KR20180113990A - 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법 - Google Patents

가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법 Download PDF

Info

Publication number
KR20180113990A
KR20180113990A KR1020187019657A KR20187019657A KR20180113990A KR 20180113990 A KR20180113990 A KR 20180113990A KR 1020187019657 A KR1020187019657 A KR 1020187019657A KR 20187019657 A KR20187019657 A KR 20187019657A KR 20180113990 A KR20180113990 A KR 20180113990A
Authority
KR
South Korea
Prior art keywords
leu
gly
ser
asp
arg
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.)
Ceased
Application number
KR1020187019657A
Other languages
English (en)
Korean (ko)
Inventor
제임스 엠. 윌슨
다니엘 제이. 레이더
Original Assignee
더 트러스티스 오브 더 유니버시티 오브 펜실바니아
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 filed Critical 더 트러스티스 오브 더 유니버시티 오브 펜실바니아
Publication of KR20180113990A publication Critical patent/KR20180113990A/ko
Ceased legal-status Critical Current

Links

Images

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

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)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Obesity (AREA)
  • General Chemical & Material Sciences (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Manufacturing & Machinery (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020187019657A 2015-12-11 2016-12-09 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법 Ceased KR20180113990A (ko)

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

Publications (1)

Publication Number Publication Date
KR20180113990A true KR20180113990A (ko) 2018-10-17

Family

ID=57794341

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020187019657A Ceased KR20180113990A (ko) 2015-12-11 2016-12-09 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법

Country Status (14)

Country Link
US (2) US10889832B2 (https=)
EP (2) EP3400304B1 (https=)
JP (2) JP7328760B2 (https=)
KR (1) KR20180113990A (https=)
CN (2) CN115957350A (https=)
AU (2) AU2016366560A1 (https=)
BR (1) BR112018011687A2 (https=)
CA (1) CA3008142A1 (https=)
CO (1) CO2018007165A2 (https=)
ES (1) ES2921450T3 (https=)
IL (2) IL259850B2 (https=)
MA (1) MA43570A (https=)
MX (1) MX2018007080A (https=)
WO (1) WO2017100682A1 (https=)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3134431B1 (en) 2014-04-25 2021-04-07 The Trustees Of The University Of Pennsylvania Ldlr variants and their use in compositions for reducing cholesterol levels
AU2016366560A1 (en) 2015-12-11 2018-06-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
IL301963A (en) 2016-07-21 2023-06-01 Spark Therapeutics Inc Scalable High Recovery Methods for High-Throughput Recombinant Adeno-Associated Viral Vector Production and Recombinant Adeno-Associated Viral Vectors Made So
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
JP2020510648A (ja) * 2017-02-20 2020-04-09 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 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
CN111989396A (zh) * 2017-11-30 2020-11-24 宾夕法尼亚州大学信托人 用于iiib型粘多糖贮积病的基因疗法
CN113710808A (zh) * 2018-12-20 2021-11-26 宾夕法尼亚州大学信托人 用于治疗家族性高胆固醇血症的基因疗法
BR112021013807A2 (pt) 2019-01-18 2021-11-30 Astrazeneca Ab Inibidores de pcsk9 e seus métodos de uso
MX2021008661A (es) 2019-01-18 2021-08-19 Astrazeneca Ab Inhibidores de la pcsk9 y metodos de uso de los mismos.
TWI908746B (zh) * 2019-11-28 2025-12-21 美商銳進科斯生物股份有限公司 微小肌縮蛋白基因療法之構築體及其用途
KR20230002594A (ko) * 2020-04-29 2023-01-05 살리오젠 테라퓨틱스 인코포레이티드 가족성 고콜레스테롤혈증 및 상승된 저밀도 지단백질 콜레스테롤의 치료를 위한 조성물 및 방법
WO2022165313A1 (en) 2021-02-01 2022-08-04 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)

* Cited by examiner, † Cited by third party
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
US6548286B1 (en) 1997-04-14 2003-04-15 Cell Genesys, Inc. 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
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
CA2379166C (en) 1999-08-09 2013-03-26 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 instrastrand base pairs
US7201898B2 (en) 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
AU2003274397A1 (en) 2002-06-05 2003-12-22 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
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
JP4495210B2 (ja) 2005-06-09 2010-06-30 パナソニック株式会社 振幅誤差補償装置及び直交度誤差補償装置
US7769466B2 (en) 2006-02-24 2010-08-03 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
JP2012516357A (ja) * 2009-01-29 2012-07-19 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 神経学的障害を治療するための皮質全体に亘る高レベルの治療薬の分布方法
US20120252877A1 (en) * 2009-08-14 2012-10-04 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex
WO2011094198A1 (en) * 2010-01-28 2011-08-04 The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2012177741A1 (en) 2011-06-20 2012-12-27 Genentech, Inc. Pcsk9-binding polypeptides and methods of use
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
HUE069234T2 (hu) 2011-09-16 2025-02-28 Regeneron Pharma Eljárások lipoprotein(a)-szint csökkentésére proprotein-konvertáz szubtilizin/kexin 9 (PCSK9) inhibitorának beadásával
MX367100B (es) * 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
WO2014127196A1 (en) * 2013-02-15 2014-08-21 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services 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
AU2014235096B2 (en) * 2013-03-15 2020-03-19 The Trustees Of The University Of Pennsylvania Compositions and methods for treating MPSI
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
EP3052521A1 (en) 2013-10-03 2016-08-10 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
EP3134431B1 (en) 2014-04-25 2021-04-07 The Trustees Of The University Of Pennsylvania Ldlr variants and their use in compositions for reducing cholesterol levels
ES2833230T3 (es) * 2014-05-13 2021-06-14 Univ Pennsylvania Composiciones que comprenden AVV que expresan construcciones y usos de doble anticuerpo del mismo
ES2934848T3 (es) 2015-12-11 2023-02-27 Univ Pennsylvania Método de purificación escalable para AAV8
AU2016366560A1 (en) 2015-12-11 2018-06-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
JP2020510648A (ja) 2017-02-20 2020-04-09 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 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

Also Published As

Publication number Publication date
JP2019504003A (ja) 2019-02-14
CO2018007165A2 (es) 2018-07-19
CA3008142A1 (en) 2017-06-15
CN109121421B (zh) 2022-11-01
ES2921450T3 (es) 2022-08-25
AU2016366560A1 (en) 2018-06-28
CN115957350A (zh) 2023-04-14
JP7328760B2 (ja) 2023-08-17
IL259850B2 (en) 2023-11-01
EP4085934A1 (en) 2022-11-09
WO2017100682A1 (en) 2017-06-15
IL259850A (en) 2018-07-31
US20190002917A1 (en) 2019-01-03
CN109121421A (zh) 2019-01-01
IL303850A (en) 2023-08-01
EP3400304B1 (en) 2022-04-06
US20210285013A1 (en) 2021-09-16
EP3400304A1 (en) 2018-11-14
IL259850B1 (en) 2023-07-01
MA43570A (fr) 2018-11-14
AU2023201657A1 (en) 2023-06-15
US10889832B2 (en) 2021-01-12
MX2018007080A (es) 2018-11-12
JP2022050575A (ja) 2022-03-30
BR112018011687A2 (pt) 2018-12-04

Similar Documents

Publication Publication Date Title
KR20180113990A (ko) 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법
JP7818558B2 (ja) 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用
AU2021202866B2 (en) Muscle-specific nucleic acid regulatory elements and methods and use thereof
ES2966692T3 (es) Vectores de virus adenoasociados poliploides y métodos de fabricación y uso de los mismos
KR20190118163A (ko) 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법
CN113950526B (zh) 通过靶向体内表观遗传阻遏实现的持久镇痛
JP7725514B2 (ja) 結節性硬化症の遺伝子治療
CN111448321A (zh) Sod1双表达载体及其用途
AU2025283486A1 (en) Gene therapy for the treatment of galactosemia
KR102877876B1 (ko) Aav-상용성 라미닌-링커 중합 단백질
CN115109788A (zh) Aav介导的人脂蛋白脂酶肝脏异位表达载体及其用途
CN114846141B (zh) 一种分离的核酸分子及其应用
KR20210106489A (ko) 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법
KR20220079857A (ko) Aav-상용성 라미닌-링커 중합 단백질
AU2023252508B2 (en) Treatment of neuromuscular diseases via gene therapy that expresses klotho protein
CN112272565A (zh) 使用慢病毒基因构建体的递送的体内基因疗法
HK40075438B (zh) 一种分离的核酸分子及其应用
HK40035051A (en) Aav-compatible laminin-linker polymerization proteins
CN116768983A (zh) 一种用于慢性乙型肝炎预防和/或治疗的蛋白、编码基因、重组腺相关病毒及其应用

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20180710

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20211209

Comment text: Request for Examination of Application

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

Comment text: Notification of reason for refusal

Patent event date: 20231124

Patent event code: PE09021S01D

E601 Decision to refuse application
PE0601 Decision on rejection of patent

Patent event date: 20240125

Comment text: Decision to Refuse Application

Patent event code: PE06012S01D

Patent event date: 20231124

Comment text: Notification of reason for refusal

Patent event code: PE06011S01I