JPWO2021178707A5 - - Google Patents
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- JPWO2021178707A5 JPWO2021178707A5 JP2022552651A JP2022552651A JPWO2021178707A5 JP WO2021178707 A5 JPWO2021178707 A5 JP WO2021178707A5 JP 2022552651 A JP2022552651 A JP 2022552651A JP 2022552651 A JP2022552651 A JP 2022552651A JP WO2021178707 A5 JPWO2021178707 A5 JP WO2021178707A5
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- Prior art keywords
- deficiency
- nucleic acid
- seq
- acid sequence
- sequence
- 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.)
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- 150000007523 nucleic acids Chemical group 0.000 claims 15
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 14
- 239000013603 viral vector Substances 0.000 claims 13
- 239000013598 vector Substances 0.000 claims 10
- 208000017855 Progressive familial intrahepatic cholestasis type 1 Diseases 0.000 claims 8
- 230000007812 deficiency Effects 0.000 claims 8
- 108091033319 polynucleotide Proteins 0.000 claims 6
- 102000040430 polynucleotide Human genes 0.000 claims 6
- 239000002157 polynucleotide Substances 0.000 claims 6
- 102000007610 Amino-acid N-acetyltransferase Human genes 0.000 claims 4
- 108010032178 Amino-acid N-acetyltransferase Proteins 0.000 claims 4
- 208000034318 Argininemia Diseases 0.000 claims 4
- 206010058298 Argininosuccinate synthetase deficiency Diseases 0.000 claims 4
- 201000011297 Citrullinemia Diseases 0.000 claims 4
- 208000034600 Hyperornithinemia-hyperammonemia-homocitrullinuria syndrome Diseases 0.000 claims 4
- 102000007981 Ornithine carbamoyltransferase Human genes 0.000 claims 4
- 101710198224 Ornithine carbamoyltransferase, mitochondrial Proteins 0.000 claims 4
- 201000003554 argininosuccinic aciduria Diseases 0.000 claims 4
- 201000011286 hyperargininemia Diseases 0.000 claims 4
- 201000003694 methylmalonic acidemia Diseases 0.000 claims 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 206010062695 Arginase deficiency Diseases 0.000 claims 2
- 101710113083 Carbamoyl-phosphate synthase Proteins 0.000 claims 2
- 101000701363 Homo sapiens Phospholipid-transporting ATPase IC Proteins 0.000 claims 2
- 208000000599 Ornithine Carbamoyltransferase Deficiency Disease Diseases 0.000 claims 2
- 208000035903 Ornithine transcarbamylase deficiency Diseases 0.000 claims 2
- 102100030448 Phospholipid-transporting ATPase IC Human genes 0.000 claims 2
- 201000002150 Progressive familial intrahepatic cholestasis Diseases 0.000 claims 2
- 208000012619 Progressive familial intrahepatic cholestasis type 3 Diseases 0.000 claims 2
- 208000031045 adult-onset type II citrullinemia Diseases 0.000 claims 2
- 208000025812 citrin deficiency Diseases 0.000 claims 2
- 208000016617 citrullinemia type I Diseases 0.000 claims 2
- 239000012212 insulator Substances 0.000 claims 2
- 208000019423 liver disease Diseases 0.000 claims 2
- 230000002503 metabolic effect Effects 0.000 claims 2
- 201000011278 ornithine carbamoyltransferase deficiency Diseases 0.000 claims 2
- 201000007976 ornithine translocase deficiency Diseases 0.000 claims 2
- 201000002148 progressive familial intrahepatic cholestasis 3 Diseases 0.000 claims 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 1
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 1
- 108020004414 DNA Proteins 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 108700019146 Transgenes Proteins 0.000 claims 1
- 125000006850 spacer group Chemical group 0.000 claims 1
Claims (18)
- ５'から３'方向に以下を含む、アデノ随伴ウイルス（ＡＡＶ）ｐｉｇｇｙＢａｃトランスポゾンポリヌクレオチド：
ａ）配列番号９３の核酸配列を含む第１のＡＡＶＩＴＲ配列、
ｂ）配列番号１２５の核酸配列を含む第１のｐｉｇｇｙＢａｃＩＴＲ配列、
ｃ）配列番号７の核酸配列を含む第１のインスレーター配列、
ｄ）配列番号１３２の核酸配列を含む少なくとも１つのプロモーター配列、
ｅ）配列番号２２の核酸配列を含む少なくとも１つのトランス遺伝子配列、
ｆ）配列番号９７の核酸配列を含むポリＡ配列、
ｇ）配列番号８の核酸配列を含む第２のインスレーター配列、
ｈ）配列番号９６の核酸配列を含む第２のｐｉｇｇｙＢａｃＩＴＲ配列、
ｉ）配列番号１３０の核酸配列を含む少なくとも１つのＤＮＡスペーサー配列、及び
ｊ）配列番号９４の核酸配列を含む第２のＡＡＶＩＴＲ配列。 - 配列番号１３９に示される核酸配列と９７％同一である核酸配列を含む、請求項１に記載のＡＡＶｐｉｇｇｙＢａｃトランスポゾンポリヌクレオチド。
- 配列番号１３９の核酸残基１２～４７８２と９７％同一である核酸配列を含む、請求項２に記載のＡＡＶｐｉｇｇｙＢａｃトランスポゾンポリヌクレオチド。
- 第１のｐｉｇｇｙＢａｃＩＴＲ配列が、配列番号９５の核酸配列を含む、請求項１に記載のＡＡＶｐｉｇｇｙＢａｃトランスポゾンポリヌクレオチド。
- 請求項１に記載のＡＡＶｐｉｇｇｙＢａｃトランスポゾンポリヌクレオチドを含むベクター。
- 前記ベクターがウイルスベクターであり、前記ウイルスベクターがＡＡＶウイルスベクターである、請求項５に記載のベクター。
- 前記ＡＡＶウイルスベクターが、ＡＡＶ１、ＡＡＶ２、ＡＡＶ３、ＡＡＶ４、ＡＡＶ５、ＡＡＶ６、ＡＡＶ７、ＡＡＶ８、ＡＡＶ９、ＡＡＶ１０、又はＡＡＶ１１ウイルスベクターである、請求項６に記載のベクター。
- 前記ＡＡＶウイルスベクターが、ＡＡＶ８ウイルスベクターである、請求項６に記載のベクター。
- 前記ＡＡＶウイルスベクターが、ＡＡＶ９ウイルスベクターである、請求項６に記載のベクター。
- 前記ＡＡＶウイルスベクターが、ＡＡＶ－ＫＰ－１又はＡＡＶ－ＮＰ５９ウイルスベクターである、請求項６に記載のベクター。
- 前記ＡＡＶウイルスベクターが、ＡＡＶ－ＫＰ－１ウイルスベクターである、請求項６に記載のベクター。
- 請求項５のいずれかに記載のベクターを含む組成物。
- 請求項１に記載のポリヌクレオチドの少なくとも１つの治療有効量を含む、被検体における少なくとも１つの代謝性肝障害（ＭＬＤ）を治療するための組成物。
- 前記少なくとも１つのＭＬＤが、Ｎ－アセチルグルタミン酸合成酵素（ＮＡＧＳ）欠損症、カルバモイルリン酸合成酵素Ｉ欠損症（ＣＰＳＩ欠損症）、オルニチントランスカルバミラーゼ（ＯＴＣ）欠損症、アルギニノコハク酸合成酵素欠損症（ＡＳＳＤ）（シトルリン血症Ｉ）、シトリン欠損症（シトルリン血症ＩＩ）、アルギニノコハク酸リアーゼ欠損症（アルギニノコハク酸尿症）、アルギナーゼ欠損症（高アルギニン血症）、オルニチントランスロカーゼ欠損症（ＨＨＨ症候群）、メチルマロン酸血症（ＭＭＡ）、進行性家族性肝内胆汁うっ滞１型（ＰＦＩＣ１）、進行性家族性肝内胆汁うっ滞１型（ＰＦＩＣ２）、進行性家族性肝内胆汁うっ滞１型（ＰＦＩＣ３）、又はこれらの任意の組み合わせである、請求項１３に記載の組成物。
- 前記ＭＬＤがＯＴＣ欠損症である、請求項１４に記載の組成物。
- 請求項５に記載のベクターの少なくとも１つの治療有効量を含む、被検体における少なくとも１つの代謝性肝障害（ＭＬＤ）を治療するための組成物。
- 前記少なくとも１つのＭＬＤが、Ｎ－アセチルグルタミン酸合成酵素（ＮＡＧＳ）欠損症、カルバモイルリン酸合成酵素Ｉ欠損症（ＣＰＳＩ欠損症）、オルニチントランスカルバミラーゼ（ＯＴＣ）欠損症、アルギニノコハク酸合成酵素欠損症（ＡＳＳＤ）（シトルリン血症Ｉ）、シトリン欠損症（シトルリン血症ＩＩ）、アルギニノコハク酸リアーゼ欠損症（アルギニノコハク酸尿症）、アルギナーゼ欠損症（高アルギニン血症）、オルニチントランスロカーゼ欠損症（ＨＨＨ症候群）、メチルマロン酸血症（ＭＭＡ）、進行性家族性肝内胆汁うっ滞１型（ＰＦＩＣ１）、進行性家族性肝内胆汁うっ滞１型（ＰＦＩＣ２）、進行性家族性肝内胆汁うっ滞１型（ＰＦＩＣ３）、又はこれらの任意の組み合わせである、請求項１６に記載の組成物。
- 前記ＭＬＤがＯＴＣ欠損症である、請求項１７に記載の組成物。
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202062985047P | 2020-03-04 | 2020-03-04 | |
| US62/985,047 | 2020-03-04 | ||
| US202063121488P | 2020-12-04 | 2020-12-04 | |
| US63/121,488 | 2020-12-04 | ||
| PCT/US2021/020929 WO2021178707A1 (en) | 2020-03-04 | 2021-03-04 | Compositions and methods for the treatment of metabolic liver disorders |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023515692A JP2023515692A (ja) | 2023-04-13 |
| JPWO2021178707A5 true JPWO2021178707A5 (ja) | 2024-03-12 |
Family
ID=75267602
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022552651A Pending JP2023515692A (ja) | 2020-03-04 | 2021-03-04 | 代謝性肝障害の治療のための組成物と方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20230104091A1 (ja) |
| EP (1) | EP4114469A1 (ja) |
| JP (1) | JP2023515692A (ja) |
| KR (1) | KR20220149588A (ja) |
| CN (1) | CN115515646A (ja) |
| AU (1) | AU2021230361A1 (ja) |
| CA (1) | CA3169529A1 (ja) |
| WO (1) | WO2021178707A1 (ja) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20230383278A1 (en) * | 2020-09-18 | 2023-11-30 | The United States Of America,As Represented By The Secretary,Department Of Health And Human Services | Novel adeno-associated viral (aav) vectors to treat hereditary methylmalonic acidemia (mma) caused by methylmalonyl-coa mutase (mmut) deficiency |
| CA3094859A1 (en) * | 2020-10-01 | 2022-04-01 | Entos Pharmaceuticals Inc. | Proteolipid vesicles formulated with fusion associated small transmembrane proteins |
| WO2022187671A1 (en) * | 2021-03-04 | 2022-09-09 | Poseida Therapeutics, Inc. | Compositions and methods for the treatment of hemophilia |
| GB202205606D0 (en) * | 2022-04-14 | 2022-06-01 | Genespire S R L | Lentiviral vector |
Family Cites Families (49)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3773919A (en) | 1969-10-23 | 1973-11-20 | Du Pont | Polylactide-drug mixtures |
| US4309989A (en) | 1976-02-09 | 1982-01-12 | The Curators Of The University Of Missouri | Topical application of medication by ultrasound with coupling agent |
| FR2374910A1 (fr) | 1976-10-23 | 1978-07-21 | Choay Sa | Preparation a base d'heparine, comprenant des liposomes, procede pour l'obtenir et medicaments contenant de telles preparations |
| US4554101A (en) | 1981-01-09 | 1985-11-19 | New York Blood Center, Inc. | Identification and preparation of epitopes on antigens and allergens on the basis of hydrophilicity |
| US4656134A (en) | 1982-01-11 | 1987-04-07 | Board Of Trustees Of Leland Stanford Jr. University | Gene amplification in eukaryotic cells |
| US5168062A (en) | 1985-01-30 | 1992-12-01 | University Of Iowa Research Foundation | Transfer vectors and microorganisms containing human cytomegalovirus immediate-early promoter-regulatory DNA sequence |
| US4683195A (en) | 1986-01-30 | 1987-07-28 | Cetus Corporation | Process for amplifying, detecting, and/or-cloning nucleic acid sequences |
| US4683202A (en) | 1985-03-28 | 1987-07-28 | Cetus Corporation | Process for amplifying nucleic acid sequences |
| US4965188A (en) | 1986-08-22 | 1990-10-23 | Cetus Corporation | Process for amplifying, detecting, and/or cloning nucleic acid sequences using a thermostable enzyme |
| US4766067A (en) | 1985-05-31 | 1988-08-23 | President And Fellows Of Harvard College | Gene amplification |
| GB8601597D0 (en) | 1986-01-23 | 1986-02-26 | Wilson R H | Nucleotide sequences |
| US4800159A (en) | 1986-02-07 | 1989-01-24 | Cetus Corporation | Process for amplifying, detecting, and/or cloning nucleic acid sequences |
| US4767402A (en) | 1986-07-08 | 1988-08-30 | Massachusetts Institute Of Technology | Ultrasound enhancement of transdermal drug delivery |
| EP0318512B1 (en) | 1986-08-18 | 1998-06-17 | Emisphere Technologies, Inc. | Delivery systems for pharmacological agents |
| US4889818A (en) | 1986-08-22 | 1989-12-26 | Cetus Corporation | Purified thermostable enzyme |
| US4921794A (en) | 1987-01-14 | 1990-05-01 | President And Fellows Of Harvard College | T7 DNA polymerase |
| US4795699A (en) | 1987-01-14 | 1989-01-03 | President And Fellows Of Harvard College | T7 DNA polymerase |
| US5130238A (en) | 1988-06-24 | 1992-07-14 | Cangene Corporation | Enhanced nucleic acid amplification process |
| US5066584A (en) | 1988-09-23 | 1991-11-19 | Cetus Corporation | Methods for generating single stranded dna by the polymerase chain reaction |
| US5091310A (en) | 1988-09-23 | 1992-02-25 | Cetus Corporation | Structure-independent dna amplification by the polymerase chain reaction |
| US5142033A (en) | 1988-09-23 | 1992-08-25 | Hoffmann-La Roche Inc. | Structure-independent DNA amplification by the polymerase chain reaction |
| US4994370A (en) | 1989-01-03 | 1991-02-19 | The United States Of America As Represented By The Department Of Health And Human Services | DNA amplification technique |
| US5266491A (en) | 1989-03-14 | 1993-11-30 | Mochida Pharmaceutical Co., Ltd. | DNA fragment and expression plasmid containing the DNA fragment |
| US5580575A (en) | 1989-12-22 | 1996-12-03 | Imarx Pharmaceutical Corp. | Therapeutic drug delivery systems |
| US5580734A (en) | 1990-07-13 | 1996-12-03 | Transkaryotic Therapies, Inc. | Method of producing a physical map contigous DNA sequences |
| US5968502A (en) | 1991-11-05 | 1999-10-19 | Transkaryotic Therapies, Inc. | Protein production and protein delivery |
| US5641670A (en) | 1991-11-05 | 1997-06-24 | Transkaryotic Therapies, Inc. | Protein production and protein delivery |
| US5643252A (en) | 1992-10-28 | 1997-07-01 | Venisect, Inc. | Laser perforator |
| US5849695A (en) | 1993-01-13 | 1998-12-15 | The Regents Of The University Of California | Parathyroid hormone analogues useful for treatment of osteoporosis and disorders of calcium meatabolism in mammals |
| ATE453709T1 (de) | 1993-02-12 | 2010-01-15 | Univ Leland Stanford Junior | Regulierte transkription von zielgerichteten genen und andere biologische ereignisse |
| US5514670A (en) | 1993-08-13 | 1996-05-07 | Pharmos Corporation | Submicron emulsions for delivery of peptides |
| US5814599A (en) | 1995-08-04 | 1998-09-29 | Massachusetts Insitiute Of Technology | Transdermal delivery of encapsulated drugs |
| US5730723A (en) | 1995-10-10 | 1998-03-24 | Visionary Medical Products Corporation, Inc. | Gas pressured needle-less injection device and method |
| US6218185B1 (en) | 1996-04-19 | 2001-04-17 | The United States Of America As Represented By The Secretary Of Agriculture | Piggybac transposon-based genetic transformation system for insects |
| US5827729A (en) | 1996-04-23 | 1998-10-27 | Advanced Tissue Sciences | Diffusion gradient bioreactor and extracorporeal liver device using a three-dimensional liver tissue |
| US5879681A (en) | 1997-02-07 | 1999-03-09 | Emisphere Technolgies Inc. | Compounds and compositions for delivering active agents |
| IL120943A (en) | 1997-05-29 | 2004-03-28 | Univ Ben Gurion | A system for administering drugs through the skin |
| US6309663B1 (en) | 1999-08-17 | 2001-10-30 | Lipocine Inc. | Triglyceride-free compositions and methods for enhanced absorption of hydrophilic therapeutic agents |
| US6962810B2 (en) | 2000-10-31 | 2005-11-08 | University Of Notre Dame Du Lac | Methods and compositions for transposition using minimal segments of the eukaryotic transformation vector piggyBac |
| CA2692501C (en) | 2007-07-04 | 2017-10-10 | Max-Delbrueck-Centrum Fuer Molekulare Medizin | Hyperactive variants of the transposase protein of the transposon system sleeping beauty |
| WO2010099296A1 (en) | 2009-02-26 | 2010-09-02 | Transposagen Biopharmaceuticals, Inc. | Hyperactive piggybac transposases |
| EP2621957B1 (en) * | 2010-09-28 | 2021-06-02 | University of Notre Dame du Lac | Chimeric spider silk and uses thereof |
| WO2012074758A1 (en) * | 2010-11-16 | 2012-06-07 | Transposagen Bioharmaceuticals, Inc. | Hyperactive piggybac transposases |
| WO2015138357A2 (en) * | 2014-03-09 | 2015-09-17 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of otc deficency |
| WO2015139093A1 (en) * | 2014-03-21 | 2015-09-24 | The Sydney Children's Hospitals Network (Randwick And Westmead) (Incorporating The Royal Alexandra Hospital For Children) | Stable gene transfer to proliferating cells |
| US10927384B2 (en) | 2014-04-09 | 2021-02-23 | Dna Twopointo Inc. | DNA vectors, transposons and transposases for eukaryotic genome modification |
| WO2017070428A1 (en) * | 2015-10-22 | 2017-04-27 | Hera Testing Laboratories, Inc. | Genetically modified non-human mammals having modified liver cells and/or tissue and methods of making same |
| JP6747517B2 (ja) | 2017-09-05 | 2020-08-26 | 東レ株式会社 | 繊維強化熱可塑性樹脂成形品 |
| CN112601583A (zh) | 2018-03-07 | 2021-04-02 | 波赛达治疗公司 | CARTyrin组合物和使用方法 |
-
2021
- 2021-03-04 EP EP21715030.9A patent/EP4114469A1/en active Pending
- 2021-03-04 CN CN202180032731.2A patent/CN115515646A/zh active Pending
- 2021-03-04 AU AU2021230361A patent/AU2021230361A1/en active Pending
- 2021-03-04 CA CA3169529A patent/CA3169529A1/en active Pending
- 2021-03-04 WO PCT/US2021/020929 patent/WO2021178707A1/en unknown
- 2021-03-04 JP JP2022552651A patent/JP2023515692A/ja active Pending
- 2021-03-04 KR KR1020227034141A patent/KR20220149588A/ko active Search and Examination
- 2021-03-04 US US17/909,101 patent/US20230104091A1/en active Pending
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