AU2016291228B2 - Compositions and methods for treating lung diseases and lung injury - Google Patents

Compositions and methods for treating lung diseases and lung injury Download PDF

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Publication number
AU2016291228B2
AU2016291228B2 AU2016291228A AU2016291228A AU2016291228B2 AU 2016291228 B2 AU2016291228 B2 AU 2016291228B2 AU 2016291228 A AU2016291228 A AU 2016291228A AU 2016291228 A AU2016291228 A AU 2016291228A AU 2016291228 B2 AU2016291228 B2 AU 2016291228B2
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mol
composition
lipid
rnai
mrna
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AU2016291228A1 (en
Inventor
Kuan-Ju CHEN
Keith DIPETRILLO
Franziska LEIFER
Vladimir Malinin
Walter Perkins
Jimin Zhang
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Insmed Inc
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Insmed Inc
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    • 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
    • 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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K47/24Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
    • 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
    • A61K9/007Pulmonary tract; Aromatherapy
    • A61K9/0073Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/0075Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a dry powder inhaler [DPI], e.g. comprising micronized drug mixed with lactose carrier particles
    • 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
    • A61K9/007Pulmonary tract; Aromatherapy
    • A61K9/0073Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/0078Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a nebulizer such as a jet nebulizer, ultrasonic nebulizer, e.g. in the form of aqueous drug solutions or dispersions
    • 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
    • A61K9/007Pulmonary tract; Aromatherapy
    • A61K9/0073Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/008Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy comprising drug dissolved or suspended in liquid propellant for inhalation via a pressurized metered dose inhaler [MDI]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • 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
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Pulmonology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Otolaryngology (AREA)
  • Organic Chemistry (AREA)
  • Biophysics (AREA)
  • Dispersion Chemistry (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
AU2016291228A 2015-07-09 2016-07-11 Compositions and methods for treating lung diseases and lung injury Active AU2016291228B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2020203367A AU2020203367A1 (en) 2015-07-09 2020-05-22 Compositions and methods for treating lung diseases and lung injury

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562190583P 2015-07-09 2015-07-09
US62/190,583 2015-07-09
PCT/US2016/041776 WO2017008076A1 (en) 2015-07-09 2016-07-11 Compositions and methods for treating lung diseases and lung injury

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2020203367A Division AU2020203367A1 (en) 2015-07-09 2020-05-22 Compositions and methods for treating lung diseases and lung injury

Publications (2)

Publication Number Publication Date
AU2016291228A1 AU2016291228A1 (en) 2017-12-07
AU2016291228B2 true AU2016291228B2 (en) 2020-04-09

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AU2016291228A Active AU2016291228B2 (en) 2015-07-09 2016-07-11 Compositions and methods for treating lung diseases and lung injury
AU2020203367A Abandoned AU2020203367A1 (en) 2015-07-09 2020-05-22 Compositions and methods for treating lung diseases and lung injury

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AU2020203367A Abandoned AU2020203367A1 (en) 2015-07-09 2020-05-22 Compositions and methods for treating lung diseases and lung injury

Country Status (7)

Country Link
US (1) US20180200186A1 (enExample)
EP (1) EP3319976B1 (enExample)
JP (1) JP6884714B2 (enExample)
AU (2) AU2016291228B2 (enExample)
CA (1) CA2989884A1 (enExample)
ES (1) ES2898340T3 (enExample)
WO (1) WO2017008076A1 (enExample)

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JP5324223B2 (ja) 2005-12-08 2013-10-23 トランセイブ, インク. 肺感染症を治療する抗感染薬の脂質ベースの組成物及びその使用法
US9119783B2 (en) 2007-05-07 2015-09-01 Insmed Incorporated Method of treating pulmonary disorders with liposomal amikacin formulations
MX2015006681A (es) 2012-11-29 2016-04-06 Insmed Inc Formulaciones de vancomicina estabilizadas.
SI3060041T1 (sl) 2013-10-25 2021-04-30 Insmed Incorporated Spojine prostaciklina
SMT202000516T1 (it) 2014-05-15 2020-11-10 Insmed Inc Metodi per trattamento di infezioni micobatteriche polmonati non tubercolari
WO2019191627A1 (en) 2018-03-30 2019-10-03 Insmed Incorporated Methods for continuous manufacture of liposomal drug products
WO2019213398A1 (en) 2018-05-02 2019-11-07 Insmed Incorporated Methods for the manufacture of liposomal drug formulations
JP2021532098A (ja) * 2018-07-19 2021-11-25 インペル ニューロファーマ インコーポレイテッド パーキンソン病の治療のためのレボドパおよびドーパデカルボキシラーゼ阻害剤の呼吸管路送達
EP3846822A4 (en) * 2018-09-04 2022-07-06 The Board Of Regents Of The University Of Texas System COMPOSITIONS AND METHODS FOR ORGAN-SPECIFIC DELIVERY OF NUCLEIC ACIDS
CN113166783B (zh) 2018-10-09 2024-10-11 不列颠哥伦比亚大学 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法
CN109432047B (zh) * 2018-10-29 2021-07-20 中国药科大学 一种逆转肺纤维化纳米制剂及其制备方法
CN114072136B (zh) 2019-04-29 2024-11-29 英斯梅德股份有限公司 曲前列素前药的干粉组合物及其使用方法
CN115297850A (zh) * 2019-12-05 2022-11-04 得克萨斯州大学系统董事会 用于肝纤维化和其他与纤维化相关的疾病的基于外排体的治疗
GB2613457B (en) 2020-04-09 2023-12-06 Verve Therapeutics Inc Base editing of angptl3 and methods of using same for treatment of disease
CN115768438A (zh) * 2020-07-10 2023-03-07 盖纳万科学有限公司 用于向肺递送治疗剂的脂质纳米颗粒
EP4213882A4 (en) 2020-09-15 2025-02-26 Verve Therapeutics, Inc. LIPID FORMULATIONS FOR GENE EDITING
CZ310613B6 (cs) 2020-09-23 2026-01-28 Ústav organické chemie a biochemie AV ČR, v. v. i. Lipidoidy pro transfekci nukleových kyselin a jejich použití
US11771652B2 (en) 2020-11-06 2023-10-03 Sanofi Lipid nanoparticles for delivering mRNA vaccines
WO2022204219A1 (en) 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Compositions and methods for targeted delivery to cells
CN113058042B (zh) * 2021-04-01 2023-06-30 易慧生物技术(上海)有限公司 一种可鼻喷的稳定递载rna分子的脂质纳米颗粒制备方法
CN115245559B (zh) * 2021-04-25 2026-05-01 上海萨美细胞技术有限公司 一种用于治疗肺损伤的药物
EP4352240A4 (en) * 2021-06-09 2025-07-09 Recode Therapeutics Inc POLYNUCLEOTIDE COMPOSITIONS, RELATED FORMULATIONS AND METHODS OF USE THEREOF
CZ310443B6 (cs) 2021-07-19 2025-06-25 Ústav organické chemie a biochemie AV ČR, v. v. i. Cyklohexanové lipidoidy pro transfekci nukleových kyselin a jejich použití
WO2023019192A1 (en) * 2021-08-11 2023-02-16 Board Of Regents, The University Of Texas System Dry liposome formulations and related methods thereof
PL249473B1 (pl) * 2021-08-14 2026-04-27 Bs Biotechna Spółka Z Ograniczoną Odpowiedzialnością Liposom kationowy wiążący i stabilizujący RNA, jego zastosowanie jako nośnika leku oraz w terapii genowej i sposób ładowania liposomu emetyną
EP4226949A1 (en) * 2022-02-14 2023-08-16 Pantherna Therapeutics GmbH Preparation for use in a method for the treatment and/or prevention of a disease
WO2024242216A1 (ko) * 2023-05-23 2024-11-28 주식회사 케이더블유바이오 대식세포 표적화 리포좀 및 이의 용도

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EP2199298A1 (en) * 2004-11-17 2010-06-23 Protiva Biotherapeutics Inc. Sirna silencing of Apolipoprotein B
WO2011108955A1 (en) * 2010-03-03 2011-09-09 Universidade De Coimbra Multi -targeting system comprising a nanocarrier, nucleic acid(s) and non-nucleic acid based drug(s)
US20140308304A1 (en) * 2011-12-07 2014-10-16 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents

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WO2011108955A1 (en) * 2010-03-03 2011-09-09 Universidade De Coimbra Multi -targeting system comprising a nanocarrier, nucleic acid(s) and non-nucleic acid based drug(s)
US20140308304A1 (en) * 2011-12-07 2014-10-16 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents

Also Published As

Publication number Publication date
EP3319976A1 (en) 2018-05-16
US20180200186A1 (en) 2018-07-19
CA2989884A1 (en) 2017-01-12
JP6884714B2 (ja) 2021-06-09
AU2016291228A1 (en) 2017-12-07
JP2018519818A (ja) 2018-07-26
EP3319976A4 (en) 2019-03-20
EP3319976B1 (en) 2021-09-01
WO2017008076A1 (en) 2017-01-12
AU2020203367A1 (en) 2020-06-11
ES2898340T3 (es) 2022-03-07

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