CN117916249A - 用于口服递送核酸的制剂 - Google Patents

用于口服递送核酸的制剂 Download PDF

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Publication number
CN117916249A
CN117916249A CN202280059708.7A CN202280059708A CN117916249A CN 117916249 A CN117916249 A CN 117916249A CN 202280059708 A CN202280059708 A CN 202280059708A CN 117916249 A CN117916249 A CN 117916249A
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formulation
nucleic acid
casein
subject
present
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CN202280059708.7A
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Chinese (zh)
Inventor
爱德华多·马尔万
艾哈迈德·G·易卜拉欣
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Cedars Sinai Medical Center
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Cedars Sinai Medical Center
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/12Carboxylic acids; Salts or anhydrides thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/36Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/42Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein
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    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration
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    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/5123Organic compounds, e.g. fats, sugars
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    • 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
    • A61K9/5161Polysaccharides, e.g. alginate, chitosan, cellulose derivatives; Cyclodextrin
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    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/513Organic macromolecular compounds; Dendrimers
    • A61K9/5169Proteins, e.g. albumin, gelatin
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    • AHUMAN NECESSITIES
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    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/04Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
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    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
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    • A01K2217/056Animals comprising random inserted nucleic acids (transgenic) inducing loss of function due to mutation of coding region of the transgene (dominant negative)
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/035Animal model for multifactorial diseases
    • A01K2267/0375Animal model for cardiovascular diseases
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    • 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
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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 delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/107Emulsions ; Emulsion preconcentrates; Micelles
    • A61K9/1075Microemulsions or submicron emulsions; Preconcentrates or solids thereof; Micelles, e.g. made of phospholipids or block copolymers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end

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CN202280059708.7A 2021-07-01 2022-06-30 用于口服递送核酸的制剂 Pending CN117916249A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163202970P 2021-07-01 2021-07-01
US63/202,970 2021-07-01
PCT/US2022/035870 WO2023278802A1 (en) 2021-07-01 2022-06-30 Formulations for oral delivery of nucleic acids

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CN117916249A true CN117916249A (zh) 2024-04-19

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CN202280059708.7A Pending CN117916249A (zh) 2021-07-01 2022-06-30 用于口服递送核酸的制剂
CN202280059718.0A Pending CN117897162A (zh) 2021-07-01 2022-06-30 治疗性核酸及其使用方法

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CN202280059718.0A Pending CN117897162A (zh) 2021-07-01 2022-06-30 治疗性核酸及其使用方法

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US (2) US20250025487A1 (https=)
EP (2) EP4363434A4 (https=)
JP (2) JP2024525512A (https=)
KR (2) KR20240028486A (https=)
CN (2) CN117916249A (https=)
AU (2) AU2022303384A1 (https=)
CA (2) CA3223495A1 (https=)
WO (2) WO2023278802A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3563859B1 (en) 2012-08-13 2021-10-13 Cedars-Sinai Medical Center Cardiosphere-derived exosomes for tissue regeneration
EP4494699A3 (en) 2014-10-03 2025-04-30 Cedars-Sinai Medical Center Cardiosphere-derived cells and exosomes secreted by such cells in the treatment of muscular dystrophy
EP3749344A4 (en) 2018-02-05 2022-01-26 Cedars-Sinai Medical Center METHODS OF THERAPEUTIC USE OF EXOSOMES AND Y-RNAS
JP2025534332A (ja) * 2022-09-30 2025-10-15 セダーズ-シナイ メディカル センター 治療用核酸及びその使用方法
AU2024340578A1 (en) * 2023-09-15 2026-03-05 Cedars-Sinai Medical Center Trex-1 augmentation to attenuate genotoxic stress and exert cardioprotection
WO2026035744A1 (en) 2024-08-08 2026-02-12 Cedars-Sinai Medical Center Combination therapies for metabolic disease
WO2026035943A1 (en) * 2024-08-08 2026-02-12 Cedars-Sinai Medical Center Formulations for oral delivery of nucleic acids

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US7780873B2 (en) * 2004-02-23 2010-08-24 Texas A&M University System Bioactive complexes compositions and methods of use thereof
US20120301463A1 (en) * 2009-09-30 2012-11-29 President And Fellows Of Harvard College Methods for Modulation of Autophagy Through the Modulation of Autophagy-Enhancing Gene Products
WO2014152211A1 (en) * 2013-03-14 2014-09-25 Moderna Therapeutics, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
ES2724549T3 (es) * 2013-10-18 2019-09-12 Inst Nat Sante Rech Med ARN pequeños derivados de RNY como biomarcadores para trastornos relacionados con la aterosclerosis
EP3247716A4 (en) * 2015-01-20 2018-10-17 Miragen Therapeutics, Inc. Mir-92 inhibitors and uses thereof
US20190365913A1 (en) * 2016-02-04 2019-12-05 Tarveda Therapeutics, Inc. Stapled peptide conjugates and particles
US11859234B2 (en) * 2016-05-06 2024-01-02 The Trustees Of Princeton University Method of monitoring RNase L activity
US12258594B2 (en) * 2018-12-05 2025-03-25 The Broad Institute, Inc. Cas proteins with reduced immunogenicity and methods of screening thereof
WO2020131986A1 (en) * 2018-12-21 2020-06-25 Pioneer Hi-Bred International, Inc. Multiplex genome targeting

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KR20240028486A (ko) 2024-03-05
KR20240027814A (ko) 2024-03-04
WO2023278799A1 (en) 2023-01-05
EP4363434A1 (en) 2024-05-08
JP2024527340A (ja) 2024-07-24
AU2022303384A1 (en) 2024-01-18
CA3223495A1 (en) 2023-01-05
US20250025487A1 (en) 2025-01-23
WO2023278802A1 (en) 2023-01-05
CN117897162A (zh) 2024-04-16
AU2022303330A1 (en) 2024-01-18
JP2024525512A (ja) 2024-07-12
CA3223686A1 (en) 2023-01-05
US20240285668A1 (en) 2024-08-29
EP4363434A4 (en) 2025-06-25
EP4362954A1 (en) 2024-05-08

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