JP6822841B2 - オルニチントランスカルバミラーゼ(otc)欠損症の処置において有用な組成物 - Google Patents

オルニチントランスカルバミラーゼ(otc)欠損症の処置において有用な組成物 Download PDF

Info

Publication number
JP6822841B2
JP6822841B2 JP2016556768A JP2016556768A JP6822841B2 JP 6822841 B2 JP6822841 B2 JP 6822841B2 JP 2016556768 A JP2016556768 A JP 2016556768A JP 2016556768 A JP2016556768 A JP 2016556768A JP 6822841 B2 JP6822841 B2 JP 6822841B2
Authority
JP
Japan
Prior art keywords
nucleic acid
sequence
hotc
acid sequence
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
JP2016556768A
Other languages
English (en)
Japanese (ja)
Other versions
JP2017512466A (ja
JP2017512466A5 (cg-RX-API-DMAC7.html
Inventor
ワング,リリ
ウイルソン,ジエームス・エム
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 JP2017512466A publication Critical patent/JP2017512466A/ja
Publication of JP2017512466A5 publication Critical patent/JP2017512466A5/ja
Priority to JP2020054236A priority Critical patent/JP6920500B2/ja
Application granted granted Critical
Publication of JP6822841B2 publication Critical patent/JP6822841B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/1003Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1018Carboxy- and carbamoyl transferases (2.1.3)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y201/00Transferases transferring one-carbon groups (2.1)
    • C12Y201/03Carboxy- and carbamoyltransferases (2.1.3)
    • C12Y201/03003Ornithine carbamoyltransferase (2.1.3.3)
    • 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
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10311Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/14032Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • C12N2750/14033Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • 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
    • C12N2750/14041Use of virus, viral particle or viral elements as a vector
    • C12N2750/14043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/14071Demonstrated in vivo effect
    • 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
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
JP2016556768A 2014-03-09 2015-03-09 オルニチントランスカルバミラーゼ(otc)欠損症の処置において有用な組成物 Active JP6822841B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2020054236A JP6920500B2 (ja) 2014-03-09 2020-03-25 オルニチントランスカルバミラーゼ(otc)欠損症の処置において有用な組成物

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461950157P 2014-03-09 2014-03-09
US61/950,157 2014-03-09
PCT/US2015/019513 WO2015138348A1 (en) 2014-03-09 2015-03-09 Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2020054236A Division JP6920500B2 (ja) 2014-03-09 2020-03-25 オルニチントランスカルバミラーゼ(otc)欠損症の処置において有用な組成物

Publications (3)

Publication Number Publication Date
JP2017512466A JP2017512466A (ja) 2017-05-25
JP2017512466A5 JP2017512466A5 (cg-RX-API-DMAC7.html) 2018-03-29
JP6822841B2 true JP6822841B2 (ja) 2021-01-27

Family

ID=52737408

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2016556768A Active JP6822841B2 (ja) 2014-03-09 2015-03-09 オルニチントランスカルバミラーゼ(otc)欠損症の処置において有用な組成物
JP2020054236A Active JP6920500B2 (ja) 2014-03-09 2020-03-25 オルニチントランスカルバミラーゼ(otc)欠損症の処置において有用な組成物

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2020054236A Active JP6920500B2 (ja) 2014-03-09 2020-03-25 オルニチントランスカルバミラーゼ(otc)欠損症の処置において有用な組成物

Country Status (22)

Country Link
US (6) US10167454B2 (cg-RX-API-DMAC7.html)
EP (2) EP3116900B1 (cg-RX-API-DMAC7.html)
JP (2) JP6822841B2 (cg-RX-API-DMAC7.html)
KR (1) KR102390075B1 (cg-RX-API-DMAC7.html)
AU (2) AU2015229750A1 (cg-RX-API-DMAC7.html)
BR (2) BR112016020688B1 (cg-RX-API-DMAC7.html)
CA (1) CA2939950C (cg-RX-API-DMAC7.html)
CL (2) CL2016002235A1 (cg-RX-API-DMAC7.html)
CY (1) CY1123378T1 (cg-RX-API-DMAC7.html)
DK (1) DK3116900T3 (cg-RX-API-DMAC7.html)
ES (1) ES2821938T3 (cg-RX-API-DMAC7.html)
HR (1) HRP20201544T1 (cg-RX-API-DMAC7.html)
HU (1) HUE051311T2 (cg-RX-API-DMAC7.html)
IL (2) IL247329B (cg-RX-API-DMAC7.html)
LT (1) LT3116900T (cg-RX-API-DMAC7.html)
MX (1) MX377804B (cg-RX-API-DMAC7.html)
PL (1) PL3116900T3 (cg-RX-API-DMAC7.html)
PT (1) PT3116900T (cg-RX-API-DMAC7.html)
RS (1) RS60902B1 (cg-RX-API-DMAC7.html)
SI (1) SI3116900T1 (cg-RX-API-DMAC7.html)
SM (1) SMT202000531T1 (cg-RX-API-DMAC7.html)
WO (2) WO2015138357A2 (cg-RX-API-DMAC7.html)

Families Citing this family (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104623666A (zh) 2011-04-29 2015-05-20 西莱克塔生物科技公司 用于诱导调节性b细胞的致耐受性合成纳米载体
MX380164B (es) 2013-05-03 2025-03-12 Selecta Biosciences Inc Uso de una macromolécula terapéutica y una composición que comprende nanoportadores sintéticos que se unen a inmunosupresores para reducir la hipersensibilidad tipo i y tipo iv en un sujeto.
CA3160394C (en) 2013-07-30 2023-11-07 Genevant Sciences Gmbh Block copolymers and their conjugates or complexes with oligonucleotides
MX377804B (es) 2014-03-09 2025-03-11 Univ Pennsylvania Composiciones utiles en el tratamiento de deficiencia de la ornitina transcarbamilasa (otc).
IL292574A (en) 2014-09-07 2022-06-01 Selecta Biosciences Inc Methods and compositions for attenuating anti-viral transfer vector immune responses
RU2020109343A (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
CN114642735A (zh) 2015-01-21 2022-06-21 菲泽尔克斯公司 用于将治疗剂和诊断剂递送到细胞中的方法、组合物和系统
WO2017059902A1 (en) * 2015-10-07 2017-04-13 Biontech Rna Pharmaceuticals Gmbh 3' utr sequences for stabilization of rna
EP3452101A2 (en) 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
KR20230161535A (ko) 2016-07-26 2023-11-27 바이오마린 파머수티컬 인크. 신규 아데노-관련 바이러스 캡시드 단백질
IL315358A (en) 2016-08-18 2024-11-01 Univ California CRISPR-CAS genome engineering using a modular AAV delivery system
EP3538136A1 (en) * 2016-11-10 2019-09-18 Translate Bio, Inc. Subcutaneous delivery of messenger rna
WO2018126112A1 (en) * 2016-12-30 2018-07-05 The Trustees Of The University Of Pennsylvania Gene therapy for treating phenylketonuria
WO2018126084A1 (en) 2016-12-30 2018-07-05 Phaserx, Inc. Branched peg molecules and related compositions and methods
WO2018127382A1 (en) * 2017-01-03 2018-07-12 Ethris Gmbh Ornithine transcarbamylase coding polyribonucleotides and formulations thereof
DK3568483T3 (da) * 2017-01-10 2025-11-10 Childrens Medical Res Institute Polynukleotider og vektorer til ekspression af transgener
WO2018169811A1 (en) 2017-03-11 2018-09-20 Selecta Biosciences, Inc. Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
US11530402B2 (en) 2017-05-31 2022-12-20 The University Of North Carolina At Chapel Hill Optimized human clotting factor IX gene expression cassettes and their use
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
KR20250040746A (ko) 2017-08-09 2025-03-24 바이오버라티브 테라퓨틱스 인크. 핵산 분자 및 이의 용도
CA3079428A1 (en) 2017-11-22 2019-05-31 Modernatx, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders
AU2019265560A1 (en) 2018-05-09 2020-11-26 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
WO2019222277A1 (en) * 2018-05-15 2019-11-21 Translate Bio, Inc. Subcutaneous delivery of messenger rna
CA3099306A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
US20200038462A1 (en) * 2018-07-16 2020-02-06 Selecta Biosciences, Inc. Methods and compositions of otc constructs and vectors
SG11202100139VA (en) 2018-07-17 2021-02-25 Neuromyon Inc Treatment of neuropathy with dna constructs expressing igf-1 isoforms
JP7602454B2 (ja) 2018-08-09 2024-12-18 バイオベラティブ セラピューティクス インコーポレイテッド 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
JP7445657B2 (ja) * 2018-12-06 2024-03-07 アークトゥラス・セラピューティクス・インコーポレイテッド オルニチントランスカルバミラーゼ欠損症を処置するための組成物および方法
AU2020216480A1 (en) 2019-01-31 2021-09-09 Oregon Health & Science University Methods for using transcription-dependent directed evolution of AAV capsids
JP2023516676A (ja) 2020-03-03 2023-04-20 アークトゥラス・セラピューティクス・インコーポレイテッド オルニチントランスカルバミラーゼ欠損症の治療のための組成物及び方法
CA3169529A1 (en) * 2020-03-04 2021-09-10 Jingjing Jiang Compositions and methods for the treatment of metabolic liver disorders
EP4121544A1 (en) * 2020-03-19 2023-01-25 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
BR112022022212A2 (pt) 2020-05-12 2022-12-13 Univ Pennsylvania Composições para redução específica de drg de expressão de transgene
JP2024505257A (ja) 2021-02-01 2024-02-05 レジェンクスバイオ インコーポレーテッド 神経セロイドリポフスチン症の遺伝子治療
WO2022224372A1 (ja) * 2021-04-21 2022-10-27 学校法人自治医科大学 オルニチントランスカルバミラーゼ欠損症の治療用アデノ随伴ウイルスビリオン
WO2023028455A1 (en) * 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Closed-end dna production with inverted terminal repeat sequences
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
US20250161493A1 (en) * 2022-01-21 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions and methods for in vivo nuclease-mediated treatment of ornithine transcarbamylase (otc) deficiency
CA3268024A1 (en) * 2022-10-11 2024-04-18 Pacific Northwest Research Institute CONSTRUCTIONS RELATED TO GENETIC DISORDERS

Family Cites Families (81)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1016745A (en) 1911-04-11 1912-02-06 Edwin C Henrikson Table-caster.
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
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US5652224A (en) 1995-02-24 1997-07-29 The Trustees Of The University Of Pennsylvania Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism
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
AU722375B2 (en) 1996-09-06 2000-08-03 Trustees Of The University Of Pennsylvania, The Methods using cre-lox for production of recombinant adeno-associated viruses
US20020037867A1 (en) 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
ES2344660T3 (es) 1996-09-06 2010-09-02 The Trustees Of The University Of Pennsylvania Uso de virus adeno-asociados recombinantes en la preparacion de un medicamento para la terapia genica mediante celulas musculares.
JP2001500015A (ja) 1996-09-06 2001-01-09 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 T7ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法
US5866552A (en) 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
WO1998011244A2 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services Aav4 vector and uses thereof
WO1998024924A1 (en) 1996-12-05 1998-06-11 Introgene B.V. Genetic modification of primate hemopoietic repopulating stem cells
WO1998044151A1 (en) 1997-04-01 1998-10-08 Glaxo Group Limited Method of nucleic acid amplification
AU728220B2 (en) 1997-04-14 2001-01-04 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
CA2304168A1 (en) 1997-09-19 1999-04-01 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
CA2304131A1 (en) 1997-09-19 1999-04-01 James M. Wilson Method for gene transfer using bcl2 and compositions useful therein
AU9319198A (en) 1997-09-19 1999-04-05 Trustees Of The University Of Pennsylvania, The Methods and vector constructs useful for production of recombinant aav
AU3097399A (en) 1998-03-20 1999-10-11 Trustees Of The University Of Pennsylvania, The Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
EP1080218A1 (en) 1998-05-27 2001-03-07 University of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient
ES2313784T3 (es) 1998-05-28 2009-03-01 The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services Vector aav5 y usos del mismo.
EP1127150B1 (en) 1998-11-05 2007-05-16 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
WO2000028004A1 (en) 1998-11-10 2000-05-18 The University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP4693244B2 (ja) 1999-03-18 2011-06-01 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 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
JP2003501067A (ja) 1999-06-02 2003-01-14 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 ヘルパーウイルスを要求する組み換えウイルス産生のために有用な組成物及び方法
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
US6365394B1 (en) 1999-09-29 2002-04-02 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus
CA2384814A1 (en) 1999-09-29 2001-04-05 The Trustees Of The University Of Pennsylvania Methods for rapid peg-modification of viral vectors, compositions for enhanced gene transduction, compositions with enhanced physical stability, and uses therefor
US7115391B1 (en) 1999-10-01 2006-10-03 Genovo, Inc. Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
CA2385823A1 (en) 1999-10-01 2001-04-12 Haifeng Chen Production of recombinant aav using adenovirus comprising aav rep/cap genes
CA2392537A1 (en) 1999-12-03 2001-06-07 The Trustees Of The University Of Pennsylvania Compositions and methods for increasing packaging and yields of recombinant adenoviruses using multiple packaging signals
US6821512B1 (en) 1999-12-03 2004-11-23 The Trustees Of The University Of Pennsylvania Compositions and methods for increasing packaging and yield of recombinant adenoviruses using multiple packaging signals
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
US20060057553A1 (en) * 2000-05-30 2006-03-16 Estuardo Aguilar-Cordova Chimeric viral vectors for gene therapy
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
JP2004520812A (ja) 2000-08-30 2004-07-15 ハプロゲン・エルエルシー 対立遺伝子を決定するための方法
ATE403013T1 (de) 2001-05-18 2008-08-15 Wisconsin Alumni Res Found Verfahren zur synthese von dna-sequenzen die photolabile linker verwenden
EP1478772A2 (en) * 2001-08-14 2004-11-24 The General Hospital Corporation Nucleic acid and amino acid sequences involved in pain
US20040086485A1 (en) * 2001-10-04 2004-05-06 Aguilar-Cordova Carlos Estuardo Chemeric viral vectors for gene therapy
MX346493B (es) 2001-11-13 2017-03-21 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
JP4810062B2 (ja) 2001-12-17 2011-11-09 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 アデノ随伴ウイルス(aav)血清型8の配列
ES2526341T3 (es) 2001-12-17 2015-01-09 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas
AU2002367943A1 (en) 2001-12-18 2003-12-22 University Of North Carolina At Chapel Hill Improved reagents and methods for producing parvoviruses
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
CN102199626B (zh) 2003-09-30 2015-06-24 宾夕法尼亚大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
EP2383346B1 (en) 2005-04-07 2014-10-08 The Trustees of the University of Pennsylvania Modified AAVrh.64 capsids, compositions containing same and uses thereof
EP1777906A1 (en) 2005-06-09 2007-04-25 Matsushita Electric Industrial Co., Ltd. Amplitude error compensating apparatus and orthogonality error compensating apparatus
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
WO2008118212A2 (en) 2006-11-08 2008-10-02 Veritas, Llc In vivo delivery of double stranded rna to a target cell
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
EP3586868B1 (en) 2008-04-22 2023-11-08 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
CA3065577C (en) 2008-05-13 2022-05-31 Phaserx, Inc. Diblock copolymers and polynucleotide complexes thereof for delivery into cells
JP2012507581A (ja) 2008-11-06 2012-03-29 ユニヴァーシティ オブ ワシントン 二重特異性細胞内送達媒体
BRPI0921357A2 (pt) 2008-11-06 2016-06-21 Phaserx Inc copolímeros em multibloco
CN104910025B (zh) 2008-11-07 2019-07-16 麻省理工学院 氨基醇类脂质和其用途
SG177744A1 (en) 2009-07-24 2012-02-28 Immune Design Corp Lentiviral vectors pseudotyped with a sindbis virus envelope glycoprotein
EP2281579A1 (en) 2009-08-05 2011-02-09 BioNTech AG Vaccine composition comprising 5'-Cap modified RNA
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
EP2553106A2 (en) 2010-03-29 2013-02-06 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
KR101811771B1 (ko) 2011-05-12 2017-12-22 히다치 겡키 가부시키 가이샤 건설기계
EP2717893B1 (en) 2011-06-08 2019-05-08 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mrna delivery
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
WO2013063468A1 (en) 2011-10-27 2013-05-02 Massachusetts Institute Of Technology Amino acid derivates functionalized on the n- terminal capable of forming drug incapsulating microspheres
WO2013143555A1 (en) 2012-03-26 2013-10-03 Biontech Ag Rna formulation for immunotherapy
AU2013243949A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
WO2013151663A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
WO2013182683A1 (en) 2012-06-08 2013-12-12 Ethris Gmbh Pulmonary delivery of messenger rna
CA3160394C (en) * 2013-07-30 2023-11-07 Genevant Sciences Gmbh Block copolymers and their conjugates or complexes with oligonucleotides
MX377804B (es) * 2014-03-09 2025-03-11 Univ Pennsylvania Composiciones utiles en el tratamiento de deficiencia de la ornitina transcarbamilasa (otc).

Also Published As

Publication number Publication date
WO2015138357A3 (en) 2015-12-10
US9890365B2 (en) 2018-02-13
US11732246B2 (en) 2023-08-22
US10167454B2 (en) 2019-01-01
IL247329B (en) 2020-07-30
HRP20201544T1 (hr) 2021-02-19
EP3778627A1 (en) 2021-02-17
US20170051259A1 (en) 2017-02-23
EP3116900B1 (en) 2020-07-08
WO2015138357A2 (en) 2015-09-17
US20180135030A1 (en) 2018-05-17
KR102390075B1 (ko) 2022-04-26
PL3116900T3 (pl) 2021-03-08
KR20160124229A (ko) 2016-10-26
LT3116900T (lt) 2020-12-10
BR122023023004A2 (pt) 2023-12-26
BR112016020688A2 (pt) 2017-10-03
IL275799B2 (en) 2023-02-01
RS60902B1 (sr) 2020-11-30
ES2821938T3 (es) 2021-04-28
CL2016002235A1 (es) 2017-06-23
JP2017512466A (ja) 2017-05-25
NZ761706A (en) 2024-04-26
NZ724165A (en) 2024-04-26
MX377804B (es) 2025-03-11
AU2020201190A1 (en) 2020-03-05
WO2015138348A1 (en) 2015-09-17
EP3116900A1 (en) 2017-01-18
MX2016011686A (es) 2016-11-07
SI3116900T1 (sl) 2021-02-26
AU2020201190B2 (en) 2022-04-07
US20170021037A1 (en) 2017-01-26
IL275799B (en) 2022-10-01
CA2939950A1 (en) 2015-09-17
CA2939950C (en) 2023-08-22
US20230416700A1 (en) 2023-12-28
JP6920500B2 (ja) 2021-08-18
US10781430B2 (en) 2020-09-22
DK3116900T3 (da) 2020-09-28
BR112016020688B1 (pt) 2024-01-30
US10626382B2 (en) 2020-04-21
CL2019002280A1 (es) 2019-11-29
HUE051311T2 (hu) 2021-03-01
CY1123378T1 (el) 2021-12-31
IL247329A0 (en) 2016-09-29
SMT202000531T1 (it) 2020-11-10
JP2020114235A (ja) 2020-07-30
US20200377866A1 (en) 2020-12-03
IL275799A (en) 2020-08-31
PT3116900T (pt) 2020-10-08
AU2015229750A1 (en) 2016-09-15
US20190071651A1 (en) 2019-03-07

Similar Documents

Publication Publication Date Title
JP6920500B2 (ja) オルニチントランスカルバミラーゼ(otc)欠損症の処置において有用な組成物
JP6851319B2 (ja) ヒト疾患のCRISPR/Cas9媒介性の修正のためのデュアルAAVベクター系
JP2022528010A (ja) メープルシロップ尿症(msud)のaav媒介遺伝子治療
EP4509606A1 (en) Nucleic acid construct for treating hereditary coagulation factor deficiency and use thereof
CN108239645B (zh) 肝脏特异性转录调控序列及其应用
US20220370638A1 (en) Compositions and methods for treatment of maple syrup urine disease
WO2024015972A2 (en) Compositions and methods for in vivo nuclease-mediated gene targeting for the treatment of genetic disorders in adult patients
CN119630433A (zh) 基因疗法组合物及其使用方法
WO2019036484A1 (en) COMPOSITIONS AND METHODS FOR THE TREATMENT OF ARGININOSUCCINIC ACIDURIA
WO2025217186A1 (en) Compositions and methods for in vivo nuclease-mediated treatment of citrullinemia type 1 (ctln1)
WO2022221276A1 (en) Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
AU2022290521A1 (en) Recombinant adeno-associated viruses for lesch-nyhan disorders and uses thereof

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20180216

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20180216

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20181220

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20181219

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20190319

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190520

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20190925

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20191225

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20200218

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200325

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20200701

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20201001

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20201126

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20201209

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20210107

R150 Certificate of patent or registration of utility model

Ref document number: 6822841

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250