JP7347933B2 - 血友病a治療に対する遺伝子療法 - Google Patents

血友病a治療に対する遺伝子療法 Download PDF

Info

Publication number
JP7347933B2
JP7347933B2 JP2018554331A JP2018554331A JP7347933B2 JP 7347933 B2 JP7347933 B2 JP 7347933B2 JP 2018554331 A JP2018554331 A JP 2018554331A JP 2018554331 A JP2018554331 A JP 2018554331A JP 7347933 B2 JP7347933 B2 JP 7347933B2
Authority
JP
Japan
Prior art keywords
raav
vector
hfviii
aav
seq
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.)
Active
Application number
JP2018554331A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019513794A (ja
JP2019513794A5 (https=
Inventor
ワン,リリ
ウイルソン,ジェームス・エム
シドレーン,ジェニー・アグネス
Original Assignee
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
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 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 filed Critical 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
Publication of JP2019513794A publication Critical patent/JP2019513794A/ja
Publication of JP2019513794A5 publication Critical patent/JP2019513794A5/ja
Priority to JP2022011052A priority Critical patent/JP2022062121A/ja
Application granted granted Critical
Publication of JP7347933B2 publication Critical patent/JP7347933B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36Blood coagulation or fibrinolysis factors
    • A61K38/37Factors VIII
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Dermatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Dispersion Chemistry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2018554331A 2016-04-15 2017-04-13 血友病a治療に対する遺伝子療法 Active JP7347933B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022011052A JP2022062121A (ja) 2016-04-15 2022-01-27 血友病a治療に対する遺伝子療法

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201662323336P 2016-04-15 2016-04-15
US62/323,336 2016-04-15
US201662331807P 2016-05-04 2016-05-04
US62/331,807 2016-05-04
US201662428866P 2016-12-01 2016-12-01
US62/428,866 2016-12-01
PCT/US2017/027396 WO2017180857A1 (en) 2016-04-15 2017-04-13 Gene therapy for treating hemophilia a

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022011052A Division JP2022062121A (ja) 2016-04-15 2022-01-27 血友病a治療に対する遺伝子療法

Publications (3)

Publication Number Publication Date
JP2019513794A JP2019513794A (ja) 2019-05-30
JP2019513794A5 JP2019513794A5 (https=) 2020-05-21
JP7347933B2 true JP7347933B2 (ja) 2023-09-20

Family

ID=59253992

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018554331A Active JP7347933B2 (ja) 2016-04-15 2017-04-13 血友病a治療に対する遺伝子療法
JP2022011052A Pending JP2022062121A (ja) 2016-04-15 2022-01-27 血友病a治療に対する遺伝子療法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022011052A Pending JP2022062121A (ja) 2016-04-15 2022-01-27 血友病a治療に対する遺伝子療法

Country Status (17)

Country Link
US (3) US11779656B2 (https=)
EP (1) EP3452102B9 (https=)
JP (2) JP7347933B2 (https=)
KR (1) KR102450833B1 (https=)
CN (1) CN109562191A (https=)
AU (1) AU2017248659B2 (https=)
BR (1) BR112018071200A2 (https=)
CA (1) CA3019425A1 (https=)
IL (1) IL262215B2 (https=)
MX (1) MX2018012537A (https=)
PE (2) PE20181922A1 (https=)
RU (1) RU2762257C2 (https=)
SA (1) SA518400233B1 (https=)
SG (2) SG10202009849WA (https=)
TW (1) TWI791433B (https=)
WO (1) WO2017180857A1 (https=)
ZA (1) ZA201806477B (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022062121A (ja) * 2016-04-15 2022-04-19 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 血友病a治療に対する遺伝子療法

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TR201813067T4 (tr) 2008-09-15 2018-09-21 Uniqure Biopharma B V Faktör IX polipeptit mutantı, bunun kullanımları ve bunu üretmeye yönelik bir yöntem.
KR102877920B1 (ko) 2015-11-16 2025-10-30 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법
US11191847B2 (en) 2016-04-15 2021-12-07 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia B
AR114350A1 (es) 2018-01-31 2020-08-26 Res Institute At Nationwide Children’S Hospital Terapia genética para la distrofia muscular de cinturas del tipo 2c
KR20210028162A (ko) 2018-06-29 2021-03-11 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법
US20210348193A1 (en) * 2018-09-21 2021-11-11 Nightstarx Limited Compositions and methods for treating retinitis pigmentosa
AU2019357039B2 (en) * 2018-10-12 2025-10-09 Genzyme Corporation Generation of improved human pah for treatment of severe PKU by liver-directed gene replacement therapy
CN112912518A (zh) * 2018-10-15 2021-06-04 再生生物股份有限公司 用于测量复制缺陷型病毒载体和病毒的感染性的方法
SG11202103917VA (en) 2018-10-16 2021-05-28 Blueallele Llc Methods for targeted insertion of dna in genes
GB201818816D0 (en) * 2018-11-19 2019-01-02 Synpromics Ltd Regulatory nucleic acid sequences
TWI851647B (zh) * 2019-01-16 2024-08-11 日商武田藥品工業股份有限公司 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體
AU2020229340A1 (en) * 2019-02-26 2021-09-16 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of B-sarcoglycan and the treatment of muscular dystrophy
SG11202109850SA (en) 2019-03-13 2021-10-28 Generation Bio Co Non-viral dna vectors and uses thereof for expressing fviii therapeutics
RS65421B1 (sr) 2019-08-21 2024-05-31 Res Inst Nationwide Childrens Hospital Isporuka adeno-asociranog virusnog vektora alfa-sarkoglikana i lečenje mišićne distrofije
CN114929735A (zh) * 2019-11-01 2022-08-19 自由行疗法有限公司 因子viii构建体
CN111218446B (zh) * 2019-12-25 2023-03-28 劲帆生物医药科技(武汉)有限公司 一种肝脏特异性启动子及其应用
JP2023510392A (ja) * 2020-01-16 2023-03-13 武田薬品工業株式会社 フェニルケトン尿症に対するアデノ随伴ウイルスベースの遺伝子治療
US20230175014A1 (en) * 2020-04-27 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for reducing nuclease expression and off-target activity using a promoter with low transcriptional activity
MX2022014258A (es) 2020-05-12 2023-02-22 Univ Pennsylvania Composiciones para la reducción específica de un fármaco de la expresión de transgén.
KR20230042513A (ko) * 2020-07-29 2023-03-28 프리라인 테라퓨틱스 리미티드 폴리펩티드
WO2022165245A1 (en) * 2021-01-29 2022-08-04 The Trustees Of The University Of Pennsylvania Compositions and method of use of mutant ace2 decoy variants
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
EP4284442A4 (en) * 2021-02-01 2025-04-23 The Trustees of The University of Pennsylvania COMPOSITIONS AND METHODS FOR TREATING NIEMANN PICK DISEASE TYPE A
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
EP4428237A4 (en) * 2021-11-02 2026-02-25 Gritgen Therapeutics Ltd ISOLATED NUCLEIC ACID MOLECULE AND ITS USES
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023205300A2 (en) * 2022-04-20 2023-10-26 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii
WO2023240222A2 (en) * 2022-06-10 2023-12-14 University Of Florida Research Foundation, Incorporated Immune tolerance induction to viral capsids
WO2025180508A1 (en) * 2024-02-29 2025-09-04 Shanghai Vitalgen Biopharma Co., Ltd. Human factor viii variant expression cassettes and uses thereof for treating hemophilia a

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002527493A (ja) 1998-10-20 2002-08-27 アヴィゲン,インコーポレーテッド 標的細胞による第viii因子の発現のためのアデノ随伴ベクター
JP2016500519A (ja) 2012-10-26 2016-01-14 フリーイェ・ユニヴェルシテイト・ブリュッセルVrije Universieit Brussel 血友病の肝臓指向性遺伝子治療のためのベクター並びにその方法及び使用

Family Cites Families (64)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US5661008A (en) 1991-03-15 1997-08-26 Kabi Pharmacia Ab Recombinant human factor VIII derivatives
US5439824A (en) 1993-05-06 1995-08-08 The United States Of America Increased expression of α-1-antitrypsin in expression vectors through the inclusion of intron II
US6268212B1 (en) 1993-10-18 2001-07-31 Amgen Inc. Tissue specific transgene expression
US6818439B1 (en) 1994-12-30 2004-11-16 Chiron Corporation Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other disorders
EP0951544A2 (en) * 1996-07-03 1999-10-27 Chiron Corporation Methods for administration of recombinant gene delivery vehicles for treatment of human disease
EP1036177A1 (en) 1997-12-05 2000-09-20 The Immune Response Corporation Novel vectors and genes exhibiting increased expression
US6783961B1 (en) 1999-02-26 2004-08-31 Genset S.A. Expressed sequence tags and encoded human proteins
US6797505B2 (en) 1998-05-27 2004-09-28 Cell Genesys, Inc. Recombinant AAV vectors for gene therapy of hemophilia A
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
EP1038959A1 (en) 1999-03-17 2000-09-27 Aventis Behring Gesellschaft mit beschränkter Haftung Factor VIII without B-domain, comprising one or more insertions of a truncated intron I of factor IX
US6610906B1 (en) 1999-06-09 2003-08-26 The Regents Of The University Of Michigan Nucleotide sequences for gene regulation and methods of use thereof
JP2003511082A (ja) 1999-10-12 2003-03-25 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 第viii因子をコードするアデノ随伴ウィルスベクターとその使用法
ES2284545T3 (es) 1999-11-16 2007-11-16 Genzyme Corporation Vectores y transgenes con elementos reguladores para la administracion de genes del higado.
EE200200538A (et) 2000-03-22 2004-04-15 Octagene Gmbh Rekombinantsete verehüübimisfaktorite produtseerimine inimese rakuliinides
US6974667B2 (en) 2000-06-14 2005-12-13 Gene Logic, Inc. Gene expression profiles in liver cancer
US7351813B2 (en) 2000-06-20 2008-04-01 The Board Of Trustees Of The Leland Stanford Junior University Liver-specific gene expression cassettes, and methods of use
US6812339B1 (en) 2000-09-08 2004-11-02 Applera Corporation Polymorphisms in known genes associated with human disease, methods of detection and uses thereof
JP2004532822A (ja) 2001-03-14 2004-10-28 アビジェン, インコーポレイテッド ビリオンの管逆行感染による組換えアデノ随伴ウイルス媒介遺伝子移入
US8021875B2 (en) 2001-08-27 2011-09-20 Roche Madison Inc. Methods for expression of transgenes
ES2319745T3 (es) 2001-10-05 2009-05-12 Expression Therapeutics, Llc Secuencias de acidos nucleicos y aminoacidos que codifican polipeptidos de factor viii de alto nivel de expresion, y metodos de uso.
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
US8005620B2 (en) 2003-08-01 2011-08-23 Dna Twopointo Inc. Systems and methods for biopolymer engineering
EP2434420A3 (en) 2003-08-01 2012-07-25 Dna Twopointo Inc. Systems and methods for biopolymer engineering
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
DE602004018349D1 (de) 2003-10-16 2009-01-22 Claude Negrier MODIFIZIERTE cDNA FÜR HOHE EXPRESSIONSNIVEAUS VON FAKTOR VIII UND DESSEN DERIVATEN
CA2547569C (en) 2003-12-03 2013-04-16 University Of Rochester Recombinant factor viii having increased specific activity
WO2006036502A2 (en) 2004-09-22 2006-04-06 St. Jude Children's Research Hospital Improved expression of factor ix in gene therapy vectors
US8008468B2 (en) 2005-02-16 2011-08-30 Benitec, Inc. RNAi expression constructs with liver-specific enhancer/promoter
WO2006102072A2 (en) 2005-03-23 2006-09-28 The Trustees Of The University Of Pennsylvania Use of a pa131 polypeptide in treatment of atherosclerosis
KR20080007226A (ko) 2005-04-14 2008-01-17 체에스엘 베링 게엠베하 증가된 안정성을 가진 변형된 응고 인자 ⅷ 및 이의 유도체
WO2007016331A2 (en) 2005-07-29 2007-02-08 Children's Hospital Medical Center Classification and diagnosis of the molecular basis of cholestasis
US7943374B2 (en) 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
JP2009519710A (ja) 2005-12-16 2009-05-21 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 遺伝子発現調節エレメントのハイスループットでの特徴付けのための機能性アレイ
WO2008016391A2 (en) 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
WO2007120533A2 (en) * 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Minigene expression cassette
ES2535877T3 (es) 2006-06-19 2015-05-18 Asklepios Biopharmaceutical, Inc. Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica
WO2008073303A2 (en) 2006-12-07 2008-06-19 Switchgear Genomics Transcriptional regulatory elements of biological pathways, tools, and methods
CA2673459C (en) 2006-12-22 2016-09-13 Stefan Schulte Modified coagulation factors with prolonged in vivo half-life
WO2008118258A2 (en) 2007-02-06 2008-10-02 Genizon Biosciences Inc. Genemap of the human genes associated with adhd
KR101235560B1 (ko) 2008-02-14 2013-02-25 재단법인 목암생명공학연구소 유전자 치료용 코딩 서열의 발현에 적합한 발현 벡터
KR100981092B1 (ko) 2008-02-29 2010-09-08 고려대학교 산학협력단 제8 혈액응고 인자와 폰 빌리 블란트 인자 변이체의 재조합발현 벡터 시스템
EP2271763A2 (en) 2008-04-03 2011-01-12 Yeda Research And Development Company Ltd. Methods of expressing a polypeptide in the brain and nucleic acid constructs capable of same
CA2722238C (en) 2008-04-22 2017-11-28 Life Sciences Research Partners Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
US20110306512A1 (en) 2008-06-25 2011-12-15 Dxterity Diagnostics Gene Expression Profiling for Identification, Monitoring, and Treatment of Osteoarthritis
KR101034811B1 (ko) 2008-08-25 2011-05-16 단국대학교 산학협력단 조직 특이적 프로모터와 암 특이 유전자를 타겟팅하는트랜스-스플라이싱 라이보자임을 포함하는 재조합아데노바이러스 및 이의 용도
US20120093775A1 (en) 2009-03-27 2012-04-19 Proyecto De Biomedicina Cima, S.L. Methods and compositions for the treatment of cirrhosis and liver fibrosis
CN101935674A (zh) * 2009-06-30 2011-01-05 中国医学科学院血液学研究所 一种用于血友病b基因治疗的腺病毒载体及其用途
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
WO2011109380A1 (en) 2010-03-01 2011-09-09 The Children's Hospital Of Philadelphia Nucleic acids for targeting multiple regions of the hcv genome
WO2011126808A2 (en) 2010-03-29 2011-10-13 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
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
SI2480678T1 (sl) 2010-09-02 2014-06-30 Molmed Spa Stabilno proizvajanje lentivirusnih vektorjev
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
US10398787B2 (en) * 2012-10-26 2019-09-03 Vrije Universiteit Brussel Vectors for liver-directed gene therapy of hemophilia and methods and use thereof
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
EP3044231B1 (en) * 2013-09-12 2020-08-05 BioMarin Pharmaceutical Inc. Aav vectors comprising a gene encoding factor viii
MX388739B (es) * 2015-02-06 2025-03-20 Univ North Carolina Chapel Hill Casetes optimizados de expresión del gen humano del factor viii de coagulación y su uso.
EP3288594B1 (en) * 2015-04-27 2022-06-29 The Trustees of The University of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
EA202190827A1 (ru) * 2015-11-13 2021-11-30 Баксалта Инкорпорейтед Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
US11028372B2 (en) 2015-12-11 2021-06-08 The Trustees Of The University Of Pennsylvania Scalable purification method for AAVRH10
MX2018012537A (es) * 2016-04-15 2019-02-25 Univ Pennsylvania Terapia de genes para tratar hemofilia a.

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002527493A (ja) 1998-10-20 2002-08-27 アヴィゲン,インコーポレーテッド 標的細胞による第viii因子の発現のためのアデノ随伴ベクター
JP2016500519A (ja) 2012-10-26 2016-01-14 フリーイェ・ユニヴェルシテイト・ブリュッセルVrije Universieit Brussel 血友病の肝臓指向性遺伝子治療のためのベクター並びにその方法及び使用

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Mol Ther.16(2):280-289,2008年
Mol Ther.18(1):118-125,2010年

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022062121A (ja) * 2016-04-15 2022-04-19 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 血友病a治療に対する遺伝子療法

Also Published As

Publication number Publication date
AU2017248659A1 (en) 2018-10-18
RU2762257C2 (ru) 2021-12-17
BR112018071200A2 (pt) 2019-04-09
WO2017180857A1 (en) 2017-10-19
KR20190032274A (ko) 2019-03-27
MX2018012537A (es) 2019-02-25
EP3452102B1 (en) 2024-06-12
SA518400233B1 (ar) 2022-08-23
JP2019513794A (ja) 2019-05-30
SG11201808422QA (en) 2018-10-30
EP3452102B9 (en) 2024-09-04
US10888628B2 (en) 2021-01-12
US20240000974A1 (en) 2024-01-04
AU2017248659B2 (en) 2022-08-11
RU2018136611A3 (https=) 2020-08-11
PE20181922A1 (es) 2018-12-11
TWI791433B (zh) 2023-02-11
CN109562191A (zh) 2019-04-02
US11779656B2 (en) 2023-10-10
IL262215A (en) 2018-11-29
ZA201806477B (en) 2024-09-25
RU2018136611A (ru) 2020-05-15
JP2022062121A (ja) 2022-04-19
US20200085975A1 (en) 2020-03-19
IL262215B2 (en) 2023-07-01
KR102450833B1 (ko) 2022-10-05
EP3452102A1 (en) 2019-03-13
SG10202009849WA (en) 2020-11-27
CA3019425A1 (en) 2017-10-19
US20190343966A1 (en) 2019-11-14
TW201741458A (zh) 2017-12-01
PE20250736A1 (es) 2025-03-10
IL262215B1 (en) 2023-03-01

Similar Documents

Publication Publication Date Title
US20240000974A1 (en) Gene therapy for treating hemophilia a
US11896652B2 (en) Modified factor IX, and compositions, methods and uses for gene transfer to cells, organs, and tissues
US20220288233A1 (en) Gene therapy for treating hemophilia b
US12385065B2 (en) Gene therapy for treating citrullenemia
US12023386B2 (en) Composition for treatment of Crigler-Najjar syndrome
US20210285013A1 (en) Gene therapy for treating familial hypercholesterolemia
JP2022544004A (ja) 操作された核酸調節エレメントならびにその使用方法
JP2023071829A (ja) 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

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200410

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20200410

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210428

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210728

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20210929

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220127

C60 Trial request (containing other claim documents, opposition documents)

Free format text: JAPANESE INTERMEDIATE CODE: C60

Effective date: 20220127

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20220207

C21 Notice of transfer of a case for reconsideration by examiners before appeal proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C21

Effective date: 20220209

A912 Re-examination (zenchi) completed and case transferred to appeal board

Free format text: JAPANESE INTERMEDIATE CODE: A912

Effective date: 20220311

C211 Notice of termination of reconsideration by examiners before appeal proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C211

Effective date: 20220316

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20230907

R150 Certificate of patent or registration of utility model

Ref document number: 7347933

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150