JPWO2020232297A5 - - Google Patents
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- JPWO2020232297A5 JPWO2020232297A5 JP2021567820A JP2021567820A JPWO2020232297A5 JP WO2020232297 A5 JPWO2020232297 A5 JP WO2020232297A5 JP 2021567820 A JP2021567820 A JP 2021567820A JP 2021567820 A JP2021567820 A JP 2021567820A JP WO2020232297 A5 JPWO2020232297 A5 JP WO2020232297A5
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- 239000013598 vector Substances 0.000 claims description 70
- 150000007523 nucleic acids Chemical class 0.000 claims description 57
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 30
- 108020004707 nucleic acids Proteins 0.000 claims description 27
- 102000039446 nucleic acids Human genes 0.000 claims description 27
- 239000000203 mixture Substances 0.000 claims description 22
- 108091006112 ATPases Proteins 0.000 claims description 19
- 102000057290 Adenosine Triphosphatases Human genes 0.000 claims description 19
- 238000000034 method Methods 0.000 claims description 13
- 102000001435 Synapsin Human genes 0.000 claims description 12
- 108050009621 Synapsin Proteins 0.000 claims description 12
- 201000010099 disease Diseases 0.000 claims description 11
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 11
- 230000001404 mediated effect Effects 0.000 claims description 11
- 230000008488 polyadenylation Effects 0.000 claims description 11
- 208000014644 Brain disease Diseases 0.000 claims description 8
- 208000032274 Encephalopathy Diseases 0.000 claims description 8
- 230000035772 mutation Effects 0.000 claims description 8
- 210000002569 neuron Anatomy 0.000 claims description 8
- 101000753178 Homo sapiens Sodium/potassium-transporting ATPase subunit alpha-3 Proteins 0.000 claims description 5
- 241000829100 Macaca mulatta polyomavirus 1 Species 0.000 claims description 5
- 102100021952 Sodium/potassium-transporting ATPase subunit alpha-3 Human genes 0.000 claims description 5
- 239000008194 pharmaceutical composition Substances 0.000 claims description 5
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 4
- 206010008025 Cerebellar ataxia Diseases 0.000 claims description 4
- 208000011580 syndromic disease Diseases 0.000 claims description 4
- 241000702421 Dependoparvovirus Species 0.000 claims description 3
- 108010006025 bovine growth hormone Proteins 0.000 claims description 3
- 238000002347 injection Methods 0.000 claims description 3
- 239000007924 injection Substances 0.000 claims description 3
- 206010003084 Areflexia Diseases 0.000 claims description 2
- 206010003591 Ataxia Diseases 0.000 claims description 2
- 206010011891 Deafness neurosensory Diseases 0.000 claims description 2
- 208000014094 Dystonic disease Diseases 0.000 claims description 2
- 206010019468 Hemiplegia Diseases 0.000 claims description 2
- 208000027089 Parkinsonian disease Diseases 0.000 claims description 2
- 206010034010 Parkinsonism Diseases 0.000 claims description 2
- 206010037660 Pyrexia Diseases 0.000 claims description 2
- 208000009966 Sensorineural Hearing Loss Diseases 0.000 claims description 2
- 230000005856 abnormality Effects 0.000 claims description 2
- 230000001154 acute effect Effects 0.000 claims description 2
- 208000011916 alternating hemiplegia Diseases 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 208000010118 dystonia Diseases 0.000 claims description 2
- 230000000632 dystonic effect Effects 0.000 claims description 2
- 208000020596 early-onset schizophrenia Diseases 0.000 claims description 2
- 230000001037 epileptic effect Effects 0.000 claims description 2
- 230000001815 facial effect Effects 0.000 claims description 2
- 238000000185 intracerebroventricular administration Methods 0.000 claims description 2
- 208000001749 optic atrophy Diseases 0.000 claims description 2
- 230000001314 paroxysmal effect Effects 0.000 claims description 2
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 2
- 230000000306 recurrent effect Effects 0.000 claims description 2
- 231100000879 sensorineural hearing loss Toxicity 0.000 claims description 2
- 208000023573 sensorineural hearing loss disease Diseases 0.000 claims description 2
- 230000035897 transcription Effects 0.000 claims description 2
- 238000013518 transcription Methods 0.000 claims description 2
- 230000002103 transcriptional effect Effects 0.000 claims description 2
- 239000013603 viral vector Substances 0.000 description 10
- 241000701022 Cytomegalovirus Species 0.000 description 6
- 102000001708 Protein Isoforms Human genes 0.000 description 6
- 108010029485 Protein Isoforms Proteins 0.000 description 6
- 239000012634 fragment Substances 0.000 description 6
- 102000017299 Synapsin-1 Human genes 0.000 description 4
- 108050005241 Synapsin-1 Proteins 0.000 description 4
- 230000002265 prevention Effects 0.000 description 3
- 101710116137 Calcium/calmodulin-dependent protein kinase II Proteins 0.000 description 2
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 description 2
- 101000756632 Homo sapiens Actin, cytoplasmic 1 Proteins 0.000 description 2
- 102000010292 Peptide Elongation Factor 1 Human genes 0.000 description 2
- 108010077524 Peptide Elongation Factor 1 Proteins 0.000 description 2
- 102000012288 Phosphopyruvate Hydratase Human genes 0.000 description 2
- 108010022181 Phosphopyruvate Hydratase Proteins 0.000 description 2
- 108010038512 Platelet-Derived Growth Factor Proteins 0.000 description 2
- 102000010780 Platelet-Derived Growth Factor Human genes 0.000 description 2
- 241000700584 Simplexvirus Species 0.000 description 2
- 102000006601 Thymidine Kinase Human genes 0.000 description 2
- 108020004440 Thymidine kinase Proteins 0.000 description 2
- 102000004243 Tubulin Human genes 0.000 description 2
- 108090000704 Tubulin Proteins 0.000 description 2
- 239000003623 enhancer Substances 0.000 description 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 description 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 description 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 description 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 description 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 description 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 description 1
- 241000649045 Adeno-associated virus 10 Species 0.000 description 1
- 241000649046 Adeno-associated virus 11 Species 0.000 description 1
- 241000649047 Adeno-associated virus 12 Species 0.000 description 1
- 241000300529 Adeno-associated virus 13 Species 0.000 description 1
- 102100029801 Calcium-transporting ATPase type 2C member 1 Human genes 0.000 description 1
- 108010022637 Copper-Transporting ATPases Proteins 0.000 description 1
- 102100027587 Copper-transporting ATPase 1 Human genes 0.000 description 1
- 102100027591 Copper-transporting ATPase 2 Human genes 0.000 description 1
- 208000009889 Herpes Simplex Diseases 0.000 description 1
- 101000728145 Homo sapiens Calcium-transporting ATPase type 2C member 1 Proteins 0.000 description 1
- 101000936280 Homo sapiens Copper-transporting ATPase 2 Proteins 0.000 description 1
- 101000923328 Homo sapiens Phospholipid-transporting ATPase IG Proteins 0.000 description 1
- 101000701334 Homo sapiens Sodium/potassium-transporting ATPase subunit alpha-1 Proteins 0.000 description 1
- 101000753197 Homo sapiens Sodium/potassium-transporting ATPase subunit alpha-2 Proteins 0.000 description 1
- 101000854873 Homo sapiens V-type proton ATPase 116 kDa subunit a 4 Proteins 0.000 description 1
- 101000806601 Homo sapiens V-type proton ATPase catalytic subunit A Proteins 0.000 description 1
- 101000670953 Homo sapiens V-type proton ATPase subunit B, kidney isoform Proteins 0.000 description 1
- 201000005505 Measles Diseases 0.000 description 1
- 208000010359 Newcastle Disease Diseases 0.000 description 1
- 102100032660 Phospholipid-transporting ATPase IG Human genes 0.000 description 1
- 241001144416 Picornavirales Species 0.000 description 1
- 102100030458 Sodium/potassium-transporting ATPase subunit alpha-1 Human genes 0.000 description 1
- 102100021955 Sodium/potassium-transporting ATPase subunit alpha-2 Human genes 0.000 description 1
- 102100020737 V-type proton ATPase 116 kDa subunit a 4 Human genes 0.000 description 1
- 102100037466 V-type proton ATPase catalytic subunit A Human genes 0.000 description 1
- 102100039468 V-type proton ATPase subunit B, kidney isoform Human genes 0.000 description 1
- -1 cationic lipid Chemical class 0.000 description 1
- 239000003814 drug Substances 0.000 description 1
- 238000002513 implantation Methods 0.000 description 1
- 238000001802 infusion Methods 0.000 description 1
- 238000010255 intramuscular injection Methods 0.000 description 1
- 239000007927 intramuscular injection Substances 0.000 description 1
- 150000002632 lipids Chemical class 0.000 description 1
- 230000011987 methylation Effects 0.000 description 1
- 238000007069 methylation reaction Methods 0.000 description 1
- 239000002105 nanoparticle Substances 0.000 description 1
- 239000002773 nucleotide Substances 0.000 description 1
- 125000003729 nucleotide group Chemical group 0.000 description 1
- 229920000642 polymer Polymers 0.000 description 1
- 108090000765 processed proteins & peptides Proteins 0.000 description 1
- 230000001177 retroviral effect Effects 0.000 description 1
- 239000002047 solid lipid nanoparticle Substances 0.000 description 1
- 230000005030 transcription termination Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962847416P | 2019-05-14 | 2019-05-14 | |
| US62/847,416 | 2019-05-14 | ||
| PCT/US2020/032978 WO2020232297A1 (en) | 2019-05-14 | 2020-05-14 | Compositions and methods for the treatment of atpase-mediated diseases |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022531809A JP2022531809A (ja) | 2022-07-11 |
| JPWO2020232297A5 true JPWO2020232297A5 (https=) | 2023-05-16 |
| JP2022531809A5 JP2022531809A5 (https=) | 2023-05-16 |
Family
ID=73289760
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021567820A Pending JP2022531809A (ja) | 2019-05-14 | 2020-05-14 | ＡＴＰａｓｅ媒介性疾患の処置のための組成物および方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US11717560B2 (https=) |
| EP (1) | EP3969597A4 (https=) |
| JP (1) | JP2022531809A (https=) |
| KR (1) | KR20220032005A (https=) |
| CN (1) | CN114072514A (https=) |
| AU (1) | AU2020274200A1 (https=) |
| BR (1) | BR112021022951A2 (https=) |
| CA (1) | CA3140049A1 (https=) |
| IL (1) | IL287796A (https=) |
| MX (1) | MX2021013913A (https=) |
| SG (1) | SG11202112078TA (https=) |
| WO (1) | WO2020232297A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3094465A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| US20210363191A1 (en) | 2018-04-03 | 2021-11-25 | Stridebio, Inc. | Antibody-evading virus vectors |
| BR112021018776A2 (pt) | 2019-03-21 | 2021-11-30 | Stridebio Inc | Vetores de vírus adenoassociado recombinante |
| CN114072514A (zh) * | 2019-05-14 | 2022-02-18 | 杜克大学 | 用于治疗atp酶介导的疾病的组合物和方法 |
| KR20220083714A (ko) | 2019-10-17 | 2022-06-20 | 스트라이드바이오 인코포레이티드 | 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터 |
| EP4200408A1 (en) | 2020-08-19 | 2023-06-28 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of rett syndrome |
| US20250144239A1 (en) * | 2021-10-18 | 2025-05-08 | Logicbio Therapeutics, Inc. | Gene therapy for the treatment of wilson's disease |
| WO2023230466A1 (en) * | 2022-05-23 | 2023-11-30 | Lutsenka Svetlana | Compositions and methods for treating wilson's disease |
| CN115851730A (zh) * | 2022-11-16 | 2023-03-28 | 泰达国际心血管病医院 | Myh6基因启动子变异及其应用与先天性心脏病基因检测试剂盒 |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2001046226A2 (en) * | 1999-12-22 | 2001-06-28 | Aventis Pasteur Limited | Chlamydia antigens and corresponding dna fragments and uses thereof |
| US7473531B1 (en) * | 2003-08-08 | 2009-01-06 | Colora Corporation | Pancreatic cancer targets and uses thereof |
| US20100095387A1 (en) * | 2005-07-15 | 2010-04-15 | Martin Smith | Methods and reagents for screening new drugs and for treating ion pump associated disorders and diseases |
| WO2010144611A2 (en) * | 2009-06-10 | 2010-12-16 | 3-V Biosciences, Inc. | Antivirals that target transporters, carriers, and ion channels |
| JP2014523884A (ja) * | 2011-06-24 | 2014-09-18 | セントローズ， エルエルシー | 細胞外標的化薬物複合体 |
| WO2014007868A2 (en) | 2012-03-15 | 2014-01-09 | The Regents Of The University Of California | Low temperature sulfur and sodium metal battery for grid-scale energy storage application |
| US20140350087A9 (en) * | 2012-03-22 | 2014-11-27 | Halozyme, Inc. | Oncovector Nucleic Acid Molecules and Methods of Use |
| AU2013243948A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| BR112015000161A2 (pt) * | 2012-07-06 | 2017-06-27 | Univ Iowa Res Found | composições de vetor de vírus adeno-associado modificado |
| KR102530118B1 (ko) * | 2012-07-25 | 2023-05-08 | 더 브로드 인스티튜트, 인코퍼레이티드 | 유도 ｄｎａ 결합 단백질 및 게놈 교란 도구 및 이의 적용 |
| ES2693465T3 (es) * | 2014-06-06 | 2018-12-11 | Uroimmun Medizinische Labordiagnostika Ag | Diagnóstico de una enfermedad neurológica |
| ES2876412T3 (es) * | 2014-12-17 | 2021-11-12 | Fundacion Para La Investig Medica Aplicada | Construcciones de ácido nucleico y vectores de terapia génica para su uso en el tratamiento de la enfermedad de Wilson |
| WO2017013648A1 (en) * | 2015-07-19 | 2017-01-26 | Yeda Research And Development Co. Ltd. | SELECTIVE INHIBITORS OF Alpha2-CONTAINING ISOFORMS OF Na,K-ATPase AND USE THEREOF FOR REDUCTION OF INTRAOCULAR PRESSURE |
| JP7049249B2 (ja) * | 2015-12-14 | 2022-04-06 | コールド スプリング ハーバー ラボラトリー | 中枢神経系疾患の処置のための組成物および方法 |
| JP2020534788A (ja) * | 2017-08-28 | 2020-12-03 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | アデノ随伴ウイルスカプシド変異体及びその使用方法 |
| WO2019199841A1 (en) * | 2018-04-09 | 2019-10-17 | Cure Ahc, Inc. | Aav-mediated delivery of atp1a3 genes to central nervous system |
| KR20210112339A (ko) * | 2019-01-04 | 2021-09-14 | 울트라제닉스 파마수티컬 인코포레이티드 | 윌슨병을 치료하기 위한 유전자 요법 구축물 |
| CN114072514A (zh) * | 2019-05-14 | 2022-02-18 | 杜克大学 | 用于治疗atp酶介导的疾病的组合物和方法 |
-
2020
- 2020-05-14 CN CN202080047216.7A patent/CN114072514A/zh active Pending
- 2020-05-14 CA CA3140049A patent/CA3140049A1/en active Pending
- 2020-05-14 AU AU2020274200A patent/AU2020274200A1/en not_active Abandoned
- 2020-05-14 BR BR112021022951A patent/BR112021022951A2/pt not_active Application Discontinuation
- 2020-05-14 WO PCT/US2020/032978 patent/WO2020232297A1/en not_active Ceased
- 2020-05-14 JP JP2021567820A patent/JP2022531809A/ja active Pending
- 2020-05-14 MX MX2021013913A patent/MX2021013913A/es unknown
- 2020-05-14 KR KR1020217040927A patent/KR20220032005A/ko not_active Withdrawn
- 2020-05-14 SG SG11202112078TA patent/SG11202112078TA/en unknown
- 2020-05-14 EP EP20805008.8A patent/EP3969597A4/en active Pending
-
2021
- 2021-11-02 IL IL287796A patent/IL287796A/en unknown
- 2021-11-11 US US17/524,466 patent/US11717560B2/en active Active
-
2023
- 2023-06-23 US US18/340,028 patent/US12533397B2/en active Active
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