JP7355335B2 - 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 - Google Patents

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 Download PDF

Info

Publication number
JP7355335B2
JP7355335B2 JP2020517886A JP2020517886A JP7355335B2 JP 7355335 B2 JP7355335 B2 JP 7355335B2 JP 2020517886 A JP2020517886 A JP 2020517886A JP 2020517886 A JP2020517886 A JP 2020517886A JP 7355335 B2 JP7355335 B2 JP 7355335B2
Authority
JP
Japan
Prior art keywords
amb
asl
cholesterol
administered
patient
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
JP2020517886A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020536859A (ja
JP2020536859A5 (enExample
Inventor
ディー バーク，マーティン
エー ムラリア，カトリーナ
エス コールガード，ラジーヴ
ジェイ ウェルシュ，マイケル
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 JP2020536859A publication Critical patent/JP2020536859A/ja
Publication of JP2020536859A5 publication Critical patent/JP2020536859A5/ja
Priority to JP2023111218A priority Critical patent/JP2023126932A/ja
Application granted granted Critical
Publication of JP7355335B2 publication Critical patent/JP7355335B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

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)
JP2020517886A 2017-10-11 2018-10-11 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 Active JP7355335B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023111218A JP2023126932A (ja) 2017-10-11 2023-07-06 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

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
JP2023111218A Division JP2023126932A (ja) 2017-10-11 2023-07-06 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

Publications (3)

Publication Number Publication Date
JP2020536859A JP2020536859A (ja) 2020-12-17
JP2020536859A5 JP2020536859A5 (enExample) 2021-11-11
JP7355335B2 true JP7355335B2 (ja) 2023-10-03

Family

ID=66100102

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020517886A Active JP7355335B2 (ja) 2017-10-11 2018-10-11 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
JP2023111218A Pending JP2023126932A (ja) 2017-10-11 2023-07-06 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

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023111218A Pending JP2023126932A (ja) 2017-10-11 2023-07-06 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

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 (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008501782A (ja) 2004-06-11 2008-01-24 ジテンドラ ナス ベルマ 塩水中のステロール濃縮混合層状アンフォテリシン（Ａｍｐｈｏｔｅｒｉｃｉｎ）挿入リポソーム及びその調製方法
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
JP7041961B2 (ja) 2016-04-08 2022-03-25 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 ヒト嚢胞性線維症肺上皮における気道表面の生理機能の小分子介在性回復

Family Cites Families (9)

* 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
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 (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008501782A (ja) 2004-06-11 2008-01-24 ジテンドラ ナス ベルマ 塩水中のステロール濃縮混合層状アンフォテリシン（Ａｍｐｈｏｔｅｒｉｃｉｎ）挿入リポソーム及びその調製方法
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
JP7041961B2 (ja) 2016-04-08 2022-03-25 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 ヒト嚢胞性線維症肺上皮における気道表面の生理機能の小分子介在性回復

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
Eur. Respi.r J.，2014年，Vol.44，pp.1308-1318
International Journal of Pediatrics，2010年，Article ID 376287，DOI: 10.1155/2010/376287
Journal of Chemotherapy，2015年，Vol.27，pp.307-311
Medical Mycology，2016年，Vol.54，pp.223-231

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
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

Similar Documents

Publication Publication Date Title
JP2023126932A (ja) 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
US11458094B2 (en) Compositions and methods for treating pulmonary edema or lung inflammation
Chorghade et al. Amphotericin B induces epithelial voltage responses in people with cystic fibrosis
Liu et al. A single dose of carbon monoxide intraperitoneal administration protects rat intestine from injury induced by lipopolysaccharide
US11850256B2 (en) Small molecule-mediated restoration of airway surface physiology in human cystic fibrosis lung epithelia
EP3215151B1 (en) Restoring physiology with small molecule mimics of missing proteins
Zhang et al. Preparation and evaluation of inhalable S-allylmercapto-N-acetylcysteine and nintedanib co-loaded liposomes for pulmonary fibrosis
JP2025510096A (ja) アムホテリシンｂの脂質被覆結晶を含むイオンチャネル補欠組成物
US20220265775A1 (en) Compositions and methods for treating pulmonary edema or lung inflammation
EP4142772A1 (en) Compositions for the prevention and/or treatment of respiratory tract infections
Liu et al. Intratracheal instillation of ethyl pyruvate nanoparticles prevents the development of shunt-flow-induced pulmonary arterial hypertension in a rat model
US20230147865A1 (en) Compositions and methods for treating pulmonary edema or lung inflammation
Chorghade Small molecule channels increase anion secretion and host defenses in cystic fibrosis airway epithelia: mechanistic studies and initial clinical translation
Liu et al. 6-Gingerol reshapes exosomal miR-148b-3p/ACSL4 axis to combat high glucose-induced retinal endothelial ferroptosis
KR20250137187A (ko) 폐 병태를 치료하기 위한 조성물 및 방법
Radadiya et al. Ciclopirox Olamine Induces Ferritinophagy and Ameliorates Disease Progression in Polycystic Kidney Disease
HK40046684A (en) Compositions and methods for treating pulmonary edema or lung inflammation
Zheng et al. SESN2 Inhibits Tubular Epithelial Cell-Derived Exosome Secretion to Protect Against Diabetic Kidney Disease by Restoring the Autophagy‒Lysosome Pathway

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211004

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20211004

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211112

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20220812

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220830

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20221017

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20221130

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20230306

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230706

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20230714

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20230814

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20230912

R150 Certificate of patent or registration of utility model

Ref document number: 7355335

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150