20240031947 A KR 20240031947A
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domain
composition
seq
binds
sirna
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Korean (ko)
Inventor
스와프닐 쿨카르니
러셀 씨. 아디스
스티븐 지. 나들러
야오 신
자나 드루지나
카린 티. 오넬
로버트 브이. 코라코프스키
스테픈 제이. 앤더슨
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에이알오 바이오테라퓨틱스 컴패니
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Publication of KR20240031947A publication Critical patent/KR20240031947A/ko
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • 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
    • C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
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    • C12N15/1137—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 against enzymes
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  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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KR1020237039175A 2021-04-14 2022-04-14 Fn3 도메인-시르나 접합체 및 이의 용도 Pending KR20240031947A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163174776P 2021-04-14 2021-04-14
US63/174,776 2021-04-14
US202163203776P 2021-07-30 2021-07-30
US63/203,776 2021-07-30
US202263324437P 2022-03-28 2022-03-28
US63/324,437 2022-03-28
PCT/US2022/024846 WO2022221550A1 (en) 2021-04-14 2022-04-14 Fn3 domain-sirna conjugates and uses thereof

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KR20240031947A true KR20240031947A (ko) 2024-03-08

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KR1020237039175A Pending KR20240031947A (ko) 2021-04-14 2022-04-14 Fn3 도메인-시르나 접합체 및 이의 용도

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US (2) US12239710B2 (https=)
EP (1) EP4323008A4 (https=)
JP (1) JP2024514266A (https=)
KR (1) KR20240031947A (https=)
AU (1) AU2022258584A1 (https=)
BR (1) BR112023021318A2 (https=)
CA (1) CA3215367A1 (https=)
IL (1) IL307595A (https=)
MX (1) MX2023012124A (https=)
TW (1) TWI906502B (https=)
WO (1) WO2022221550A1 (https=)

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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
CN119604311A (zh) * 2022-04-14 2025-03-11 Aro生物疗法公司 FN3结构域-siRNA缀合物与酶替代疗法
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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