JPWO2023177885A5 - - Google Patents
Info
- Publication number
- JPWO2023177885A5 JPWO2023177885A5 JP2024555349A JP2024555349A JPWO2023177885A5 JP WO2023177885 A5 JPWO2023177885 A5 JP WO2023177885A5 JP 2024555349 A JP2024555349 A JP 2024555349A JP 2024555349 A JP2024555349 A JP 2024555349A JP WO2023177885 A5 JPWO2023177885 A5 JP WO2023177885A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- aav vector
- recombinant aav
- nos
- signal peptide
- 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
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263321336P | 2022-03-18 | 2022-03-18 | |
| US63/321,336 | 2022-03-18 | ||
| US202263348862P | 2022-06-03 | 2022-06-03 | |
| US63/348,862 | 2022-06-03 | ||
| US202363444804P | 2023-02-10 | 2023-02-10 | |
| US63/444,804 | 2023-02-10 | ||
| PCT/US2023/015531 WO2023177885A2 (en) | 2022-03-18 | 2023-03-17 | Therapeutic adeno-associated virus using codon optimized nucleic acid encoding alpha-glucosidase (gaa) for treating pompe disease, with signal peptide modifications |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2025510017A JP2025510017A (ja) | 2025-04-14 |
| JP2025510017A5 JP2025510017A5 (https=) | 2026-03-26 |
| JPWO2023177885A5 true JPWO2023177885A5 (https=) | 2026-03-26 |
Family
ID=88024211
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024555349A Pending JP2025510017A (ja) | 2022-03-18 | 2023-03-17 | シグナルペプチド改変を有する、ポンペ病を処置するためのアルファ－グルコシダーゼ（ｇａａ）をコードするコドン最適化核酸を使用する治療用アデノ随伴ウイルス |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20250213726A1 (https=) |
| EP (1) | EP4493704A2 (https=) |
| JP (1) | JP2025510017A (https=) |
| KR (1) | KR20250005569A (https=) |
| CN (1) | CN119213137A (https=) |
| AU (1) | AU2023235203A1 (https=) |
| CA (1) | CA3254578A1 (https=) |
| IL (1) | IL315711A (https=) |
| MX (1) | MX2024011396A (https=) |
| TW (1) | TW202403048A (https=) |
| WO (1) | WO2023177885A2 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2023164060A2 (en) * | 2022-02-25 | 2023-08-31 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease with long term cessation of gaa enzyme replacement therapy |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ATE521701T1 (de) * | 2003-01-22 | 2011-09-15 | Univ Duke | Verbesserte konstrukte zur expression lysosomaler polypeptide |
| EP3293259A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| WO2020102667A2 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence |
| CA3159018A1 (en) * | 2019-11-19 | 2021-05-27 | Michael W. O'CALLAGHAN | Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders |
-
2023
- 2023-03-17 EP EP23771464.7A patent/EP4493704A2/en active Pending
- 2023-03-17 TW TW112110096A patent/TW202403048A/zh unknown
- 2023-03-17 KR KR1020247032558A patent/KR20250005569A/ko active Pending
- 2023-03-17 CN CN202380041220.6A patent/CN119213137A/zh active Pending
- 2023-03-17 CA CA3254578A patent/CA3254578A1/en active Pending
- 2023-03-17 JP JP2024555349A patent/JP2025510017A/ja active Pending
- 2023-03-17 WO PCT/US2023/015531 patent/WO2023177885A2/en not_active Ceased
- 2023-03-17 AU AU2023235203A patent/AU2023235203A1/en active Pending
- 2023-03-17 US US18/848,389 patent/US20250213726A1/en active Pending
- 2023-03-17 IL IL315711A patent/IL315711A/en unknown
-
2024
- 2024-09-17 MX MX2024011396A patent/MX2024011396A/es unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20230048025A1 (en) | Adeno associated viral vectors | |
| CN113316639A (zh) | 用于治疗庞贝氏病的治疗性腺相关病毒 | |
| IL299325B1 (en) | Variants of acid alpha-glucosidase and their uses | |
| JP7654562B2 (ja) | 線維芽細胞増殖因子２３関連低リン血症性疾患の遺伝子療法 | |
| JP2021533805A5 (https=) | ||
| JPWO2021102107A5 (https=) | ||
| WO2023060269A4 (en) | Recombinant adeno-associated viruses for targeted delivery | |
| CN114829391A (zh) | 用肝特异性基因疗法载体治疗遗传性血管性水肿 | |
| CA3151442A1 (en) | Sortilin receptor ligand based chimeric polypeptides and uses thereof | |
| IL315711A (en) | Therapeutic adeno-associated virus using codon optimized nucleic acid encoding alpha-glucosidase (gaa) for treating pompe disease, with signal peptide modifications | |
| CN119173269A (zh) | 用于递送glp-1受体激动剂融合物的aav载体 | |
| JPWO2023177885A5 (https=) | ||
| JPWO2020041773A5 (https=) | ||
| JPWO2020167996A5 (https=) | ||
| JPWO2021226267A5 (https=) | ||
| JP7745307B2 (ja) | 連結ペプチド、連結ペプチドを含む血液凝固ｖｉｉｉ因子タンパク質又はその変異体及びその用途 | |
| CN107073078A (zh) | 利用肝细胞生长因子和基质细胞衍生因子1α的用于预防或治疗外周动脉疾病的组合物 | |
| JPWO2021108809A5 (https=) | ||
| KR20240049295A (ko) | 방사선 유발 타액 기능저하를 예방하기 위한 aqp1 유전자 요법 | |
| WO2021078834A1 (en) | Chimeric acid-alpha glucosidase polypeptides and uses thereof | |
| JPWO2020237130A5 (https=) | ||
| JP2612362B2 (ja) | トリコデルマ・ビリデ由来のｄｎａ断片 | |
| JP2022514271A5 (https=) | ||
| TWI920044B (zh) | 用於治療龐貝症及溶酶體病症之包含肝特異性啟動子之治療性腺相關病毒 | |
| KR20250143161A (ko) | 시누클레인병증의 치료를 위한 유전자 요법 |