IL314102B2 - Transferrin receptor-targeting conjugates and their uses - Google Patents

Transferrin receptor-targeting conjugates and their uses

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Publication number
IL314102B2
IL314102B2 IL314102A IL31410224A IL314102B2 IL 314102 B2 IL314102 B2 IL 314102B2 IL 314102 A IL314102 A IL 314102A IL 31410224 A IL31410224 A IL 31410224A IL 314102 B2 IL314102 B2 IL 314102B2
Authority
IL
Israel
Prior art keywords
transferrin receptor
muscle
oligonucleotide
antibody
complex
Prior art date
Application number
IL314102A
Other languages
English (en)
Hebrew (he)
Other versions
IL314102B1 (en
IL314102A (en
Original Assignee
Dyne Therapeutics Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Dyne Therapeutics Inc filed Critical Dyne Therapeutics Inc
Publication of IL314102A publication Critical patent/IL314102A/en
Publication of IL314102B1 publication Critical patent/IL314102B1/en
Publication of IL314102B2 publication Critical patent/IL314102B2/en

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    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • 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
    • A61K47/54—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 an organic compound
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    • 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
    • A61K47/68—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 an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
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    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
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    • C07K16/2881—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD71
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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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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
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    • C—CHEMISTRY; METALLURGY
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    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
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IL314102A 2018-08-02 2019-08-02 Transferrin receptor-targeting conjugates and their uses IL314102B2 (en)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201862714010P 2018-08-02 2018-08-02
US201862779173P 2018-12-13 2018-12-13
US201962855781P 2019-05-31 2019-05-31
US201962858925P 2019-06-07 2019-06-07
US201962859694P 2019-06-10 2019-06-10
PCT/US2019/044982 WO2020028857A1 (en) 2018-08-02 2019-08-02 Muscle-targeting complexes and uses thereof

Publications (3)

Publication Number Publication Date
IL314102A IL314102A (en) 2024-09-01
IL314102B1 IL314102B1 (en) 2025-06-01
IL314102B2 true IL314102B2 (en) 2025-10-01

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ID=69232332

Family Applications (3)

Application Number Title Priority Date Filing Date
IL314102A IL314102B2 (en) 2018-08-02 2019-08-02 Transferrin receptor-targeting conjugates and their uses
IL280536A IL280536B2 (en) 2018-08-02 2019-08-02 Complexes targeting transferrin receptor and uses thereof
IL320597A IL320597A (en) 2018-08-02 2019-08-02 Transferrin receptor-targeting conjugates and their uses

Family Applications After (2)

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IL280536A IL280536B2 (en) 2018-08-02 2019-08-02 Complexes targeting transferrin receptor and uses thereof
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Families Citing this family (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
CN118638787A (zh) 2017-12-06 2024-09-13 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
CA3108386A1 (en) * 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating friedreich's ataxia
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
AU2019315593B2 (en) * 2018-08-02 2026-05-21 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
EP4087653A4 (en) * 2020-01-10 2024-06-19 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2021154477A1 (en) 2020-01-31 2021-08-05 Dyne Therapeutics, Inc. Anti-transferrin receptor (tfr) antibody and uses thereof
KR102927945B1 (ko) 2020-03-27 2026-02-23 어비디티 바이오사이언시스 인크. 근이영양증의 치료용 조성물 및 방법
US20230330246A1 (en) * 2020-05-05 2023-10-19 Avidity Biosciences, Inc. Compositions and methods of treating pompe disease
WO2021226454A1 (en) * 2020-05-08 2021-11-11 Mpeg La, L.L.C. Linker compounds
MX2023000984A (es) * 2020-07-23 2023-05-03 Dyne Therapeutics Inc Complejos dirigidos al musculo y usos de los mismos para el tratamiento de distrofinopatias.
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
MX2023000958A (es) * 2020-07-23 2023-04-14 Dyne Therapeutics Inc Complejos dirigidos al musculo y usos de estos para el tratamiento de la distrofia miotonica.
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
AU2021313058A1 (en) * 2020-07-23 2023-03-30 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
KR20230117182A (ko) * 2020-12-04 2023-08-07 다인 세라퓨틱스, 인크. 항체-올리고뉴클레오티드 복합체 및 그의 용도
AU2021414227A1 (en) * 2020-12-31 2023-07-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2022197554A1 (en) * 2021-03-16 2022-09-22 University Of Massachusetts Photocaged citrulline analogs and methods for site-specific incorporation of citrulline into proteins
EP4359534A4 (en) * 2021-06-21 2026-07-22 Dyne Therapeutics Inc MUSCLE TARGETTING COMPLEXES AND THEIR USES IN THE TREATMENT OF POMPE DISEASE
CA3222816A1 (en) * 2021-06-21 2022-12-29 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating friedreich's ataxia
CA3226301A1 (en) * 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
JP2024525613A (ja) * 2021-07-09 2024-07-12 ダイン セラピューティクス,インコーポレーテッド 筋標的化複合体およびジストロフィノパチーを処置するためのその使用
WO2023283623A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
EP4366774A4 (en) * 2021-07-09 2025-11-19 Dyne Therapeutics Inc MUSCLE TARGETTING COMPLEXES AND THEIR USES FOR MODULATING GENES ASSOCIATED WITH MUSCLE HEALTH
US20240318176A1 (en) * 2021-07-09 2024-09-26 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2023023031A2 (en) * 2021-08-17 2023-02-23 Blaze Bioscience, Inc. Transferrin receptor targeting peptide oligonucleotide complexes and methods of use thereof
CA3230796A1 (en) 2021-09-01 2023-03-09 Biogen Ma Inc. Anti-transferrin receptor antibodies and uses thereof
KR20240067118A (ko) * 2021-09-29 2024-05-16 아이오니스 파마수티컬즈, 인코포레이티드 접합된 올리고뉴클레오타이드 및 이의 용도
CA3240612A1 (en) 2021-12-22 2023-06-29 Miriam Verena Bujny Targeted saponin-nucleic acid conjugates for treatment of muscle wasting disorders
US20250082759A1 (en) 2021-12-22 2025-03-13 Sapreme Technologies B.V. Compositions comprising therapeutic nucleic acid and targeted saponin for the treatment of muscle-wasting disorders
EP4452281A1 (en) 2021-12-22 2024-10-30 Sapreme Technologies B.V. Compositions comprising therapeutic nucleic acid and saponin for the treatment of muscle-wasting disorders
IL314093A (en) * 2022-01-07 2024-09-01 Mirecule Inc Agents used for tissue-specific imaging and as therapeutic agents targeting proteins expressed on the surface of muscle cells
IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
AU2023371614A1 (en) * 2022-11-04 2025-05-15 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
EP4480964A1 (en) * 2023-06-22 2024-12-25 Vect-Horus Transferrin receptor-binding molecules, conjugates thereof and their uses to prevent or treat muscular diseases
WO2024261332A2 (en) * 2023-06-22 2024-12-26 Vect-Horus Transferrin receptor-binding molecules, conjugates thereof and their uses to prevent or treat muscular diseases
US12527877B2 (en) 2024-01-24 2026-01-20 Arrowhead Pharmaceuticals, Inc. Anti-transferrin receptor antibodies, conjugates thereof, and uses thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120122801A1 (en) * 2005-01-05 2012-05-17 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
US20140193436A1 (en) * 2011-06-24 2014-07-10 Centrose, Llc Extracellular targeted drug conjugates

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040063654A1 (en) * 2001-11-02 2004-04-01 Davis Mark E. Methods and compositions for therapeutic use of RNA interference
AU2010306940A1 (en) * 2009-10-12 2012-06-07 Smith, Larry Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro
HRP20180268T4 (hr) * 2012-10-23 2026-03-27 Synaffix B.V. Modificirano antitijelo, konjugat antitijela i postupak za njihovu pripremu
EP3922643A1 (en) * 2013-01-07 2021-12-15 Omniox, Inc. Polymeric forms of h-nox proteins
GB201507926D0 (en) * 2015-05-08 2015-06-24 Proqr Therapeutics N V Improved treatments using oligonucleotides
DK3473270T5 (da) * 2016-06-20 2024-09-09 Genahead Bio Inc Konjugat af antistof-lægemiddel
KR20240172236A (ko) * 2017-01-06 2024-12-09 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
AU2019315593B2 (en) 2018-08-02 2026-05-21 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120122801A1 (en) * 2005-01-05 2012-05-17 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
US20140193436A1 (en) * 2011-06-24 2014-07-10 Centrose, Llc Extracellular targeted drug conjugates

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