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
Authority
CA
Canada
Prior art keywords
amb
sterol
administered
molar ratio
f508del
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.)
Pending
Application number
CA3076246A
Other languages
English (en)
French (fr)
Inventor
Martin D. Burke
Katrina A. MURAGLIA
Rajeev S. CHORGHADE
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
Original Assignee
University of Iowa Research Foundation UIRF
University of Illinois System
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 University of Iowa Research Foundation UIRF, University of Illinois System filed Critical University of Iowa Research Foundation UIRF
Publication of CA3076246A1 publication Critical patent/CA3076246A1/en
Pending legal-status Critical Current

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Classifications

    • 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)
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
AU2018348214C1 (en) 2017-10-11 2025-01-30 The Board Of Trustees Of The University Of Illinois Genotype-agnostic rescue of cystic fibrosis with small molecule bicarbonate channels
AU2023238751A1 (en) 2022-03-21 2024-10-31 Cystetic Medicines, Inc. Ion channel prosthetic compositions comprising lipid-coated crystals of amphotericin b
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

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
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
MX353044B (es) * 2002-10-29 2017-12-18 Insmed Inc Liberacion sostenida de anti-infecciosos.
TWI249408B (en) * 2003-12-10 2006-02-21 Ind Tech Res Inst New antifungal formulation and manufacturing method thereof
US20070218119A1 (en) 2004-06-11 2007-09-20 Verma Jitendra N Sterol enriched mixed lammelarity amphotericin intercalating liposomes in saline and the process for their preparation
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
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
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
EP3442512A4 (en) * 2016-04-15 2020-07-22 The UAB Research Foundation METHODS AND COMPOUNDS FOR STIMULATING THE TRANSLECTURE OF PREMATURE TERMINATION CODONS
AU2018348214C1 (en) 2017-10-11 2025-01-30 The Board Of Trustees Of The University Of Illinois Genotype-agnostic rescue of cystic fibrosis with small molecule bicarbonate channels

Also Published As

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

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