JP2024133657A5 - - Google Patents
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- JP2024133657A5 JP2024133657A5 JP2024109621A JP2024109621A JP2024133657A5 JP 2024133657 A5 JP2024133657 A5 JP 2024133657A5 JP 2024109621 A JP2024109621 A JP 2024109621A JP 2024109621 A JP2024109621 A JP 2024109621A JP 2024133657 A5 JP2024133657 A5 JP 2024133657A5
- Authority
- JP
- Japan
- Prior art keywords
- cell
- aav
- poloxamer
- composition
- raav
- 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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- 210000004027 cell Anatomy 0.000 claims 39
- 239000000203 mixture Substances 0.000 claims 15
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims 12
- RVGRUAULSDPKGF-UHFFFAOYSA-N Poloxamer Chemical compound C1CO1.CC1CO1 RVGRUAULSDPKGF-UHFFFAOYSA-N 0.000 claims 8
- 239000002157 polynucleotide Substances 0.000 claims 8
- 102000040430 polynucleotide Human genes 0.000 claims 8
- 108091033319 polynucleotide Proteins 0.000 claims 8
- 241000702421 Dependoparvovirus Species 0.000 claims 6
- 239000003795 chemical substances by application Substances 0.000 claims 6
- 239000011780 sodium chloride Substances 0.000 claims 6
- 239000002773 nucleotide Substances 0.000 claims 5
- 125000003729 nucleotide group Chemical group 0.000 claims 5
- 239000007983 Tris buffer Substances 0.000 claims 4
- 238000000034 method Methods 0.000 claims 4
- 239000004094 surface-active agent Substances 0.000 claims 4
- LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 claims 4
- TWRXJAOTZQYOKJ-UHFFFAOYSA-L Magnesium chloride Chemical compound [Mg+2].[Cl-].[Cl-] TWRXJAOTZQYOKJ-UHFFFAOYSA-L 0.000 claims 3
- WCUXLLCKKVVCTQ-UHFFFAOYSA-M Potassium chloride Chemical compound [Cl-].[K+] WCUXLLCKKVVCTQ-UHFFFAOYSA-M 0.000 claims 3
- 239000000872 buffer Substances 0.000 claims 3
- 239000013612 plasmid Substances 0.000 claims 3
- 229920001983 poloxamer Polymers 0.000 claims 3
- 229960000502 poloxamer Drugs 0.000 claims 3
- 229920001993 poloxamer 188 Polymers 0.000 claims 3
- 229940044519 poloxamer 188 Drugs 0.000 claims 3
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 2
- NLXLAEXVIDQMFP-UHFFFAOYSA-N Ammonia chloride Chemical compound [NH4+].[Cl-] NLXLAEXVIDQMFP-UHFFFAOYSA-N 0.000 claims 2
- 241000255789 Bombyx mori Species 0.000 claims 2
- 241000255925 Diptera Species 0.000 claims 2
- 241000238631 Hexapoda Species 0.000 claims 2
- CSNNHWWHGAXBCP-UHFFFAOYSA-L Magnesium sulfate Chemical compound [Mg+2].[O-][S+2]([O-])([O-])[O-] CSNNHWWHGAXBCP-UHFFFAOYSA-L 0.000 claims 2
- SEQKRHFRPICQDD-UHFFFAOYSA-N N-tris(hydroxymethyl)methylglycine Chemical compound OCC(CO)(CO)[NH2+]CC([O-])=O SEQKRHFRPICQDD-UHFFFAOYSA-N 0.000 claims 2
- 239000007997 Tricine buffer Substances 0.000 claims 2
- 230000001580 bacterial effect Effects 0.000 claims 2
- 210000004962 mammalian cell Anatomy 0.000 claims 2
- SCVFZCLFOSHCOH-UHFFFAOYSA-M potassium acetate Chemical compound [K+].CC([O-])=O SCVFZCLFOSHCOH-UHFFFAOYSA-M 0.000 claims 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 claims 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 claims 1
- DVLFYONBTKHTER-UHFFFAOYSA-N 3-(N-morpholino)propanesulfonic acid Chemical compound OS(=O)(=O)CCCN1CCOCC1 DVLFYONBTKHTER-UHFFFAOYSA-N 0.000 claims 1
- USFZMSVCRYTOJT-UHFFFAOYSA-N Ammonium acetate Chemical compound N.CC(O)=O USFZMSVCRYTOJT-UHFFFAOYSA-N 0.000 claims 1
- 239000005695 Ammonium acetate Substances 0.000 claims 1
- 101150044789 Cap gene Proteins 0.000 claims 1
- 108090000565 Capsid Proteins Proteins 0.000 claims 1
- 102100023321 Ceruloplasmin Human genes 0.000 claims 1
- 239000007995 HEPES buffer Substances 0.000 claims 1
- 239000007993 MOPS buffer Substances 0.000 claims 1
- 229910021380 Manganese Chloride Inorganic materials 0.000 claims 1
- GLFNIEUTAYBVOC-UHFFFAOYSA-L Manganese chloride Chemical compound Cl[Mn]Cl GLFNIEUTAYBVOC-UHFFFAOYSA-L 0.000 claims 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 claims 1
- 239000007990 PIPES buffer Substances 0.000 claims 1
- 229910019142 PO4 Inorganic materials 0.000 claims 1
- 229920002507 Poloxamer 124 Polymers 0.000 claims 1
- 229920002508 Poloxamer 181 Polymers 0.000 claims 1
- 229920002511 Poloxamer 237 Polymers 0.000 claims 1
- 229920002516 Poloxamer 331 Polymers 0.000 claims 1
- 229920002517 Poloxamer 338 Polymers 0.000 claims 1
- VMHLLURERBWHNL-UHFFFAOYSA-M Sodium acetate Chemical compound [Na+].CC([O-])=O VMHLLURERBWHNL-UHFFFAOYSA-M 0.000 claims 1
- QAOWNCQODCNURD-UHFFFAOYSA-L Sulfate Chemical compound [O-]S([O-])(=O)=O QAOWNCQODCNURD-UHFFFAOYSA-L 0.000 claims 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 claims 1
- 239000007994 TES buffer Substances 0.000 claims 1
- 208000034953 Twin anemia-polycythemia sequence Diseases 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 229940043376 ammonium acetate Drugs 0.000 claims 1
- 235000019257 ammonium acetate Nutrition 0.000 claims 1
- 235000019270 ammonium chloride Nutrition 0.000 claims 1
- BFNBIHQBYMNNAN-UHFFFAOYSA-N ammonium sulfate Chemical compound N.N.OS(O)(=O)=O BFNBIHQBYMNNAN-UHFFFAOYSA-N 0.000 claims 1
- 229910052921 ammonium sulfate Inorganic materials 0.000 claims 1
- 235000011130 ammonium sulphate Nutrition 0.000 claims 1
- 239000006172 buffering agent Substances 0.000 claims 1
- 239000003093 cationic surfactant Substances 0.000 claims 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 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- XIXADJRWDQXREU-UHFFFAOYSA-M lithium acetate Chemical compound [Li+].CC([O-])=O XIXADJRWDQXREU-UHFFFAOYSA-M 0.000 claims 1
- KWGKDLIKAYFUFQ-UHFFFAOYSA-M lithium chloride Chemical compound [Li+].[Cl-] KWGKDLIKAYFUFQ-UHFFFAOYSA-M 0.000 claims 1
- UEGPKNKPLBYCNK-UHFFFAOYSA-L magnesium acetate Chemical compound [Mg+2].CC([O-])=O.CC([O-])=O UEGPKNKPLBYCNK-UHFFFAOYSA-L 0.000 claims 1
- 239000011654 magnesium acetate Substances 0.000 claims 1
- 235000011285 magnesium acetate Nutrition 0.000 claims 1
- 229940069446 magnesium acetate Drugs 0.000 claims 1
- 229910001629 magnesium chloride Inorganic materials 0.000 claims 1
- 229910052943 magnesium sulfate Inorganic materials 0.000 claims 1
- 229960003390 magnesium sulfate Drugs 0.000 claims 1
- 235000019341 magnesium sulphate Nutrition 0.000 claims 1
- 229940071125 manganese acetate Drugs 0.000 claims 1
- 239000011565 manganese chloride Substances 0.000 claims 1
- 235000002867 manganese chloride Nutrition 0.000 claims 1
- 229940099607 manganese chloride Drugs 0.000 claims 1
- 229940099596 manganese sulfate Drugs 0.000 claims 1
- 239000011702 manganese sulphate Substances 0.000 claims 1
- 235000007079 manganese sulphate Nutrition 0.000 claims 1
- UOGMEBQRZBEZQT-UHFFFAOYSA-L manganese(2+);diacetate Chemical compound [Mn+2].CC([O-])=O.CC([O-])=O UOGMEBQRZBEZQT-UHFFFAOYSA-L 0.000 claims 1
- SQQMAOCOWKFBNP-UHFFFAOYSA-L manganese(II) sulfate Chemical compound [Mn+2].[O-]S([O-])(=O)=O SQQMAOCOWKFBNP-UHFFFAOYSA-L 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000004806 packaging method and process Methods 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
- UEZVMMHDMIWARA-UHFFFAOYSA-M phosphonate Chemical compound [O-]P(=O)=O UEZVMMHDMIWARA-UHFFFAOYSA-M 0.000 claims 1
- 229940093448 poloxamer 124 Drugs 0.000 claims 1
- 229940085692 poloxamer 181 Drugs 0.000 claims 1
- -1 poloxamer 184 Polymers 0.000 claims 1
- 229940116406 poloxamer 184 Drugs 0.000 claims 1
- 229920001992 poloxamer 407 Polymers 0.000 claims 1
- 229940044476 poloxamer 407 Drugs 0.000 claims 1
- 235000011056 potassium acetate Nutrition 0.000 claims 1
- 239000001103 potassium chloride Substances 0.000 claims 1
- 235000011164 potassium chloride Nutrition 0.000 claims 1
- OTYBMLCTZGSZBG-UHFFFAOYSA-L potassium sulfate Chemical compound [K+].[K+].[O-]S([O-])(=O)=O OTYBMLCTZGSZBG-UHFFFAOYSA-L 0.000 claims 1
- 229910052939 potassium sulfate Inorganic materials 0.000 claims 1
- 235000011151 potassium sulphates Nutrition 0.000 claims 1
- 101150066583 rep gene Proteins 0.000 claims 1
- 239000001632 sodium acetate Substances 0.000 claims 1
- 235000017281 sodium acetate Nutrition 0.000 claims 1
- 229960002668 sodium chloride Drugs 0.000 claims 1
- BDHFUVZGWQCTTF-UHFFFAOYSA-M sulfonate Chemical compound [O-]S(=O)=O BDHFUVZGWQCTTF-UHFFFAOYSA-M 0.000 claims 1
Applications Claiming Priority (14)
| 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 |
| JP2021549782A 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 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021549782A Division 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 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024133657A JP2024133657A (ja) | 2024-10-02 |
| JP2024133657A5 true JP2024133657A5 (https=) | 2025-02-05 |
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 Before (1)
| 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
|---|---|---|---|---|
| KR102877920B1 (ko) | 2015-11-16 | 2025-10-30 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법 |
| EP3596112A2 (en) | 2017-03-17 | 2020-01-22 | Newcastle University | Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy |
| AR114350A1 (es) | 2018-01-31 | 2020-08-26 | Res Institute At Nationwide ChildrenS Hospital | Terapia genética para la distrofia muscular de cinturas del tipo 2c |
| MY208145A (en) | 2018-06-18 | 2025-04-18 | Res Inst Nationwide Childrens Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| KR20210028162A (ko) | 2018-06-29 | 2021-03-11 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법 |
| RS65421B1 (sr) | 2019-08-21 | 2024-05-31 | Res Inst Nationwide Childrens Hospital | Isporuka adeno-asociranog virusnog vektora alfa-sarkoglikana i lečenje mišićne distrofije |
| IL299094A (en) | 2020-06-15 | 2023-02-01 | Res Inst Nationwide Childrens Hospital | Administration of an adeno-associated virus vector for muscular dystrophies |
| TWI887479B (zh) | 2020-09-08 | 2025-06-21 | 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 |
| EP4219726A1 (en) | 2021-10-15 | 2023-08-02 | Research Institute at Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| 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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