MX2021009388A - Tratamiento de ciliopatias. - Google Patents
Tratamiento de ciliopatias.Info
- Publication number
- MX2021009388A MX2021009388A MX2021009388A MX2021009388A MX2021009388A MX 2021009388 A MX2021009388 A MX 2021009388A MX 2021009388 A MX2021009388 A MX 2021009388A MX 2021009388 A MX2021009388 A MX 2021009388A MX 2021009388 A MX2021009388 A MX 2021009388A
- Authority
- MX
- Mexico
- Prior art keywords
- polyribonucleotide
- ciliopathy
- subject
- ciliogenesis
- present disclosure
- Prior art date
Links
- 208000031214 ciliopathy Diseases 0.000 title abstract 4
- 102000040430 polynucleotide Human genes 0.000 abstract 4
- 108091033319 polynucleotide Proteins 0.000 abstract 4
- 239000008194 pharmaceutical composition Substances 0.000 abstract 2
- 102000004169 proteins and genes Human genes 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 210000002345 respiratory system Anatomy 0.000 abstract 2
- 206010061218 Inflammation Diseases 0.000 abstract 1
- 230000007547 defect Effects 0.000 abstract 1
- 230000000694 effects Effects 0.000 abstract 1
- 230000004054 inflammatory process Effects 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
- A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
- A61K31/19—Carboxylic acids, e.g. valproic acid
- A61K31/195—Carboxylic acids, e.g. valproic acid having an amino group
- A61K31/197—Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid
- A61K31/198—Alpha-amino acids, e.g. alanine or edetic acid [EDTA]
-
- 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
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
- A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
- A61K47/183—Amino acids, e.g. glycine, EDTA or aspartame
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
- G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
- G01N33/5044—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics involving specific cell types
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
-
- 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
- A61K9/0043—Nose
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Chemical & Material Sciences (AREA)
- Pulmonology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biochemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Zoology (AREA)
- Marine Sciences & Fisheries (AREA)
- Biomedical Technology (AREA)
- Otolaryngology (AREA)
- Biotechnology (AREA)
- Urology & Nephrology (AREA)
- Hematology (AREA)
- Cell Biology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Genetics & Genomics (AREA)
- Dispersion Chemistry (AREA)
- General Physics & Mathematics (AREA)
- Pathology (AREA)
- Tropical Medicine & Parasitology (AREA)
- Food Science & Technology (AREA)
- Physics & Mathematics (AREA)
- Analytical Chemistry (AREA)
- Toxicology (AREA)
Abstract
La presente divulgación proporciona una composición farmacéutica que comprende un polirribonucleótido para uso en el tratamiento de una ciliopatía en un sujeto que padece una ciliopatía, en la que el polirribonucleótido codifica una versión funcional de una proteína cuyo defecto está asociado con dicha ciliopatía, y en la que la administración de dicha composición farmacéutica al sistema respiratorio de dicho sujeto se efectúa cuando el sujeto muestra una inflamación del sistema respiratorio. Además, la presente divulgación se refiere a un procedimiento para analizar el efecto de un polirribonucleótido sobre la ciliogénesis, en el que dicho polirribonucleótido codifica una proteína implicada y/o necesaria para la ciliogénesis.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP19157210 | 2019-02-14 | ||
PCT/EP2020/053774 WO2020165352A1 (en) | 2019-02-14 | 2020-02-13 | Treatment of ciliopathies |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2021009388A true MX2021009388A (es) | 2021-09-10 |
Family
ID=65440876
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2021009388A MX2021009388A (es) | 2019-02-14 | 2020-02-13 | Tratamiento de ciliopatias. |
Country Status (13)
Country | Link |
---|---|
US (1) | US20220211807A1 (es) |
EP (2) | EP4223306A3 (es) |
JP (1) | JP2022522412A (es) |
KR (1) | KR20210127718A (es) |
CN (1) | CN113423418A (es) |
AU (1) | AU2020220636A1 (es) |
BR (1) | BR112021014528A2 (es) |
CA (2) | CA3129912A1 (es) |
HU (1) | HUE065017T2 (es) |
MX (1) | MX2021009388A (es) |
PL (1) | PL3923971T3 (es) |
WO (1) | WO2020165352A1 (es) |
ZA (1) | ZA202106769B (es) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
DK3463483T3 (da) | 2016-05-27 | 2024-03-04 | Transcriptx Inc | Behandling af primær ciliedyskinesi med syntetisk messenger-RNA |
US20240123087A1 (en) * | 2021-03-19 | 2024-04-18 | Recode Therapeutics, Inc. | Polynucleotide compositions, related formulations, and methods of use thereof |
EP4313002A1 (en) * | 2021-03-22 | 2024-02-07 | Recode Therapeutics, Inc. | Polynucleotide compositions, related formulations, and methods of use thereof |
WO2023014887A1 (en) * | 2021-08-04 | 2023-02-09 | The Trustees Of Indiana University | Methods of treating compromised lung function and assessing clinical improvement |
Family Cites Families (12)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
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 |
CA2299429C (en) | 1997-08-13 | 2010-04-20 | Biontex Laboratories Gmbh | New lipopolyamines,their preparation and the use thereof |
WO2001000708A1 (de) | 1999-06-25 | 2001-01-04 | Christian Plank | Kombinationen zur einführung von nucleinsäuren in zellen |
CA2569664C (en) | 2004-06-07 | 2013-07-16 | Protiva Biotherapeutics, Inc. | Lipid encapsulated interfering rna |
FR2925491B1 (fr) | 2007-12-19 | 2010-09-03 | Oz Biosciences Sas | Nouvelle classe de lipides cationiques pour le transport d'agents actifs dans les cellules |
DK2858677T3 (da) * | 2012-06-08 | 2020-08-31 | Ethris Gmbh | Pulmonær levering af messenger rna |
EA201492055A1 (ru) * | 2012-06-08 | 2015-11-30 | Шир Хьюман Дженетик Терапис, Инк. | ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ |
WO2014020723A1 (ja) | 2012-08-01 | 2014-02-06 | 株式会社コナミデジタルエンタテインメント | 処理装置、処理装置の制御方法、及び、処理装置のプログラム |
DK3013964T3 (da) * | 2013-06-28 | 2020-06-15 | Ethris Gmbh | Sammensætninger til indføring af rna i celler |
JP2017534649A (ja) | 2014-11-10 | 2017-11-24 | エスリス ゲーエムベーハーethris GmbH | Bmpをコードするrnaの送達による骨形成誘導 |
DK3463483T3 (da) * | 2016-05-27 | 2024-03-04 | Transcriptx Inc | Behandling af primær ciliedyskinesi med syntetisk messenger-RNA |
GB201707212D0 (en) * | 2017-05-05 | 2017-06-21 | Ucl Business Plc | Gene therapy for ciliopathies |
-
2020
- 2020-02-13 CA CA3129912A patent/CA3129912A1/en active Pending
- 2020-02-13 JP JP2021547511A patent/JP2022522412A/ja active Pending
- 2020-02-13 EP EP23164871.8A patent/EP4223306A3/en active Pending
- 2020-02-13 AU AU2020220636A patent/AU2020220636A1/en active Pending
- 2020-02-13 CN CN202080014297.0A patent/CN113423418A/zh active Pending
- 2020-02-13 KR KR1020217028846A patent/KR20210127718A/ko active Search and Examination
- 2020-02-13 BR BR112021014528-1A patent/BR112021014528A2/pt unknown
- 2020-02-13 CA CA3225057A patent/CA3225057A1/en active Pending
- 2020-02-13 HU HUE20704031A patent/HUE065017T2/hu unknown
- 2020-02-13 PL PL20704031.2T patent/PL3923971T3/pl unknown
- 2020-02-13 US US17/431,146 patent/US20220211807A1/en active Pending
- 2020-02-13 EP EP20704031.2A patent/EP3923971B1/en active Active
- 2020-02-13 WO PCT/EP2020/053774 patent/WO2020165352A1/en unknown
- 2020-02-13 MX MX2021009388A patent/MX2021009388A/es unknown
-
2021
- 2021-09-13 ZA ZA2021/06769A patent/ZA202106769B/en unknown
Also Published As
Publication number | Publication date |
---|---|
EP3923971B1 (en) | 2023-12-20 |
CN113423418A (zh) | 2021-09-21 |
CA3225057A1 (en) | 2020-08-20 |
EP3923971A1 (en) | 2021-12-22 |
US20220211807A1 (en) | 2022-07-07 |
EP4223306A2 (en) | 2023-08-09 |
CA3129912A1 (en) | 2020-08-20 |
ZA202106769B (en) | 2024-01-31 |
PL3923971T3 (pl) | 2024-03-25 |
BR112021014528A2 (pt) | 2021-10-13 |
AU2020220636A1 (en) | 2021-07-29 |
EP3923971C0 (en) | 2023-12-20 |
KR20210127718A (ko) | 2021-10-22 |
WO2020165352A1 (en) | 2020-08-20 |
HUE065017T2 (hu) | 2024-04-28 |
EP4223306A3 (en) | 2023-11-29 |
JP2022522412A (ja) | 2022-04-19 |
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