AU2018348214C1 - Genotype-agnostic rescue of cystic fibrosis with small molecule bicarbonate channels - Google Patents

Genotype-agnostic rescue of cystic fibrosis with small molecule bicarbonate channels Download PDF

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Publication number
AU2018348214C1
AU2018348214C1 AU2018348214A AU2018348214A AU2018348214C1 AU 2018348214 C1 AU2018348214 C1 AU 2018348214C1 AU 2018348214 A AU2018348214 A AU 2018348214A AU 2018348214 A AU2018348214 A AU 2018348214A AU 2018348214 C1 AU2018348214 C1 AU 2018348214C1
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AU
Australia
Prior art keywords
amb
cufi
f508del
cholesterol
patient
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AU2018348214A
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English (en)
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AU2018348214B2 (en
AU2018348214A1 (en
Inventor
Martin D. Burke
Rajeev S. CHORGHADE
Katrina A. Muraglia
Michael J. Welsh
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.)
University of Iowa Research Foundation UIRF
University of Illinois System
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University of Iowa Research Foundation UIRF
University of Illinois at Urbana Champaign
University of Illinois System
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Publication of AU2018348214A1 publication Critical patent/AU2018348214A1/en
Publication of AU2018348214B2 publication Critical patent/AU2018348214B2/en
Priority to AU2024259758A priority Critical patent/AU2024259758A1/en
Application granted granted Critical
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7048—Compounds having saccharide radicals and heterocyclic rings having oxygen as a ring hetero atom, e.g. leucoglucosan, hesperidin, erythromycin, nystatin, digitoxin or digoxin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/575—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of three or more carbon atoms, e.g. cholane, cholestane, ergosterol, sitosterol
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/0078—Sprays 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/008—Sprays 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]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pulmonology (AREA)
  • Dispersion Chemistry (AREA)
  • Otolaryngology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2018348214A 2017-10-11 2018-10-11 Genotype-agnostic rescue of cystic fibrosis with small molecule bicarbonate channels Active AU2018348214C1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2024259758A AU2024259758A1 (en) 2017-10-11 2024-11-06 Genotype-agnostic rescue of cystic fibrosis with small molecule bicarbonate channels

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762570795P 2017-10-11 2017-10-11
US62/570,795 2017-10-11
PCT/US2018/055435 WO2019075214A2 (en) 2017-10-11 2018-10-11 INDEPENDENT RESCUEING OF THE GENOTYPE OF CYSTIC FIBROSIS WITH BICARBONATE CHANNELS WITH SMALL MOLECULES

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2024259758A Division AU2024259758A1 (en) 2017-10-11 2024-11-06 Genotype-agnostic rescue of cystic fibrosis with small molecule bicarbonate channels

Publications (3)

Publication Number Publication Date
AU2018348214A1 AU2018348214A1 (en) 2020-04-23
AU2018348214B2 AU2018348214B2 (en) 2024-08-08
AU2018348214C1 true AU2018348214C1 (en) 2025-01-30

Family

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Family Applications (2)

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AU2018348214A Active AU2018348214C1 (en) 2017-10-11 2018-10-11 Genotype-agnostic rescue of cystic fibrosis with small molecule bicarbonate channels
AU2024259758A Pending AU2024259758A1 (en) 2017-10-11 2024-11-06 Genotype-agnostic rescue of cystic fibrosis with small molecule bicarbonate channels

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2024259758A Pending AU2024259758A1 (en) 2017-10-11 2024-11-06 Genotype-agnostic rescue of cystic fibrosis with small molecule bicarbonate channels

Country Status (10)

Country Link
US (3) US12090164B2 (enExample)
EP (1) EP3694526B1 (enExample)
JP (2) JP7355335B2 (enExample)
AU (2) AU2018348214C1 (enExample)
CA (1) CA3076246A1 (enExample)
DK (1) DK3694526T3 (enExample)
ES (1) ES3009563T3 (enExample)
FI (1) FI3694526T3 (enExample)
PT (1) PT3694526T (enExample)
WO (1) WO2019075214A2 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7355335B2 (ja) 2017-10-11 2023-10-03 ザ ボード オブ トラスティーズ オブ ザ ユニヴァーシティ オブ イリノイ 小分子重炭酸イオンチャネルを用いた嚢胞性線維症の遺伝子型非依存性救済
KR20240164801A (ko) 2022-03-21 2024-11-20 더 보오드 오브 트러스티스 오브 더 유니버시티 오브 일리노이즈 암포테리신 b의 지질-코팅된 결정을 포함하는 이온 채널 보형 조성물
WO2025019806A2 (en) * 2023-07-19 2025-01-23 The Board Of Trustees Of The University Of Illinois Restoring physiology with small molecule mimics of missing proteins
WO2025064626A1 (en) * 2023-09-20 2025-03-27 The Board Of Trustees Of The University Of Illinois Additive effects for molecular prosthetcs and cftr modulators in cf epithelia

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016073462A1 (en) * 2014-11-04 2016-05-12 The Board Of Trustees Of The University Of Illinois Restoring physiology with small molecule mimics of missing proteins
WO2017177228A1 (en) * 2016-04-08 2017-10-12 The Board Of Trustees Of The University Of Illinois Small molecule-mediated restoration of airway surface physiology in human cystic fibrosis lung epithelia

Family Cites Families (10)

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AU598958B2 (en) * 1987-11-12 1990-07-05 Vestar, Inc. Improved amphotericin b liposome preparation
AU643141B2 (en) 1991-03-15 1993-11-04 Amgen, Inc. Pulmonary administration of granulocyte colony stimulating factor
US5451569A (en) 1994-04-19 1995-09-19 Hong Kong University Of Science And Technology R & D Corporation Limited Pulmonary drug delivery system
JP5118302B2 (ja) * 2002-10-29 2013-01-16 トランセイブ， インク． 抗感染剤の徐放
TWI249408B (en) 2003-12-10 2006-02-21 Ind Tech Res Inst New antifungal formulation and manufacturing method thereof
ATE476965T1 (de) * 2004-06-11 2010-08-15 Verma Jitendra Nath Sterol-angereicherte multilamellare amphotericin- enthaltende liposomen in kochsalzlösung und herstellungsverfahren
WO2011050311A1 (en) 2009-10-22 2011-04-28 University Of Pittsburgh-Of The Commonwealth System Of Higher Education Use of endostatin peptides for the treatment of fibrosis
US8945605B2 (en) * 2011-06-07 2015-02-03 Parion Sciences, Inc. Aerosol delivery systems, compositions and methods
WO2017181193A2 (en) * 2016-04-15 2017-10-19 The Uab Research Foundation Methods and compounds for stimulating read-through of premature termination codons
JP7355335B2 (ja) 2017-10-11 2023-10-03 ザ ボード オブ トラスティーズ オブ ザ ユニヴァーシティ オブ イリノイ 小分子重炭酸イオンチャネルを用いた嚢胞性線維症の遺伝子型非依存性救済

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016073462A1 (en) * 2014-11-04 2016-05-12 The Board Of Trustees Of The University Of Illinois Restoring physiology with small molecule mimics of missing proteins
WO2017177228A1 (en) * 2016-04-08 2017-10-12 The Board Of Trustees Of The University Of Illinois Small molecule-mediated restoration of airway surface physiology in human cystic fibrosis lung epithelia

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
"AmBisome (Amphotericin B) liposome for injection", March 2012 (2012-03-01), pages 1 - 27, XP055692942, Retrieved from the Internet *
M. PROESMANS ET AL.: "Use of Nebulized Amphotericin B in the Treatment of Allergic Bronchopulmonary Aspergillosis in Cystic Fibrosis", INTERNATIONAL JOURNAL OF PEDIATRICS, vol. 2010, 376287, (2010), pages 1 - 9, DOI: 10.1155/2010/376287 *

Also Published As

Publication number Publication date
WO2019075214A2 (en) 2019-04-18
JP2020536859A (ja) 2020-12-17
PT3694526T (pt) 2025-01-20
US20200352970A1 (en) 2020-11-12
DK3694526T3 (da) 2025-02-03
CA3076246A1 (en) 2019-04-18
EP3694526A2 (en) 2020-08-19
WO2019075214A3 (en) 2020-04-02
ES3009563T3 (en) 2025-03-27
AU2018348214B2 (en) 2024-08-08
US20250009775A1 (en) 2025-01-09
JP7355335B2 (ja) 2023-10-03
AU2024259758A1 (en) 2024-11-21
JP2023126932A (ja) 2023-09-12
FI3694526T3 (fi) 2025-02-06
AU2018348214A1 (en) 2020-04-23
US20250000886A1 (en) 2025-01-02
EP3694526B1 (en) 2024-12-04
EP3694526A4 (en) 2021-06-09
US12090164B2 (en) 2024-09-17

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