MX2018007080A - Terapia genica para tratar hipercolesterolemia familiar. - Google Patents
Terapia genica para tratar hipercolesterolemia familiar.Info
- Publication number
- MX2018007080A MX2018007080A MX2018007080A MX2018007080A MX2018007080A MX 2018007080 A MX2018007080 A MX 2018007080A MX 2018007080 A MX2018007080 A MX 2018007080A MX 2018007080 A MX2018007080 A MX 2018007080A MX 2018007080 A MX2018007080 A MX 2018007080A
- Authority
- MX
- Mexico
- Prior art keywords
- gene therapy
- familial hypercholesterolemia
- ldl
- raav
- treating familial
- Prior art date
Links
- 208000000563 Hyperlipoproteinemia Type II Diseases 0.000 title 1
- 102100024640 Low-density lipoprotein receptor Human genes 0.000 title 1
- 206010045261 Type IIa hyperlipidaemia Diseases 0.000 title 1
- 201000001386 familial hypercholesterolemia Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 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 abstract 2
- 101150102415 Apob gene Proteins 0.000 abstract 1
- 101710095342 Apolipoprotein B Proteins 0.000 abstract 1
- 102100040202 Apolipoprotein B-100 Human genes 0.000 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 108010028554 LDL Cholesterol Proteins 0.000 abstract 1
- 238000008214 LDL Cholesterol Methods 0.000 abstract 1
- 108010007622 LDL Lipoproteins Proteins 0.000 abstract 1
- 230000001925 catabolic effect Effects 0.000 abstract 1
- 235000012000 cholesterol Nutrition 0.000 abstract 1
- 230000002950 deficient Effects 0.000 abstract 1
- 238000001802 infusion Methods 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 230000002093 peripheral effect Effects 0.000 abstract 1
- 230000010076 replication Effects 0.000 abstract 1
- 239000000725 suspension Substances 0.000 abstract 1
- 150000003626 triacylglycerols Chemical class 0.000 abstract 1
- 210000003462 vein Anatomy 0.000 abstract 1
Classifications
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- 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
-
- 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
- A61K48/0008—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
- 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
-
- 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
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Obesity (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Diabetes (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Hematology (AREA)
- Cell Biology (AREA)
- Dermatology (AREA)
- Manufacturing & Machinery (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562266383P | 2015-12-11 | 2015-12-11 | |
| US201562269440P | 2015-12-18 | 2015-12-18 | |
| PCT/US2016/065984 WO2017100682A1 (en) | 2015-12-11 | 2016-12-09 | Gene therapy for treating familial hypercholesterolemia |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2018007080A true MX2018007080A (es) | 2018-11-12 |
Family
ID=57794341
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2018007080A MX2018007080A (es) | 2015-12-11 | 2016-12-09 | Terapia genica para tratar hipercolesterolemia familiar. |
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 |
|---|---|---|---|---|
| JP6741590B2 (ja) | 2014-04-25 | 2020-08-19 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
| CA3008142A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| KR20240126073A (ko) | 2016-07-21 | 2024-08-20 | 스파크 테라퓨틱스, 인코포레이티드 | 재조합 아데노-관련 바이러스(rAAV) 벡터를 고수율로 생산하기 위한 확장 가능한 고회수 방법 및 이에 의해 생산된 재조합 아데노-관련 바이러스(rAAV) 벡터 |
| 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 |
| CA3051011A1 (en) * | 2017-02-20 | 2018-08-23 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| US11723989B2 (en) * | 2017-11-30 | 2023-08-15 | 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 |
| US20230270886A1 (en) * | 2019-11-28 | 2023-08-31 | Regenxbio Inc. | Microdystrophin gene therapy constructs and uses thereof |
| KR20230002594A (ko) * | 2020-04-29 | 2023-01-05 | 살리오젠 테라퓨틱스 인코포레이티드 | 가족성 고콜레스테롤혈증 및 상승된 저밀도 지단백질 콜레스테롤의 치료를 위한 조성물 및 방법 |
| BR112023015303A2 (pt) | 2021-02-01 | 2023-11-14 | Regenxbio Inc | Método para tratar doença cln2 devido a deficiência de tpp1 em um sujeito |
| 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)
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| 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 |
| 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 |
| CA2287478C (en) | 1997-04-14 | 2007-06-19 | Richard J. Samulski | Methods for increasing the efficiency of recombinant aav product |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| US6491907B1 (en) | 1998-11-10 | 2002-12-10 | The University Of North Carolina At Chapel Hill | Recombinant parvovirus vectors and method of making |
| WO2001011034A2 (en) | 1999-08-09 | 2001-02-15 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs |
| AU2001268149B2 (en) | 2000-06-01 | 2005-08-18 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| ATE317916T1 (de) | 2001-11-13 | 2006-03-15 | Univ Pennsylvania | Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode |
| EP1453547B1 (en) | 2001-12-17 | 2016-09-21 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
| US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | 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 |
| ES2874298T3 (es) | 2003-09-30 | 2021-11-04 | Univ Pennsylvania | Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos |
| ES2428218T3 (es) | 2005-04-07 | 2013-11-06 | The Trustees Of The University Of Pennsylvania | Cápsides de AAV rh48 modificadas, composiciones que las contienen y usos de las mismas |
| EP1777906A1 (en) | 2005-06-09 | 2007-04-25 | Matsushita Electric Industrial Co., Ltd. | Amplitude error compensating apparatus and orthogonality error compensating apparatus |
| EP1986737A1 (en) | 2006-02-24 | 2008-11-05 | Kenergy Inc | Class-e radio frequency amplifier for use with an implantable medical device |
| 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 |
| WO2010088560A1 (en) * | 2009-01-29 | 2010-08-05 | University Of California, San Francisco | Methods for distributing high levels of therapeutic agent throughout the cortex to treat neurological disorders |
| US20120252877A1 (en) * | 2009-08-14 | 2012-10-04 | Theramind Research, Llc | Methods and compositions for treatment of tuberous sclerosis complex |
| ES2680915T3 (es) * | 2010-01-28 | 2018-09-11 | The Children's Hospital Of Philadelphia | Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica |
| US20130023033A1 (en) | 2010-03-29 | 2013-01-24 | The Trustees Of The University Of 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 |
| MX2013015311A (es) | 2011-06-20 | 2014-03-31 | Genentech Inc | Polipeptidos de enlace de pcsk9 y metodos de uso. |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| US9169299B2 (en) | 2011-08-24 | 2015-10-27 | The Board Of Trustees Of The Leleand Stanford Junior University | AAV capsid proteins for nucleic acid transfer |
| MX363642B (es) | 2011-09-16 | 2019-03-28 | Regeneron Pharma | Metodos para reducir los niveles de lipoproteina (a) administrando un inhibidor de proproteina convertasa subtilisina kexina-9 (pcsk9). |
| JP6373763B2 (ja) * | 2012-02-17 | 2018-08-22 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ | Ａａｖベクター成分、及び、細胞、臓器並びに組織への遺伝子導入のための方法 |
| WO2014124282A1 (en) | 2013-02-08 | 2014-08-14 | 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 |
| US8980864B2 (en) * | 2013-03-15 | 2015-03-17 | Moderna Therapeutics, Inc. | Compositions and methods of altering cholesterol levels |
| KR102346455B1 (ko) * | 2013-03-15 | 2022-01-04 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ｍｐｓｉ 치료를 위한 조성물 및 방법 |
| 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 |
| BR112016007255A2 (pt) | 2013-10-03 | 2017-09-12 | Moderna Therapeutics Inc | polinucleotídeos que codificam receptor de lipoproteína de baixa densidade |
| JP6741590B2 (ja) * | 2014-04-25 | 2020-08-19 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
| JP6974943B2 (ja) * | 2014-05-13 | 2021-12-01 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ２種の抗体構築物を発現するａａｖを含む組成物およびこの使用 |
| EP3387117B1 (en) | 2015-12-11 | 2022-11-23 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| CA3008142A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| CA3051011A1 (en) | 2017-02-20 | 2018-08-23 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
-
2016
- 2016-12-09 CA CA3008142A patent/CA3008142A1/en active Pending
- 2016-12-09 WO PCT/US2016/065984 patent/WO2017100682A1/en not_active Ceased
- 2016-12-09 KR KR1020187019657A patent/KR20180113990A/ko not_active Ceased
- 2016-12-09 MA MA043570A patent/MA43570A/fr unknown
- 2016-12-09 ES ES16825936T patent/ES2921450T3/es active Active
- 2016-12-09 IL IL259850A patent/IL259850B2/en unknown
- 2016-12-09 US US16/060,409 patent/US10889832B2/en active Active
- 2016-12-09 CN CN202211286244.1A patent/CN115957350A/zh active Pending
- 2016-12-09 JP JP2018530566A patent/JP7328760B2/ja active Active
- 2016-12-09 EP EP16825936.4A patent/EP3400304B1/en active Active
- 2016-12-09 IL IL303850A patent/IL303850A/en unknown
- 2016-12-09 BR BR112018011687A patent/BR112018011687A2/pt not_active IP Right Cessation
- 2016-12-09 CN CN201680081580.9A patent/CN109121421B/zh active Active
- 2016-12-09 MX MX2018007080A patent/MX2018007080A/es unknown
- 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
Also Published As
| Publication number | Publication date |
|---|---|
| CN109121421A (zh) | 2019-01-01 |
| US20190002917A1 (en) | 2019-01-03 |
| EP3400304B1 (en) | 2022-04-06 |
| JP7328760B2 (ja) | 2023-08-17 |
| CN115957350A (zh) | 2023-04-14 |
| WO2017100682A1 (en) | 2017-06-15 |
| JP2019504003A (ja) | 2019-02-14 |
| CO2018007165A2 (es) | 2018-07-19 |
| AU2023201657A1 (en) | 2023-06-15 |
| EP4085934A1 (en) | 2022-11-09 |
| IL259850A (en) | 2018-07-31 |
| MA43570A (fr) | 2018-11-14 |
| EP3400304A1 (en) | 2018-11-14 |
| ES2921450T3 (es) | 2022-08-25 |
| IL259850B2 (en) | 2023-11-01 |
| KR20180113990A (ko) | 2018-10-17 |
| US20210285013A1 (en) | 2021-09-16 |
| IL259850B1 (en) | 2023-07-01 |
| JP2022050575A (ja) | 2022-03-30 |
| AU2016366560A1 (en) | 2018-06-28 |
| IL303850A (en) | 2023-08-01 |
| CN109121421B (zh) | 2022-11-01 |
| US10889832B2 (en) | 2021-01-12 |
| CA3008142A1 (en) | 2017-06-15 |
| BR112018011687A2 (pt) | 2018-12-04 |
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