JP2025500482A - 筋消耗性障害の治療用の標的型サポニン－核酸コンジュゲート - Google Patents

筋消耗性障害の治療用の標的型サポニン－核酸コンジュゲート Download PDF

Info

Publication number
JP2025500482A
JP2025500482A JP2024538258A JP2024538258A JP2025500482A JP 2025500482 A JP2025500482 A JP 2025500482A JP 2024538258 A JP2024538258 A JP 2024538258A JP 2024538258 A JP2024538258 A JP 2024538258A JP 2025500482 A JP2025500482 A JP 2025500482A
Authority
JP
Japan
Prior art keywords
saponin
bond
conjugate
linker
ligand
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
JP2024538258A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025500482A5 (https=
Inventor
ヴェレナ ブイニー，ミリアム
ポステル，ルーベン
ヘアマンス，ガイ
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sapreme Technologies BV
Original Assignee
Sapreme Technologies BV
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 Sapreme Technologies BV filed Critical Sapreme Technologies BV
Publication of JP2025500482A publication Critical patent/JP2025500482A/ja
Publication of JP2025500482A5 publication Critical patent/JP2025500482A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K47/68—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
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K47/54—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
    • A61K47/554—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 the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K47/68—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
    • A61K47/6835—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 the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6849—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 the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a receptor, a cell surface antigen or a cell surface determinant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Botany (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
JP2024538258A 2021-12-22 2022-12-20 筋消耗性障害の治療用の標的型サポニン－核酸コンジュゲート Pending JP2025500482A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
NL2030215 2021-12-22
NL2030215 2021-12-22
PCT/NL2022/050736 WO2023121446A1 (en) 2021-12-22 2022-12-20 Targeted saponin-nucleic acid conjugates for treatment of muscle wasting disorders

Publications (2)

Publication Number Publication Date
JP2025500482A true JP2025500482A (ja) 2025-01-09
JP2025500482A5 JP2025500482A5 (https=) 2025-12-26

Family

ID=81585344

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024538258A Pending JP2025500482A (ja) 2021-12-22 2022-12-20 筋消耗性障害の治療用の標的型サポニン－核酸コンジュゲート

Country Status (11)

Country Link
US (1) US20250057971A1 (https=)
EP (2) EP4452331B8 (https=)
JP (1) JP2025500482A (https=)
KR (1) KR20240121778A (https=)
CN (1) CN118414172A (https=)
CA (1) CA3240612A1 (https=)
DK (1) DK4452331T3 (https=)
ES (1) ES3052234T3 (https=)
IL (1) IL313728A (https=)
PT (1) PT4452331T (https=)
WO (1) WO2023121446A1 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
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
CA3108282A1 (en) 2018-08-02 2020-02-06 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
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
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
IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
KR20260025101A (ko) * 2023-06-02 2026-02-23 사프렘 테크놀로지스 비.브이. 세포내 분자 표적을 통해 작용하는 치료적 이펙터 성분을 위한 개선된 치료 요법

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX9204374A (es) 1991-07-25 1993-03-01 Idec Pharma Corp Anticuerpo recombinante y metodo para su produccion.
US10626395B2 (en) 2016-10-27 2020-04-21 The General Hospital Corporation Therapeutic targeting of a microRNA to treat Duchenne muscular dystrophy
MX2019008199A (es) 2017-01-06 2019-11-25 Avidity Biosciences Llc Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.
IL280536B2 (en) 2018-08-02 2024-12-01 Dyne Therapeutics Inc Complexes targeting transferrin receptor and uses thereof
SG11202106603UA (en) 2018-12-21 2021-07-29 Sapreme Tech Bv Biologically active cluster of molecules
CA3163283A1 (en) 2020-01-10 2021-07-15 Romesh R. Subramanian Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health

Also Published As

Publication number Publication date
US20250057971A1 (en) 2025-02-20
WO2023121446A1 (en) 2023-06-29
IL313728A (en) 2024-08-01
EP4610278A2 (en) 2025-09-03
PT4452331T (pt) 2025-10-28
DK4452331T3 (da) 2025-09-29
ES3052234T3 (en) 2026-01-02
EP4610278A3 (en) 2025-12-10
CN118414172A (zh) 2024-07-30
EP4452331A1 (en) 2024-10-30
EP4452331B1 (en) 2025-08-06
EP4452331B8 (en) 2025-09-17
KR20240121778A (ko) 2024-08-09
CA3240612A1 (en) 2023-06-29

Similar Documents

Publication Publication Date Title
US20250051770A1 (en) Compositions comprising therapeutic nucleic acid and saponin for the treatment of muscle-wasting disorders
EP4452331B1 (en) Targeted saponin-nucleic acid conjugates for treatment of muscle wasting disorders
US20250082759A1 (en) Compositions comprising therapeutic nucleic acid and targeted saponin for the treatment of muscle-wasting disorders
EP4355369B1 (en) Conjugate of saponin, oligonucleotide and galnac
US20250295800A1 (en) Conjugate of a single domain antibody, a saponin and an effector molecule, pharmaceutical composition comprising the same, therapeutic use of said pharmaceutical composition
EP4210760B1 (en) Conjugate of saponin, oligonucleotide and galnac
HK40129943A (en) Targeted saponin-nucleic acid conjugates for treatment of muscle wasting disorders
HK40118872A (en) Targeted saponin-nucleic acid conjugates for treatment of muscle wasting disorders
HK40118872B (en) Targeted saponin-nucleic acid conjugates for treatment of muscle wasting disorders
KR20230043117A (ko) 단일 도메인 항체, 사포닌 및 이펙터 분자의 접합체, 이를 포함하는 약학적 조성물, 상기 약학적 조성물의 치료 용도
US20250205361A1 (en) Treatment regimens for therapeutic effector components that act via an intracellular molecular target
WO2026087680A1 (en) Treatment for cardiovascular disease
KR20260026556A (ko) 중추신경계 장애의 치료를 위한 약제학적 조성물
WO2026087678A1 (en) Treatment for renal disorders
HK40110483A (en) Conjugate of saponin, oligonucleotide and galnac
HK40110483B (en) Conjugate of saponin, oligonucleotide and galnac
HK40091234B (en) Conjugate of saponin, oligonucleotide and galnac
HK40091234A (en) Conjugate of saponin, oligonucleotide and galnac
CN117881427A (zh) 皂苷、寡核苷酸和galnac的缀合物

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20251217

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20251217