JPWO2020210698A5 - - Google Patents
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- JPWO2020210698A5 JPWO2020210698A5 JP2021559727A JP2021559727A JPWO2020210698A5 JP WO2020210698 A5 JPWO2020210698 A5 JP WO2020210698A5 JP 2021559727 A JP2021559727 A JP 2021559727A JP 2021559727 A JP2021559727 A JP 2021559727A JP WO2020210698 A5 JPWO2020210698 A5 JP WO2020210698A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- raav
- itr
- vector
- composition according
- 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
- 239000013608 rAAV vector Substances 0.000 claims description 11
- 108020004707 nucleic acids Proteins 0.000 claims description 9
- 102000039446 nucleic acids Human genes 0.000 claims description 9
- 150000007523 nucleic acids Chemical class 0.000 claims description 9
- 239000013598 vector Substances 0.000 claims description 9
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims description 8
- 239000002773 nucleotide Substances 0.000 claims description 8
- 125000003729 nucleotide group Chemical group 0.000 claims description 8
- 108090000565 Capsid Proteins Proteins 0.000 claims description 5
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 5
- 108090000623 proteins and genes Proteins 0.000 claims description 4
- 108010006025 bovine growth hormone Proteins 0.000 claims description 3
- 239000003623 enhancer Substances 0.000 claims description 3
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims description 2
- 230000001124 posttranscriptional effect Effects 0.000 claims description 2
- 230000001105 regulatory effect Effects 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 18
- 108700019146 Transgenes Proteins 0.000 claims 8
- 102000019204 Progranulins Human genes 0.000 claims 7
- 108010012809 Progranulins Proteins 0.000 claims 7
- 210000001175 cerebrospinal fluid Anatomy 0.000 claims 5
- TWRXJAOTZQYOKJ-UHFFFAOYSA-L Magnesium chloride Chemical compound [Mg+2].[Cl-].[Cl-] TWRXJAOTZQYOKJ-UHFFFAOYSA-L 0.000 claims 4
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims 4
- 239000000203 mixture Substances 0.000 claims 4
- 239000000523 sample Substances 0.000 claims 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 3
- 241000701022 Cytomegalovirus Species 0.000 claims 3
- 201000011240 Frontotemporal dementia Diseases 0.000 claims 3
- 102100037632 Progranulin Human genes 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- 230000035772 mutation Effects 0.000 claims 3
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- CTKXFMQHOOWWEB-UHFFFAOYSA-N Ethylene oxide/propylene oxide copolymer Chemical compound CCCOC(C)COCCO CTKXFMQHOOWWEB-UHFFFAOYSA-N 0.000 claims 2
- 239000007983 Tris buffer Substances 0.000 claims 2
- VHJLVAABSRFDPM-QWWZWVQMSA-N dithiothreitol Chemical compound SC[C@@H](O)[C@H](O)CS VHJLVAABSRFDPM-QWWZWVQMSA-N 0.000 claims 2
- 238000003018 immunoassay Methods 0.000 claims 2
- 229910001629 magnesium chloride Inorganic materials 0.000 claims 2
- 229920001993 poloxamer 188 Polymers 0.000 claims 2
- 229940044519 poloxamer 188 Drugs 0.000 claims 2
- 239000011780 sodium chloride Substances 0.000 claims 2
- LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 claims 2
- 238000001262 western blot Methods 0.000 claims 2
- 238000007865 diluting Methods 0.000 claims 1
- HWYHZTIRURJOHG-UHFFFAOYSA-N luminol Chemical compound O=C1NNC(=O)C2=C1C(N)=CC=C2 HWYHZTIRURJOHG-UHFFFAOYSA-N 0.000 claims 1
- 150000002978 peroxides Chemical class 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 239000012723 sample buffer Substances 0.000 claims 1
- 101000997662 Homo sapiens Lysosomal acid glucosylceramidase Proteins 0.000 description 3
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 description 3
- 102100033342 Lysosomal acid glucosylceramidase Human genes 0.000 description 3
- 238000004806 packaging method and process Methods 0.000 description 3
- 108091026890 Coding region Proteins 0.000 description 2
- 101150003696 gba-1 gene Proteins 0.000 description 2
- HVCOBJNICQPDBP-UHFFFAOYSA-N 3-[3-[3,5-dihydroxy-6-methyl-4-(3,4,5-trihydroxy-6-methyloxan-2-yl)oxyoxan-2-yl]oxydecanoyloxy]decanoic acid;hydrate Chemical compound O.OC1C(OC(CC(=O)OC(CCCCCCC)CC(O)=O)CCCCCCC)OC(C)C(O)C1OC1C(O)C(O)C(O)C(C)O1 HVCOBJNICQPDBP-UHFFFAOYSA-N 0.000 description 1
- YBSQGNFRWZKFMJ-UHFFFAOYSA-N Cerebroside B Natural products CCCCCCCCCCCCCCC(O)C(=O)NC(C(O)C=CCCC=C(C)CCCCCCCCC)COC1OC(CO)C(O)C(O)C1O YBSQGNFRWZKFMJ-UHFFFAOYSA-N 0.000 description 1
- 108020004414 DNA Proteins 0.000 description 1
- 108010017544 Glucosylceramidase Proteins 0.000 description 1
- 229930186217 Glycolipid Natural products 0.000 description 1
- 241000282412 Homo Species 0.000 description 1
- 101000934372 Homo sapiens Macrosialin Proteins 0.000 description 1
- 108091092195 Intron Proteins 0.000 description 1
- 102100025136 Macrosialin Human genes 0.000 description 1
- 102000009572 RNA Polymerase II Human genes 0.000 description 1
- 108010009460 RNA Polymerase II Proteins 0.000 description 1
- 108091093126 WHP Posttrascriptional Response Element Proteins 0.000 description 1
- 210000000349 chromosome Anatomy 0.000 description 1
- 238000010586 diagram Methods 0.000 description 1
- 102000045630 human GBA Human genes 0.000 description 1
- 210000005260 human cell Anatomy 0.000 description 1
- 229930027917 kanamycin Natural products 0.000 description 1
- SBUJHOSQTJFQJX-NOAMYHISSA-N kanamycin Chemical compound O[C@@H]1[C@@H](O)[C@H](O)[C@@H](CN)O[C@@H]1O[C@H]1[C@H](O)[C@@H](O[C@@H]2[C@@H]([C@@H](N)[C@H](O)[C@@H](CO)O2)O)[C@H](N)C[C@@H]1N SBUJHOSQTJFQJX-NOAMYHISSA-N 0.000 description 1
- 229960000318 kanamycin Drugs 0.000 description 1
- 229930182823 kanamycin A Natural products 0.000 description 1
- 108010045758 lysosomal proteins Proteins 0.000 description 1
- 210000004962 mammalian cell Anatomy 0.000 description 1
- 230000004060 metabolic process Effects 0.000 description 1
- 108090000765 processed proteins & peptides Proteins 0.000 description 1
- 239000000126 substance Substances 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024176952A JP2025016496A (ja) | 2019-04-10 | 2024-10-09 | リソソーム障害のための遺伝子療法 |
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962831846P | 2019-04-10 | 2019-04-10 | |
| US62/831,846 | 2019-04-10 | ||
| US201962934450P | 2019-11-12 | 2019-11-12 | |
| US62/934,450 | 2019-11-12 | ||
| US201962954089P | 2019-12-27 | 2019-12-27 | |
| US62/954,089 | 2019-12-27 | ||
| US202062960471P | 2020-01-13 | 2020-01-13 | |
| US62/960,471 | 2020-01-13 | ||
| US202062988665P | 2020-03-12 | 2020-03-12 | |
| US62/988,665 | 2020-03-12 | ||
| PCT/US2020/027764 WO2020210698A1 (en) | 2019-04-10 | 2020-04-10 | Gene therapies for lysosomal disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024176952A Division JP2025016496A (ja) | 2019-04-10 | 2024-10-09 | リソソーム障害のための遺伝子療法 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022526823A JP2022526823A (ja) | 2022-05-26 |
| JPWO2020210698A5 true JPWO2020210698A5 (https=) | 2024-01-19 |
| JP2022526823A5 JP2022526823A5 (https=) | 2024-01-19 |
| JP7571044B2 JP7571044B2 (ja) | 2024-10-22 |
Family
ID=70554180
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021559727A Active JP7571044B2 (ja) | 2019-04-10 | 2020-04-10 | リソソーム障害のための遺伝子療法 |
| JP2024176952A Pending JP2025016496A (ja) | 2019-04-10 | 2024-10-09 | リソソーム障害のための遺伝子療法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024176952A Pending JP2025016496A (ja) | 2019-04-10 | 2024-10-09 | リソソーム障害のための遺伝子療法 |
Country Status (20)
| Country | Link |
|---|---|
| US (2) | US11999974B2 (https=) |
| EP (2) | EP3953377B1 (https=) |
| JP (2) | JP7571044B2 (https=) |
| KR (1) | KR20210150486A (https=) |
| CN (1) | CN114174324A (https=) |
| AU (2) | AU2020273182B2 (https=) |
| BR (1) | BR112021020183A2 (https=) |
| CA (1) | CA3136004A1 (https=) |
| DK (1) | DK3953377T3 (https=) |
| ES (1) | ES3055985T3 (https=) |
| FI (1) | FI3953377T3 (https=) |
| HR (1) | HRP20251506T1 (https=) |
| IL (1) | IL286903A (https=) |
| LT (1) | LT3953377T (https=) |
| MX (1) | MX2021011958A (https=) |
| PL (1) | PL3953377T3 (https=) |
| PT (1) | PT3953377T (https=) |
| RS (1) | RS67476B1 (https=) |
| SI (1) | SI3953377T1 (https=) |
| WO (1) | WO2020210698A1 (https=) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN110506057B (zh) | 2017-02-17 | 2023-09-29 | 百时美施贵宝公司 | Alpha突触核蛋白抗体及其应用 |
| BR112020006671A2 (pt) | 2017-10-03 | 2020-12-01 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| PL3953377T3 (pl) | 2019-04-10 | 2026-02-16 | Prevail Therapeutics, Inc. | Terapie genowe zaburzeń lizosomalnych |
| US20230346979A1 (en) * | 2020-08-10 | 2023-11-02 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
| WO2023202637A1 (en) | 2022-04-19 | 2023-10-26 | Shanghai Vitalgen Biopharma Co., Ltd. | Recombinant aav vectors for treating neurodegenerative disorders |
| KR20250131772A (ko) * | 2022-11-13 | 2025-09-03 | 알렉시온 파마 인터내셔널 오퍼레이션즈 리미티드 | 전두측두엽 치매에 대한 유전자 요법 |
| EP4669361A2 (en) * | 2023-02-24 | 2025-12-31 | The Board of Trustees of the Leland Stanford Junior University | BMP SYNTHASE ACTIVATORS AND INHIBITORS |
| WO2025251041A1 (en) * | 2024-05-31 | 2025-12-04 | Bluerock Therapeutics Lp | METHODS AND COMPOSITIONS FOR TREATING PROGRANULIN DEFICIENCIES USING iPSC-DERIVED CELLS |
Family Cites Families (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6538119B2 (en) | 1997-11-18 | 2003-03-25 | Abbott Laboratories | Reagents and methods useful for detecting diseases of the breast |
| 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 |
| AU2001290984A1 (en) | 2000-09-18 | 2002-04-02 | Genzyme Corporation | Expression vectors containing hybrid ubiquitin promoters |
| 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 |
| US20030133924A1 (en) | 2001-12-21 | 2003-07-17 | Novazyme Pharmaceuticals, Inc. | Highly phosphorylated acid beta-glucocerebrosidase and methods of treating gaucher's disease |
| AU2003221733A1 (en) | 2002-04-17 | 2003-11-03 | University Of Florida Research Foundation, Inc. | Improved raav vectors |
| WO2006036465A2 (en) | 2004-09-03 | 2006-04-06 | University Of Florida | Compositions and methods for treating cystic fibrosis |
| EP2687223B2 (en) | 2006-05-30 | 2024-11-20 | Mayo Foundation For Medical Education And Research | Detecting and treating dementia |
| CA2653974A1 (en) | 2006-05-30 | 2008-02-14 | Mayo Foundation For Medical Education And Research | Detecting and treating dementia |
| WO2007146046A2 (en) | 2006-06-07 | 2007-12-21 | Genzyme Corporation | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
| ES2385679T3 (es) | 2006-08-24 | 2012-07-30 | Virovek, Inc. | Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos |
| WO2009079399A2 (en) | 2007-12-14 | 2009-06-25 | Alnylam Pharmaceuticals, Inc. | Method of treating neurodegenerative disease |
| WO2009089635A1 (en) | 2008-01-16 | 2009-07-23 | Neurodyn, Inc. | Treating neurodegenerative diseases with progranulin (pgrn) |
| WO2009120978A2 (en) | 2008-03-27 | 2009-10-01 | The Ohio State University | Treatment of metabolic-related disorders using hypothalamic gene transfer of bdnf and compositions therfor |
| 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 |
| US20150352185A1 (en) | 2010-11-16 | 2015-12-10 | Denis G. Kay | Method for increasing neprilysin expression and activity |
| JP2016503405A (ja) | 2012-11-05 | 2016-02-04 | ジェンザイム・コーポレーション | タンパク質症を処置するための組成物および方法 |
| CN105377039A (zh) | 2013-05-15 | 2016-03-02 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| WO2016025523A1 (en) | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting and uses thereof |
| WO2016081927A2 (en) | 2014-11-21 | 2016-05-26 | University Of Florida Research Foundation, Inc. | Genome-modified recombinant adeno-associated virus vectors |
| EP3292206B8 (en) | 2015-05-07 | 2022-02-09 | Takeda Pharmaceutical Company Limited | Glucocerebrosidase gene therapy for parkinson's disease |
| PL3411484T3 (pl) | 2016-02-05 | 2024-02-19 | Emory University | Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego |
| WO2017151884A1 (en) | 2016-03-02 | 2017-09-08 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
| WO2017178826A1 (en) * | 2016-04-14 | 2017-10-19 | The University Of Liverpool | Diagnosing acute bacterial meningitis |
| KR20230079511A (ko) | 2016-04-21 | 2023-06-07 | 바이로베크 인코포레이티드 | 곤충 세포에서의 aav 생산, 및 이를 위한 방법 및 조성물 |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| BR112020006671A2 (pt) | 2017-10-03 | 2020-12-01 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| EP3692075A4 (en) | 2017-10-03 | 2022-02-09 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISEASES |
| AU2018354195A1 (en) | 2017-10-23 | 2020-06-04 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disease |
| MX2020004830A (es) * | 2017-11-08 | 2020-11-11 | Avexis Inc | Medios y metodo para preparar vectores virales y usos de los mismos. |
| PL3953377T3 (pl) | 2019-04-10 | 2026-02-16 | Prevail Therapeutics, Inc. | Terapie genowe zaburzeń lizosomalnych |
| CA3134841A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| CA3136117A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| US20230346979A1 (en) | 2020-08-10 | 2023-11-02 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
-
2020
- 2020-04-10 PL PL20724262.9T patent/PL3953377T3/pl unknown
- 2020-04-10 US US16/846,065 patent/US11999974B2/en active Active
- 2020-04-10 EP EP20724262.9A patent/EP3953377B1/en active Active
- 2020-04-10 LT LTEPPCT/US2020/027764T patent/LT3953377T/lt unknown
- 2020-04-10 PT PT207242629T patent/PT3953377T/pt unknown
- 2020-04-10 BR BR112021020183A patent/BR112021020183A2/pt unknown
- 2020-04-10 HR HRP20251506TT patent/HRP20251506T1/hr unknown
- 2020-04-10 CA CA3136004A patent/CA3136004A1/en active Pending
- 2020-04-10 FI FIEP20724262.9T patent/FI3953377T3/fi active
- 2020-04-10 KR KR1020217036234A patent/KR20210150486A/ko active Pending
- 2020-04-10 CN CN202080041413.8A patent/CN114174324A/zh active Pending
- 2020-04-10 RS RS20251192A patent/RS67476B1/sr unknown
- 2020-04-10 WO PCT/US2020/027764 patent/WO2020210698A1/en not_active Ceased
- 2020-04-10 JP JP2021559727A patent/JP7571044B2/ja active Active
- 2020-04-10 EP EP25202531.7A patent/EP4707400A2/en active Pending
- 2020-04-10 DK DK20724262.9T patent/DK3953377T3/da active
- 2020-04-10 AU AU2020273182A patent/AU2020273182B2/en active Active
- 2020-04-10 MX MX2021011958A patent/MX2021011958A/es unknown
- 2020-04-10 ES ES20724262T patent/ES3055985T3/es active Active
- 2020-04-10 SI SI202030683T patent/SI3953377T1/sl unknown
-
2021
- 2021-10-03 IL IL286903A patent/IL286903A/en unknown
-
2024
- 2024-04-26 US US18/646,957 patent/US12516298B2/en active Active
- 2024-10-09 JP JP2024176952A patent/JP2025016496A/ja active Pending
-
2026
- 2026-02-17 AU AU2026201184A patent/AU2026201184A1/en active Pending
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