MX2018008185A - Alfa-glucosidasa con mayor cantidad de acido para el tratamiento de la enfermedad de pompe. - Google Patents
Alfa-glucosidasa con mayor cantidad de acido para el tratamiento de la enfermedad de pompe.Info
- Publication number
- MX2018008185A MX2018008185A MX2018008185A MX2018008185A MX2018008185A MX 2018008185 A MX2018008185 A MX 2018008185A MX 2018008185 A MX2018008185 A MX 2018008185A MX 2018008185 A MX2018008185 A MX 2018008185A MX 2018008185 A MX2018008185 A MX 2018008185A
- Authority
- MX
- Mexico
- Prior art keywords
- glucosidase
- pompe disease
- treatment
- acid alpha
- recombinant human
- Prior art date
Links
- 239000002253 acid Substances 0.000 title abstract 4
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 title abstract 3
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 title abstract 2
- 206010053185 Glycogen storage disease type II Diseases 0.000 title abstract 2
- 201000004502 glycogen storage disease II Diseases 0.000 title abstract 2
- 108010028144 alpha-Glucosidases Proteins 0.000 title 1
- 230000003190 augmentative effect Effects 0.000 title 1
- NBSCHQHZLSJFNQ-QTVWNMPRSA-N D-Mannose-6-phosphate Chemical group OC1O[C@H](COP(O)(O)=O)[C@@H](O)[C@H](O)[C@@H]1O NBSCHQHZLSJFNQ-QTVWNMPRSA-N 0.000 abstract 1
- 230000002255 enzymatic effect Effects 0.000 abstract 1
- 230000013595 glycosylation Effects 0.000 abstract 1
- 238000006206 glycosylation reaction Methods 0.000 abstract 1
- 230000005764 inhibitory process Effects 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- UQRORFVVSGFNRO-UTINFBMNSA-N miglustat Chemical compound CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO UQRORFVVSGFNRO-UTINFBMNSA-N 0.000 abstract 1
- 229960001512 miglustat Drugs 0.000 abstract 1
Classifications
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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
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- 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
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
-
- 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
- A61K9/0012—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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
-
- 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
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)
- Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Zoology (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Obesity (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Physiology (AREA)
- Nutrition Science (AREA)
- Biomedical Technology (AREA)
- Dermatology (AREA)
- Microbiology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
Un método para tratar la enfermedad de Pompe que incluye la administración de a-glucosidasa ácida humana recombinante que tiene una glucosilación óptima con residuos de manosa-6-fosfato junto con una cantidad de miglustat eficaz para maximizar la captación en tejidos de la a-glucosidasa ácida humana recombinante a la vez que se minimiza la inhibición de la actividad enzimática de la a-glucosidasa ácida humana recombinante.
Applications Claiming Priority (8)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562272890P | 2015-12-30 | 2015-12-30 | |
| US201662300479P | 2016-02-26 | 2016-02-26 | |
| US201662315412P | 2016-03-30 | 2016-03-30 | |
| US201662402454P | 2016-09-30 | 2016-09-30 | |
| US201662428867P | 2016-12-01 | 2016-12-01 | |
| US201662431791P | 2016-12-08 | 2016-12-08 | |
| US15/394,135 US20170189497A1 (en) | 2015-12-30 | 2016-12-29 | Augmented Acid Alpha-Glucosidase For The Treatment Of Pompe Disease |
| PCT/US2016/069243 WO2017117407A1 (en) | 2015-12-30 | 2016-12-29 | Augmented acid alpha-glucosidase for the treatment of pompe disease |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| MX2018008185A true MX2018008185A (es) | 2018-08-28 |
| MX394968B MX394968B (es) | 2025-03-24 |
Family
ID=57822129
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2018008185A MX394968B (es) | 2015-12-30 | 2016-12-29 | Alfa-glucosidasa con mayor cantidad de acido para el tratamiento de la enfermedad de pompe. |
| MX2022010311A MX2022010311A (es) | 2015-12-30 | 2018-06-29 | Alfa-glucosidasa con mayor cantidad de acido para el tratamiento de la enfermedad de pompe. |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2022010311A MX2022010311A (es) | 2015-12-30 | 2018-06-29 | Alfa-glucosidasa con mayor cantidad de acido para el tratamiento de la enfermedad de pompe. |
Country Status (15)
| Country | Link |
|---|---|
| US (5) | US20170189497A1 (es) |
| JP (4) | JP2019501178A (es) |
| KR (3) | KR102510941B1 (es) |
| AU (2) | AU2016381832B2 (es) |
| BR (1) | BR112018013151A2 (es) |
| CA (1) | CA3010205A1 (es) |
| CL (1) | CL2018001773A1 (es) |
| CO (1) | CO2018007680A2 (es) |
| EA (1) | EA201891507A1 (es) |
| IL (3) | IL299470A (es) |
| MX (2) | MX394968B (es) |
| MY (1) | MY198085A (es) |
| PH (1) | PH12018501313B1 (es) |
| SG (1) | SG11201804965SA (es) |
| WO (1) | WO2017117407A1 (es) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3900702A1 (en) | 2015-03-19 | 2021-10-27 | Translate Bio, Inc. | Mrna therapy for pompe disease |
| KR102510941B1 (ko) | 2015-12-30 | 2023-03-20 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
| CA3019128A1 (en) * | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
| US11944628B2 (en) | 2017-05-12 | 2024-04-02 | Duke University | Methods for the use of low-dose immune modulators transiently for treating patients undergoing protein replacement therapy |
| DK3624831T5 (da) * | 2017-05-15 | 2024-09-02 | Amicus Therapeutics Inc | Rekombinant human sur alfa-glucosidase |
| MA50546A (fr) | 2017-06-07 | 2020-09-16 | Regeneron Pharma | Compositions et méthodes pour l'internalisation d'enzymes |
| AU2019218892B2 (en) | 2018-02-07 | 2025-08-14 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for therapeutic protein delivery |
| AU2019269685B2 (en) | 2018-05-17 | 2025-12-04 | Regeneron Pharmaceuticals, Inc. | Anti-CD63 antibodies, conjugates, and uses thereof |
| WO2020163480A1 (en) * | 2019-02-05 | 2020-08-13 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase and uses thereof |
| AU2020343680A1 (en) | 2019-09-06 | 2022-03-24 | Amicus Therapeutics, Inc. | Method for capturing and purification of biologics |
| AU2020408066A1 (en) | 2019-12-20 | 2022-06-30 | Codexis, Inc. | Engineered acid alpha-glucosidase variants |
| US20240197839A1 (en) * | 2021-02-11 | 2024-06-20 | Amicus Therapeutics, Inc. | Recombinant Human Acid Alpha-Glucosidase and Uses Thereof |
| EP4518885A1 (en) * | 2022-05-05 | 2025-03-12 | Amicus Therapeutics, Inc. | Methods for treating pompe disease |
| WO2025064350A1 (en) * | 2023-09-18 | 2025-03-27 | AskBio Inc. | Therapeutic adeno-associated virus using nucleic acid encoding alpha-glucosidase (gaa) for treating pompe disease, with heterologous signal peptide |
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| WO2017049161A1 (en) | 2015-09-18 | 2017-03-23 | Duke University | ACID ALPHA-GLUCOSIDASE AND A β-2 AGONIST FOR THE TREATMENT OF LYSOSOMAL STORAGE DISORDER |
| KR102510941B1 (ko) | 2015-12-30 | 2023-03-20 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
| NZ786723A (en) | 2016-03-30 | 2025-07-25 | Amicus Therapeutics Inc | Method for selection of high m6p recombinant proteins |
| CA3019128A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
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2016
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