IL284480A - Preparations and methods for the treatment of primary ciliary movement disorder - Google Patents
Preparations and methods for the treatment of primary ciliary movement disorderInfo
- Publication number
- IL284480A IL284480A IL284480A IL28448021A IL284480A IL 284480 A IL284480 A IL 284480A IL 284480 A IL284480 A IL 284480A IL 28448021 A IL28448021 A IL 28448021A IL 284480 A IL284480 A IL 284480A
- Authority
- IL
- Israel
- Prior art keywords
- treatment
- composition
- methods
- ciliary dyskinesia
- primary ciliary
- Prior art date
Links
- 208000025678 Ciliary Motility disease Diseases 0.000 title 1
- 201000009266 primary ciliary dyskinesia Diseases 0.000 title 1
Classifications
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- 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)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962789414P | 2019-01-07 | 2019-01-07 | |
| PCT/US2020/012529 WO2020146344A1 (en) | 2019-01-07 | 2020-01-07 | Composition and methods for treatment of primary ciliary dyskinesia |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL284480A true IL284480A (en) | 2021-08-31 |
Family
ID=69423424
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL284480A IL284480A (en) | 2019-01-07 | 2021-06-29 | Preparations and methods for the treatment of primary ciliary movement disorder |
Country Status (8)
| Country | Link |
|---|---|
| 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=) |
| WO (1) | WO2020146344A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
Family Cites Families (49)
| Publication number | Priority date | Publication date | Assignee | Title |
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| US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
| US4500707A (en) | 1980-02-29 | 1985-02-19 | University Patents, Inc. | Nucleosides useful in the preparation of polynucleotides |
| US5132418A (en) | 1980-02-29 | 1992-07-21 | University Patents, Inc. | Process for preparing polynucleotides |
| US4668777A (en) | 1981-03-27 | 1987-05-26 | University Patents, Inc. | Phosphoramidite nucleoside compounds |
| US4415732A (en) | 1981-03-27 | 1983-11-15 | University Patents, Inc. | Phosphoramidite compounds and processes |
| US4973679A (en) | 1981-03-27 | 1990-11-27 | University Patents, Inc. | Process for oligonucleo tide synthesis using phosphormidite intermediates |
| US4373071A (en) | 1981-04-30 | 1983-02-08 | City Of Hope Research Institute | Solid-phase synthesis of polynucleotides |
| US4401796A (en) | 1981-04-30 | 1983-08-30 | City Of Hope Research Institute | Solid-phase synthesis of polynucleotides |
| US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4737323A (en) | 1986-02-13 | 1988-04-12 | Liposome Technology, Inc. | Liposome extrusion method |
| US5153319A (en) | 1986-03-31 | 1992-10-06 | University Patents, Inc. | Process for preparing polynucleotides |
| US5262530A (en) | 1988-12-21 | 1993-11-16 | Applied Biosystems, Inc. | Automated system for polynucleotide synthesis and purification |
| US5047524A (en) | 1988-12-21 | 1991-09-10 | Applied Biosystems, Inc. | Automated system for polynucleotide synthesis and purification |
| FR2645866B1 (fr) | 1989-04-17 | 1991-07-05 | Centre Nat Rech Scient | Nouvelles lipopolyamines, leur preparation et leur emploi |
| US5334761A (en) | 1992-08-28 | 1994-08-02 | Life Technologies, Inc. | Cationic lipids |
| US5885613A (en) | 1994-09-30 | 1999-03-23 | The University Of British Columbia | Bilayer stabilizing components and their use in forming programmable fusogenic liposomes |
| US5700642A (en) | 1995-05-22 | 1997-12-23 | Sri International | Oligonucleotide sizing using immobilized cleavable primers |
| US5744335A (en) | 1995-09-19 | 1998-04-28 | Mirus Corporation | Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein |
| EP1327684A1 (en) * | 2002-01-14 | 2003-07-16 | Universitätsklinikum Freiburg | DNAH5 and its use in diagnosis |
| US7799565B2 (en) | 2004-06-07 | 2010-09-21 | Protiva Biotherapeutics, Inc. | Lipid encapsulated interfering RNA |
| EP2049665A2 (en) | 2006-07-28 | 2009-04-22 | Applera Corporation | Dinucleotide mrna cap analogs |
| US8252757B2 (en) * | 2006-11-09 | 2012-08-28 | Dynavax Technologies Corporation | Long term disease modification using immunostimulatory oligonucleotides |
| WO2009058911A2 (en) | 2007-10-31 | 2009-05-07 | Applied Biosystems Inc. | Preparation and isolation of 5' capped mrna |
| CN104119242B (zh) | 2008-10-09 | 2017-07-07 | 泰米拉制药公司 | 改善的氨基脂质和递送核酸的方法 |
| ES2646630T3 (es) | 2008-11-07 | 2017-12-14 | Massachusetts Institute Of Technology | Lipidoides aminoalcohólicos y usos de los mismos |
| MX342785B (es) | 2009-06-10 | 2016-10-12 | Alnylam Pharmaceuticals Inc | Formulacion mejorada de lipido. |
| EP3354644A1 (en) | 2011-06-08 | 2018-08-01 | Translate Bio, Inc. | Cleavable lipids |
| KR102451116B1 (ko) | 2011-10-27 | 2022-10-06 | 메사추세츠 인스티튜트 오브 테크놀로지 | 약물 캡슐화 마이크로스피어를 형성할 수 있는, ｎ-말단 상에 관능화된 아미노산 유도체 |
| ES2762873T3 (es) | 2012-03-29 | 2020-05-26 | Translate Bio Inc | Lípidos catiónicos ionizables |
| ES2895651T3 (es) | 2013-12-19 | 2022-02-22 | Novartis Ag | Lípidos y composiciones lipídicas para la administración de agentes activos |
| US20170143848A1 (en) * | 2014-03-24 | 2017-05-25 | Shire Human Genetic Therapies, Inc. | Mrna therapy for the treatment of ocular diseases |
| JP6557722B2 (ja) | 2014-05-30 | 2019-08-07 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | 核酸の送達のための生分解性脂質 |
| SMT202200502T1 (it) | 2014-06-25 | 2023-01-13 | Acuitas Therapeutics Inc | Nuovi lipidi e formulazioni di nanoparticelle lipidiche per l'erogazione di acidi nucleici |
| US9840479B2 (en) | 2014-07-02 | 2017-12-12 | Massachusetts Institute Of Technology | Polyamine-fatty acid derived lipidoids and uses thereof |
| US20180000953A1 (en) | 2015-01-21 | 2018-01-04 | Moderna Therapeutics, Inc. | Lipid nanoparticle compositions |
| WO2016118725A1 (en) | 2015-01-23 | 2016-07-28 | Moderna Therapeutics, Inc. | Lipid nanoparticle compositions |
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| ES2949540T3 (es) | 2015-06-19 | 2023-09-29 | Massachusetts Inst Technology | 2,5-piperazinadionas sustituidas con alquenilo y su uso en composiciones para suministrar un agente a un sujeto o una célula |
| LT3313829T (lt) | 2015-06-29 | 2024-08-12 | Acuitas Therapeutics Inc. | Lipidai ir lipidų nanodalelių sudėtys, skirtos nukleorūgščių tiekimui |
| EP4286012A3 (en) | 2015-09-17 | 2024-05-29 | ModernaTX, Inc. | Compounds and compositions for intracellular delivery of therapeutic agents |
| IL307179A (en) | 2015-10-28 | 2023-11-01 | Acuitas Therapeutics Inc | Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids |
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| TWI773666B (zh) | 2016-03-30 | 2022-08-11 | 美商英特利亞醫療公司 | Crispr/cas 組分之脂質奈米粒子調配物 |
| EP4316588A3 (en) | 2016-05-27 | 2024-05-15 | Transcriptx, Inc. | Treatment of primary ciliary dyskinesia with synthetic messenger rna |
| MX2019005470A (es) | 2016-11-10 | 2019-11-21 | Translate Bio Inc | Formulación de nanopartículas lipídicas basadas en ice mejorada para el suministro de arnm. |
| WO2018089801A1 (en) | 2016-11-10 | 2018-05-17 | Translate Bio, Inc. | Improved process of preparing mrna-loaded lipid nanoparticles |
| MA47824A (fr) * | 2017-03-07 | 2020-01-15 | Translate Bio Inc | Administration polyanionique d'acides nucléiques |
| MX2020011166A (es) | 2018-04-25 | 2022-10-19 | Ethris Gmbh | Agentes crioprotectores para formulaciones en forma de particulas. |
| 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 |
-
2020
- 2020-01-07 JP JP2021539435A patent/JP2022516356A/ja active Pending
- 2020-01-07 CA CA3125588A patent/CA3125588A1/en active Pending
- 2020-01-07 CN CN202080012787.7A patent/CN113412273B/zh active Active
- 2020-01-07 WO PCT/US2020/012529 patent/WO2020146344A1/en not_active Ceased
- 2020-01-07 EP EP20703340.8A patent/EP3908597A1/en active Pending
- 2020-01-07 US US16/736,417 patent/US11559561B2/en active Active
- 2020-01-07 AU AU2020206109A patent/AU2020206109B2/en active Active
- 2020-01-07 US US17/420,346 patent/US20220087935A1/en not_active Abandoned
-
2021
- 2021-06-29 IL IL284480A patent/IL284480A/en unknown
-
2022
- 2022-12-06 US US18/062,435 patent/US12370238B2/en active Active
-
2024
- 2024-01-26 JP JP2024010105A patent/JP7789103B2/ja active Active
-
2025
- 2025-03-05 AU AU2025201623A patent/AU2025201623A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| 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 |
| CA3125588A1 (en) | 2020-07-16 |
| WO2020146344A1 (en) | 2020-07-16 |
| JP2022516356A (ja) | 2022-02-25 |
| 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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