JP2020510648A5 - - Google Patents
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- Publication number
- JP2020510648A5 JP2020510648A5 JP2019545363A JP2019545363A JP2020510648A5 JP 2020510648 A5 JP2020510648 A5 JP 2020510648A5 JP 2019545363 A JP2019545363 A JP 2019545363A JP 2019545363 A JP2019545363 A JP 2019545363A JP 2020510648 A5 JP2020510648 A5 JP 2020510648A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- suspension
- raav
- patient
- genome
- 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
- 239000008194 pharmaceutical composition Substances 0.000 claims 22
- 239000000725 suspension Substances 0.000 claims 15
- 239000007971 pharmaceutical suspension Substances 0.000 claims 8
- 102100024640 Low-density lipoprotein receptor Human genes 0.000 claims 7
- 101001051093 Homo sapiens Low-density lipoprotein receptor Proteins 0.000 claims 6
- 208000000563 Hyperlipoproteinemia Type II Diseases 0.000 claims 6
- 206010045261 Type IIa hyperlipidaemia Diseases 0.000 claims 6
- 210000000234 capsid Anatomy 0.000 claims 6
- 201000001386 familial hypercholesterolemia Diseases 0.000 claims 6
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 5
- 241000702421 Dependoparvovirus Species 0.000 claims 5
- 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 4
- 239000002245 particle Substances 0.000 claims 4
- 230000002093 peripheral effect Effects 0.000 claims 3
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- RVGRUAULSDPKGF-UHFFFAOYSA-N Poloxamer Chemical compound C1CO1.CC1CO1 RVGRUAULSDPKGF-UHFFFAOYSA-N 0.000 claims 2
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims 2
- 238000002617 apheresis Methods 0.000 claims 2
- 230000001506 immunosuppresive effect Effects 0.000 claims 2
- 210000004185 liver Anatomy 0.000 claims 2
- 238000010172 mouse model Methods 0.000 claims 2
- 230000003472 neutralizing effect Effects 0.000 claims 2
- 150000007523 nucleic acids Chemical group 0.000 claims 2
- 210000003462 vein Anatomy 0.000 claims 2
- 208000035150 Hypercholesterolemia Diseases 0.000 claims 1
- 241000699666 Mus <mouse, genus> Species 0.000 claims 1
- 241000699670 Mus sp. Species 0.000 claims 1
- 229940127355 PCSK9 Inhibitors Drugs 0.000 claims 1
- 229910019142 PO4 Inorganic materials 0.000 claims 1
- 239000007864 aqueous solution Substances 0.000 claims 1
- 239000000872 buffer Substances 0.000 claims 1
- 239000007853 buffer solution Substances 0.000 claims 1
- 238000011278 co-treatment Methods 0.000 claims 1
- 230000002950 deficient Effects 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
- 230000009977 dual effect Effects 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 239000012537 formulation buffer Substances 0.000 claims 1
- 238000001802 infusion Methods 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 1
- 239000010452 phosphate 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
- 229920001993 poloxamer 188 Polymers 0.000 claims 1
- 229940044519 poloxamer 188 Drugs 0.000 claims 1
- 230000010076 replication Effects 0.000 claims 1
- 239000011780 sodium chloride Substances 0.000 claims 1
- 239000004575 stone Substances 0.000 claims 1
- 238000005303 weighing Methods 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023030651A JP2023071829A (ja) | 2017-02-20 | 2023-03-01 | 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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762461015P | 2017-02-20 | 2017-02-20 | |
| US62/461,015 | 2017-02-20 | ||
| PCT/US2018/018678 WO2018152485A1 (en) | 2017-02-20 | 2018-02-20 | Gene therapy for treating familial hypercholesterolemia |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023030651A Division JP2023071829A (ja) | 2017-02-20 | 2023-03-01 | 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 (2)
| Publication Number | Publication Date |
|---|---|
| JP2020510648A JP2020510648A (ja) | 2020-04-09 |
| JP2020510648A5 true JP2020510648A5 (enExample) | 2021-03-25 |
Family
ID=63170484
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019545363A Pending JP2020510648A (ja) | 2017-02-20 | 2018-02-20 | 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 |
| JP2023030651A Pending JP2023071829A (ja) | 2017-02-20 | 2023-03-01 | 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 |
|---|---|---|---|
| JP2023030651A Pending JP2023071829A (ja) | 2017-02-20 | 2023-03-01 | 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 (8)
| Country | Link |
|---|---|
| US (2) | US11554147B2 (enExample) |
| EP (1) | EP3595688A4 (enExample) |
| JP (2) | JP2020510648A (enExample) |
| KR (1) | KR20190118163A (enExample) |
| AU (1) | AU2018220212A1 (enExample) |
| CA (1) | CA3051011A1 (enExample) |
| IL (1) | IL268276A (enExample) |
| WO (1) | WO2018152485A1 (enExample) |
Families Citing this family (6)
| 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 |
| US11554147B2 (en) | 2017-02-20 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| WO2021080975A1 (en) * | 2019-10-21 | 2021-04-29 | The Trustees Of The University Of Pennsylvania | Compositions and methods for reducing cholesterol levels |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2023081807A1 (en) * | 2021-11-04 | 2023-05-11 | The Trustees of the University of Pennsylvania Penn Center for Innovation | Compositions and methods for reducing pcsk9 levels in a subject |
Family Cites Families (41)
| 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 |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| 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 |
| 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 |
| 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 |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| SG10201502270TA (en) | 2010-03-29 | 2015-05-28 | Univ 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 |
| 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 |
| AU2013355327A1 (en) | 2012-12-05 | 2015-06-11 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of metabolic disorders |
| 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 |
| US9254332B2 (en) | 2013-03-15 | 2016-02-09 | Arecor Limited | Stable aqueous formulations of adenovirus vectors |
| 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 |
| ES2934848T3 (es) | 2015-12-11 | 2023-02-27 | Univ Pennsylvania | Método de purificación escalable para AAV8 |
| EP3400304B1 (en) | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| US11554147B2 (en) * | 2017-02-20 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
-
2018
- 2018-02-20 US US16/486,981 patent/US11554147B2/en active Active
- 2018-02-20 AU AU2018220212A patent/AU2018220212A1/en not_active Abandoned
- 2018-02-20 CA CA3051011A patent/CA3051011A1/en active Pending
- 2018-02-20 WO PCT/US2018/018678 patent/WO2018152485A1/en not_active Ceased
- 2018-02-20 KR KR1020197024773A patent/KR20190118163A/ko not_active Ceased
- 2018-02-20 EP EP18754347.5A patent/EP3595688A4/en not_active Withdrawn
- 2018-02-20 JP JP2019545363A patent/JP2020510648A/ja active Pending
-
2019
- 2019-07-25 IL IL268276A patent/IL268276A/en unknown
-
2022
- 2022-11-22 US US18/058,126 patent/US20230190837A1/en not_active Abandoned
-
2023
- 2023-03-01 JP JP2023030651A patent/JP2023071829A/ja active Pending
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