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MA46761A - Administration sous-cutanée d'arn messager - Google Patents

Administration sous-cutanée d'arn messager

Info

Publication number
MA46761A
MA46761A MA046761A MA46761A MA46761A MA 46761 A MA46761 A MA 46761A MA 046761 A MA046761 A MA 046761A MA 46761 A MA46761 A MA 46761A MA 46761 A MA46761 A MA 46761A
Authority
MA
Morocco
Prior art keywords
subcutaneous administration
messenger rna
messenger
rna
subcutaneous
Prior art date
Application number
MA046761A
Other languages
English (en)
Inventor
Zarna Bhavsar
Frank Derosa
Michael Heartlein
Shrirang Karve
Original Assignee
Translate Bio 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 Translate Bio Inc filed Critical Translate Bio Inc
Publication of MA46761A publication Critical patent/MA46761A/fr

Links

Classifications

    • 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
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45Transferases (2)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A61K47/50Medicinal 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
    • A61K47/6925Medicinal 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 microcapsule, nanocapsule, microbubble or nanobubble
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2474Hyaluronoglucosaminidase (3.2.1.35), i.e. hyaluronidase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y201/00Transferases transferring one-carbon groups (2.1)
    • C12Y201/03Carboxy- and carbamoyltransferases (2.1.3)
    • C12Y201/03003Ornithine carbamoyltransferase (2.1.3.3)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01035Hyaluronoglucosaminidase (3.2.1.35), i.e. hyaluronidase

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Nanotechnology (AREA)
  • Dispersion Chemistry (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Dermatology (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
MA046761A 2016-11-10 2017-11-10 Administration sous-cutanée d'arn messager MA46761A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201662420435P 2016-11-10 2016-11-10

Publications (1)

Publication Number Publication Date
MA46761A true MA46761A (fr) 2019-09-18

Family

ID=60574723

Family Applications (1)

Application Number Title Priority Date Filing Date
MA046761A MA46761A (fr) 2016-11-10 2017-11-10 Administration sous-cutanée d'arn messager

Country Status (7)

Country Link
US (2) US11400109B2 (fr)
EP (1) EP3538136A1 (fr)
JP (3) JP7560946B2 (fr)
AU (1) AU2017356190B2 (fr)
CA (1) CA3041350A1 (fr)
MA (1) MA46761A (fr)
WO (1) WO2018089846A1 (fr)

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US8853377B2 (en) * 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
AU2017266932B2 (en) 2016-05-18 2023-04-20 Modernatx, Inc. Polynucleotides encoding alpha-galactosidase A for the treatment of Fabry disease
US11400109B2 (en) * 2016-11-10 2022-08-02 Translate Bio, Inc. Subcutaneous delivery of messenger RNA
WO2019104152A1 (fr) 2017-11-22 2019-05-31 Modernatx, Inc. Polynucléotides codant pour l'ornithine transcarbamylase pour le traitement de troubles du cycle de l'urée
US11939601B2 (en) 2017-11-22 2024-03-26 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
JP7448488B2 (ja) * 2018-05-15 2024-03-12 トランスレイト バイオ, インコーポレイテッド メッセンジャーrnaの皮下送達
AU2019394996B2 (en) 2018-12-06 2025-11-06 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
CN114599789A (zh) * 2019-10-17 2022-06-07 公立大学法人名古屋市立大学 人工合成mRNA及其利用
WO2021127394A2 (fr) * 2019-12-20 2021-06-24 Translate Bio, Inc. Administration par voie rectale d'arn messager
WO2021173840A1 (fr) * 2020-02-25 2021-09-02 Translate Bio, Inc. Procédés améliorés de préparation de nanoparticules lipidiques chargées d'arnm
WO2021178510A1 (fr) * 2020-03-03 2021-09-10 Arcturus Therapeutics, Inc. Compositions et procédés pour le traitement d'une déficience en ornithine transcarbamylase
IL297948A (en) * 2020-05-07 2023-01-01 Translate Bio Inc Production of optimal nucleotide sequences
US20220218622A1 (en) 2020-10-14 2022-07-14 George Mason Research Foundation, Inc. Ionizable lipids and methods of manufacture and use thereof
CA3196877A1 (fr) * 2020-11-17 2022-05-27 Janssen Sciences Ireland Unlimited Company Traitement ou prevention d'une maladie ou d'un trouble
CN113941005A (zh) * 2021-10-29 2022-01-18 上海唯可生物科技有限公司 二硫键功能化的二氧化硅纳米粒子、制备、复合物、用途
CA3240127A1 (fr) * 2021-12-07 2023-06-15 Zihao Wang Formulations d'arn et lipides
CN115813848B (zh) * 2021-12-10 2023-08-08 上海臻上医药科技有限公司 微针注射mRNA编码双特异性抗体药物的试剂、方法和应用
CN115957332B (zh) * 2022-11-01 2023-10-10 北京华睿鼎信科技有限公司 透明质酸酶稳定的布瑞诺龙纳米晶及其制备方法与应用
KR20250150005A (ko) * 2023-02-20 2025-10-17 린디 바이오사이언시스, 아이엔씨. 히알루로니다제 입자, 이를 포함하는 조성물, 및 이의 제조 및 사용 방법
WO2025212951A1 (fr) * 2024-04-05 2025-10-09 Elixirgen Therapeutics, Inc. Procédés se rapportant à l'expression de protéine dans de l'arnm synthétique administré par injection

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US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
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US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
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Also Published As

Publication number Publication date
US20190298755A1 (en) 2019-10-03
US11400109B2 (en) 2022-08-02
US20230065040A1 (en) 2023-03-02
AU2017356190B2 (en) 2024-11-21
JP2024150601A (ja) 2024-10-23
JP2019533708A (ja) 2019-11-21
JP7560946B2 (ja) 2024-10-03
JP2022173314A (ja) 2022-11-18
CA3041350A1 (fr) 2018-05-17
EP3538136A1 (fr) 2019-09-18
WO2018089846A1 (fr) 2018-05-17
AU2017356190A1 (en) 2019-05-16

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