JP2022522166A - 眼咽頭型筋ジストロフィー(opmd)を治療するための組成物及び方法 - Google Patents
眼咽頭型筋ジストロフィー(opmd)を治療するための組成物及び方法 Download PDFInfo
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- JP2022522166A JP2022522166A JP2021549908A JP2021549908A JP2022522166A JP 2022522166 A JP2022522166 A JP 2022522166A JP 2021549908 A JP2021549908 A JP 2021549908A JP 2021549908 A JP2021549908 A JP 2021549908A JP 2022522166 A JP2022522166 A JP 2022522166A
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
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- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
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- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
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- C12N2310/53—Physical structure partially self-complementary or closed
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- C12N2750/14011—Parvoviridae
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962812187P | 2019-02-28 | 2019-02-28 | |
| US62/812,187 | 2019-02-28 | ||
| PCT/AU2020/050182 WO2020172720A1 (en) | 2019-02-28 | 2020-02-28 | Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd) |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022522166A true JP2022522166A (ja) | 2022-04-14 |
| JP2022522166A5 JP2022522166A5 (https=) | 2023-03-03 |
| JPWO2020172720A5 JPWO2020172720A5 (https=) | 2023-03-03 |
Family
ID=72238736
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021549908A Pending JP2022522166A (ja) | 2019-02-28 | 2020-02-28 | 眼咽頭型筋ジストロフィー(opmd)を治療するための組成物及び方法 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20220098614A1 (https=) |
| EP (1) | EP3931317A4 (https=) |
| JP (1) | JP2022522166A (https=) |
| CN (2) | CN121160804A (https=) |
| AU (1) | AU2020229886B2 (https=) |
| BR (1) | BR112021017054A2 (https=) |
| CA (1) | CA3130221A1 (https=) |
| IL (1) | IL285841A (https=) |
| MX (1) | MX2021010412A (https=) |
| SG (1) | SG11202108469XA (https=) |
| WO (1) | WO2020172720A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN112867511A (zh) * | 2018-10-17 | 2021-05-28 | 贝尼泰克生物制药有限公司 | 用于治疗眼咽肌营养不良(opmd)的方法 |
| KR20260030147A (ko) * | 2023-06-28 | 2026-03-05 | 베니텍 아이피 홀딩스 아이엔씨. | 인두 근육에 치료 조성물을 투여하기 위한 장치 및 방법 |
| WO2026011009A1 (en) * | 2024-07-02 | 2026-01-08 | Kate Therapeutics, Inc. | Compositions and methods for muscle disorders |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018139634A1 (ja) * | 2017-01-30 | 2018-08-02 | 学校法人日本医科大学 | アデノ随伴ウイルス(aav)キャプシドタンパク質の変異体 |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
| EP1983057A3 (en) | 1995-09-08 | 2009-01-07 | Genzyme Corporation | Improved AAV vectors for gene therapy |
| US7208315B2 (en) | 2000-01-05 | 2007-04-24 | Fred Hutchinson Cancer Research Center | Compositions and methods for efficient AAV vector production |
| EP2017338A1 (en) | 2001-05-24 | 2009-01-21 | Genzyme Corporation | Muscle-specific expression vectors |
| WO2003042361A2 (en) | 2001-11-09 | 2003-05-22 | Government Of The United States Of America, Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| 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 |
| WO2003074714A1 (en) | 2002-03-05 | 2003-09-12 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
| WO2005072364A2 (en) * | 2004-01-27 | 2005-08-11 | University Of Florida | A modified baculovirus expression system for production of pseudotyped raav vector |
| US8008468B2 (en) | 2005-02-16 | 2011-08-30 | Benitec, Inc. | RNAi expression constructs with liver-specific enhancer/promoter |
| EP2311967B1 (en) | 2005-10-20 | 2017-09-20 | UniQure IP B.V. | Improved AAV vectors produced in insect cells |
| WO2007120533A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Minigene expression cassette |
| EP2018421B1 (en) | 2006-04-28 | 2012-12-19 | The Trustees of the University of Pennsylvania | Scalable production method for aav |
| KR101589259B1 (ko) | 2006-06-21 | 2016-02-01 | 유니큐어 아이피 비.브이. | 곤충세포 내 aav의 생산에 유용한 aav-rep78의 번역을 위한 변형된 개시 코돈을 갖는 벡터 |
| DK2242840T3 (da) | 2008-01-29 | 2019-10-21 | Applied Genetic Tech Corporation | Produktion af rekombinant adeno-associeret virus under anvendelse af bhk-celler i suspension |
| EP3024497B1 (en) * | 2013-07-26 | 2021-01-13 | University of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| EP3039129B1 (en) | 2013-08-30 | 2018-06-27 | Amgen Inc. | High titer recombinant aav vector production in adherent and suspension cells |
| CN104815338A (zh) * | 2015-05-01 | 2015-08-05 | 许瑞安 | 一种携带kal基因重组腺相关病毒载体的基因治疗药物 |
| KR20230079511A (ko) | 2016-04-21 | 2023-06-07 | 바이로베크 인코포레이티드 | 곤충 세포에서의 aav 생산, 및 이를 위한 방법 및 조성물 |
| EP3555294A4 (en) * | 2016-12-14 | 2020-08-26 | Benitec Biopharma Limited | REAGENTS FOR THE TREATMENT OF OCULOPHARYNGEAL MUSCLE DYSTROPHY (PDD) AND THEIR USE |
| UY37863A (es) | 2017-08-31 | 2019-03-29 | Benitec Biopharma Ltd | Virus adenoasociado (aav) con dominio de fosfolipasa modificado |
-
2020
- 2020-02-28 US US17/434,666 patent/US20220098614A1/en active Pending
- 2020-02-28 CN CN202511142034.9A patent/CN121160804A/zh active Pending
- 2020-02-28 WO PCT/AU2020/050182 patent/WO2020172720A1/en not_active Ceased
- 2020-02-28 JP JP2021549908A patent/JP2022522166A/ja active Pending
- 2020-02-28 BR BR112021017054A patent/BR112021017054A2/pt unknown
- 2020-02-28 SG SG11202108469XA patent/SG11202108469XA/en unknown
- 2020-02-28 EP EP20762817.3A patent/EP3931317A4/en active Pending
- 2020-02-28 CA CA3130221A patent/CA3130221A1/en active Pending
- 2020-02-28 AU AU2020229886A patent/AU2020229886B2/en active Active
- 2020-02-28 MX MX2021010412A patent/MX2021010412A/es unknown
- 2020-02-28 CN CN202080017400.7A patent/CN113631706A/zh active Pending
-
2021
- 2021-08-24 IL IL285841A patent/IL285841A/en unknown
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018139634A1 (ja) * | 2017-01-30 | 2018-08-02 | 学校法人日本医科大学 | アデノ随伴ウイルス(aav)キャプシドタンパク質の変異体 |
Non-Patent Citations (1)
| Title |
|---|
| MOLECULAR THERAPY, vol. Vol. 26, No. 5S1, JPN6024004474, 2018, pages 165, ISSN: 0005255281 * |
Also Published As
| Publication number | Publication date |
|---|---|
| CA3130221A1 (en) | 2020-09-03 |
| AU2020229886B2 (en) | 2025-09-18 |
| KR20210138030A (ko) | 2021-11-18 |
| IL285841A (en) | 2021-10-31 |
| CN113631706A (zh) | 2021-11-09 |
| EP3931317A4 (en) | 2023-01-04 |
| AU2020229886A1 (en) | 2021-09-09 |
| WO2020172720A1 (en) | 2020-09-03 |
| US20220098614A1 (en) | 2022-03-31 |
| SG11202108469XA (en) | 2021-09-29 |
| CN121160804A (zh) | 2025-12-19 |
| BR112021017054A2 (pt) | 2021-11-09 |
| MX2021010412A (es) | 2022-01-24 |
| EP3931317A1 (en) | 2022-01-05 |
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