HRP20240041T1 - Vrlo snažna kisela alfa-glukozidaza s pojačanim ugljikohidratima - Google Patents
Vrlo snažna kisela alfa-glukozidaza s pojačanim ugljikohidratima Download PDFInfo
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- HRP20240041T1 HRP20240041T1 HRP20240041TT HRP20240041T HRP20240041T1 HR P20240041 T1 HRP20240041 T1 HR P20240041T1 HR P20240041T T HRP20240041T T HR P20240041TT HR P20240041 T HRP20240041 T HR P20240041T HR P20240041 T1 HRP20240041 T1 HR P20240041T1
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- Prior art keywords
- rhgaa
- preparation
- glycans
- average
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Links
- 108010028144 alpha-Glucosidases Proteins 0.000 title claims 2
- 102100024295 Maltase-glucoamylase Human genes 0.000 title 1
- 239000002253 acid Substances 0.000 title 1
- 150000001720 carbohydrates Chemical class 0.000 title 1
- 235000014633 carbohydrates Nutrition 0.000 title 1
- 230000003389 potentiating effect Effects 0.000 title 1
- 101001018026 Homo sapiens Lysosomal alpha-glucosidase Proteins 0.000 claims 16
- 102000045921 human GAA Human genes 0.000 claims 16
- NBSCHQHZLSJFNQ-QTVWNMPRSA-N D-Mannose-6-phosphate Chemical compound OC1O[C@H](COP(O)(O)=O)[C@@H](O)[C@H](O)[C@@H]1O NBSCHQHZLSJFNQ-QTVWNMPRSA-N 0.000 claims 5
- LXBIFEVIBLOUGU-JGWLITMVSA-N duvoglustat Chemical compound OC[C@H]1NC[C@H](O)[C@@H](O)[C@@H]1O LXBIFEVIBLOUGU-JGWLITMVSA-N 0.000 claims 4
- 230000000144 pharmacologic effect Effects 0.000 claims 4
- 150000003839 salts Chemical class 0.000 claims 4
- LXBIFEVIBLOUGU-UHFFFAOYSA-N Deoxymannojirimycin Natural products OCC1NCC(O)C(O)C1O LXBIFEVIBLOUGU-UHFFFAOYSA-N 0.000 claims 2
- UQRORFVVSGFNRO-UTINFBMNSA-N miglustat Chemical compound CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO UQRORFVVSGFNRO-UTINFBMNSA-N 0.000 claims 2
- 229960001512 miglustat Drugs 0.000 claims 2
- 125000005629 sialic acid group Chemical group 0.000 claims 2
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 1
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims 1
- 230000002378 acidificating effect Effects 0.000 claims 1
- 102000016679 alpha-Glucosidases Human genes 0.000 claims 1
- SQVRNKJHWKZAKO-UHFFFAOYSA-N beta-N-Acetyl-D-neuraminic acid Natural products CC(=O)NC1C(O)CC(O)(C(O)=O)OC1C(O)C(O)CO SQVRNKJHWKZAKO-UHFFFAOYSA-N 0.000 claims 1
- 210000004978 chinese hamster ovary cell Anatomy 0.000 claims 1
- 201000004502 glycogen storage disease II Diseases 0.000 claims 1
- 210000004165 myocardium Anatomy 0.000 claims 1
- SQVRNKJHWKZAKO-OQPLDHBCSA-N sialic acid Chemical compound CC(=O)N[C@@H]1[C@@H](O)C[C@@](O)(C(O)=O)OC1[C@H](O)[C@H](O)CO SQVRNKJHWKZAKO-OQPLDHBCSA-N 0.000 claims 1
- 210000002027 skeletal muscle Anatomy 0.000 claims 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
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- 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)
- C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
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- 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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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- 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
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
-
- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
-
- 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)
-
- 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
- C12N2510/00—Genetically modified cells
- C12N2510/02—Cells for production
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Microbiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Emergency Medicine (AREA)
- Endocrinology (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Claims (15)
1. Pripravak koji sadrži rekombinantnu ljudsku kiselu alfa-glukozidazu (rhGAA) koju proizvode stanice jajnika kineskog hrčka, naznačen time što su 40%-60% ukupnih N-glikana na rhGAA u pripravku kompleksni tip N-glikana; i pri čemu je prosječni sadržaj N-glikana koji nose manoza-6-fosfat (M6P) u rasponu od 3,0 do 5,0 mola po molu rhGAA.
2. Pripravak prema zahtjevu 1, naznačen time što je prosječni sadržaj N-glikana koji nose M6P u rasponu od 3,0 do 4,0 mola po molu rhGAA.
3. Pripravak prema zahtjevu 1, naznačen time što je prosječni sadržaj N-glikana koji nose M6P u rasponu od 4,0 do 5,0 mola po molu rhGAA.
4. Pripravak prema bilo kojem od zahtjeva 1-3, naznačen time što u prosjeku N-glikani sadrže više od 4 mola sijalične kiseline po molu rhGAA.
5. Pripravak prema bilo kojem od zahtjeva 1-3, naznačen time što rhGAA sadrži u prosjeku 2,0 do 8,0 mol ostataka sijalične kiseline po molu rhGAA.
6. Pripravak prema bilo kojem od zahtjeva 1-5, naznačen time što rhGAA sadrži u prosjeku najmanje jedan bis-fosforilirani N-glikan po rhGAA.
7. Pripravak prema bilo kojem od zahtjeva 1-6, naznačen time što su 45%-55% ukupnih N-glikana na rhGAA u pripravku kompleksni tip N-glikana.
8. Pripravak prema bilo kojem od zahtjeva 1-7, naznačen time što su 50% ukupnih N-glikana na rhGAA u pripravku kompleksni tip N-glikana.
9. Pripravak prema bilo kojem od zahtjeva 1-8, naznačen time što prosječni sadržaj N-glikana koji nose M6P iznosi 4 mola po molu rhGAA, te pri čemu rhGAA sadrži u prosjeku 4,5 mola ostataka sijalične kiseline po molu rhGAA i u prosjeku najmanje jedan bis-fosforilirani N-glikan po rhGAA.
10. Pripravak prema bilo kojem od zahtjeva 1-9, naznačen time što nadalje sadrži farmakološki pratilac.
11. Pripravak prema zahtjevu 10, naznačen time što je farmakološki pratilac odabran iz skupine koju čine 1-deoksinojirimicin ili njegova sol i N-butil-deoksinojirimicin ili njegova sol.
12. Pripravak prema bilo kojem od zahtjeva 1-11, za upotrebu u liječenju Pompeove bolesti.
13. Pripravak za upotrebu prema zahtjevu 12, naznačen time što se navedeni pripravak primjenjuje na (a) srčani mišić subjekta, (b) kvadriceps, triceps ili drugi skeletni mišić subjekta, ili (c) dijafragmu subjekta.
14. Pripravak za upotrebu prema zahtjevu 12 ili zahtjevu 13, naznačen time što je navedeni pripravak kombiniran s, primijenjen zajedno s, ili odvojeno primijenjen zajedno s farmakološkim pratiocem.
15. Pripravak za upotrebu prema zahtjevu 14, naznačen time što je farmakološki pratilac odabran iz skupine koju čine 1-deoksinojirimicin ili njegova sol i N-butil-deoksinojirimicin ili njegova sol.
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462057842P | 2014-09-30 | 2014-09-30 | |
| US201462057847P | 2014-09-30 | 2014-09-30 | |
| US201562112463P | 2015-02-05 | 2015-02-05 | |
| US201562135345P | 2015-03-19 | 2015-03-19 | |
| PCT/US2015/053252 WO2016054231A1 (en) | 2014-09-30 | 2015-09-30 | Highly potent acid alpha-glucosidase with enhanced carbohydrates |
| EP15845664.0A EP3201320B1 (en) | 2014-09-30 | 2015-09-30 | Highly potent acid alpha-glucosidase with enhanced carbohydrates |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20240041T1 true HRP20240041T1 (hr) | 2024-03-29 |
Family
ID=55631441
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20240041TT HRP20240041T1 (hr) | 2014-09-30 | 2015-09-30 | Vrlo snažna kisela alfa-glukozidaza s pojačanim ugljikohidratima |
Country Status (36)
| Country | Link |
|---|---|
| US (5) | US10208299B2 (hr) |
| EP (2) | EP4273241A3 (hr) |
| JP (3) | JP6851964B2 (hr) |
| KR (3) | KR102306577B1 (hr) |
| CN (2) | CN114540327A (hr) |
| AU (2) | AU2015325028B2 (hr) |
| BR (1) | BR112017005810A2 (hr) |
| CA (2) | CA3228032A1 (hr) |
| CL (1) | CL2017000722A1 (hr) |
| CO (1) | CO2017002776A2 (hr) |
| CR (1) | CR20170107A (hr) |
| DK (1) | DK3201320T3 (hr) |
| DO (1) | DOP2017000079A (hr) |
| EA (1) | EA038986B1 (hr) |
| EC (1) | ECSP17019103A (hr) |
| ES (1) | ES2967620T3 (hr) |
| FI (2) | FI3201320T3 (hr) |
| FR (1) | FR24C1012I1 (hr) |
| HR (1) | HRP20240041T1 (hr) |
| HU (2) | HUE064629T2 (hr) |
| IL (4) | IL289383B2 (hr) |
| LT (1) | LT3201320T (hr) |
| MX (2) | MX2017003989A (hr) |
| MY (1) | MY186336A (hr) |
| NI (1) | NI201700039A (hr) |
| PE (1) | PE20170906A1 (hr) |
| PH (1) | PH12017500455A1 (hr) |
| PL (1) | PL3201320T3 (hr) |
| PT (1) | PT3201320T (hr) |
| RS (1) | RS65066B1 (hr) |
| SG (2) | SG10202003753PA (hr) |
| SI (1) | SI3201320T1 (hr) |
| SV (1) | SV2017005416A (hr) |
| TN (1) | TN2017000082A1 (hr) |
| WO (1) | WO2016054231A1 (hr) |
| ZA (2) | ZA201701945B (hr) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EA038986B1 (ru) | 2014-09-30 | 2021-11-18 | Амикус Терапьютикс, Инк. | Высокоактивная кислая альфа-глюкозидаза с повышенным содержанием углеводов |
| EP3397273B1 (en) * | 2015-12-30 | 2021-05-19 | Amicus Therapeutics, Inc. | Augmented acid alpha-glucosidase for the treatment of pompe disease |
| EA201891507A1 (ru) * | 2015-12-30 | 2018-12-28 | Амикус Терапьютикс, Инк. | Кислая альфа-глюкозидаза усиленного действия для лечения болезни помпе |
| WO2017173060A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
| TWI823272B (zh) * | 2016-03-30 | 2023-11-21 | 美商阿米庫斯醫療股份有限公司 | 用於選擇高m6p重組蛋白之方法 |
| IL295551A (en) * | 2016-03-30 | 2022-10-01 | Amicus Therapeutics Inc | A method for selecting recombinant proteins with a high amount of mannose-6-phosphate |
| JP2020503900A (ja) | 2017-01-10 | 2020-02-06 | アミカス セラピューティックス インコーポレイテッド | ファブリー病の処置のための組換えアルファ−ガラクトシダーゼａ |
| EA202290114A1 (ru) * | 2017-03-30 | 2022-03-29 | Амикус Терапьютикс, Инк. | Способ отбора рекомбинантных белков с высоким содержанием m6p |
| EP3624831B1 (en) * | 2017-05-15 | 2023-03-29 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase |
| BR112022004000A2 (pt) | 2019-09-06 | 2022-05-31 | Amicus Therapeutics Inc | Método para captura e purificação de biológicas |
| TW202245830A (zh) * | 2021-02-11 | 2022-12-01 | 美商阿米庫斯醫療股份有限公司 | 重組人類酸性α-葡萄糖苷酶及其用途 |
| WO2023150387A1 (en) * | 2022-02-07 | 2023-08-10 | M6P Therapeutics, Inc. | Compositions comprising acid alpha glucosidase and methods of use thereof |
| WO2023215865A1 (en) | 2022-05-05 | 2023-11-09 | Amicus Therapeutics, Inc. | Methods for treating pompe disease |
| WO2024119091A1 (en) | 2022-12-02 | 2024-06-06 | Amicus Therapeutics, Inc. | Fexamethods for treating infantile-onset pompe disease in pediatric patients |
| WO2024119070A1 (en) | 2022-12-02 | 2024-06-06 | Amicus Therapeutics, Inc. | Methods for treating late onset pompe disease in pediatric patients |
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