CA3024625A1 - Polynucleotides encoding citrin for the treatment of citrullinemia type 2 - Google Patents
Polynucleotides encoding citrin for the treatment of citrullinemia type 2 Download PDFInfo
- Publication number
- CA3024625A1 CA3024625A1 CA3024625A CA3024625A CA3024625A1 CA 3024625 A1 CA3024625 A1 CA 3024625A1 CA 3024625 A CA3024625 A CA 3024625A CA 3024625 A CA3024625 A CA 3024625A CA 3024625 A1 CA3024625 A1 CA 3024625A1
- Authority
- CA
- Canada
- Prior art keywords
- pharmaceutical composition
- group
- polynucleotide
- alkyl
- citrin
- 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
Links
Classifications
-
- 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
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5123—Organic compounds, e.g. fats, sugars
-
- 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
- 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
-
- 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
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/513—Organic macromolecular compounds; Dendrimers
- A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Optics & Photonics (AREA)
- Nanotechnology (AREA)
- Biomedical Technology (AREA)
- Physics & Mathematics (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Chemical & Material Sciences (AREA)
- Urology & Nephrology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Toxicology (AREA)
- Zoology (AREA)
- Gastroenterology & Hepatology (AREA)
- Biophysics (AREA)
- Genetics & Genomics (AREA)
- Dermatology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662338479P | 2016-05-18 | 2016-05-18 | |
| US62/338,479 | 2016-05-18 | ||
| PCT/US2017/033421 WO2017201349A1 (en) | 2016-05-18 | 2017-05-18 | Polynucleotides encoding citrin for the treatment of citrullinemia type 2 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3024625A1 true CA3024625A1 (en) | 2017-11-23 |
Family
ID=59009788
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3024625A Pending CA3024625A1 (en) | 2016-05-18 | 2017-05-18 | Polynucleotides encoding citrin for the treatment of citrullinemia type 2 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US10993918B2 (enExample) |
| EP (1) | EP3458081A1 (enExample) |
| JP (2) | JP7210287B2 (enExample) |
| AU (2) | AU2017268396B2 (enExample) |
| CA (1) | CA3024625A1 (enExample) |
| MA (1) | MA45036A (enExample) |
| MY (1) | MY201498A (enExample) |
| SG (1) | SG11201810162PA (enExample) |
| WO (1) | WO2017201349A1 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12458604B2 (en) | 2020-10-14 | 2025-11-04 | The Trustees Of The University Of Pennsylvania | Methods of lipid nanoparticle manufacture and compositions derived therefrom |
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-
2017
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- 2017-05-18 JP JP2018560776A patent/JP7210287B2/ja active Active
- 2017-05-18 EP EP17728001.3A patent/EP3458081A1/en active Pending
- 2017-05-18 US US16/302,360 patent/US10993918B2/en active Active
- 2017-05-18 CA CA3024625A patent/CA3024625A1/en active Pending
- 2017-05-18 WO PCT/US2017/033421 patent/WO2017201349A1/en not_active Ceased
- 2017-05-18 MY MYPI2018001974A patent/MY201498A/en unknown
- 2017-05-18 AU AU2017268396A patent/AU2017268396B2/en not_active Ceased
- 2017-05-18 MA MA045036A patent/MA45036A/fr unknown
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Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12458604B2 (en) | 2020-10-14 | 2025-11-04 | The Trustees Of The University Of Pennsylvania | Methods of lipid nanoparticle manufacture and compositions derived therefrom |
Also Published As
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|---|---|
| AU2017268396B2 (en) | 2023-05-18 |
| MY201498A (en) | 2024-02-27 |
| US20220071915A1 (en) | 2022-03-10 |
| US20190175517A1 (en) | 2019-06-13 |
| EP3458081A1 (en) | 2019-03-27 |
| JP2023055716A (ja) | 2023-04-18 |
| AU2023216799A1 (en) | 2023-09-07 |
| MA45036A (fr) | 2019-03-27 |
| US10993918B2 (en) | 2021-05-04 |
| SG11201810162PA (en) | 2018-12-28 |
| JP2019519511A (ja) | 2019-07-11 |
| WO2017201349A1 (en) | 2017-11-23 |
| JP7210287B2 (ja) | 2023-01-23 |
| US12239742B2 (en) | 2025-03-04 |
| AU2017268396A1 (en) | 2018-12-06 |
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