CA3076246A1 - 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
CA3076246A1
CA3076246A1 CA3076246A CA3076246A CA3076246A1 CA 3076246 A1 CA3076246 A1 CA 3076246A1 CA 3076246 A CA3076246 A CA 3076246A CA 3076246 A CA3076246 A CA 3076246A CA 3076246 A1 CA3076246 A1 CA 3076246A1
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CA
Canada
Prior art keywords
amb
sterol
administered
molar ratio
f508del
Prior art date
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Pending
Application number
CA3076246A
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English (en)
French (fr)
Inventor
D. Burke Martin
A. Muraglia Katrina
S. Chorghade Rajeev
J. Welsh Michael
Original Assignee
University of Iowa Research Foundation
University of Illinois System
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Application filed by University of Iowa Research Foundation, University of Illinois System filed Critical University of Iowa Research Foundation
Publication of CA3076246A1 publication Critical patent/CA3076246A1/en
Pending legal-status Critical Current

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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

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  • 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)
CA3076246A 2017-10-11 2018-10-11 Genotype-agnostic rescue of cystic fibrosis with small molecule bicarbonate channels Pending CA3076246A1 (en)

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

Publications (1)

Publication Number Publication Date
CA3076246A1 true CA3076246A1 (en) 2019-04-18

Family

ID=66100102

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3076246A Pending CA3076246A1 (en) 2017-10-11 2018-10-11 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
PT3694526T (pt) 2017-10-11 2025-01-20 Univ Illinois Resgate independente do genótipo da fibrose quística com canais de bicarbonato de moléculas pequenas
IL315719A (en) 2022-03-21 2024-11-01 Univ Illinois Prosthetic assemblies of ion channels containing lipid-coated amphotericin B crystals
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
EP4780466A1 (en) * 2023-09-20 2026-07-29 The Board Of Trustees Of The University Of Illinois Additive effects for molecular prosthetcs and cftr modulators in cf epithelia

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU598958B2 (en) * 1987-11-12 1990-07-05 Vestar, Inc. Improved amphotericin b liposome preparation
JP3507486B2 (ja) 1991-03-15 2004-03-15 アムジエン・インコーポレーテツド 顆粒球コロニー刺激因子の肺内投与
US5451569A (en) 1994-04-19 1995-09-19 Hong Kong University Of Science And Technology R & D Corporation Limited Pulmonary drug delivery system
AU2003304204B2 (en) * 2002-10-29 2009-12-24 Insmed Incorporated Sustained release of antiinfectives
TWI249408B (en) 2003-12-10 2006-02-21 Ind Tech Res Inst New antifungal formulation and manufacturing method thereof
CN101014322A (zh) * 2004-06-11 2007-08-08 吉藤德拉·纳提·维玛 盐水中富含固醇的添加两性霉素的混合层状脂质体及其制备方法
US8507441B2 (en) 2009-10-22 2013-08-13 University of Pittsburgh—Of the Commonwealth Systems 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
JP6796581B2 (ja) * 2014-11-04 2020-12-09 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Ｔｈｅ Ｂｏａｒｄ Ｏｆ Ｔｒｕｓｔｅｅｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｉｌｌｉｎｏｉｓ ミッシングタンパク質の小分子模倣体を用いた生理機能の回復
ES2983639T3 (es) 2016-04-08 2024-10-24 The Board Of Trustees Of The Univ Illinois Restauración mediada por moléculas pequeñas de la fisiología de la superficie de las vías respiratorias en epitelios pulmonares humanos de fibrosis quística
US10960018B2 (en) * 2016-04-15 2021-03-30 The Uab Research Foundation Methods and compounds for stimulating read-through of premature termination codons
PT3694526T (pt) 2017-10-11 2025-01-20 Univ Illinois Resgate independente do genótipo da fibrose quística com canais de bicarbonato de moléculas pequenas

Also Published As

Publication number Publication date
US20250000886A1 (en) 2025-01-02
WO2019075214A3 (en) 2020-04-02
ES3009563T3 (en) 2025-03-27
JP7355335B2 (ja) 2023-10-03
FI3694526T3 (fi) 2025-02-06
US20250009775A1 (en) 2025-01-09
JP2020536859A (ja) 2020-12-17
AU2018348214B2 (en) 2024-08-08
EP3694526A4 (en) 2021-06-09
DK3694526T3 (da) 2025-02-03
JP2023126932A (ja) 2023-09-12
EP3694526A2 (en) 2020-08-19
US12090164B2 (en) 2024-09-17
US20200352970A1 (en) 2020-11-12
AU2024259758A1 (en) 2024-11-21
AU2018348214C1 (en) 2025-01-30
AU2018348214A1 (en) 2020-04-23
EP3694526B1 (en) 2024-12-04
WO2019075214A2 (en) 2019-04-18
PT3694526T (pt) 2025-01-20

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