MA43570A - Thérapie génique pour traiter l'hypercholestérolémie familiale - Google Patents
Thérapie génique pour traiter l'hypercholestérolémie familialeInfo
- Publication number
- MA43570A MA43570A MA043570A MA43570A MA43570A MA 43570 A MA43570 A MA 43570A MA 043570 A MA043570 A MA 043570A MA 43570 A MA43570 A MA 43570A MA 43570 A MA43570 A MA 43570A
- Authority
- MA
- Morocco
- Prior art keywords
- treat
- gene therapy
- family hypercholesterolemia
- hypercholesterolemia
- family
- Prior art date
Links
- 208000035150 Hypercholesterolemia Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N15/861—Adenoviral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
- A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
- A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562266383P | 2015-12-11 | 2015-12-11 | |
US201562269440P | 2015-12-18 | 2015-12-18 |
Publications (1)
Publication Number | Publication Date |
---|---|
MA43570A true MA43570A (fr) | 2018-11-14 |
Family
ID=57794341
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MA043570A MA43570A (fr) | 2015-12-11 | 2016-12-09 | Thérapie génique pour traiter l'hypercholestérolémie familiale |
Country Status (14)
Country | Link |
---|---|
US (2) | US10889832B2 (fr) |
EP (2) | EP4085934A1 (fr) |
JP (2) | JP7328760B2 (fr) |
KR (1) | KR20180113990A (fr) |
CN (2) | CN115957350A (fr) |
AU (2) | AU2016366560A1 (fr) |
BR (1) | BR112018011687A2 (fr) |
CA (1) | CA3008142A1 (fr) |
CO (1) | CO2018007165A2 (fr) |
ES (1) | ES2921450T3 (fr) |
IL (2) | IL303850A (fr) |
MA (1) | MA43570A (fr) |
MX (1) | MX2018007080A (fr) |
WO (1) | WO2017100682A1 (fr) |
Families Citing this family (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CA2946392A1 (fr) | 2014-04-25 | 2015-10-29 | James M. Wilson | Variants de ldlr et leur utilisation dans des compositions permettant de reduire les taux de cholesterol |
EP4085934A1 (fr) | 2015-12-11 | 2022-11-09 | The Trustees of The University of Pennsylvania | Thérapie génique pour traiter l'hypercholestérolémie familiale |
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 |
EP3595688A4 (fr) * | 2017-02-20 | 2020-12-30 | The Trustees Of The University Of Pennsylvania | Thérapie génique pour traiter l'hypercholestérolémie familiale |
BR112020010977A2 (pt) * | 2017-11-30 | 2020-11-17 | The Trustees Of The University Of Pennsylvania | terapia de gene para mucopolissacaridose iiib |
CA3123844A1 (fr) * | 2018-12-20 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Therapie genique pour le traitement de l'hypercholesterolemie familiale |
CA3209779A1 (fr) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Therapie genique de ceroides-lipofuscinoses neuronales |
Family Cites Families (40)
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 |
US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
US5478745A (en) | 1992-12-04 | 1995-12-26 | 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 |
AU728220B2 (en) | 1997-04-14 | 2001-01-04 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant AAV product |
EP1080218A1 (fr) | 1998-05-27 | 2001-03-07 | University of Florida | Procede de preparation de compositions de virus adeno-associes de recombinaison a l'aide d'un gradient d'iodixananol |
CA2348382C (fr) | 1998-11-10 | 2013-09-17 | The University Of North Carolina At Chapel Hill | Vecteurs de papirovirus chimeriques et procedes de production et d'administration connexes |
CA2379166C (fr) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Vecteurs viraux de recombinaison actives metaboliquement et procede de preparation et d'utilisation de ceux-ci |
WO2001091803A2 (fr) | 2000-06-01 | 2001-12-06 | University Of North Carolina At Chapel Hill | Procedes et composes pour la liberation controlee de vecteurs de parvovirus de recombinaison |
NZ578982A (en) | 2001-11-13 | 2011-03-31 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby |
ES2717377T3 (es) | 2001-12-17 | 2019-06-20 | Univ Pennsylvania | Secuencias de serotipo 8 de virus adeno-asociado (AAV), vectores que las contienen y usos de las mismas |
AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
EP3910063A1 (fr) | 2003-09-30 | 2021-11-17 | The Trustees of The University of Pennsylvania | Clades de virus adéno associés (aav), séquences, vecteurs les contenant et leurs utilisations |
EP2359865B1 (fr) | 2005-04-07 | 2013-10-02 | The Trustees of The University of Pennsylvania | Procédé d'amélioration de la fonction d'un vecteur AAV |
US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
WO2007100697A2 (fr) | 2006-02-24 | 2007-09-07 | Kenergy, Inc. | Amplificateur de puissance de classe e à commande de rétroaction |
US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
KR101010352B1 (ko) | 2008-05-30 | 2011-01-25 | 삼성중공업 주식회사 | 전력 제어 장치 및 방법 |
CN102369024A (zh) * | 2009-01-29 | 2012-03-07 | 加利福尼亚大学董事会 | 用于向整个皮质中分布高水平治疗剂以治疗神经障碍的方法 |
US20120252877A1 (en) * | 2009-08-14 | 2012-10-04 | Theramind Research, Llc | Methods and compositions for treatment of tuberous sclerosis complex |
DK2529020T3 (en) * | 2010-01-28 | 2018-08-06 | Childrens Hospital Philadelphia | SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY |
MX342858B (es) | 2010-03-29 | 2016-10-13 | The Trustees Of The Univ Of Pennsylvania * | Sistema de ablacion transgenica inducida farmacologicamente. |
US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
JP2014519848A (ja) | 2011-06-20 | 2014-08-21 | ジェネンテック, インコーポレイテッド | Pcsk9結合ポリペプチドと使用方法 |
FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
WO2013029030A1 (fr) | 2011-08-24 | 2013-02-28 | The Board Of Trustees Of The Leland Stanford Junior University | Protéines capsidiques d'aav inédites pouvant être utilisées pour le transfert d'acides nucléiques |
JP6158813B2 (ja) | 2011-09-16 | 2017-07-05 | リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. | プロタンパク質転換酵素サブチリシンケキシン−9(PCSK9)の阻害剤を投与することによってリポタンパク質(a)レベルを低下させる方法 |
EP2814958B1 (fr) * | 2012-02-17 | 2019-08-07 | The Children's Hospital of Philadelphia | Compositions de vecteurs de virus adéno-associés et méthodes de transfert de gènes dans des cellules, des organes et des tissus |
EP2954051B1 (fr) | 2013-02-08 | 2019-03-27 | The Trustees Of The University Of Pennsylvania | Capsides aav8 modifiées pour transfert génétique pour thérapies rétiniennes |
AU2014216160B2 (en) * | 2013-02-15 | 2017-03-30 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | AAV8 retinoschisin expression vector for treating X-linked retinoschisis |
WO2015012924A2 (fr) | 2013-04-29 | 2015-01-29 | The Trustees Of The University Of Pennsylvania | Cassettes d'expression préférentielle pour tissus modifiées par un codon, vecteurs les contenant, et utilisation |
AU2014329452B2 (en) | 2013-10-03 | 2019-06-20 | Moderna Therapeutics, Inc. | Polynucleotides encoding low density lipoprotein receptor |
CA2946392A1 (fr) | 2014-04-25 | 2015-10-29 | James M. Wilson | Variants de ldlr et leur utilisation dans des compositions permettant de reduire les taux de cholesterol |
EP4215605A1 (fr) | 2015-12-11 | 2023-07-26 | The Trustees of The University of Pennsylvania | Procédé de purification évolutif d'aav8 |
EP4085934A1 (fr) | 2015-12-11 | 2022-11-09 | The Trustees of The University of Pennsylvania | Thérapie génique pour traiter l'hypercholestérolémie familiale |
EP3595688A4 (fr) | 2017-02-20 | 2020-12-30 | The Trustees Of The University Of Pennsylvania | Thérapie génique pour traiter l'hypercholestérolémie familiale |
-
2016
- 2016-12-09 EP EP22165782.8A patent/EP4085934A1/fr active Pending
- 2016-12-09 WO PCT/US2016/065984 patent/WO2017100682A1/fr active Application Filing
- 2016-12-09 EP EP16825936.4A patent/EP3400304B1/fr active Active
- 2016-12-09 MA MA043570A patent/MA43570A/fr unknown
- 2016-12-09 CN CN202211286244.1A patent/CN115957350A/zh active Pending
- 2016-12-09 BR BR112018011687A patent/BR112018011687A2/pt active Search and Examination
- 2016-12-09 CA CA3008142A patent/CA3008142A1/fr active Pending
- 2016-12-09 MX MX2018007080A patent/MX2018007080A/es unknown
- 2016-12-09 IL IL303850A patent/IL303850A/en unknown
- 2016-12-09 US US16/060,409 patent/US10889832B2/en active Active
- 2016-12-09 AU AU2016366560A patent/AU2016366560A1/en not_active Abandoned
- 2016-12-09 ES ES16825936T patent/ES2921450T3/es active Active
- 2016-12-09 KR KR1020187019657A patent/KR20180113990A/ko not_active Application Discontinuation
- 2016-12-09 JP JP2018530566A patent/JP7328760B2/ja active Active
- 2016-12-09 IL IL259850A patent/IL259850B2/en unknown
- 2016-12-09 CN CN201680081580.9A patent/CN109121421B/zh active Active
-
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 active Pending
Also Published As
Publication number | Publication date |
---|---|
US20210285013A1 (en) | 2021-09-16 |
JP7328760B2 (ja) | 2023-08-17 |
JP2022050575A (ja) | 2022-03-30 |
EP3400304A1 (fr) | 2018-11-14 |
US10889832B2 (en) | 2021-01-12 |
CO2018007165A2 (es) | 2018-07-19 |
KR20180113990A (ko) | 2018-10-17 |
CA3008142A1 (fr) | 2017-06-15 |
JP2019504003A (ja) | 2019-02-14 |
ES2921450T3 (es) | 2022-08-25 |
WO2017100682A1 (fr) | 2017-06-15 |
EP4085934A1 (fr) | 2022-11-09 |
BR112018011687A2 (pt) | 2018-12-04 |
IL303850A (en) | 2023-08-01 |
IL259850B1 (en) | 2023-07-01 |
MX2018007080A (es) | 2018-11-12 |
US20190002917A1 (en) | 2019-01-03 |
AU2023201657A1 (en) | 2023-06-15 |
EP3400304B1 (fr) | 2022-04-06 |
IL259850B2 (en) | 2023-11-01 |
CN109121421B (zh) | 2022-11-01 |
AU2016366560A1 (en) | 2018-06-28 |
CN109121421A (zh) | 2019-01-01 |
CN115957350A (zh) | 2023-04-14 |
IL259850A (en) | 2018-07-31 |
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