ES3009563T3 - 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
ES3009563T3
ES3009563T3 ES18865483T ES18865483T ES3009563T3 ES 3009563 T3 ES3009563 T3 ES 3009563T3 ES 18865483 T ES18865483 T ES 18865483T ES 18865483 T ES18865483 T ES 18865483T ES 3009563 T3 ES3009563 T3 ES 3009563T3
Authority
ES
Spain
Prior art keywords
amb
asl
cholesterol
patient
epithelia
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
ES18865483T
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English (en)
Spanish (es)
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.)
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Publication date
Application filed by University of Iowa Research Foundation UIRF, University of Illinois System filed Critical University of Iowa Research Foundation UIRF
Application granted granted Critical
Publication of ES3009563T3 publication Critical patent/ES3009563T3/es
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Classifications

    • 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
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7048Compounds having saccharide radicals and heterocyclic rings having oxygen as a ring hetero atom, e.g. leucoglucosan, hesperidin, erythromycin, nystatin, digitoxin or digoxin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/56Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/575Compounds 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • 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
    • A61K9/0019Injectable 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)
ES18865483T 2017-10-11 2018-10-11 Genotype-agnostic rescue of cystic fibrosis with small molecule bicarbonate channels Active ES3009563T3 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762570795P 2017-10-11 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
ES3009563T3 true ES3009563T3 (en) 2025-03-27

Family

ID=66100102

Family Applications (1)

Application Number Title Priority Date Filing Date
ES18865483T Active ES3009563T3 (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
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

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
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
US20180015115A1 (en) * 2014-11-04 2018-01-18 The Board Of Trustees Of The University Of Illinois Restoring physiology with small molecule mimics of missing proteins
DK3439668T3 (da) * 2016-04-08 2024-09-02 Univ Illinois Småmolekylemedieret genoprettelse af luftvejsoverfladefysiologi i lungeepiteler ved human cystisk fibrose
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

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
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
AU2018348214C1 (en) 2025-01-30

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