JP2019504003A5 - - Google Patents
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- Publication number
- JP2019504003A5 JP2019504003A5 JP2018530566A JP2018530566A JP2019504003A5 JP 2019504003 A5 JP2019504003 A5 JP 2019504003A5 JP 2018530566 A JP2018530566 A JP 2018530566A JP 2018530566 A JP2018530566 A JP 2018530566A JP 2019504003 A5 JP2019504003 A5 JP 2019504003A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- composition
- composition according
- human subject
- genome copies
- 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
- 239000000203 mixture Substances 0.000 claims 16
- 101001051093 Homo sapiens Low-density lipoprotein receptor Proteins 0.000 claims 8
- 102100024640 Low-density lipoprotein receptor Human genes 0.000 claims 8
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 8
- 208000000563 Hyperlipoproteinemia Type II Diseases 0.000 claims 7
- 206010045261 Type IIa hyperlipidaemia Diseases 0.000 claims 7
- 201000001386 familial hypercholesterolemia Diseases 0.000 claims 7
- 239000000725 suspension Substances 0.000 claims 7
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 5
- 241000702421 Dependoparvovirus Species 0.000 claims 5
- 230000002093 peripheral effect Effects 0.000 claims 5
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims 4
- 230000037396 body weight Effects 0.000 claims 4
- 210000000234 capsid Anatomy 0.000 claims 4
- 239000012537 formulation buffer Substances 0.000 claims 4
- 238000002347 injection Methods 0.000 claims 4
- 239000007924 injection Substances 0.000 claims 4
- 239000008194 pharmaceutical composition Substances 0.000 claims 4
- 210000003462 vein Anatomy 0.000 claims 4
- RVGRUAULSDPKGF-UHFFFAOYSA-N Poloxamer Chemical compound C1CO1.CC1CO1 RVGRUAULSDPKGF-UHFFFAOYSA-N 0.000 claims 3
- 239000002245 particle Substances 0.000 claims 3
- 101150102415 Apob gene Proteins 0.000 claims 2
- 101710095342 Apolipoprotein B Proteins 0.000 claims 2
- 102100040202 Apolipoprotein B-100 Human genes 0.000 claims 2
- 238000008214 LDL Cholesterol Methods 0.000 claims 2
- 102000007330 LDL Lipoproteins Human genes 0.000 claims 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- 229910019142 PO4 Inorganic materials 0.000 claims 2
- 230000015556 catabolic process Effects 0.000 claims 2
- 235000012000 cholesterol Nutrition 0.000 claims 2
- 210000004185 liver Anatomy 0.000 claims 2
- 238000010172 mouse model Methods 0.000 claims 2
- 150000007523 nucleic acids Chemical group 0.000 claims 2
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 2
- 239000010452 phosphate Substances 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
- 239000007787 solid Substances 0.000 claims 2
- 150000003626 triacylglycerols Chemical class 0.000 claims 2
- 229940121710 HMGCoA reductase inhibitor Drugs 0.000 claims 1
- 241000699666 Mus <mouse, genus> Species 0.000 claims 1
- 241000699670 Mus sp. Species 0.000 claims 1
- PVNIIMVLHYAWGP-UHFFFAOYSA-N Niacin Chemical compound OC(=O)C1=CC=CN=C1 PVNIIMVLHYAWGP-UHFFFAOYSA-N 0.000 claims 1
- 229940122392 PCSK9 inhibitor Drugs 0.000 claims 1
- 238000011529 RT qPCR Methods 0.000 claims 1
- 239000007864 aqueous solution Substances 0.000 claims 1
- 229920000080 bile acid sequestrant Polymers 0.000 claims 1
- LOKCTEFSRHRXRJ-UHFFFAOYSA-I dipotassium trisodium dihydrogen phosphate hydrogen phosphate dichloride Chemical compound P(=O)(O)(O)[O-].[K+].P(=O)(O)([O-])[O-].[Na+].[Na+].[Cl-].[K+].[Cl-].[Na+] LOKCTEFSRHRXRJ-UHFFFAOYSA-I 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- OLNTVTPDXPETLC-XPWALMASSA-N ezetimibe Chemical compound N1([C@@H]([C@H](C1=O)CC[C@H](O)C=1C=CC(F)=CC=1)C=1C=CC(O)=CC=1)C1=CC=C(F)C=C1 OLNTVTPDXPETLC-XPWALMASSA-N 0.000 claims 1
- 229960000815 ezetimibe Drugs 0.000 claims 1
- 238000001802 infusion Methods 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 229960003512 nicotinic acid Drugs 0.000 claims 1
- 235000001968 nicotinic acid Nutrition 0.000 claims 1
- 239000011664 nicotinic acid Substances 0.000 claims 1
- 239000002953 phosphate buffered saline Substances 0.000 claims 1
- 229920001983 poloxamer Polymers 0.000 claims 1
- 229960000502 poloxamer Drugs 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022002190A JP2022050575A (ja) | 2015-12-11 | 2022-01-11 | 家族性高コレステロール血症を処置するための遺伝子治療 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562266383P | 2015-12-11 | 2015-12-11 | |
| US62/266,383 | 2015-12-11 | ||
| US201562269440P | 2015-12-18 | 2015-12-18 | |
| US62/269,440 | 2015-12-18 | ||
| PCT/US2016/065984 WO2017100682A1 (en) | 2015-12-11 | 2016-12-09 | Gene therapy for treating familial hypercholesterolemia |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022002190A Division JP2022050575A (ja) | 2015-12-11 | 2022-01-11 | 家族性高コレステロール血症を処置するための遺伝子治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019504003A JP2019504003A (ja) | 2019-02-14 |
| JP2019504003A5 true JP2019504003A5 (enExample) | 2020-01-23 |
| JP7328760B2 JP7328760B2 (ja) | 2023-08-17 |
Family
ID=57794341
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018530566A Active JP7328760B2 (ja) | 2015-12-11 | 2016-12-09 | 家族性高コレステロール血症を処置するための遺伝子治療 |
| JP2022002190A Pending JP2022050575A (ja) | 2015-12-11 | 2022-01-11 | 家族性高コレステロール血症を処置するための遺伝子治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022002190A Pending JP2022050575A (ja) | 2015-12-11 | 2022-01-11 | 家族性高コレステロール血症を処置するための遺伝子治療 |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US10889832B2 (enExample) |
| EP (2) | EP3400304B1 (enExample) |
| JP (2) | JP7328760B2 (enExample) |
| KR (1) | KR20180113990A (enExample) |
| CN (2) | CN115957350A (enExample) |
| AU (2) | AU2016366560A1 (enExample) |
| BR (1) | BR112018011687A2 (enExample) |
| CA (1) | CA3008142A1 (enExample) |
| CO (1) | CO2018007165A2 (enExample) |
| ES (1) | ES2921450T3 (enExample) |
| IL (2) | IL259850B2 (enExample) |
| MA (1) | MA43570A (enExample) |
| MX (1) | MX2018007080A (enExample) |
| WO (1) | WO2017100682A1 (enExample) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015164778A1 (en) | 2014-04-25 | 2015-10-29 | The Trustees Of The University Of Pennysylvania | Ldlr variants and their use in compositions for reducing cholesterol levels |
| EP3400304B1 (en) | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| DK3488007T3 (da) | 2016-07-21 | 2024-02-19 | Spark Therapeutics Inc | Skalerbare fremgangsmåder med høj indvinding til fremstilling af højt udbytte rekombinant adeno-associeret viral (raav) vektor og rekombinante adeno-associeret virale (raav) vektorer produceret derved |
| US11883470B2 (en) | 2016-07-25 | 2024-01-30 | The Trustees Of The University Of Pennsylvania | Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof |
| US11554147B2 (en) * | 2017-02-20 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| CA3083761A1 (en) | 2017-11-30 | 2019-06-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for mucopolysaccharidosis iiib |
| CA3123844A1 (en) * | 2018-12-20 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| KR20220107222A (ko) * | 2019-11-28 | 2022-08-02 | 리젠엑스바이오 인크. | 마이크로디스트로핀 유전자 치료 작제물 및 이의 용도 |
| AU2021263938A1 (en) * | 2020-04-29 | 2022-10-20 | Saliogen Therapeutics, Inc. | Compositions and methods for treatment of familial hypercholesterolemia and elevated low-density lipoprotein cholesterol |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| CN117887723A (zh) * | 2024-01-08 | 2024-04-16 | 苏州诺洁贝生物技术有限公司 | 密码子优化的ldlr基因及其应用 |
Family Cites Families (44)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| DE69824859T2 (de) | 1997-04-14 | 2005-08-04 | Cell Genesys, Inc., Foster City | Methoden zur erhöhung der effizienz rekombinanter aav produkte |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| DE60039766D1 (de) | 1999-08-09 | 2008-09-18 | Targeted Genetics Corp | Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
| CA2915124C (en) | 2001-11-13 | 2018-08-14 | The Trustees Of The University Of Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| ES2602352T3 (es) * | 2001-12-17 | 2017-02-20 | The Trustees Of The University Of Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas |
| WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
| CN1780910A (zh) * | 2003-01-28 | 2006-05-31 | 拉塞有限公司 | 对与家族性高胆固醇血症有关的分离的低密度脂蛋白受体(ldl－r)的基因序列中的突变进行检测的方法和装置 |
| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| EP2357010B1 (en) | 2005-04-07 | 2013-06-12 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
| WO2007100697A2 (en) | 2006-02-24 | 2007-09-07 | Kenergy, Inc. | Class-e radio frequency power amplifier with feedback control |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| KR101010352B1 (ko) | 2008-05-30 | 2011-01-25 | 삼성중공업 주식회사 | 전력 제어 장치 및 방법 |
| BRPI1007155A2 (pt) * | 2009-01-29 | 2017-05-30 | Univ Of California San Francisco | métodos para tratar um distúrbio neurológico cortical, e para dispensar um agente terapêutico para o córtex em um primata |
| WO2011020118A1 (en) * | 2009-08-14 | 2011-02-17 | Theramind Research, Llc | Methods and compositions for treatment of tuberous sclerosis complex |
| PT2529020T (pt) * | 2010-01-28 | 2018-07-30 | Childrens Hospital Philadelphia | Plataforma de fabrico escalável para purificação de vetor viral e vetores virais assim purificados para utilização na terapia génica |
| SG10201502270TA (en) | 2010-03-29 | 2015-05-28 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| CN103717615A (zh) | 2011-06-20 | 2014-04-09 | 霍夫曼-拉罗奇有限公司 | 结合pcsk9的多肽及使用方法 |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| ES2857773T5 (es) | 2011-08-24 | 2024-06-04 | Univ Leland Stanford Junior | Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos |
| CA2848201C (en) | 2011-09-16 | 2020-10-27 | Regeneron Pharmaceuticals, Inc. | Methods for reducing lipoprotein(a) levels by administering an inhibitor of proprotein convertase subtilisin kexin-9 (pcsk9) |
| BR112014020325A2 (pt) * | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| US9567376B2 (en) | 2013-02-08 | 2017-02-14 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| CA2900231C (en) * | 2013-02-15 | 2019-07-30 | Paul Albert Sieving | Aav8 retinoschisin expression vector for treating x-linked retinoschisis |
| EP3747998B1 (en) * | 2013-03-15 | 2024-07-24 | The Trustees of the University of Pennsylvania | Compositions for treating mpsi |
| US8980864B2 (en) * | 2013-03-15 | 2015-03-17 | Moderna Therapeutics, Inc. | Compositions and methods of altering cholesterol levels |
| WO2015012924A2 (en) | 2013-04-29 | 2015-01-29 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof |
| EA201690675A1 (ru) | 2013-10-03 | 2016-08-31 | Модерна Терапьютикс, Инк. | Полинуклеотиды, кодирующие рецептор липопротеинов низкой плотности |
| WO2015164778A1 (en) | 2014-04-25 | 2015-10-29 | The Trustees Of The University Of Pennysylvania | Ldlr variants and their use in compositions for reducing cholesterol levels |
| SG11201609207SA (en) * | 2014-05-13 | 2016-12-29 | Univ Pennsylvania | Compositions comprising aav expressing dual antibody constructs and uses thereof |
| EP3400304B1 (en) | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| ES2934848T3 (es) | 2015-12-11 | 2023-02-27 | Univ Pennsylvania | Método de purificación escalable para AAV8 |
| US11554147B2 (en) | 2017-02-20 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
-
2016
- 2016-12-09 EP EP16825936.4A patent/EP3400304B1/en active Active
- 2016-12-09 KR KR1020187019657A patent/KR20180113990A/ko not_active Ceased
- 2016-12-09 MX MX2018007080A patent/MX2018007080A/es unknown
- 2016-12-09 CA CA3008142A patent/CA3008142A1/en active Pending
- 2016-12-09 CN CN202211286244.1A patent/CN115957350A/zh active Pending
- 2016-12-09 WO PCT/US2016/065984 patent/WO2017100682A1/en not_active Ceased
- 2016-12-09 JP JP2018530566A patent/JP7328760B2/ja active Active
- 2016-12-09 MA MA043570A patent/MA43570A/fr unknown
- 2016-12-09 US US16/060,409 patent/US10889832B2/en active Active
- 2016-12-09 IL IL259850A patent/IL259850B2/en unknown
- 2016-12-09 IL IL303850A patent/IL303850A/en unknown
- 2016-12-09 CN CN201680081580.9A patent/CN109121421B/zh active Active
- 2016-12-09 BR BR112018011687A patent/BR112018011687A2/pt not_active IP Right Cessation
- 2016-12-09 ES ES16825936T patent/ES2921450T3/es active Active
- 2016-12-09 EP EP22165782.8A patent/EP4085934A1/en not_active Withdrawn
- 2016-12-09 AU AU2016366560A patent/AU2016366560A1/en not_active Abandoned
-
2018
- 2018-07-09 CO CONC2018/0007165A patent/CO2018007165A2/es unknown
-
2020
- 2020-11-17 US US16/950,272 patent/US20210285013A1/en not_active Abandoned
-
2022
- 2022-01-11 JP JP2022002190A patent/JP2022050575A/ja active Pending
-
2023
- 2023-03-16 AU AU2023201657A patent/AU2023201657A1/en not_active Abandoned
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