KR20240035821A - 단일 사슬 가변 단편(scFv) 변형 지질 나노입자 조성물 및 이의 용도 - Google Patents

단일 사슬 가변 단편(scFv) 변형 지질 나노입자 조성물 및 이의 용도 Download PDF

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KR20240035821A
KR20240035821A KR1020247004144A KR20247004144A KR20240035821A KR 20240035821 A KR20240035821 A KR 20240035821A KR 1020247004144 A KR1020247004144 A KR 1020247004144A KR 20247004144 A KR20247004144 A KR 20247004144A KR 20240035821 A KR20240035821 A KR 20240035821A
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formula
pharmaceutical composition
lipid
alkyl
itr
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필립 사마요아
나다니엘 실버
프루던스 유이 퉁 리
랜달 뉴턴 토이
비르테 놀팅
랄리타 운쏜판
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제너레이션 바이오 컴퍼니
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    • AHUMAN NECESSITIES
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/69Medicinal 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6921Medicinal 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere
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    • A61K47/6929Medicinal 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle
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    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/5123Organic compounds, e.g. fats, sugars
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    • A61K47/68Medicinal 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
    • A61K47/6835Medicinal 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
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    • A61K47/6855Medicinal 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 determinant of a tumour cell the tumour determinant being from breast cancer cell
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    • A61K47/6931Medicinal 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle the material constituting the nanoparticle being a polymer
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • A61K48/0041Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/513Organic macromolecular compounds; Dendrimers
    • A61K9/5169Proteins, e.g. albumin, gelatin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/32Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against translation products of oncogenes
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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    • A61K2039/51Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/60Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622Single chain antibody (scFv)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/70Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/77Internalization into the cell

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
KR1020247004144A 2021-07-13 2022-07-13 단일 사슬 가변 단편(scFv) 변형 지질 나노입자 조성물 및 이의 용도 Pending KR20240035821A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163221290P 2021-07-13 2021-07-13
US63/221,290 2021-07-13
PCT/US2022/036930 WO2023287861A2 (en) 2021-07-13 2022-07-13 Single chain variable fragment (scfv) modified lipid nanoparticle compositions and uses thereof

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KR20240035821A true KR20240035821A (ko) 2024-03-18

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KR1020247004144A Pending KR20240035821A (ko) 2021-07-13 2022-07-13 단일 사슬 가변 단편(scFv) 변형 지질 나노입자 조성물 및 이의 용도

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US (1) US20240382432A1 (https=)
EP (1) EP4370135A4 (https=)
JP (1) JP2024529343A (https=)
KR (1) KR20240035821A (https=)
CN (1) CN118159278A (https=)
AU (1) AU2022311904A1 (https=)
CA (1) CA3225694A1 (https=)
IL (1) IL309767A (https=)
MX (1) MX2024000670A (https=)
WO (1) WO2023287861A2 (https=)
ZA (1) ZA202400158B (https=)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL309767A (en) * 2021-07-13 2024-02-01 Generation Bio Co Single chain variable fragment (scfv) modified lipid nanoparticle compositions and uses thereof
US20240226133A1 (en) * 2023-01-09 2024-07-11 Claudia Chimisso Dos Santos Microrna-based particle for the treatment of dysregulated immune response
US12311033B2 (en) 2023-05-31 2025-05-27 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025090417A1 (en) * 2023-10-24 2025-05-01 Generation Bio Co. Bispecific stealth lipid nanoparticle compositions for cell targeting
WO2025090138A1 (en) * 2023-10-24 2025-05-01 Generation Bio Co. Stealth lipid nanoparticle compositions for cell targeting
WO2025150516A1 (ja) * 2024-01-12 2025-07-17 日油株式会社 核酸内封脂質ナノ粒子を含む凍結組成物

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WO2017027325A1 (en) * 2015-08-07 2017-02-16 Imaginab, Inc. Antigen binding constructs to target molecules
WO2018200951A1 (en) * 2017-04-28 2018-11-01 Tran David T Multi-targeted multi-valent ligand-drug particles for the treatment and prevention of diseases and conditions
US20200093936A1 (en) * 2018-09-21 2020-03-26 The Trustees Of The University Of Pennsylvania Therapeutic Targeting of Lipid Nanoparticles
AU2020397956A1 (en) * 2019-12-04 2022-07-07 Orna Therapeutics, Inc. Circular RNA compositions and methods
IL309767A (en) * 2021-07-13 2024-02-01 Generation Bio Co Single chain variable fragment (scfv) modified lipid nanoparticle compositions and uses thereof

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WO2023287861A3 (en) 2023-05-25
ZA202400158B (en) 2024-10-30
JP2024529343A (ja) 2024-08-06
IL309767A (en) 2024-02-01
AU2022311904A1 (en) 2024-02-08
US20240382432A1 (en) 2024-11-21
CN118159278A (zh) 2024-06-07
CA3225694A1 (en) 2023-01-19
EP4370135A4 (en) 2025-11-26
EP4370135A2 (en) 2024-05-22
WO2023287861A2 (en) 2023-01-19
MX2024000670A (es) 2024-03-14

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