JP2021502123A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2021502123A5 JP2021502123A5 JP2020544560A JP2020544560A JP2021502123A5 JP 2021502123 A5 JP2021502123 A5 JP 2021502123A5 JP 2020544560 A JP2020544560 A JP 2020544560A JP 2020544560 A JP2020544560 A JP 2020544560A JP 2021502123 A5 JP2021502123 A5 JP 2021502123A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- viral vector
- less
- dose
- protein
- 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.)
- Granted
Links
- 239000013603 viral vector Substances 0.000 claims description 22
- 230000004083 survival effect Effects 0.000 claims description 18
- 239000000825 pharmaceutical preparation Substances 0.000 claims description 14
- 238000000034 method Methods 0.000 claims description 12
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims description 11
- 238000004519 manufacturing process Methods 0.000 claims description 6
- 239000013598 vector Substances 0.000 claims description 6
- 239000000872 buffer Substances 0.000 claims description 5
- 210000000234 capsid Anatomy 0.000 claims description 5
- 239000011780 sodium chloride Substances 0.000 claims description 5
- 239000002245 particle Substances 0.000 claims description 4
- 239000000243 solution Substances 0.000 claims description 3
- 239000002299 complementary DNA Substances 0.000 claims description 2
- 108091033319 polynucleotide Proteins 0.000 claims 14
- 102000040430 polynucleotide Human genes 0.000 claims 14
- 239000002157 polynucleotide Substances 0.000 claims 14
- 210000004027 cell Anatomy 0.000 claims 13
- 238000005303 weighing Methods 0.000 claims 11
- 239000000203 mixture Substances 0.000 claims 10
- 239000008194 pharmaceutical composition Substances 0.000 claims 9
- 108090000623 proteins and genes Proteins 0.000 claims 9
- 102000004169 proteins and genes Human genes 0.000 claims 9
- 230000001464 adherent effect Effects 0.000 claims 8
- 238000009295 crossflow filtration Methods 0.000 claims 8
- AIYUHDOJVYHVIT-UHFFFAOYSA-M caesium chloride Chemical compound [Cl-].[Cs+] AIYUHDOJVYHVIT-UHFFFAOYSA-M 0.000 claims 7
- 239000013592 cell lysate Substances 0.000 claims 7
- 239000013612 plasmid Substances 0.000 claims 6
- 238000001890 transfection Methods 0.000 claims 6
- CTKXFMQHOOWWEB-UHFFFAOYSA-N Ethylene oxide/propylene oxide copolymer Chemical compound CCCOC(C)COCCO CTKXFMQHOOWWEB-UHFFFAOYSA-N 0.000 claims 5
- 239000007983 Tris buffer Substances 0.000 claims 5
- 238000005277 cation exchange chromatography Methods 0.000 claims 5
- 229920001993 poloxamer 188 Polymers 0.000 claims 5
- 229940044519 poloxamer 188 Drugs 0.000 claims 5
- 208000002320 spinal muscular atrophy Diseases 0.000 claims 5
- LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 claims 5
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 4
- 241000701022 Cytomegalovirus Species 0.000 claims 4
- 108010006025 bovine growth hormone Proteins 0.000 claims 4
- 238000001914 filtration Methods 0.000 claims 4
- 210000002161 motor neuron Anatomy 0.000 claims 4
- 229940127557 pharmaceutical product Drugs 0.000 claims 4
- 241000702421 Dependoparvovirus Species 0.000 claims 3
- 101000617738 Homo sapiens Survival motor neuron protein Proteins 0.000 claims 3
- 102000006890 Methyl-CpG-Binding Protein 2 Human genes 0.000 claims 3
- 108010072388 Methyl-CpG-Binding Protein 2 Proteins 0.000 claims 3
- -1 MgCl 2 Chemical compound 0.000 claims 3
- 108091027967 Small hairpin RNA Proteins 0.000 claims 3
- 102000008221 Superoxide Dismutase-1 Human genes 0.000 claims 3
- 108010021188 Superoxide Dismutase-1 Proteins 0.000 claims 3
- 102100021947 Survival motor neuron protein Human genes 0.000 claims 3
- 230000020477 pH reduction Effects 0.000 claims 3
- 239000004055 small Interfering RNA Substances 0.000 claims 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 2
- 101150044789 Cap gene Proteins 0.000 claims 2
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 2
- 229920002873 Polyethylenimine Polymers 0.000 claims 2
- 108010034546 Serratia marcescens nuclease Proteins 0.000 claims 2
- 241000700605 Viruses Species 0.000 claims 2
- 239000003623 enhancer Substances 0.000 claims 2
- 238000010899 nucleation Methods 0.000 claims 2
- 230000008488 polyadenylation Effects 0.000 claims 2
- 229920000136 polysorbate Polymers 0.000 claims 2
- 101150066583 rep gene Proteins 0.000 claims 2
- 230000003612 virological effect Effects 0.000 claims 2
- 239000013607 AAV vector Substances 0.000 claims 1
- 206010003694 Atrophy Diseases 0.000 claims 1
- OYPRJOBELJOOCE-UHFFFAOYSA-N Calcium Chemical compound [Ca] OYPRJOBELJOOCE-UHFFFAOYSA-N 0.000 claims 1
- 206010021118 Hypotonia Diseases 0.000 claims 1
- 208000005137 Joint instability Diseases 0.000 claims 1
- ZDXPYRJPNDTMRX-VKHMYHEASA-N L-glutamine Chemical compound OC(=O)[C@@H](N)CCC(N)=O ZDXPYRJPNDTMRX-VKHMYHEASA-N 0.000 claims 1
- 208000007379 Muscle Hypotonia Diseases 0.000 claims 1
- 101150015954 SMN2 gene Proteins 0.000 claims 1
- 208000003954 Spinal Muscular Atrophies of Childhood Diseases 0.000 claims 1
- 230000037444 atrophy Effects 0.000 claims 1
- 230000037396 body weight Effects 0.000 claims 1
- 229910052792 caesium Inorganic materials 0.000 claims 1
- TVFDJXOCXUVLDH-UHFFFAOYSA-N caesium atom Chemical compound [Cs] TVFDJXOCXUVLDH-UHFFFAOYSA-N 0.000 claims 1
- 229910052791 calcium Inorganic materials 0.000 claims 1
- 239000011575 calcium Substances 0.000 claims 1
- 238000004113 cell culture Methods 0.000 claims 1
- 239000006143 cell culture medium Substances 0.000 claims 1
- 229920002678 cellulose Polymers 0.000 claims 1
- 239000001913 cellulose Substances 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 claims 1
- 238000005352 clarification Methods 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 230000009089 cytolysis Effects 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 230000008014 freezing Effects 0.000 claims 1
- 238000007710 freezing Methods 0.000 claims 1
- ZDXPYRJPNDTMRX-UHFFFAOYSA-N glutamine Natural products OC(=O)C(N)CCC(N)=O ZDXPYRJPNDTMRX-UHFFFAOYSA-N 0.000 claims 1
- 238000001802 infusion Methods 0.000 claims 1
- 238000007913 intrathecal administration Methods 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 239000006166 lysate Substances 0.000 claims 1
- 239000012139 lysis buffer Substances 0.000 claims 1
- 239000002609 medium Substances 0.000 claims 1
- 239000012528 membrane Substances 0.000 claims 1
- 210000003205 muscle Anatomy 0.000 claims 1
- 230000003387 muscular Effects 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 238000002360 preparation method Methods 0.000 claims 1
- 239000003755 preservative agent Substances 0.000 claims 1
- 230000002335 preservative effect Effects 0.000 claims 1
- 238000000746 purification Methods 0.000 claims 1
- 239000011347 resin Substances 0.000 claims 1
- 229920005989 resin Polymers 0.000 claims 1
- 210000002966 serum Anatomy 0.000 claims 1
- 238000006467 substitution reaction Methods 0.000 claims 1
- 125000000472 sulfonyl group Chemical group *S(*)(=O)=O 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
- 239000003656 tris buffered saline Substances 0.000 claims 1
- 208000032471 type 1 spinal muscular atrophy Diseases 0.000 claims 1
- 238000005199 ultracentrifugation Methods 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 229940126534 drug product Drugs 0.000 description 9
- 241000699670 Mus sp. Species 0.000 description 4
- 231100000673 dose–response relationship Toxicity 0.000 description 4
- 238000011304 droplet digital PCR Methods 0.000 description 4
- 238000012417 linear regression Methods 0.000 description 4
- 238000010172 mouse model Methods 0.000 description 4
- 239000000463 material Substances 0.000 description 3
- 229950009805 onasemnogene abeparvovec Drugs 0.000 description 3
- 238000012356 Product development Methods 0.000 description 2
- 238000003556 assay Methods 0.000 description 2
- 238000004364 calculation method Methods 0.000 description 2
- 238000011088 calibration curve Methods 0.000 description 2
- 238000011156 evaluation Methods 0.000 description 2
- 239000013642 negative control Substances 0.000 description 2
- 239000013641 positive control Substances 0.000 description 2
- 208000022074 proximal spinal muscular atrophy Diseases 0.000 description 2
- 239000000126 substance Substances 0.000 description 2
- 238000011287 therapeutic dose Methods 0.000 description 2
- 238000002560 therapeutic procedure Methods 0.000 description 2
- 241001465754 Metazoa Species 0.000 description 1
- 238000004458 analytical method Methods 0.000 description 1
- 239000007853 buffer solution Substances 0.000 description 1
- 229940121657 clinical drug Drugs 0.000 description 1
- 208000015181 infectious disease Diseases 0.000 description 1
- 230000002458 infectious effect Effects 0.000 description 1
- 238000007619 statistical method Methods 0.000 description 1
- 239000012906 subvisible particle Substances 0.000 description 1
Images
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024150419A JP2025017361A (ja) | 2017-11-08 | 2024-09-02 | ウイルスベクターの調製手段及び方法並びにその使用 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762583035P | 2017-11-08 | 2017-11-08 | |
| US62/583,035 | 2017-11-08 | ||
| PCT/US2018/058744 WO2019094253A1 (en) | 2017-11-08 | 2018-11-01 | Means and method for preparing viral vectors and uses of same |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024150419A Division JP2025017361A (ja) | 2017-11-08 | 2024-09-02 | ウイルスベクターの調製手段及び方法並びにその使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021502123A JP2021502123A (ja) | 2021-01-28 |
| JP2021502123A5 true JP2021502123A5 (https=) | 2021-12-09 |
| JP7587422B2 JP7587422B2 (ja) | 2024-11-20 |
Family
ID=64362706
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020544560A Active JP7587422B2 (ja) | 2017-11-08 | 2018-11-01 | ウイルスベクターの調製手段及び方法並びにその使用 |
| JP2024150419A Pending JP2025017361A (ja) | 2017-11-08 | 2024-09-02 | ウイルスベクターの調製手段及び方法並びにその使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024150419A Pending JP2025017361A (ja) | 2017-11-08 | 2024-09-02 | ウイルスベクターの調製手段及び方法並びにその使用 |
Country Status (17)
| Country | Link |
|---|---|
| US (2) | US12168777B2 (https=) |
| EP (2) | EP4585229A3 (https=) |
| JP (2) | JP7587422B2 (https=) |
| KR (1) | KR20200086292A (https=) |
| CN (4) | CN111566220A (https=) |
| AU (2) | AU2018365677C1 (https=) |
| BR (1) | BR112019013268A2 (https=) |
| CA (1) | CA3082136A1 (https=) |
| CL (3) | CL2020001193A1 (https=) |
| CO (1) | CO2020006900A2 (https=) |
| IL (2) | IL314692A (https=) |
| MX (3) | MX2020004830A (https=) |
| MY (1) | MY208779A (https=) |
| SA (1) | SA520411930B1 (https=) |
| SG (1) | SG11202004406VA (https=) |
| TW (1) | TWI827560B (https=) |
| WO (1) | WO2019094253A1 (https=) |
Families Citing this family (49)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11219696B2 (en) * | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
| MX2020000713A (es) * | 2017-07-17 | 2020-08-31 | Spark Therapeutics Inc | Metodos de aferesis y sus usos. |
| MX2020004830A (es) | 2017-11-08 | 2020-11-11 | Avexis Inc | Medios y metodo para preparar vectores virales y usos de los mismos. |
| CN112601557B (zh) * | 2018-06-08 | 2025-06-06 | 诺华股份有限公司 | 用于测量药物产品效力的基于细胞的测定 |
| EP3837374A4 (en) * | 2018-08-15 | 2022-06-08 | Biogen MA Inc. | COMBINATION THERAPY FOR SPINAL MUSCLE ATROPHY |
| WO2020113034A1 (en) * | 2018-11-30 | 2020-06-04 | Avexis, Inc. | Aav viral vectors and uses thereof |
| PL3953377T3 (pl) * | 2019-04-10 | 2026-02-16 | Prevail Therapeutics, Inc. | Terapie genowe zaburzeń lizosomalnych |
| US10801042B1 (en) * | 2019-07-15 | 2020-10-13 | Vigene Biosciences, Inc. | Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus |
| WO2021016075A1 (en) | 2019-07-19 | 2021-01-28 | Flagship Pioneering Innovations Vi, Llc | Recombinase compositions and methods of use |
| WO2021014428A1 (en) | 2019-07-25 | 2021-01-28 | Novartis Ag | Regulatable expression systems |
| CA3149825A1 (en) * | 2019-08-08 | 2021-02-11 | Biogen Ma Inc. | Potency assays for viral vector production |
| CA3156506A1 (en) * | 2019-10-30 | 2021-05-06 | Amicus Therapeutics, Inc. | Recombinant cdkl5 proteins, gene therapy and production methods |
| KR20220133908A (ko) * | 2020-01-28 | 2022-10-05 | 론자 워커스빌 아이엔씨. | 바이러스 역가를 결정하는 방법 |
| EP4136231A1 (en) | 2020-04-17 | 2023-02-22 | Friedrich Miescher Institute for Biomedical Research | Use of an orphan motif to increase expression of a heterologous transgene |
| RU2742837C1 (ru) * | 2020-06-02 | 2021-02-11 | Общество С Ограниченной Ответственностью "Анабион" | Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение |
| CN115996940A (zh) * | 2020-07-01 | 2023-04-21 | 免疫医疗有限责任公司 | 用于纯化生物治疗剂的洗涤剂和方法 |
| CN112121179A (zh) * | 2020-09-15 | 2020-12-25 | 山东兴瑞生物科技有限公司 | 一种组合物及其在治疗脊髓性肌萎缩症中的应用 |
| KR20230088393A (ko) * | 2020-10-15 | 2023-06-19 | 프리베일 테라퓨틱스, 인크. | 재조합 아데노 연관 바이러스 조성물 및 이의 제조 방법 |
| CN112251440A (zh) * | 2020-10-29 | 2021-01-22 | 大连理工大学 | 一种CRISPR/Cas9基因载体、其制备方法及应用 |
| WO2022118237A1 (en) | 2020-12-03 | 2022-06-09 | Friedrich Miescher Institute For Biomedical Research | Use of a combination of an orphan motif and cpg density to control expression of a heterologous transgene |
| EP4324907A4 (en) | 2021-04-15 | 2025-06-18 | The University of Tokyo | METHOD FOR PRODUCING VIRUS PARTICLES |
| EP4377459A2 (en) | 2021-07-30 | 2024-06-05 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of frataxin (fxn) |
| WO2023010135A1 (en) | 2021-07-30 | 2023-02-02 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) |
| CA3226803A1 (en) * | 2021-08-04 | 2023-02-09 | Christian Fiedler | Adeno-associated virus separation on a cation exchanger |
| WO2023042104A1 (en) | 2021-09-16 | 2023-03-23 | Novartis Ag | Novel transcription factors |
| CN118119710A (zh) * | 2021-10-15 | 2024-05-31 | 上海天泽云泰生物医药有限公司 | 用于治疗脊髓性肌萎缩的重组腺相关病毒载体 |
| US20250235554A1 (en) | 2021-10-25 | 2025-07-24 | Novartis Ag | Methods for improving adeno-associated virus (aav) delivery |
| WO2023085382A1 (ja) * | 2021-11-12 | 2023-05-19 | Ａｇｃ株式会社 | ウイルスの精製方法 |
| EP4436982A1 (en) | 2021-11-24 | 2024-10-02 | Novartis AG | Modulators of adeno-associated virus transduction and uses thereof |
| US20250353883A1 (en) | 2022-05-06 | 2025-11-20 | Novartis Ag | Novel recombinant aav vp2 fusion polypeptides |
| CA3261865A1 (en) | 2022-07-12 | 2024-01-18 | Tune Therapeutics, Inc. | TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS |
| CN114990162A (zh) * | 2022-07-18 | 2022-09-02 | 苏州吉纳星辰生物技术有限公司 | 一种降低aav病毒载体生产中质粒残留的方法 |
| WO2024027632A1 (en) * | 2022-08-01 | 2024-02-08 | Skyline Therapeutics (Shanghai) Co., Ltd. | Novel plasmid backbone to reduce dna impurities in raav preparation |
| CN115354071A (zh) * | 2022-08-10 | 2022-11-18 | 上海药明生基医药科技有限公司 | 一种检测重组腺相关病毒宿主细胞dna残留的方法 |
| CN116350801A (zh) | 2022-11-22 | 2023-06-30 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
| WO2024163678A2 (en) | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods |
| WO2024163683A2 (en) | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist) |
| CN120835797A (zh) | 2023-03-21 | 2025-10-24 | 豪夫迈·罗氏有限公司 | 用于产生重组aav颗粒制备物的方法 |
| WO2024252182A1 (en) * | 2023-06-08 | 2024-12-12 | Meiragtx Uk Ii Limited | Cation exchange chromatography for aav capture |
| CN120677245A (zh) * | 2024-01-25 | 2025-09-19 | 鼐济医药科技(杭州)有限公司 | Aav载体、载体组合物、嵌合载体和治疗或预防sma的方法 |
| WO2025168663A1 (en) | 2024-02-09 | 2025-08-14 | F. Hoffmann-La Roche Ag | Method for producing recombinant adeno-associated viral particles |
| WO2025188316A1 (en) * | 2024-03-08 | 2025-09-12 | Jaguar Gene Therapy, Llc | Aav pharmaceutical formulations and methods thereof |
| WO2025190867A1 (en) * | 2024-03-11 | 2025-09-18 | Sartorius Stedim Biotech Gmbh | Two-step intensified cell lysis to release a viral vector |
| WO2025217224A1 (en) * | 2024-04-08 | 2025-10-16 | Advanced Medicine Partners, LLC | Method of manufacture of aav particles |
| KR102915956B1 (ko) * | 2024-04-17 | 2026-01-20 | 한국로봇융합연구원 | 3차원 오비탈 용접로봇 |
| WO2025252480A1 (en) | 2024-06-07 | 2025-12-11 | F. Hoffmann-La Roche Ag | Method for purifying plasmid dna |
| US20260022401A1 (en) | 2024-07-22 | 2026-01-22 | Hoffmann-La Roche Inc. | Novel AAV rep ORFs and Rep polypeptides |
| WO2026028089A1 (en) | 2024-07-29 | 2026-02-05 | Novartis Ag | Compositions and methods for the treatment of lysosomal acid lipase deficiency (lal-d) |
| WO2026037757A1 (en) | 2024-08-13 | 2026-02-19 | F. Hoffmann-La Roche Ag | Modified aav particles |
Family Cites Families (59)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173418A (en) | 1985-05-10 | 1992-12-22 | Benzon Pharma, A/S | Production in Escherichia coli of extracellular Serratia spp. hydrolases |
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| 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 |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| 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 |
| US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
| 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 |
| 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 |
| US6756207B1 (en) | 1997-02-27 | 2004-06-29 | Cellomics, Inc. | System for cell-based screening |
| 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 |
| 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 |
| WO2001048155A2 (en) * | 1999-12-29 | 2001-07-05 | Genzyme Corporation | Method using filtration aids for the separation of virus vectors from nucleic acids and other cellular contaminants |
| 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 |
| AU2002359284A1 (en) | 2001-12-17 | 2003-06-30 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor |
| DE602004030327D1 (de) * | 2003-05-21 | 2011-01-13 | Genzyme Corp | Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US20050153446A1 (en) | 2003-11-07 | 2005-07-14 | Steindler Dennis A. | Culturing and differentiating neural precursor cells |
| EP1751275B1 (en) * | 2004-06-01 | 2017-08-16 | Avigen, Inc. | Compositions and methods to prevent aav vector aggregation |
| SI2548560T1 (sl) | 2005-06-23 | 2015-12-31 | Isis Pharmaceuticals, Inc. | Sestavki in postopki za moduliranje izrezovanja smn2 |
| EP2058401A1 (en) * | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| PL2310492T3 (pl) | 2008-06-30 | 2015-12-31 | Janssen Biotech Inc | Różnocowanie pluripotencjalnych komórek macierzystych |
| US11219696B2 (en) * | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
| WO2010071832A1 (en) | 2008-12-19 | 2010-06-24 | Nationwide Children's Hospital | Delivery of polynucleotides across the blood brain barrier using recombinant aav9 |
| US9415121B2 (en) | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| JP5879256B2 (ja) | 2009-05-02 | 2016-03-08 | ジェンザイム・コーポレーション | 神経変性障害のための遺伝子治療 |
| PT3305302T (pt) | 2009-06-17 | 2018-12-14 | Biogen Ma Inc | Composições e métodos de modulação de excisões de smn2 em um sujeito |
| CA2784425A1 (en) | 2009-12-23 | 2011-06-30 | Centocor Ortho Biotech Inc. | Differentiation of human embryonic stem cells |
| DK2529020T3 (en) * | 2010-01-28 | 2018-08-06 | Childrens Hospital Philadelphia | SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| EP2861723A4 (en) | 2012-06-14 | 2016-01-20 | Janssen Biotech Inc | DIFFERENTIATION OF HUMAN EMBRYONAL STEM CELLS IN ENDOCRINE PANCREAS CELLS |
| WO2013190059A1 (en) | 2012-06-21 | 2013-12-27 | Association Institut De Myologie | Widespread gene delivery of gene therapy vectors |
| AU2013296425B2 (en) | 2012-08-01 | 2018-06-07 | Nationwide Children's Hospital | Intrathecal delivery of recombinant adeno-associated virus 9 |
| MX380973B (es) | 2013-05-01 | 2025-03-12 | Genzyme Corp | Composiciones y metodos para tratar la atrofia muscular espinal. |
| EP3020808B1 (en) | 2013-07-11 | 2019-09-11 | Takara Bio Inc. | Method for manufacturing adeno-associated virus |
| EP3024497B1 (en) | 2013-07-26 | 2021-01-13 | University of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| LT3702466T (lt) | 2013-08-27 | 2023-02-27 | Research Institute At Nationwide Children's Hospital | Produktai ir būdai, skirti šoninės amiotrofinės sklerozės gydymui |
| 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 |
| 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 |
| EP2933335A1 (en) | 2014-04-18 | 2015-10-21 | Genethon | A method of treating peripheral neuropathies and motor neuron diseases |
| US10526583B2 (en) * | 2014-07-02 | 2020-01-07 | University Of Florida Research Foundation, Incorporated | Compositions and methods for purifying recombinant adeno-associated virus |
| 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 |
| CN107249646B (zh) | 2014-12-16 | 2021-06-29 | 内布拉斯加大学董事会 | 用于青少年巴滕病的基因疗法 |
| EP3054007A1 (en) | 2015-02-09 | 2016-08-10 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Recombinant adeno-associated virus particle purification comprising an immuno-affinity purification step |
| EP3054006A1 (en) | 2015-02-09 | 2016-08-10 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Recombinant adeno-associated virus particle purification with multiple-step anion exchange chromatography |
| EP3387138B1 (en) * | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| BR112018070256A2 (pt) * | 2016-03-31 | 2019-01-29 | Spark Therapeutics, Inc. | processo de fabricação de raav totalmente escalável à base de colunas |
| IL305449B2 (en) * | 2016-04-15 | 2026-01-01 | Univ Pennsylvania | Gene therapy for the treatment of mucocutaneous type ii diabetes |
| EP3299456A1 (en) | 2016-09-26 | 2018-03-28 | Université de Strasbourg | Recombinant dgkk gene for fragile x syndrome gene therapy |
| BR112019009834B1 (pt) | 2016-11-17 | 2022-05-17 | Ohio State Innovation Foundation | Vetor viral adeno-associado recombinante (raav), vírus, composição farmacêutica e uso de um vetor raav |
| US20200370069A1 (en) | 2017-07-08 | 2020-11-26 | Genethon | Treatment of spinal muscular atrophy |
| MX2020004830A (es) | 2017-11-08 | 2020-11-11 | Avexis Inc | Medios y metodo para preparar vectores virales y usos de los mismos. |
| CN112601557B (zh) | 2018-06-08 | 2025-06-06 | 诺华股份有限公司 | 用于测量药物产品效力的基于细胞的测定 |
-
2018
- 2018-11-01 MX MX2020004830A patent/MX2020004830A/es unknown
- 2018-11-01 CN CN201880085757.1A patent/CN111566220A/zh active Pending
- 2018-11-01 EP EP25159502.1A patent/EP4585229A3/en active Pending
- 2018-11-01 CN CN202411444917.0A patent/CN119033965A/zh active Pending
- 2018-11-01 SG SG11202004406VA patent/SG11202004406VA/en unknown
- 2018-11-01 BR BR112019013268-6A patent/BR112019013268A2/pt not_active Application Discontinuation
- 2018-11-01 EP EP18804830.0A patent/EP3707264A1/en active Pending
- 2018-11-01 CN CN202510662244.4A patent/CN120505370A/zh active Pending
- 2018-11-01 IL IL314692A patent/IL314692A/en unknown
- 2018-11-01 IL IL274430A patent/IL274430B2/en unknown
- 2018-11-01 US US16/761,869 patent/US12168777B2/en active Active
- 2018-11-01 AU AU2018365677A patent/AU2018365677C1/en active Active
- 2018-11-01 CN CN202510662252.9A patent/CN120501895A/zh active Pending
- 2018-11-01 CA CA3082136A patent/CA3082136A1/en active Pending
- 2018-11-01 TW TW107138852A patent/TWI827560B/zh active
- 2018-11-01 MY MYPI2020002052A patent/MY208779A/en unknown
- 2018-11-01 KR KR1020207014865A patent/KR20200086292A/ko not_active Ceased
- 2018-11-01 JP JP2020544560A patent/JP7587422B2/ja active Active
- 2018-11-01 WO PCT/US2018/058744 patent/WO2019094253A1/en not_active Ceased
-
2020
- 2020-05-06 CL CL2020001193A patent/CL2020001193A1/es unknown
- 2020-05-08 SA SA520411930A patent/SA520411930B1/ar unknown
- 2020-06-04 CO CONC2020/0006900A patent/CO2020006900A2/es unknown
- 2020-07-13 MX MX2025001503A patent/MX2025001503A/es unknown
- 2020-07-13 MX MX2025009360A patent/MX2025009360A/es unknown
-
2024
- 2024-09-02 JP JP2024150419A patent/JP2025017361A/ja active Pending
- 2024-11-08 US US18/940,967 patent/US20250066813A1/en active Pending
- 2024-12-26 CL CL2024004045A patent/CL2024004045A1/es unknown
-
2025
- 2025-05-14 AU AU2025203480A patent/AU2025203480B2/en active Active
- 2025-05-15 CL CL2025001440A patent/CL2025001440A1/es unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2021502123A5 (https=) | ||
| JP7808674B2 (ja) | Ａａｖウイルスベクター及びその使用 | |
| US11578341B2 (en) | Compositions useful in treatment of spinal muscular atrophy | |
| Rodrigues et al. | Pharmaceutical Development of AAV-Based Gene Therapy Products for the Eye: Rodrigues et al. | |
| EP4349852A1 (en) | Recombinant adeno-associated virus having variant capsid, and application thereof | |
| JP2021121638A (ja) | 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法 | |
| US12594348B2 (en) | Compositions for treating Friedreich's ataxia | |
| US20170007720A1 (en) | Methods and compositions for gene delivery to on bipolar cells | |
| CN117821513A (zh) | 治疗退行性眼部疾病的药物组合物及方法 | |
| JP2025527658A (ja) | 組換えアデノ随伴ウイルス及びその使用 | |
| CN113574176A (zh) | 腺相关病毒对cln6多核苷酸的递送 | |
| WO2024094044A1 (zh) | 优化的pah基因和表达盒及其用途 | |
| WO2023169115A1 (zh) | 一种神经系统高亲和性的aav载体及其应用 | |
| TW202508610A (zh) | 醫藥組成物及其用途 | |
| CN116042719B (zh) | 一种重组腺相关病毒载体及其应用 | |
| US12612647B2 (en) | Compositions useful in treatment of spinal muscular atrophy | |
| CN119971081B (zh) | 用于治疗智力障碍的组合物、其应用以及药物筛选方法 | |
| RU2020118557A (ru) | Средства и способ получения вирусных векторов и варианты их применения | |
| CN117281923A (zh) | 包含aav的药物组合物及其制备方法 | |
| CN121175424A (zh) | 一种携带smn基因表达框的病毒载体及其用途 | |
| HK40063258A (zh) | 腺相关病毒对cln6多核苷酸的递送 | |
| Engelhardt et al. | Virus-Mediated Transduction of Murine | |
| RU2024110309A (ru) | Средства и способ получения вирусных векторов и варианты их применения | |
| NZ792477A (en) | Aav capsid designs |