JPWO2020176614A5 - - Google Patents
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- JPWO2020176614A5 JPWO2020176614A5 JP2021549782A JP2021549782A JPWO2020176614A5 JP WO2020176614 A5 JPWO2020176614 A5 JP WO2020176614A5 JP 2021549782 A JP2021549782 A JP 2021549782A JP 2021549782 A JP2021549782 A JP 2021549782A JP WO2020176614 A5 JPWO2020176614 A5 JP WO2020176614A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- raav
- subject
- sarcoglycan
- seq
- 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
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- 238000000034 method Methods 0.000 claims description 65
- 108010083379 Sarcoglycans Proteins 0.000 claims description 58
- 102000006308 Sarcoglycans Human genes 0.000 claims description 56
- 239000000203 mixture Substances 0.000 claims description 51
- 210000004027 cell Anatomy 0.000 claims description 33
- 239000002773 nucleotide Substances 0.000 claims description 24
- 125000003729 nucleotide group Chemical group 0.000 claims description 24
- 239000013612 plasmid Substances 0.000 claims description 19
- 230000014509 gene expression Effects 0.000 claims description 12
- 102000004420 Creatine Kinase Human genes 0.000 claims description 11
- 108010042126 Creatine kinase Proteins 0.000 claims description 11
- RVGRUAULSDPKGF-UHFFFAOYSA-N Poloxamer Chemical compound C1CO1.CC1CO1 RVGRUAULSDPKGF-UHFFFAOYSA-N 0.000 claims description 11
- 201000006938 muscular dystrophy Diseases 0.000 claims description 10
- 241000702421 Dependoparvovirus Species 0.000 claims description 9
- 108090000623 proteins and genes Proteins 0.000 claims description 8
- 210000002966 serum Anatomy 0.000 claims description 8
- 239000003795 chemical substances by application Substances 0.000 claims description 7
- 239000002157 polynucleotide Substances 0.000 claims description 7
- 102000040430 polynucleotide Human genes 0.000 claims description 7
- 108091033319 polynucleotide Proteins 0.000 claims description 7
- 210000003205 muscle Anatomy 0.000 claims description 6
- 239000004094 surface-active agent Substances 0.000 claims description 6
- 229920001983 poloxamer Polymers 0.000 claims description 5
- 102100030686 Beta-sarcoglycan Human genes 0.000 claims description 4
- 210000000170 cell membrane Anatomy 0.000 claims description 4
- 238000001802 infusion Methods 0.000 claims description 4
- 229960000502 poloxamer Drugs 0.000 claims description 4
- 102100021790 Delta-sarcoglycan Human genes 0.000 claims description 3
- 102100021792 Gamma-sarcoglycan Human genes 0.000 claims description 3
- 101000616408 Homo sapiens Delta-sarcoglycan Proteins 0.000 claims description 3
- 201000009342 Limb-girdle muscular dystrophy Diseases 0.000 claims description 3
- 238000001990 intravenous administration Methods 0.000 claims description 3
- 229920001993 poloxamer 188 Polymers 0.000 claims description 3
- 229940044519 poloxamer 188 Drugs 0.000 claims description 3
- 208000034067 Beta-sarcoglycan-related limb-girdle muscular dystrophy R4 Diseases 0.000 claims description 2
- 241000255925 Diptera Species 0.000 claims description 2
- 241000238631 Hexapoda Species 0.000 claims description 2
- 101000703495 Homo sapiens Beta-sarcoglycan Proteins 0.000 claims description 2
- 229920002507 Poloxamer 124 Polymers 0.000 claims description 2
- 229920002508 Poloxamer 181 Polymers 0.000 claims description 2
- 229920002511 Poloxamer 237 Polymers 0.000 claims description 2
- 229920002516 Poloxamer 331 Polymers 0.000 claims description 2
- 229920002517 Poloxamer 338 Polymers 0.000 claims description 2
- 241000700605 Viruses Species 0.000 claims description 2
- 239000000872 buffer Substances 0.000 claims description 2
- 238000002513 implantation Methods 0.000 claims description 2
- 238000002347 injection Methods 0.000 claims description 2
- 239000007924 injection Substances 0.000 claims description 2
- 230000004807 localization Effects 0.000 claims description 2
- 210000004962 mammalian cell Anatomy 0.000 claims description 2
- 238000004806 packaging method and process Methods 0.000 claims description 2
- 229940093448 poloxamer 124 Drugs 0.000 claims description 2
- 229940085692 poloxamer 181 Drugs 0.000 claims description 2
- -1 poloxamer 184 Polymers 0.000 claims description 2
- 229940116406 poloxamer 184 Drugs 0.000 claims description 2
- 229920001992 poloxamer 407 Polymers 0.000 claims description 2
- 229940044476 poloxamer 407 Drugs 0.000 claims description 2
- 230000009885 systemic effect Effects 0.000 claims description 2
- 241000255789 Bombyx mori Species 0.000 claims 1
- 230000001580 bacterial effect Effects 0.000 claims 1
- 238000011002 quantification Methods 0.000 claims 1
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 10
- 239000000835 fiber Substances 0.000 description 6
- 230000007659 motor function Effects 0.000 description 5
- 239000011780 sodium chloride Substances 0.000 description 5
- 238000001964 muscle biopsy Methods 0.000 description 4
- NLXLAEXVIDQMFP-UHFFFAOYSA-N Ammonia chloride Chemical compound [NH4+].[Cl-] NLXLAEXVIDQMFP-UHFFFAOYSA-N 0.000 description 3
- TWRXJAOTZQYOKJ-UHFFFAOYSA-L Magnesium chloride Chemical compound [Mg+2].[Cl-].[Cl-] TWRXJAOTZQYOKJ-UHFFFAOYSA-L 0.000 description 3
- WCUXLLCKKVVCTQ-UHFFFAOYSA-M Potassium chloride Chemical group [Cl-].[K+] WCUXLLCKKVVCTQ-UHFFFAOYSA-M 0.000 description 3
- 239000007983 Tris buffer Substances 0.000 description 3
- 239000006172 buffering agent Substances 0.000 description 3
- 230000003247 decreasing effect Effects 0.000 description 3
- LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 description 3
- CSNNHWWHGAXBCP-UHFFFAOYSA-L Magnesium sulfate Chemical compound [Mg+2].[O-][S+2]([O-])([O-])[O-] CSNNHWWHGAXBCP-UHFFFAOYSA-L 0.000 description 2
- 229910021380 Manganese Chloride Inorganic materials 0.000 description 2
- GLFNIEUTAYBVOC-UHFFFAOYSA-L Manganese chloride Chemical compound Cl[Mn]Cl GLFNIEUTAYBVOC-UHFFFAOYSA-L 0.000 description 2
- SEQKRHFRPICQDD-UHFFFAOYSA-N N-tris(hydroxymethyl)methylglycine Chemical compound OCC(CO)(CO)[NH2+]CC([O-])=O SEQKRHFRPICQDD-UHFFFAOYSA-N 0.000 description 2
- 239000007997 Tricine buffer Substances 0.000 description 2
- 235000019270 ammonium chloride Nutrition 0.000 description 2
- 238000003364 immunohistochemistry Methods 0.000 description 2
- 235000002867 manganese chloride Nutrition 0.000 description 2
- 239000011565 manganese chloride Substances 0.000 description 2
- 230000035772 mutation Effects 0.000 description 2
- 239000008194 pharmaceutical composition Substances 0.000 description 2
- SCVFZCLFOSHCOH-UHFFFAOYSA-M potassium acetate Chemical compound [K+].CC([O-])=O SCVFZCLFOSHCOH-UHFFFAOYSA-M 0.000 description 2
- 238000012360 testing method Methods 0.000 description 2
- 238000001262 western blot Methods 0.000 description 2
- IHPYMWDTONKSCO-UHFFFAOYSA-N 2,2'-piperazine-1,4-diylbisethanesulfonic acid Chemical compound OS(=O)(=O)CCN1CCN(CCS(O)(=O)=O)CC1 IHPYMWDTONKSCO-UHFFFAOYSA-N 0.000 description 1
- JKMHFZQWWAIEOD-UHFFFAOYSA-N 2-[4-(2-hydroxyethyl)piperazin-1-yl]ethanesulfonic acid Chemical compound OCC[NH+]1CCN(CCS([O-])(=O)=O)CC1 JKMHFZQWWAIEOD-UHFFFAOYSA-N 0.000 description 1
- DVLFYONBTKHTER-UHFFFAOYSA-N 3-(N-morpholino)propanesulfonic acid Chemical compound OS(=O)(=O)CCCN1CCOCC1 DVLFYONBTKHTER-UHFFFAOYSA-N 0.000 description 1
- 108700028369 Alleles Proteins 0.000 description 1
- USFZMSVCRYTOJT-UHFFFAOYSA-N Ammonium acetate Chemical compound N.CC(O)=O USFZMSVCRYTOJT-UHFFFAOYSA-N 0.000 description 1
- 239000005695 Ammonium acetate Substances 0.000 description 1
- 239000007995 HEPES buffer Substances 0.000 description 1
- 239000007993 MOPS buffer Substances 0.000 description 1
- JOCBASBOOFNAJA-UHFFFAOYSA-N N-tris(hydroxymethyl)methyl-2-aminoethanesulfonic acid Chemical compound OCC(CO)(CO)NCCS(O)(=O)=O JOCBASBOOFNAJA-UHFFFAOYSA-N 0.000 description 1
- 239000007990 PIPES buffer Substances 0.000 description 1
- 229910019142 PO4 Inorganic materials 0.000 description 1
- ABLZXFCXXLZCGV-UHFFFAOYSA-N Phosphorous acid Chemical class OP(O)=O ABLZXFCXXLZCGV-UHFFFAOYSA-N 0.000 description 1
- VMHLLURERBWHNL-UHFFFAOYSA-M Sodium acetate Chemical compound [Na+].CC([O-])=O VMHLLURERBWHNL-UHFFFAOYSA-M 0.000 description 1
- UZMAPBJVXOGOFT-UHFFFAOYSA-N Syringetin Natural products COC1=C(O)C(OC)=CC(C2=C(C(=O)C3=C(O)C=C(O)C=C3O2)O)=C1 UZMAPBJVXOGOFT-UHFFFAOYSA-N 0.000 description 1
- 239000007994 TES buffer Substances 0.000 description 1
- 208000034953 Twin anemia-polycythemia sequence Diseases 0.000 description 1
- 229940043376 ammonium acetate Drugs 0.000 description 1
- 235000019257 ammonium acetate Nutrition 0.000 description 1
- BFNBIHQBYMNNAN-UHFFFAOYSA-N ammonium sulfate Chemical compound N.N.OS(O)(=O)=O BFNBIHQBYMNNAN-UHFFFAOYSA-N 0.000 description 1
- 229910052921 ammonium sulfate Inorganic materials 0.000 description 1
- 235000011130 ammonium sulphate Nutrition 0.000 description 1
- 239000003093 cationic surfactant Substances 0.000 description 1
- KCFYHBSOLOXZIF-UHFFFAOYSA-N dihydrochrysin Natural products COC1=C(O)C(OC)=CC(C2OC3=CC(O)=CC(O)=C3C(=O)C2)=C1 KCFYHBSOLOXZIF-UHFFFAOYSA-N 0.000 description 1
- XIXADJRWDQXREU-UHFFFAOYSA-M lithium acetate Chemical compound [Li+].CC([O-])=O XIXADJRWDQXREU-UHFFFAOYSA-M 0.000 description 1
- KWGKDLIKAYFUFQ-UHFFFAOYSA-M lithium chloride Chemical compound [Li+].[Cl-] KWGKDLIKAYFUFQ-UHFFFAOYSA-M 0.000 description 1
- UEGPKNKPLBYCNK-UHFFFAOYSA-L magnesium acetate Chemical compound [Mg+2].CC([O-])=O.CC([O-])=O UEGPKNKPLBYCNK-UHFFFAOYSA-L 0.000 description 1
- 235000011285 magnesium acetate Nutrition 0.000 description 1
- 239000011654 magnesium acetate Substances 0.000 description 1
- 229940069446 magnesium acetate Drugs 0.000 description 1
- 229910001629 magnesium chloride Inorganic materials 0.000 description 1
- 229910052943 magnesium sulfate Inorganic materials 0.000 description 1
- 229960003390 magnesium sulfate Drugs 0.000 description 1
- 235000019341 magnesium sulphate Nutrition 0.000 description 1
- 229940071125 manganese acetate Drugs 0.000 description 1
- 229940099607 manganese chloride Drugs 0.000 description 1
- 229940099596 manganese sulfate Drugs 0.000 description 1
- 235000007079 manganese sulphate Nutrition 0.000 description 1
- 239000011702 manganese sulphate Substances 0.000 description 1
- UOGMEBQRZBEZQT-UHFFFAOYSA-L manganese(2+);diacetate Chemical compound [Mn+2].CC([O-])=O.CC([O-])=O UOGMEBQRZBEZQT-UHFFFAOYSA-L 0.000 description 1
- SQQMAOCOWKFBNP-UHFFFAOYSA-L manganese(II) sulfate Chemical compound [Mn+2].[O-]S([O-])(=O)=O SQQMAOCOWKFBNP-UHFFFAOYSA-L 0.000 description 1
- 239000012528 membrane Substances 0.000 description 1
- 230000004220 muscle function Effects 0.000 description 1
- 210000001087 myotubule Anatomy 0.000 description 1
- 230000002093 peripheral effect Effects 0.000 description 1
- 235000021317 phosphate Nutrition 0.000 description 1
- 150000003013 phosphoric acid derivatives Chemical class 0.000 description 1
- 235000011056 potassium acetate Nutrition 0.000 description 1
- 239000001103 potassium chloride Substances 0.000 description 1
- 235000011164 potassium chloride Nutrition 0.000 description 1
- OTYBMLCTZGSZBG-UHFFFAOYSA-L potassium sulfate Chemical compound [K+].[K+].[O-]S([O-])(=O)=O OTYBMLCTZGSZBG-UHFFFAOYSA-L 0.000 description 1
- 229910052939 potassium sulfate Inorganic materials 0.000 description 1
- 235000011151 potassium sulphates Nutrition 0.000 description 1
- 239000001632 sodium acetate Substances 0.000 description 1
- 235000017281 sodium acetate Nutrition 0.000 description 1
- 229960002668 sodium chloride Drugs 0.000 description 1
- 150000003871 sulfonates Chemical class 0.000 description 1
- 150000003467 sulfuric acid derivatives Chemical class 0.000 description 1
- 210000003462 vein Anatomy 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024109621A JP2024133657A (ja) | 2019-02-26 | 2024-07-08 | Β－サルコグリカンのアデノ随伴ウイルスベクター送達および筋ジストロフィーの治療 |
| JP2025139290A JP2025170362A (ja) | 2019-02-26 | 2025-08-22 | Β－サルコグリカンのアデノ随伴ウイルスベクター送達および筋ジストロフィーの治療 |
Applications Claiming Priority (13)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962810917P | 2019-02-26 | 2019-02-26 | |
| US62/810,917 | 2019-02-26 | ||
| US201962834012P | 2019-04-15 | 2019-04-15 | |
| US62/834,012 | 2019-04-15 | ||
| US201962858644P | 2019-06-07 | 2019-06-07 | |
| US62/858,644 | 2019-06-07 | ||
| US201962881901P | 2019-08-01 | 2019-08-01 | |
| US62/881,901 | 2019-08-01 | ||
| US201962909564P | 2019-10-02 | 2019-10-02 | |
| US62/909,564 | 2019-10-02 | ||
| US201962910779P | 2019-10-04 | 2019-10-04 | |
| US62/910,779 | 2019-10-04 | ||
| PCT/US2020/019892 WO2020176614A1 (en) | 2019-02-26 | 2020-02-26 | Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024109621A Division JP2024133657A (ja) | 2019-02-26 | 2024-07-08 | Β－サルコグリカンのアデノ随伴ウイルスベクター送達および筋ジストロフィーの治療 |
| JP2025139290A Division JP2025170362A (ja) | 2019-02-26 | 2025-08-22 | Β－サルコグリカンのアデノ随伴ウイルスベクター送達および筋ジストロフィーの治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022521776A JP2022521776A (ja) | 2022-04-12 |
| JPWO2020176614A5 true JPWO2020176614A5 (https=) | 2023-03-06 |
| JP2022521776A5 JP2022521776A5 (https=) | 2023-03-06 |
Family
ID=70005777
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021549782A Pending JP2022521776A (ja) | 2019-02-26 | 2020-02-26 | Β－サルコグリカンのアデノ随伴ウイルスベクター送達および筋ジストロフィーの治療 |
| JP2024109621A Pending JP2024133657A (ja) | 2019-02-26 | 2024-07-08 | Β－サルコグリカンのアデノ随伴ウイルスベクター送達および筋ジストロフィーの治療 |
| JP2025139290A Pending JP2025170362A (ja) | 2019-02-26 | 2025-08-22 | Β－サルコグリカンのアデノ随伴ウイルスベクター送達および筋ジストロフィーの治療 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024109621A Pending JP2024133657A (ja) | 2019-02-26 | 2024-07-08 | Β－サルコグリカンのアデノ随伴ウイルスベクター送達および筋ジストロフィーの治療 |
| JP2025139290A Pending JP2025170362A (ja) | 2019-02-26 | 2025-08-22 | Β－サルコグリカンのアデノ随伴ウイルスベクター送達および筋ジストロフィーの治療 |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US12377170B2 (https=) |
| EP (1) | EP3930765A1 (https=) |
| JP (3) | JP2022521776A (https=) |
| KR (1) | KR20210133242A (https=) |
| CN (1) | CN113766935A (https=) |
| AU (1) | AU2020229340A1 (https=) |
| BR (1) | BR112021016570A2 (https=) |
| CA (1) | CA3131390A1 (https=) |
| CL (1) | CL2021002236A1 (https=) |
| CO (1) | CO2021012074A2 (https=) |
| IL (1) | IL285849A (https=) |
| MX (1) | MX2021010356A (https=) |
| SG (1) | SG11202109113TA (https=) |
| TW (1) | TW202045531A (https=) |
| WO (1) | WO2020176614A1 (https=) |
| ZA (1) | ZA202106017B (https=) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3005633C (en) | 2015-11-16 | 2023-11-21 | Research Institute Of Nationwide Children's Hospital | Materials and methods for treatment of titin-based myopathies and other titinopathies |
| WO2019012336A2 (en) | 2017-03-17 | 2019-01-17 | Newcastle University | ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY |
| MX2020007876A (es) | 2018-01-31 | 2020-12-03 | Res Inst Nationwide Childrens Hospital | Terapia genica para la distrofia muscular de cinturas tipo 2c. |
| CN120393053A (zh) | 2018-06-18 | 2025-08-01 | 全国儿童医院研究所 | 用于治疗肌营养不良的肌肉特异性微小肌营养不良蛋白的腺相关病毒载体递送 |
| MX2020014119A (es) | 2018-06-29 | 2021-06-18 | Res Inst Nationwide Childrens Hospital | Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a. |
| PT4017871T (pt) | 2019-08-21 | 2024-04-22 | Res Inst Nationwide Childrens Hospital | Distribuição de vetor de vírus adeno-associado de alfasarcoglicano e o tratamento da distrofia muscular |
| EP4164668A1 (en) | 2020-06-15 | 2023-04-19 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery for muscular dystrophies |
| TW202541851A (zh) * | 2020-09-08 | 2025-11-01 | 美商薩羅塔治療公司 | 表現γ-肌聚醣之腺相關病毒載體之全身性遞送及肌肉失養症之治療 |
| JP2023059858A (ja) | 2021-10-15 | 2023-04-27 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用 |
| EP4198048A1 (en) | 2021-12-16 | 2023-06-21 | Genethon | Calpain-3 gene transfer increase using modified itr sequences |
| TWI889013B (zh) * | 2023-11-17 | 2025-07-01 | 矽品精密工業股份有限公司 | 電子封裝件及其製法 |
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