JP2023553005A5 - - Google Patents
Info
- Publication number
- JP2023553005A5 JP2023553005A5 JP2023534050A JP2023534050A JP2023553005A5 JP 2023553005 A5 JP2023553005 A5 JP 2023553005A5 JP 2023534050 A JP2023534050 A JP 2023534050A JP 2023534050 A JP2023534050 A JP 2023534050A JP 2023553005 A5 JP2023553005 A5 JP 2023553005A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- viral vector
- composition according
- pmp22
- nucleic acid
- 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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063120190P | 2020-12-01 | 2020-12-01 | |
| US63/120,190 | 2020-12-01 | ||
| PCT/US2021/061177 WO2022119826A1 (en) | 2020-12-01 | 2021-11-30 | Products and methods for inhibition of expression of peripheral myelin protein-22 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023553005A JP2023553005A (ja) | 2023-12-20 |
| JP2023553005A5 true JP2023553005A5 (https=) | 2024-11-18 |
Family
ID=81853504
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023534050A Pending JP2023553005A (ja) | 2020-12-01 | 2021-11-30 | 末梢ミエリンタンパク質２２の発現の阻害のための生成物及び方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20240318172A1 (https=) |
| EP (1) | EP4256069A4 (https=) |
| JP (1) | JP2023553005A (https=) |
| KR (1) | KR20230134477A (https=) |
| CN (1) | CN116917492A (https=) |
| AU (1) | AU2021390471A1 (https=) |
| CA (1) | CA3203748A1 (https=) |
| IL (1) | IL303308A (https=) |
| WO (1) | WO2022119826A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20240103029A (ko) * | 2021-11-18 | 2024-07-03 | 노파르티스 아게 | 샤르코 마리 투스 질환 치료용 pmp22 표적화 화합물 |
| WO2024229211A2 (en) | 2023-05-02 | 2024-11-07 | Research Institute At Nationwide Children's Hospital | A modular system to convert therapeutic microrna expression cassettes from polymerase iii-based to polymerase ii-based promoters |
| WO2025150052A1 (en) * | 2024-01-12 | 2025-07-17 | Potentia Ltd. | Targeted delivery of drug conjugates to schwann cells and treatment methods in schwann cell-related diseases |
| WO2025242147A1 (zh) * | 2024-05-21 | 2025-11-27 | 北京安龙生物医药有限公司 | 靶向外周髓鞘蛋白-22基因的寡核苷酸及其用途 |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2012145597A2 (en) * | 2011-04-21 | 2012-10-26 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of myotilin |
| WO2017156242A1 (en) * | 2016-03-09 | 2017-09-14 | Ionis Pharmaceuticals, Inc. | Methods and compositions for inhibiting pmp22 expression |
| JP7305534B2 (ja) * | 2016-07-06 | 2023-07-10 | バーテックス ファーマシューティカルズ インコーポレイテッド | 疼痛関連障害を処置するための物質及び方法 |
| KR101872105B1 (ko) * | 2016-09-05 | 2018-06-27 | 사회복지법인 삼성생명공익재단 | 샤르코 마리 투스병 치료용 약학 조성물 |
| AU2018224380A1 (en) * | 2017-02-22 | 2019-08-29 | Crispr Therapeutics Ag | Compositions and methods for treatment of proprotein convertase subtilisin/kexin type 9 (PCSK9)-related disorders |
| JP2020536501A (ja) * | 2017-09-28 | 2020-12-17 | ツールゲン インコーポレイテッドＴｏｏｌｇｅｎ Ｉｎｃｏｒｐｏｒａｔｅｄ | 遺伝子発現調節のための人為的なゲノム操作 |
| WO2020047268A1 (en) * | 2018-08-29 | 2020-03-05 | Research Institute At Nationwide Children's Hospital | Products and methods for inhibition of expression of mutant gars protein |
| EP3628735A1 (en) * | 2018-09-25 | 2020-04-01 | Centre National De La Recherche Scientifique | Antisense rna targeting pmp22 for the treatment of charcot-marie-tooth 1a disease |
| JP7561129B2 (ja) * | 2018-12-21 | 2024-10-03 | アイオーニス ファーマシューティカルズ， インコーポレーテッド | Ｐｍｐ２２の発現を低減するための化合物及び方法 |
| KR20200124038A (ko) * | 2019-04-23 | 2020-11-02 | 사회복지법인 삼성생명공익재단 | 마이크로rna-381을 유효성분으로 포함하는, pmp22 과발현에 의한 말초신경병증의 예방 또는 치료용 약학적 조성물 |
-
2021
- 2021-11-30 US US18/038,900 patent/US20240318172A1/en active Pending
- 2021-11-30 CA CA3203748A patent/CA3203748A1/en active Pending
- 2021-11-30 KR KR1020237022382A patent/KR20230134477A/ko active Pending
- 2021-11-30 WO PCT/US2021/061177 patent/WO2022119826A1/en not_active Ceased
- 2021-11-30 CN CN202180092622.XA patent/CN116917492A/zh active Pending
- 2021-11-30 JP JP2023534050A patent/JP2023553005A/ja active Pending
- 2021-11-30 AU AU2021390471A patent/AU2021390471A1/en active Pending
- 2021-11-30 IL IL303308A patent/IL303308A/en unknown
- 2021-11-30 EP EP21901321.6A patent/EP4256069A4/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2023553005A5 (https=) | ||
| JP7395550B2 (ja) | 新規アデノ随伴ウイルスキャプシドタンパク質 | |
| US11939597B2 (en) | Restrictive inverted terminal repeats for viral vectors | |
| US20220331409A1 (en) | Factor ix gene therapy | |
| JP2020019772A5 (https=) | ||
| JP2020522269A5 (https=) | ||
| JP2025162557A5 (https=) | ||
| JP2007507223A5 (https=) | ||
| IL320714A (en) | Gene therapy for limb-girdle muscular dystrophy type 2c | |
| IL320791A (en) | Muscle-specific delivery of microdystrophin using an adeno-associated virus vector for the treatment of muscular dystrophy | |
| CN112424217A (zh) | 肝脏靶向的腺病毒相关病毒载体 | |
| JP2017510298A5 (https=) | ||
| JP2021534755A5 (https=) | ||
| JP2021534794A5 (https=) | ||
| JP2023524414A5 (https=) | ||
| JP2022530095A5 (https=) | ||
| JP2025539750A (ja) | 前頭側頭型認知症の遺伝子治療 | |
| JP2024543211A5 (https=) | ||
| JP2025540074A (ja) | 繰り返しエレメントの適切なパッケージングのためのアデノ随伴ウイルスベクター | |
| JP2023529371A5 (https=) | ||
| AU2022282057B2 (en) | Novel dual helper plasmid | |
| JP2024514160A5 (https=) | ||
| JP2022522166A5 (https=) | ||
| JP2023541444A5 (https=) | ||
| JP2025509262A5 (https=) |