JPWO2022174037A5 - - Google Patents
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- Publication number
- JPWO2022174037A5 JPWO2022174037A5 JP2023548587A JP2023548587A JPWO2022174037A5 JP WO2022174037 A5 JPWO2022174037 A5 JP WO2022174037A5 JP 2023548587 A JP2023548587 A JP 2023548587A JP 2023548587 A JP2023548587 A JP 2023548587A JP WO2022174037 A5 JPWO2022174037 A5 JP WO2022174037A5
- Authority
- JP
- Japan
- Prior art keywords
- rhgaa
- subject
- treatment
- mol
- pharmaceutical composition
- 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
- 101001018026 Homo sapiens Lysosomal alpha-glucosidase Proteins 0.000 claims 46
- 102000045921 human GAA Human genes 0.000 claims 46
- 239000008194 pharmaceutical composition Substances 0.000 claims 23
- 238000000034 method Methods 0.000 claims 15
- 230000004988 N-glycosylation Effects 0.000 claims 8
- 238000001294 liquid chromatography-tandem mass spectrometry Methods 0.000 claims 8
- 102000004420 Creatine Kinase Human genes 0.000 claims 7
- 108010042126 Creatine kinase Proteins 0.000 claims 7
- 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 6
- 239000003814 drug Substances 0.000 claims 6
- 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 6
- 230000002485 urinary effect Effects 0.000 claims 6
- UQRORFVVSGFNRO-UTINFBMNSA-N miglustat Chemical group CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO UQRORFVVSGFNRO-UTINFBMNSA-N 0.000 claims 5
- 229960001512 miglustat Drugs 0.000 claims 5
- 150000003839 salts Chemical class 0.000 claims 5
- 239000000872 buffer Substances 0.000 claims 4
- 230000003247 decreasing effect Effects 0.000 claims 4
- 239000003550 marker Substances 0.000 claims 4
- FBPFZTCFMRRESA-KVTDHHQDSA-N D-Mannitol Chemical compound OC[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-KVTDHHQDSA-N 0.000 claims 3
- 229930195725 Mannitol Natural products 0.000 claims 3
- 108010006519 Molecular Chaperones Proteins 0.000 claims 3
- 239000000594 mannitol Substances 0.000 claims 3
- 235000010355 mannitol Nutrition 0.000 claims 3
- 230000000144 pharmacologic effect Effects 0.000 claims 3
- 239000000244 polyoxyethylene sorbitan monooleate Substances 0.000 claims 3
- 235000010482 polyoxyethylene sorbitan monooleate Nutrition 0.000 claims 3
- 229920000053 polysorbate 80 Polymers 0.000 claims 3
- 229940068968 polysorbate 80 Drugs 0.000 claims 3
- 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 claims 2
- 229920002527 Glycogen Polymers 0.000 claims 2
- 208000029549 Muscle injury Diseases 0.000 claims 2
- ZAIHWUXYHDAQEV-YXTXVXLGSA-N P(=O)(O)(O)O.O=C[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO.O=C[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO Chemical compound P(=O)(O)(O)O.O=C[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO.O=C[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO ZAIHWUXYHDAQEV-YXTXVXLGSA-N 0.000 claims 2
- 238000009825 accumulation Methods 0.000 claims 2
- 239000002535 acidifier Substances 0.000 claims 2
- 230000003113 alkalizing effect Effects 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 239000003795 chemical substances by application Substances 0.000 claims 2
- 229940096919 glycogen Drugs 0.000 claims 2
- 230000007659 motor function Effects 0.000 claims 2
- 239000000546 pharmaceutical excipient Substances 0.000 claims 2
- 125000005629 sialic acid group Chemical group 0.000 claims 2
- 239000001509 sodium citrate Substances 0.000 claims 2
- NLJMYIDDQXHKNR-UHFFFAOYSA-K sodium citrate Chemical group O.O.[Na+].[Na+].[Na+].[O-]C(=O)CC(O)(CC([O-])=O)C([O-])=O NLJMYIDDQXHKNR-UHFFFAOYSA-K 0.000 claims 2
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 claims 2
- KRKNYBCHXYNGOX-UHFFFAOYSA-K Citrate Chemical compound [O-]C(=O)CC(O)(CC([O-])=O)C([O-])=O KRKNYBCHXYNGOX-UHFFFAOYSA-K 0.000 claims 1
- 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
- 229910019142 PO4 Inorganic materials 0.000 claims 1
- 229960004593 alglucosidase alfa Drugs 0.000 claims 1
- 210000004978 chinese hamster ovary cell Anatomy 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 229940079593 drug Drugs 0.000 claims 1
- 201000004502 glycogen storage disease II Diseases 0.000 claims 1
- -1 hexose tetrasaccharide Chemical class 0.000 claims 1
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 1
- 239000010452 phosphate Substances 0.000 claims 1
- 239000000902 placebo Substances 0.000 claims 1
- 229940068196 placebo Drugs 0.000 claims 1
- 230000009325 pulmonary function Effects 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163148596P | 2021-02-11 | 2021-02-11 | |
| US63/148,596 | 2021-02-11 | ||
| US202163162683P | 2021-03-18 | 2021-03-18 | |
| US63/162,683 | 2021-03-18 | ||
| PCT/US2022/016124 WO2022174037A1 (en) | 2021-02-11 | 2022-02-11 | Recombinant human acid alpha-glucosidase and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024506346A JP2024506346A (ja) | 2024-02-13 |
| JPWO2022174037A5 true JPWO2022174037A5 (es) | 2025-02-19 |
Family
ID=82837922
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023548587A Pending JP2024506346A (ja) | 2021-02-11 | 2022-02-11 | 組換えヒト酸性アルファグルコシダーゼ及びその使用 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20240197839A1 (es) |
| EP (1) | EP4291225A4 (es) |
| JP (1) | JP2024506346A (es) |
| KR (1) | KR20230155622A (es) |
| AU (1) | AU2022218792A1 (es) |
| BR (1) | BR112023016212A2 (es) |
| CA (1) | CA3207917A1 (es) |
| IL (1) | IL305103A (es) |
| TW (1) | TW202245830A (es) |
| WO (1) | WO2022174037A1 (es) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN119343147A (zh) * | 2022-05-05 | 2025-01-21 | 阿米库斯治疗学公司 | 用于治疗庞贝病的方法 |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2013234042B2 (en) * | 2012-03-15 | 2017-11-02 | Oxyrane Uk Limited | Methods and materials for treatment of Pompe's disease |
| HRP20250058T1 (hr) * | 2014-09-30 | 2025-03-28 | Amicus Therapeutics, Inc. | Vrlo snažna kisela alfa-glukozidaza s pojačanim ugljikohidratima |
| KR20250007693A (ko) * | 2015-12-30 | 2025-01-14 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
| KR102758570B1 (ko) * | 2016-03-30 | 2025-01-23 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 재조합 산 알파-글루코시다제를 포함하는 제형 |
| CN119139461A (zh) * | 2017-05-15 | 2024-12-17 | 阿米库斯治疗学公司 | 重组人类酸性α-葡萄糖苷酶 |
| WO2020163480A1 (en) * | 2019-02-05 | 2020-08-13 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase and uses thereof |
-
2022
- 2022-02-11 CA CA3207917A patent/CA3207917A1/en active Pending
- 2022-02-11 TW TW111105104A patent/TW202245830A/zh unknown
- 2022-02-11 BR BR112023016212A patent/BR112023016212A2/pt unknown
- 2022-02-11 US US18/276,954 patent/US20240197839A1/en active Pending
- 2022-02-11 AU AU2022218792A patent/AU2022218792A1/en active Pending
- 2022-02-11 KR KR1020237031038A patent/KR20230155622A/ko active Pending
- 2022-02-11 JP JP2023548587A patent/JP2024506346A/ja active Pending
- 2022-02-11 IL IL305103A patent/IL305103A/en unknown
- 2022-02-11 EP EP22753411.2A patent/EP4291225A4/en active Pending
- 2022-02-11 WO PCT/US2022/016124 patent/WO2022174037A1/en active Application Filing
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