JPWO2021156769A5 - - Google Patents
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- JPWO2021156769A5 JPWO2021156769A5 JP2021564953A JP2021564953A JPWO2021156769A5 JP WO2021156769 A5 JPWO2021156769 A5 JP WO2021156769A5 JP 2021564953 A JP2021564953 A JP 2021564953A JP 2021564953 A JP2021564953 A JP 2021564953A JP WO2021156769 A5 JPWO2021156769 A5 JP WO2021156769A5
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- 239000008194 pharmaceutical composition Substances 0.000 claims 23
- 150000001875 compounds Chemical class 0.000 claims 19
- 210000005013 brain tissue Anatomy 0.000 claims 18
- 210000004556 brain Anatomy 0.000 claims 16
- 238000000034 method Methods 0.000 claims 12
- 102100033342 Lysosomal acid glucosylceramidase Human genes 0.000 claims 7
- -1 fluorine) Chemical class 0.000 claims 7
- 229910052736 halogen Inorganic materials 0.000 claims 7
- 150000002367 halogens Chemical class 0.000 claims 7
- 239000003814 drug Substances 0.000 claims 6
- 238000002599 functional magnetic resonance imaging Methods 0.000 claims 6
- 108010039650 imiglucerase Proteins 0.000 claims 6
- 229960002127 imiglucerase Drugs 0.000 claims 6
- 210000002637 putamen Anatomy 0.000 claims 6
- 208000012902 Nervous system disease Diseases 0.000 claims 5
- 230000001965 increasing effect Effects 0.000 claims 5
- 229940002612 prodrug Drugs 0.000 claims 5
- 239000000651 prodrug Substances 0.000 claims 5
- 150000003839 salts Chemical class 0.000 claims 5
- YFHRCLAKZBDRHN-MRXNPFEDSA-N [(3s)-1-azabicyclo[2.2.2]octan-3-yl] n-[2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl]carbamate Chemical compound O([C@H]1C2CCN(CC2)C1)C(=O)NC(C)(C)C(N=1)=CSC=1C1=CC=C(F)C=C1 YFHRCLAKZBDRHN-MRXNPFEDSA-N 0.000 claims 4
- 238000002641 enzyme replacement therapy Methods 0.000 claims 4
- 238000004519 manufacturing process Methods 0.000 claims 4
- 230000001537 neural effect Effects 0.000 claims 4
- 125000003545 alkoxy group Chemical group 0.000 claims 3
- 125000000217 alkyl group Chemical group 0.000 claims 3
- 210000004727 amygdala Anatomy 0.000 claims 3
- 210000001353 entorhinal cortex Anatomy 0.000 claims 3
- 150000004701 malic acid derivatives Chemical class 0.000 claims 3
- 210000001009 nucleus accumben Anatomy 0.000 claims 3
- 210000004233 talus Anatomy 0.000 claims 3
- 208000015872 Gaucher disease Diseases 0.000 claims 2
- 208000037311 Gaucher disease type 3 Diseases 0.000 claims 2
- 208000015439 Lysosomal storage disease Diseases 0.000 claims 2
- 125000004093 cyano group Chemical group *C#N 0.000 claims 2
- 229940079593 drug Drugs 0.000 claims 2
- 230000002708 enhancing effect Effects 0.000 claims 2
- 150000002431 hydrogen Chemical class 0.000 claims 2
- 229910052739 hydrogen Inorganic materials 0.000 claims 2
- 239000001257 hydrogen Substances 0.000 claims 2
- 238000003384 imaging method Methods 0.000 claims 2
- 238000005259 measurement Methods 0.000 claims 2
- 238000012034 trail making test Methods 0.000 claims 2
- OJCLMSVFVVKWJX-UHFFFAOYSA-N 1-azabicyclo[2.2.2]octan-3-yl n-[2-[3-(4-fluorophenyl)phenyl]propan-2-yl]carbamate Chemical compound C1N(CC2)CCC2C1OC(=O)NC(C)(C)C(C=1)=CC=CC=1C1=CC=C(F)C=C1 OJCLMSVFVVKWJX-UHFFFAOYSA-N 0.000 claims 1
- 125000000882 C2-C6 alkenyl group Chemical group 0.000 claims 1
- 125000003320 C2-C6 alkenyloxy group Chemical group 0.000 claims 1
- 125000003601 C2-C6 alkynyl group Chemical group 0.000 claims 1
- 102000004328 Cytochrome P-450 CYP3A Human genes 0.000 claims 1
- 108010081668 Cytochrome P-450 CYP3A Proteins 0.000 claims 1
- XPOQHMRABVBWPR-UHFFFAOYSA-N Efavirenz Natural products O1C(=O)NC2=CC=C(Cl)C=C2C1(C(F)(F)F)C#CC1CC1 XPOQHMRABVBWPR-UHFFFAOYSA-N 0.000 claims 1
- PXGOKWXKJXAPGV-UHFFFAOYSA-N Fluorine Chemical compound FF PXGOKWXKJXAPGV-UHFFFAOYSA-N 0.000 claims 1
- 108010017544 Glucosylceramidase Proteins 0.000 claims 1
- 102000001554 Hemoglobins Human genes 0.000 claims 1
- 108010054147 Hemoglobins Proteins 0.000 claims 1
- UMWIMNNPYBWNRY-XMMPIXPASA-N [(3s)-1-azabicyclo[2.2.2]octan-3-yl] n-[2-[4-[4-(2-methoxyethoxy)phenyl]phenyl]propan-2-yl]carbamate Chemical compound C1=CC(OCCOC)=CC=C1C1=CC=C(C(C)(C)NC(=O)O[C@H]2C3CCN(CC3)C2)C=C1 UMWIMNNPYBWNRY-XMMPIXPASA-N 0.000 claims 1
- 125000003342 alkenyl group Chemical group 0.000 claims 1
- 125000003302 alkenyloxy group Chemical group 0.000 claims 1
- 125000000304 alkynyl group Chemical group 0.000 claims 1
- 125000005133 alkynyloxy group Chemical group 0.000 claims 1
- 125000003118 aryl group Chemical group 0.000 claims 1
- 239000002585 base Substances 0.000 claims 1
- 230000017531 blood circulation Effects 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 claims 1
- 230000001149 cognitive effect Effects 0.000 claims 1
- 125000001995 cyclobutyl group Chemical group [H]C1([H])C([H])([H])C([H])(*)C1([H])[H] 0.000 claims 1
- 125000001559 cyclopropyl group Chemical group [H]C1([H])C([H])([H])C1([H])* 0.000 claims 1
- XPOQHMRABVBWPR-ZDUSSCGKSA-N efavirenz Chemical compound C([C@]1(C2=CC(Cl)=CC=C2NC(=O)O1)C(F)(F)F)#CC1CC1 XPOQHMRABVBWPR-ZDUSSCGKSA-N 0.000 claims 1
- 229960003804 efavirenz Drugs 0.000 claims 1
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims 1
- 229910052731 fluorine Inorganic materials 0.000 claims 1
- 239000011737 fluorine Substances 0.000 claims 1
- 239000012458 free base Substances 0.000 claims 1
- 210000001652 frontal lobe Anatomy 0.000 claims 1
- 125000001072 heteroaryl group Chemical group 0.000 claims 1
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims 1
- 239000000411 inducer Substances 0.000 claims 1
- 239000003112 inhibitor Substances 0.000 claims 1
- 230000000977 initiatory effect Effects 0.000 claims 1
- 230000002197 limbic effect Effects 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 238000002595 magnetic resonance imaging Methods 0.000 claims 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 238000012544 monitoring process Methods 0.000 claims 1
- 125000000449 nitro group Chemical group [O-][N+](*)=O 0.000 claims 1
- 210000000869 occipital lobe Anatomy 0.000 claims 1
- 230000001936 parietal effect Effects 0.000 claims 1
- 210000001152 parietal lobe Anatomy 0.000 claims 1
- DDBREPKUVSBGFI-UHFFFAOYSA-N phenobarbital Chemical compound C=1C=CC=CC=1C1(CC)C(=O)NC(=O)NC1=O DDBREPKUVSBGFI-UHFFFAOYSA-N 0.000 claims 1
- 229960002695 phenobarbital Drugs 0.000 claims 1
- 125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 0.000 claims 1
- 238000002600 positron emission tomography Methods 0.000 claims 1
- 230000003389 potentiating effect Effects 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims 1
- 230000000284 resting effect Effects 0.000 claims 1
- JQXXHWHPUNPDRT-WLSIYKJHSA-N rifampicin Chemical compound O([C@](C1=O)(C)O/C=C/[C@@H]([C@H]([C@@H](OC(C)=O)[C@H](C)[C@H](O)[C@H](C)[C@@H](O)[C@@H](C)\C=C\C=C(C)/C(=O)NC=2C(O)=C3C([O-])=C4C)C)OC)C4=C1C3=C(O)C=2\C=N\N1CC[NH+](C)CC1 JQXXHWHPUNPDRT-WLSIYKJHSA-N 0.000 claims 1
- 229960001225 rifampicin Drugs 0.000 claims 1
- 210000000952 spleen Anatomy 0.000 claims 1
- 230000002123 temporal effect Effects 0.000 claims 1
- 210000003478 temporal lobe Anatomy 0.000 claims 1
- 230000001225 therapeutic effect Effects 0.000 claims 1
- 238000002560 therapeutic procedure Methods 0.000 claims 1
- 125000000335 thiazolyl group Chemical group 0.000 claims 1
- 230000003442 weekly effect Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025153760A JP2026001009A (ja) | 2020-02-03 | 2025-09-17 | リソソーム蓄積性疾患と関連する神経学的症状を処置するための方法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202062969568P | 2020-02-03 | 2020-02-03 | |
| US62/969,568 | 2020-02-03 | ||
| US202063029154P | 2020-05-22 | 2020-05-22 | |
| US63/029,154 | 2020-05-22 | ||
| US202063072714P | 2020-08-31 | 2020-08-31 | |
| US63/072,714 | 2020-08-31 | ||
| PCT/IB2021/050879 WO2021156769A1 (en) | 2020-02-03 | 2021-02-03 | Methods for treating neurological symptoms associated with lysosomal storage diseases |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025153760A Division JP2026001009A (ja) | 2020-02-03 | 2025-09-17 | リソソーム蓄積性疾患と関連する神経学的症状を処置するための方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023512366A JP2023512366A (ja) | 2023-03-27 |
| JP2023512366A5 JP2023512366A5 (https=) | 2024-02-13 |
| JPWO2021156769A5 true JPWO2021156769A5 (https=) | 2024-02-13 |
Family
ID=74592326
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021564953A Pending JP2023512366A (ja) | 2020-02-03 | 2021-02-03 | リソソーム蓄積性疾患と関連する神経学的症状を処置するための方法 |
| JP2025153760A Pending JP2026001009A (ja) | 2020-02-03 | 2025-09-17 | リソソーム蓄積性疾患と関連する神経学的症状を処置するための方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025153760A Pending JP2026001009A (ja) | 2020-02-03 | 2025-09-17 | リソソーム蓄積性疾患と関連する神経学的症状を処置するための方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US12083115B2 (https=) |
| EP (1) | EP4100009A1 (https=) |
| JP (2) | JP2023512366A (https=) |
| KR (1) | KR20220136887A (https=) |
| CN (1) | CN115427038A (https=) |
| AU (1) | AU2021215396B2 (https=) |
| BR (1) | BR112022014553A2 (https=) |
| CA (1) | CA3166777A1 (https=) |
| IL (1) | IL295255A (https=) |
| MX (1) | MX2022009477A (https=) |
| TW (1) | TW202142236A (https=) |
| WO (1) | WO2021156769A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MA37975B2 (fr) | 2012-09-11 | 2021-03-31 | Genzyme Corp | Inhibiteurs de synthase de glucosylcéramide |
| CN114040762A (zh) | 2019-02-04 | 2022-02-11 | 建新公司 | 使用葡糖神经酰胺合酶(gcs)的抑制剂治疗纤毛疾病 |
| TW202142236A (zh) | 2020-02-03 | 2021-11-16 | 美商健臻公司 | 用於治療與溶體儲積症相關的神經症狀之方法 |
| KR20230043024A (ko) | 2020-07-24 | 2023-03-30 | 젠자임 코포레이션 | 벤글루스타트를 포함하는 제약 조성물 |
| WO2025219952A1 (en) * | 2024-04-19 | 2025-10-23 | Genzyme Corporation | Venglustat for use in methods for reducing disease related biomarker levels in patients with ganglioside storage disorders |
| WO2025219951A1 (en) * | 2024-04-19 | 2025-10-23 | Genzyme Corporation | Venglustat for use in methods for reducing disease related biomarker levels in patients with ganglioside storage disorders |
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| TW201642855A (zh) * | 2015-03-10 | 2016-12-16 | 健臻公司 | 用於治療蛋白質病變之方法 |
| EP3412296B1 (en) * | 2016-02-05 | 2025-11-12 | Kyungpook National University Industry-Academic Cooperation Foundation | Pharmaceutical composition containing stem cell in which vascular endothelial growth factor is overexpressed as effective ingredient for preventing or treating neurodegenerative disease |
| EP3920924A4 (en) * | 2019-02-04 | 2022-11-16 | Prilenia Neurotherapeutics Ltd. | LOW-DOSE PRIDOIDIN FOR THE TREATMENT OF PARKINSON'S DISEASE AND OTHER PARKINSONIC-RELATED DISEASES |
| CN114040762A (zh) | 2019-02-04 | 2022-02-11 | 建新公司 | 使用葡糖神经酰胺合酶(gcs)的抑制剂治疗纤毛疾病 |
| AU2020218496B2 (en) | 2019-02-04 | 2024-12-19 | Genzyme Corporation | Methods for treating symptoms and disorders associated with lysosomal storage diseases |
| TW202142236A (zh) | 2020-02-03 | 2021-11-16 | 美商健臻公司 | 用於治療與溶體儲積症相關的神經症狀之方法 |
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2021
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- 2021-02-03 US US17/166,863 patent/US12083115B2/en active Active
- 2021-02-03 CN CN202180025025.5A patent/CN115427038A/zh active Pending
- 2021-02-03 CA CA3166777A patent/CA3166777A1/en active Pending
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- 2021-02-03 WO PCT/IB2021/050879 patent/WO2021156769A1/en not_active Ceased
- 2021-02-03 JP JP2021564953A patent/JP2023512366A/ja active Pending
- 2021-02-03 AU AU2021215396A patent/AU2021215396B2/en active Active
- 2021-02-03 KR KR1020217038776A patent/KR20220136887A/ko active Pending
- 2021-02-03 EP EP21704944.4A patent/EP4100009A1/en active Pending
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