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

Info

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
Authority
AU
Australia
Prior art keywords
mol
composition
lipid
rnai
mrna
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.)
Active
Application number
AU2016291228A
Other languages
English (en)
Other versions
AU2016291228A1 (en
Inventor
Kuan-Ju CHEN
Keith DIPETRILLO
Franziska LEIFER
Vladimir Malinin
Walter Perkins
Jimin Zhang
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.)
Insmed Inc
Original Assignee
Insmed 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 Insmed Inc filed Critical Insmed Inc
Publication of AU2016291228A1 publication Critical patent/AU2016291228A1/en
Application granted granted Critical
Publication of AU2016291228B2 publication Critical patent/AU2016291228B2/en
Priority to AU2020203367A priority Critical patent/AU2020203367A1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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

Landscapes

  • 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)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Pulmonology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Otolaryngology (AREA)
  • Organic Chemistry (AREA)
  • Biophysics (AREA)
  • Dispersion Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Plant Pathology (AREA)
  • Microbiology (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

Family

ID=57686097

Family Applications (2)

Application Number Title Priority Date Filing Date
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

Family Applications After (1)

Application Number Title Priority Date Filing Date
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 (cg-RX-API-DMAC7.html)
EP (1) EP3319976B1 (cg-RX-API-DMAC7.html)
JP (1) JP6884714B2 (cg-RX-API-DMAC7.html)
AU (2) AU2016291228B2 (cg-RX-API-DMAC7.html)
CA (1) CA2989884A1 (cg-RX-API-DMAC7.html)
ES (1) ES2898340T3 (cg-RX-API-DMAC7.html)
WO (1) WO2017008076A1 (cg-RX-API-DMAC7.html)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK1962805T3 (en) 2005-12-08 2016-09-26 Insmed Inc Lipid-based compositions of the anti-infective agents for the treatment of lung infections
US9119783B2 (en) 2007-05-07 2015-09-01 Insmed Incorporated Method of treating pulmonary disorders with liposomal amikacin formulations
CA2891487A1 (en) 2012-11-29 2014-06-05 Insmed Incorporated Stabilized vancomycin formulations
EA031604B1 (ru) 2013-10-25 2019-01-31 Инсмед Инкорпорейтед Трепростинильные соединения, композиции и способы их использования
ES2926985T3 (es) 2014-05-15 2022-10-31 Insmed Inc Métodos para tratar infecciones pulmonares micobacterianas no tuberculosas
US11571386B2 (en) 2018-03-30 2023-02-07 Insmed Incorporated Methods for continuous manufacture of liposomal drug products
WO2020018959A1 (en) 2018-07-19 2020-01-23 Impel Neuropharma, Inc. Respiratory tract delivery of levodopa and dopa decarboxylase inhibitor for treatment of parkinson's disease
JP7425066B2 (ja) * 2018-09-04 2024-01-30 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム 核酸を臓器特異的送達するための組成物および方法
WO2020077007A1 (en) 2018-10-09 2020-04-16 The University Of British Columbia Compositions and systems comprising transfection-competent vesicles free of organic-solvents and detergents and methods related thereto
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
EP3962472A4 (en) 2019-04-29 2023-01-25 Insmed Incorporated DRY POWDER COMPOSITIONS OF TREPROSTINIL PRODRUGS AND METHODS OF USE THEREOF
AU2020398177A1 (en) * 2019-12-05 2022-07-07 Board Of Regents, The University Of Texas System Exosomes-based therapy for liver fibrosis and other diseases associated with fibrosis
AU2021253959B2 (en) 2020-04-09 2025-04-24 Verve Therapeutics, Inc. Base editing of PCSK9 and methods of using same for treatment of disease
AU2021305214A1 (en) * 2020-07-10 2023-02-23 Genevant Sciences Gmbh Lipid nanoparticles for delivering therapeutics to lungs
EP4213882A4 (en) 2020-09-15 2025-02-26 Verve Therapeutics, Inc. LIPID FORMULATIONS FOR GENE EDITING
TW202233232A (zh) 2020-11-06 2022-09-01 法商賽諾菲公司 遞送mRNA疫苗的脂質奈米顆粒
BR112023019229A2 (pt) 2021-03-22 2023-11-28 Recode Therapeutics Inc Composições e métodos para a liberação direcionada às células
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
CN115245559A (zh) * 2021-04-25 2022-10-28 上海萨美细胞技术有限公司 一种用于治疗肺损伤的药物
CN117881789A (zh) * 2021-06-09 2024-04-12 瑞科德治疗公司 多核苷酸组合物、相关制剂、及其使用方法
US20230057155A1 (en) * 2021-08-11 2023-02-23 Board Of Regents, The University Of Texas System Dry liposome formulations and related methods thereof
PL438742A1 (pl) * 2021-08-14 2023-02-20 Bs Biotechna Spółka Z Ograniczoną Odpowiedzialnością Liposom kationowy wiążący i stabilizujący RNA, jego zastosowanie 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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4721612A (en) * 1984-04-12 1988-01-26 The Liposome Company, Inc. Steroidal liposomes
US4861580A (en) * 1985-10-15 1989-08-29 The Liposome Company, Inc. Composition using salt form of organic acid derivative of alpha-tocopheral
DE19746287A1 (de) * 1997-10-20 1999-04-22 Hoechst Marion Roussel De Gmbh Substituierte Isochinolin-2-Carbonsäureamide, ihre Herstellung und ihre Verwendung als Arzneimittel
WO2005019472A1 (ja) * 2003-08-20 2005-03-03 Locomogene, Inc. シノビオリン活性調節作用の検出方法
JP5777519B2 (ja) * 2008-10-09 2015-09-09 テクミラ ファーマシューティカルズ コーポレイション 改良されたアミノ脂質および核酸の送達方法
AU2011261434B2 (en) * 2010-06-02 2015-11-26 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
US20150246137A1 (en) * 2012-09-27 2015-09-03 The University Of North Carolina At Chapel Hill Lipid coated nanoparticles containing agents having low aqueous and lipid solubilities and methods thereof

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Also Published As

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

Similar Documents

Publication Publication Date Title
AU2016291228B2 (en) Compositions and methods for treating lung diseases and lung injury
JP6990176B2 (ja) メッセンジャーリボ核酸薬物の治療投与のための方法
JP6023126B2 (ja) 標的遺伝子の発現を抑制する組成物
US20080076701A1 (en) Dicer substrate rna peptide conjugates and methods for rna therapeutics
JP2024541993A (ja) Rnaコンストラクト及びその使用
JP2008504827A (ja) 免疫賦活性siRNA分子およびその使用方法
CN101346393A (zh) 修饰的siRNA分子及其应用
CN114096274A (zh) 免疫治疗构建体以及其使用方法
EP4041400B1 (en) Messenger rnas encoding sting and il-12 and use in the treatment of cancer
CN118319880B (zh) 靶向肺部高效递送核酸的脂质纳米颗粒、吸入式制剂及应用
EP4561638A2 (en) Engineered polynucleotides for cell selective expression
JP2024534066A (ja) 脂質ナノ粒子製剤
CN118766874B (zh) 一种吸入式制剂、迭代优化流程及其应用
JP2025533111A (ja) Rnaコンストラクト及びその使用
KR20230144472A (ko) 대식세포 유래 소포로 코팅된 지질 나노입자 및 이의 제조 방법
US20220257614A1 (en) Use of 12-lipoxygenase inhibitors in the treatment of covid-19
JP2023039115A (ja) 物質送達キャリア及び組成物
JP5872898B2 (ja) 標的遺伝子の発現を抑制する組成物
WO2025149492A1 (en) Rna encoding an immune inhibitory il-1 family member
CN118401547A (zh) 白细胞介素-12自我复制rna和方法
WO2012098692A1 (ja) 標的遺伝子の発現を抑制する組成物

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)