JP2022516356A - 原発性線毛機能不全症の治療のための組成物および方法 - Google Patents

原発性線毛機能不全症の治療のための組成物および方法 Download PDF

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JP2022516356A
JP2022516356A JP2021539435A JP2021539435A JP2022516356A JP 2022516356 A JP2022516356 A JP 2022516356A JP 2021539435 A JP2021539435 A JP 2021539435A JP 2021539435 A JP2021539435 A JP 2021539435A JP 2022516356 A JP2022516356 A JP 2022516356A
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mrna
seq
coding sequence
dnah5
cationic lipids
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JPWO2020146344A5 (https=
JP2022516356A5 (https=
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アヌシャ ディアス，
ダーシャン パレク，
ジェフリー エス． ドゥビンス，
クリスティアン コバー，
シュリラング カーブ，
ザルナ パテル，
サラ ジェイ． ドゥナジ，
フランク デローサ，
マイケル ハートレイン，
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トランスレイト バイオ， インコーポレイテッド
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Publication of JPWO2020146344A5 publication Critical patent/JPWO2020146344A5/ja
Publication of JP2022516356A5 publication Critical patent/JP2022516356A5/ja
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • 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
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • 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
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Organic Chemistry (AREA)
  • Dispersion Chemistry (AREA)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Genetics & Genomics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pulmonology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021539435A 2019-01-07 2020-01-07 原発性線毛機能不全症の治療のための組成物および方法 Pending JP2022516356A (ja)

Priority Applications (1)

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JP2024010105A JP7789103B2 (ja) 2019-01-07 2024-01-26 原発性線毛機能不全症の治療のための組成物および方法

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US201962789414P 2019-01-07 2019-01-07
US62/789,414 2019-01-07
PCT/US2020/012529 WO2020146344A1 (en) 2019-01-07 2020-01-07 Composition and methods for treatment of primary ciliary dyskinesia

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US (3) US11559561B2 (https=)
EP (1) EP3908597A1 (https=)
JP (2) JP2022516356A (https=)
CN (1) CN113412273B (https=)
AU (2) AU2020206109B2 (https=)
CA (1) CA3125588A1 (https=)
IL (1) IL284480A (https=)
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EP4316588A3 (en) 2016-05-27 2024-05-15 Transcriptx, Inc. Treatment of primary ciliary dyskinesia with synthetic messenger rna
JP2022516356A (ja) 2019-01-07 2022-02-25 トランスレイト バイオ， インコーポレイテッド 原発性線毛機能不全症の治療のための組成物および方法
US20240123087A1 (en) * 2021-03-19 2024-04-18 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
JP2024511463A (ja) 2021-03-22 2024-03-13 リコード セラピューティクス，インク． 細胞への標的送達のための組成物および方法
JP2024545572A (ja) * 2021-11-10 2024-12-10 トランスレイト バイオ， インコーポレイテッド 原発性線毛機能不全の治療のための組成物及び方法

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JP2018511588A (ja) * 2015-03-19 2018-04-26 トランスレイト バイオ， インコーポレイテッド ポンペ病のｍＲＮＡ治療

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AU2020206109B2 (en) 2024-12-19
US20230226147A1 (en) 2023-07-20
US20220087935A1 (en) 2022-03-24
JP2024028651A (ja) 2024-03-04
JP7789103B2 (ja) 2025-12-19
AU2020206109A1 (en) 2021-07-29
CN113412273B (zh) 2025-08-22
IL284480A (en) 2021-08-31
CA3125588A1 (en) 2020-07-16
WO2020146344A1 (en) 2020-07-16
CN113412273A (zh) 2021-09-17
US11559561B2 (en) 2023-01-24
US20200215157A1 (en) 2020-07-09
AU2025201623A1 (en) 2025-03-27
US12370238B2 (en) 2025-07-29
EP3908597A1 (en) 2021-11-17

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