JP2021523198A5 - - Google Patents
Info
- Publication number
- JP2021523198A5 JP2021523198A5 JP2020564142A JP2020564142A JP2021523198A5 JP 2021523198 A5 JP2021523198 A5 JP 2021523198A5 JP 2020564142 A JP2020564142 A JP 2020564142A JP 2020564142 A JP2020564142 A JP 2020564142A JP 2021523198 A5 JP2021523198 A5 JP 2021523198A5
- Authority
- JP
- Japan
- Prior art keywords
- composition according
- therapeutic
- aav virus
- young
- concentration
- 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023191118A JP2024028696A (ja) | 2018-05-14 | 2023-11-08 | 幼若対象におけるａａｖベクターの安定的発現 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862671271P | 2018-05-14 | 2018-05-14 | |
| US62/671,271 | 2018-05-14 | ||
| PCT/US2019/032092 WO2019222132A1 (en) | 2018-05-14 | 2019-05-14 | Stable expression of aav vectors in juvenile subjects |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023191118A Division JP2024028696A (ja) | 2018-05-14 | 2023-11-08 | 幼若対象におけるａａｖベクターの安定的発現 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021523198A JP2021523198A (ja) | 2021-09-02 |
| JP2021523198A5 true JP2021523198A5 (https=) | 2022-05-24 |
Family
ID=66770562
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020564142A Pending JP2021523198A (ja) | 2018-05-14 | 2019-05-14 | 幼若対象におけるａａｖベクターの安定的発現 |
| JP2023191118A Pending JP2024028696A (ja) | 2018-05-14 | 2023-11-08 | 幼若対象におけるａａｖベクターの安定的発現 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023191118A Pending JP2024028696A (ja) | 2018-05-14 | 2023-11-08 | 幼若対象におけるａａｖベクターの安定的発現 |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US20200069819A1 (https=) |
| EP (1) | EP3794112A1 (https=) |
| JP (2) | JP2021523198A (https=) |
| KR (1) | KR20210008491A (https=) |
| CN (1) | CN112424345A (https=) |
| AR (1) | AR117427A1 (https=) |
| AU (1) | AU2019270972A1 (https=) |
| BR (1) | BR112020023159A2 (https=) |
| CA (1) | CA3100000A1 (https=) |
| MX (1) | MX2020012167A (https=) |
| SG (1) | SG11202010832YA (https=) |
| TW (1) | TW202016298A (https=) |
| WO (1) | WO2019222132A1 (https=) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL305149A (en) | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| TW202016314A (zh) | 2018-05-09 | 2020-05-01 | 美商拜奧馬林製藥公司 | 治療苯丙酮尿症之方法 |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| US20230407328A1 (en) | 2020-11-02 | 2023-12-21 | Biomarin Pharmaceutical Inc. | Process for enriching adeno-associated virus |
| WO2023034990A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| JP2024534888A (ja) | 2021-09-03 | 2024-09-26 | ビオマリン プハルマセウトイカル インコーポレイテッド | Ａａｖカプシド組成物及び送達方法 |
| WO2023034980A1 (en) | 2021-09-03 | 2023-03-09 | Bomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| WO2023034996A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| JP2024533174A (ja) | 2021-09-03 | 2024-09-12 | ビオマリン プハルマセウトイカル インコーポレイテッド | Ａａｖカプシド組成物及び送達方法 |
| EP4396203A1 (en) | 2021-09-03 | 2024-07-10 | BioMarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| CN120380156A (zh) | 2022-09-22 | 2025-07-25 | 迪纳柯公司 | 用aav基因治疗载体治疗心肌病 |
| TW202421788A (zh) | 2022-09-22 | 2024-06-01 | 美商拜奧馬林製藥公司 | 用ａａｖ基因療法載體治療致心律不整性心肌病 |
| EP4598590A1 (en) * | 2022-10-08 | 2025-08-13 | Lingyi Biotech Co., Ltd. | Construct for enhancing gene expression |
| 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 |
| WO2025214477A1 (en) | 2024-04-12 | 2025-10-16 | Skyline Therapeutics (Shanghai) Co., Ltd. | Treatment of genetic cardiomyopathies with aav gene therapy vectors |
Family Cites Families (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US298A (en) | 1837-07-29 | Process for purifying salt-water preparatory to manufacturing salt | ||
| US6531A (en) | 1849-06-19 | Beed musical instrument | ||
| US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
| EP0127839B1 (en) | 1983-05-27 | 1992-07-15 | THE TEXAS A&M UNIVERSITY SYSTEM | 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 |
| US4994371A (en) | 1987-08-28 | 1991-02-19 | Davie Earl W | DNA preparation of Christmas factor and use of DNA sequences |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US6531298B2 (en) | 1997-07-21 | 2003-03-11 | The University Of North Carolina At Chapel Hill | Factor IX antihemophilic factor with increased clotting activity |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| AU2003212708A1 (en) | 2002-03-05 | 2003-09-16 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
| US7943374B2 (en) * | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| US7943379B2 (en) | 2008-04-30 | 2011-05-17 | Nationwide Children's Hospital, Inc. | Production of rAAV in vero cells using particular adenovirus helpers |
| TR201813067T4 (tr) | 2008-09-15 | 2018-09-21 | Uniqure Biopharma B V | Faktör IX polipeptit mutantı, bunun kullanımları ve bunu üretmeye yönelik bir yöntem. |
| GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
| EP3044231B1 (en) | 2013-09-12 | 2020-08-05 | BioMarin Pharmaceutical Inc. | Aav vectors comprising a gene encoding factor viii |
| TW202446416A (zh) * | 2015-09-24 | 2024-12-01 | 美商拜奧馬林製藥公司 | 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物 |
| MY189674A (en) * | 2015-10-28 | 2022-02-24 | Sangamo Therapeutics Inc | Liver-specific constructs, factor viii expression cassettes and methods of use thereof |
| PE20231949A1 (es) | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| NZ756587A (en) * | 2017-03-15 | 2025-12-19 | Univ North Carolina Chapel Hill | Polyploid adeno-associated virus vectors and methods of making and using the same |
-
2019
- 2019-05-14 TW TW108116635A patent/TW202016298A/zh unknown
- 2019-05-14 AU AU2019270972A patent/AU2019270972A1/en not_active Abandoned
- 2019-05-14 EP EP19728796.4A patent/EP3794112A1/en not_active Withdrawn
- 2019-05-14 MX MX2020012167A patent/MX2020012167A/es unknown
- 2019-05-14 CN CN201980047182.9A patent/CN112424345A/zh active Pending
- 2019-05-14 WO PCT/US2019/032092 patent/WO2019222132A1/en not_active Ceased
- 2019-05-14 CA CA3100000A patent/CA3100000A1/en active Pending
- 2019-05-14 AR ARP190101290A patent/AR117427A1/es not_active Application Discontinuation
- 2019-05-14 BR BR112020023159-2A patent/BR112020023159A2/pt unknown
- 2019-05-14 SG SG11202010832YA patent/SG11202010832YA/en unknown
- 2019-05-14 US US16/411,841 patent/US20200069819A1/en not_active Abandoned
- 2019-05-14 KR KR1020207034760A patent/KR20210008491A/ko not_active Withdrawn
- 2019-05-14 JP JP2020564142A patent/JP2021523198A/ja active Pending
-
2023
- 2023-11-08 JP JP2023191118A patent/JP2024028696A/ja active Pending
-
2024
- 2024-05-13 US US18/662,491 patent/US20240285806A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2021523198A5 (https=) | ||
| Gane et al. | Evaluation of RNAi therapeutics VIR-2218 and ALN-HBV for chronic hepatitis B: results from randomized clinical trials | |
| Day et al. | Advances and limitations for the treatment of spinal muscular atrophy | |
| Martinez et al. | Can we cure hepatitis B virus with novel direct‐acting antivirals? | |
| TWI620568B (zh) | 用於治療ｂ型肝炎及ｄ型肝炎感染之方法 | |
| JP2018535929A5 (https=) | ||
| Andrade | Intranasal drug delivery in neuropsychiatry: focus on intranasal ketamine for refractory depression | |
| CN109467566B (zh) | 用于治疗和/或预防与肝炎病毒相关的疾病或病症的双二氮杂双环化合物 | |
| KR20170029577A (ko) | B형 간염 및 d형 간염 바이러스 감염의 치료 방법 | |
| CA2817787C (en) | Composition comprising a peptide and an inhibitor of viral neuraminidase | |
| JPWO2018193902A1 (ja) | マイクロｒｎａによるｂ型肝炎ウイルスに対する抗ウイルス効果 | |
| CN114729360A (zh) | 降低病毒分子水平的方法 | |
| Marrapu et al. | Hepatitis B functional cure: Current and future perspective | |
| Ho et al. | Targeting the innate immune system in treating hepatitis B: prospects for functional cure | |
| JP2023513487A5 (https=) | ||
| JP7850132B2 (ja) | ＳＡＲＳ－ＣｏＶ－２感染患者におけるアウトカムを改善するための吸入インターフェロン－ベータ | |
| JP2022518354A5 (https=) | ||
| RU2020140634A (ru) | Стабильная экспрессия векторов на основе аденоассоциированного вируса у несовершеннолетних пациентов | |
| RU2023105585A (ru) | Стабильная экспрессия векторов на основе аденоассоциированного вируса у несовершеннолетних пациентов | |
| RU2595862C2 (ru) | Способ профилактики или лечения заболеваний, связанных с пониженной плотностью интерфероновых рецепторов | |
| CN115835877A (zh) | 病毒感染引起的器官衰竭的预防和治疗 | |
| Mtewa et al. | SARS-CoV-2 vaccine development | |
| Shafer | A SARS-CoV-2 antiviral therapy score card | |
| RU2668538C1 (ru) | Способ восстановления плотности интерфероновых рецепторов для профилактики или лечения заболеваний, связанных с пониженной плотностью интерфероновых рецепторов | |
| Harford et al. | TP53 Gene Therapy as a Potential Treatment for Patients with COVID-19. Viruses 2022, 14, 739 |