JP2024133657A5 - - Google Patents
Download PDFInfo
- Publication number
- 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
Links
- 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 |
|---|---|---|---|---|
| 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 |
Family Cites Families (111)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IE58011B1 (en) | 1983-05-27 | 1993-06-16 | Texas A & M Univ Sys | Method for producing a recombinant baculovirus expression vector |
| ZA848495B (en) | 1984-01-31 | 1985-09-25 | Idaho Res Found | Production of polypeptides in insect cells |
| US5449616A (en) | 1990-05-23 | 1995-09-12 | University Of Iowa Research Foundation | Nucleic acid encoding dystrophin-associated protein |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| BR9407135A (pt) | 1993-07-23 | 1996-09-17 | Unichema Chemie Bv | Processo para produzir material de sabão transparente e barras macarrões ou pelotas de sabão transparentes |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| AU707866B2 (en) | 1994-12-06 | 1999-07-22 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant AAV vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| AU722196B2 (en) | 1995-08-30 | 2000-07-27 | Genzyme Corporation | Chromatographic purification of adenovirus and AAV |
| EP1983057A3 (en) | 1995-09-08 | 2009-01-07 | Genzyme Corporation | Improved AAV vectors for gene therapy |
| US5672694A (en) | 1995-10-24 | 1997-09-30 | University Of Iowa Research Foundation | β-sarcoglycan nucleic acid sequence, and nucleic acid probes |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| EP0932418B1 (en) | 1996-09-06 | 2007-12-05 | The Trustees Of The University Of Pennsylvania | Method for recombinant adeno-associated virus-directed gene therapy |
| US5843078A (en) | 1997-07-01 | 1998-12-01 | Sharkey; Hugh R. | Radio frequency device for resurfacing skin and method |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| JP2001514845A (ja) | 1997-09-05 | 2001-09-18 | ターゲティッド ジェネティクス コーポレイション | 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法 |
| WO1999043360A1 (en) | 1998-02-26 | 1999-09-02 | The Trustees Of The University Of Pennsylvania | Stable protection from dystrophic sarcolemmal degeneration and restoration of the sarcoglycan complex |
| US6262035B1 (en) | 1998-10-01 | 2001-07-17 | University Of Iowa Research Foundation | Gene replacement therapy for muscular dystrophy |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US6632800B1 (en) | 1999-08-17 | 2003-10-14 | Mayo Foundation For Medical Education And Research | System for monitoring the expression of transgenes |
| JP4520569B2 (ja) | 2000-02-18 | 2010-08-04 | 照彦 豊岡 | 拡張型心筋症の遺伝子治療剤 |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| AU2002248297A1 (en) | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
| US7759314B2 (en) | 2001-08-15 | 2010-07-20 | Brown University | Treatment of muscular dystrophies and related disorders |
| US20040126762A1 (en) | 2002-12-17 | 2004-07-01 | Morris David W. | Novel compositions and methods in cancer |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| WO2003074714A1 (en) | 2002-03-05 | 2003-09-12 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
| US20030225260A1 (en) | 2002-04-30 | 2003-12-04 | Snyder Richard O. | Production of recombinant AAV virions |
| AU2003223775A1 (en) | 2002-04-30 | 2003-11-17 | Duke University | Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use |
| EP1751275B1 (en) | 2004-06-01 | 2017-08-16 | Avigen, Inc. | Compositions and methods to prevent aav vector aggregation |
| US7972593B2 (en) | 2004-06-10 | 2011-07-05 | Saint Louis University | Delivery of therapeutic agents to the bone |
| WO2006039218A2 (en) | 2004-09-30 | 2006-04-13 | The Trustees Of The University Of Pennsylvania | Perfusion circuit and use therein in targeted delivery of macromolecules |
| JP2006121961A (ja) | 2004-10-28 | 2006-05-18 | Univ Of Tokushima | 顎骨骨幹異形成症ｇｄｄの原因遺伝子ｇｄｄ１とその用途 |
| US7883858B2 (en) | 2005-01-27 | 2011-02-08 | Institute For Systems Biology | Methods for identifying and monitoring drug side effects |
| US7788045B2 (en) | 2005-09-01 | 2010-08-31 | Meditasks, Llc | Systems and method for homeostatic blood states |
| CA2625403A1 (en) | 2005-10-17 | 2007-04-26 | Institute For System Biology | Tissue-and serum-derived glycoproteins and methods of their use |
| WO2007049095A1 (en) | 2005-10-25 | 2007-05-03 | Cellectis | Laglidadg homing endonuclease variants having mutations in two functional subdomains and use thereof |
| EP2018421B1 (en) | 2006-04-28 | 2012-12-19 | The Trustees of the University of Pennsylvania | Scalable production method for aav |
| US20100120627A1 (en) | 2006-08-02 | 2010-05-13 | Abdelmajid Belouchi | Genemap of the human genes associated with psoriasis |
| EP2520935A3 (en) | 2006-08-09 | 2013-02-13 | Homestead Clinical Corporation | Organ-specific proteins and methods of their use |
| KR101240487B1 (ko) | 2006-11-09 | 2013-03-08 | 더 존스 홉킨스 유니버시티 | 성체 포유동물 심근세포의 심장 줄기 세포로의 역분화 |
| JP5575486B2 (ja) | 2007-01-18 | 2014-08-20 | ユニヴァーシティ オブ ミズーリー−コロンビア | 筋鞘にｎＮＯＳを回復させる合成ミニ／ミクロジストロフィン遺伝子 |
| FR2919305B1 (fr) | 2007-07-26 | 2009-09-18 | Genethon Ass Loi De 1901 | Vecteurs viraux adeno-associes pour l'expression de la dysferline. |
| GB0715087D0 (en) | 2007-08-03 | 2007-09-12 | Summit Corp Plc | Drug combinations for the treatment of duchenne muscular dystrophy |
| AU2008317566B2 (en) | 2007-10-26 | 2014-05-01 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| WO2009114702A2 (en) | 2008-03-14 | 2009-09-17 | Humanzyme Limited | Recombinant production of authentic human proteins using human cell expression systems |
| US20090280103A1 (en) | 2008-04-04 | 2009-11-12 | Martin Flueck | Regulation of muscle repair |
| US8632764B2 (en) | 2008-04-30 | 2014-01-21 | University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
| US8236557B2 (en) | 2008-05-28 | 2012-08-07 | University Of Missouri-Columbia | Hybrid-AAV vectors to deliver large gene expression cassette |
| US20100026655A1 (en) | 2008-07-31 | 2010-02-04 | Avago Technologies Ecbu Ip (Singapore) Pte. Ltd. | Capacitive Touchscreen or Touchpad for Finger or Stylus |
| US8729041B2 (en) | 2008-12-03 | 2014-05-20 | The Johns Hopkins University | Compositions and methods for treating hepatic neoplasia |
| US20110023139A1 (en) | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in cardiovascular disease |
| US20100247495A1 (en) | 2009-03-30 | 2010-09-30 | Tom Ichim | Treatment of Muscular Dystrophy |
| US8946451B2 (en) | 2009-10-05 | 2015-02-03 | Catabasis Pharmaceuticals, Inc. | Lipoic acid acylated salicylate derivatives and their uses |
| KR20120099709A (ko) | 2009-11-03 | 2012-09-11 | 더 리전츠 오브 더 유니버시티 오브 캘리포니아 | 하나 이상의 표면 위의 아연 산화물 나노로드 어레이들을 이용하는 발광 다이오드 구조물 및 이러한 아연 산화물 나노로드 어레이들을 저비용으로 제조하는 방법 |
| FR2962041B1 (fr) | 2010-07-01 | 2012-07-27 | Genethon | Inhibiteurs de la calpaine 3 pour le traitement de dystrophies musculaires et de cardiomyopathies |
| AU2012286994B2 (en) | 2011-07-25 | 2017-08-10 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of DUX4 |
| WO2013075008A1 (en) | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| US9434928B2 (en) | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| AU2013221212B2 (en) | 2012-02-17 | 2018-08-09 | The Children's Hospital Of Philadelphia | AAV vector compositions and methods for gene transfer to cells, organs and tissues |
| AU2013243948A1 (en) | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| US9254311B2 (en) | 2012-04-02 | 2016-02-09 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| EP3241902B1 (en) | 2012-05-25 | 2018-02-28 | The Regents of The University of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| CA2884245C (en) | 2012-09-06 | 2023-03-14 | The University Of Chicago | Antisense polynucleotides to induce exon skipping and methods of treating dystrophies |
| EP2893038A1 (en) | 2012-09-07 | 2015-07-15 | INSERM - Institut National de la Santé et de la Recherche Médicale | In vitro genetic diagnostic of inherited neuromuscular disorders |
| KR102240217B1 (ko) | 2012-09-25 | 2021-04-14 | 젠자임 코포레이션 | 근긴장성 이영양증의 치료용 펩티드-결합 모르폴리노 안티센스 올리고뉴클레오티드 |
| US9624282B2 (en) | 2012-11-26 | 2017-04-18 | The Curators Of The University Of Missouri | Microdystrophin peptides and methods for treating muscular dystrophy using the same |
| DK2931897T3 (en) | 2012-12-12 | 2018-02-05 | Broad Inst Inc | CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS |
| AU2013359262C1 (en) | 2012-12-12 | 2021-05-13 | Massachusetts Institute Of Technology | CRISPR-Cas component systems, methods and compositions for sequence manipulation |
| EP2931899A1 (en) | 2012-12-12 | 2015-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| CN105121648B (zh) | 2012-12-12 | 2021-05-07 | 布罗德研究所有限公司 | 用于序列操纵的系统、方法和优化的指导组合物的工程化 |
| FR3004463A1 (fr) | 2013-04-11 | 2014-10-17 | Genethon | Systeme d'expression pour une therapie genique selective |
| EP3011030B1 (en) | 2013-06-17 | 2023-11-08 | The Broad Institute, Inc. | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
| ITTO20130669A1 (it) | 2013-08-05 | 2015-02-06 | Consiglio Nazionale Ricerche | Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari |
| TW201536329A (zh) | 2013-08-09 | 2015-10-01 | Isis Pharmaceuticals Inc | 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法 |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| HUE037179T2 (hu) | 2013-10-08 | 2018-08-28 | Ystem S R L | Gyógyászati kompozíciók izom-rendellenességek kezelésére |
| US9850497B2 (en) | 2013-11-04 | 2017-12-26 | Regents Of The University Of Minnesota | Gene targeting methods and tools |
| EP3470089A1 (en) | 2013-12-12 | 2019-04-17 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components |
| EP3097197B1 (en) | 2014-01-21 | 2020-12-16 | Vrije Universiteit Brussel | Muscle-specific nucleic acid regulatory elements and methods and use thereof |
| JP6202701B2 (ja) | 2014-03-21 | 2017-09-27 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及びプログラム |
| DE102014207498A1 (de) | 2014-04-17 | 2015-10-22 | Universitätsklinikum Hamburg-Eppendorf | Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark |
| EP2960336A1 (en) | 2014-06-27 | 2015-12-30 | Genethon | Efficient systemic treatment of dystrophic muscle pathologies |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| IL286316B2 (en) | 2015-01-16 | 2023-03-01 | Univ Washington | New micro-dystrophins and related method of use |
| 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 |
| WO2017165859A1 (en) | 2016-03-24 | 2017-09-28 | Research Institute At Nationwide Children's Hospital | Modified viral capsid proteins |
| KR102450833B1 (ko) * | 2016-04-15 | 2022-10-05 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 혈우병 a 치료용 유전자 요법 |
| PT3442600T (pt) * | 2016-04-15 | 2024-05-09 | Res Inst Nationwide Childrens Hospital | Distribuição de vetor de vírus adeno-associado de bsarcoglicano e microrna-29 e o tratamento da distrofia muscular |
| AU2017250791B2 (en) | 2016-04-15 | 2022-11-24 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy |
| CA2971303C (en) | 2016-06-21 | 2026-03-03 | Bamboo Therapeutics, Inc. | Optimized mini-dystrophin genes and expression cassettes and their use |
| WO2019012336A2 (en) | 2017-03-17 | 2019-01-17 | Newcastle University | ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY |
| MX2019011046A (es) | 2017-03-17 | 2019-10-17 | Res Inst Nationwide Childrens Hospital | Administracion de vectores del virus adenoasociado de microdistrofina especifica del musculo para tratar la distrofia muscular. |
| JP7213238B2 (ja) | 2017-10-18 | 2023-01-26 | 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 | 筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| AU2018351528B2 (en) | 2017-10-20 | 2025-02-27 | Research Institute At Nationwide Children's Hospital | Methods and materials for NT-3 gene therapy |
| WO2019118806A1 (en) | 2017-12-14 | 2019-06-20 | Solid Biosciences Inc. | Non-viral production and delivery of genes |
| MX2020007876A (es) | 2018-01-31 | 2020-12-03 | Res Inst Nationwide Childrens Hospital | Terapia genica para la distrofia muscular de cinturas tipo 2c. |
| JP2021520196A (ja) | 2018-04-03 | 2021-08-19 | ザ・キュレイターズ・オブ・ザ・ユニバーシティー・オブ・ミズーリＴｈｅ Ｃｕｒａｔｏｒｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍｉｓｓｏｕｒｉ | ジストロフィン異常症（ｄｙｓｔｒｏｐｈｉｎｏｐａｔｈｙ）治療のためのヒンジ１および／または４の改変ジストロフィン |
| US12152242B2 (en) | 2018-04-23 | 2024-11-26 | The Curators Of The University Of Missouri | CRISPR therapy |
| 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. |
| WO2020123645A1 (en) | 2018-12-12 | 2020-06-18 | Solid Biosciences Inc. | Combination therapy for treating muscular dystrophy |
| 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 |
| JP2023534127A (ja) | 2020-06-17 | 2023-08-08 | アクセルロン ファーマ インコーポレイテッド | ＡｃｔＲＩＩ－ＡＬＫ４アンタゴニストおよび心不全を処置する方法 |
| US20230390417A1 (en) | 2022-06-02 | 2023-12-07 | Research Institute At Nationwide Children's Hospital | Recombinant Adeno-Associated Virus Products and Methods for Treating Limb Girdle Muscular Dystrophy 2A |
-
2020
- 2020-02-26 MX MX2021010356A patent/MX2021010356A/es unknown
- 2020-02-26 CA CA3131390A patent/CA3131390A1/en active Pending
- 2020-02-26 AU AU2020229340A patent/AU2020229340A1/en active Pending
- 2020-02-26 WO PCT/US2020/019892 patent/WO2020176614A1/en not_active Ceased
- 2020-02-26 TW TW109106292A patent/TW202045531A/zh unknown
- 2020-02-26 SG SG11202109113TA patent/SG11202109113TA/en unknown
- 2020-02-26 KR KR1020217030293A patent/KR20210133242A/ko active Pending
- 2020-02-26 JP JP2021549782A patent/JP2022521776A/ja active Pending
- 2020-02-26 US US17/432,417 patent/US12377170B2/en active Active
- 2020-02-26 CN CN202080031591.2A patent/CN113766935A/zh active Pending
- 2020-02-26 EP EP20714395.9A patent/EP3930765A1/en active Pending
- 2020-02-26 BR BR112021016570A patent/BR112021016570A2/pt unknown
-
2021
- 2021-08-20 ZA ZA2021/06017A patent/ZA202106017B/en unknown
- 2021-08-24 CL CL2021002236A patent/CL2021002236A1/es unknown
- 2021-08-25 IL IL285849A patent/IL285849A/en unknown
- 2021-09-15 CO CONC2021/0012074A patent/CO2021012074A2/es unknown
-
2024
- 2024-07-08 JP JP2024109621A patent/JP2024133657A/ja active Pending
-
2025
- 2025-08-22 JP JP2025139290A patent/JP2025170362A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2024133657A5 (https=) | ||
| US10858632B2 (en) | Restrictive inverted terminal repeats for viral vectors | |
| Wu et al. | Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism | |
| Salganik et al. | Adeno‐associated virus as a mammalian DNA vector | |
| Rayaprolu et al. | Comparative analysis of adeno-associated virus capsid stability and dynamics | |
| Venkatakrishnan et al. | Structure and dynamics of adeno-associated virus serotype 1 VP1-unique N-terminal domain and its role in capsid trafficking | |
| Walters et al. | Structure of adeno-associated virus serotype 5 | |
| Rabinowitz et al. | Insertional mutagenesis of AAV2 capsid and the production of recombinant virus | |
| US5622856A (en) | High efficiency helper system for AAV vector production | |
| CN111117985B (zh) | 一种拆分Cas9的方法及其应用 | |
| US12398403B2 (en) | Methods for the manufacture of recombinant viral vectors | |
| US20150184197A1 (en) | Modified adeno-associated virus vector compositions | |
| JP7134240B2 (ja) | 組換え型アデノ随伴ウイルスの調製方法、システム及び組換え型バクミド | |
| Bando et al. | Terminal structure of a densovirus implies a hairpin transfer replication which is similar to the model for AAV | |
| Urabe et al. | Charged-to-alanine scanning mutagenesis of the N-terminal half of adeno-associated virus type 2 Rep78 protein | |
| US7091029B2 (en) | High titer recombinant AAV production | |
| CN114981442A (zh) | 用于生产重组aav的新颖组合物及方法 | |
| Smith et al. | Comparative characterization of Rep proteins from the helper-dependent adeno-associated virus type 2 and the autonomous goose parvovirus | |
| CN116063404B (zh) | 可提高AAV包装能力的衣壳蛋白MutD及其应用 | |
| US20060286545A1 (en) | Viral vectors with improved properties | |
| Leonard et al. | Adeno-associated virus type 2: a latent life cycle | |
| Yoon et al. | Amino-terminal domain exchange redirects origin-specific interactions of adeno-associated virus rep78 in vitro | |
| Guo et al. | Complete sequence and organization of Periplaneta fuliginosa densovirus genome | |
| Weitzman | The parvovirus life cycle: an introduction to molecular interactions important for infection | |
| Youjin et al. | The treatment of hemophilia A: from protein replacement to AAV-mediated gene therapy |