[go: up one dir, main page]

CL2019002280A1 - Vectores virales, adenovirales y composiciones a base de una secuencia de ornitina transcarbamilasa humana (hotch) modificada genéticamente y su uso en el tratamiento de deficiencia de la otc. (divisional solicitud 201602235) - Google Patents

Vectores virales, adenovirales y composiciones a base de una secuencia de ornitina transcarbamilasa humana (hotch) modificada genéticamente y su uso en el tratamiento de deficiencia de la otc. (divisional solicitud 201602235)

Info

Publication number
CL2019002280A1
CL2019002280A1 CL2019002280A CL2019002280A CL2019002280A1 CL 2019002280 A1 CL2019002280 A1 CL 2019002280A1 CL 2019002280 A CL2019002280 A CL 2019002280A CL 2019002280 A CL2019002280 A CL 2019002280A CL 2019002280 A1 CL2019002280 A1 CL 2019002280A1
Authority
CL
Chile
Prior art keywords
hotc
nucleic acid
acid sequence
sequence
treatment
Prior art date
Application number
CL2019002280A
Other languages
English (en)
Inventor
Lili Wang
James M Wilson
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of CL2019002280A1 publication Critical patent/CL2019002280A1/es

Links

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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1018—Carboxy- and carbamoyl transferases (2.1.3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/03—Carboxy- and carbamoyltransferases (2.1.3)
    • C12Y201/03003—Ornithine carbamoyltransferase (2.1.3.3)
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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
    • 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
    • C12N2750/14032—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • C12N2750/14033—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • 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
    • C12N2750/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14043—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
    • 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
    • C12N2750/14071—Demonstrated in vivo effect
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors 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)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Un vector viral recombinante que Comprende una secuencia del ácido nucleico que codifica la ornitina transcarbamilasa humana (hOTC) y secuencias de control de la expresión que dirigen la expresión de hOTO en una célula del hígado, en donde la secuencia del ácido nucleico de hOTC es menos del 80% idéntica a la secuencia de hOTC de tipo salvaje sobre la secuencia madura o hOTC de longitud completa de SEQ ID N0:1, y expresa una hOTC funcional, en donde dicha secuencia del ácido nucleico de hOTC se selecciona de la secuencia del ácido nucleico que comprende SEQ ID NO:9 o una secuencia del ácido nucleico al menos aproximadamente 96 a aproximadamente 99% idéntica a ésta. Se provee una composición farmacéutica, un virus adenoasociado recombinante (rAAV)que comprenden secuencias de ADN y ARN de hOTC modificadas genéticamente que cuando se suministran a un sujeto en necesidad del mismo son útiles para el tratamiento de la hiperamonemia, deficiencia de ornitina transcarbamilasa y síntomas asociados con la misma. También se provee el uso de hOTC para el tratamiento de la cirrosis fibrosis hepática en pacientes mediante de OTCD mediante la administración de hOTC.
CL2019002280A 2014-03-09 2019-08-12 Vectores virales, adenovirales y composiciones a base de una secuencia de ornitina transcarbamilasa humana (hotch) modificada genéticamente y su uso en el tratamiento de deficiencia de la otc. (divisional solicitud 201602235) CL2019002280A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201461950157P 2014-03-09 2014-03-09

Publications (1)

Publication Number Publication Date
CL2019002280A1 true CL2019002280A1 (es) 2019-11-29

Family

ID=52737408

Family Applications (2)

Application Number Title Priority Date Filing Date
CL2016002235A CL2016002235A1 (es) 2014-03-09 2016-09-06 Composiciones útiles en el tratamiento de deficiencia de ornitina-transcarbamilasa (otc)
CL2019002280A CL2019002280A1 (es) 2014-03-09 2019-08-12 Vectores virales, adenovirales y composiciones a base de una secuencia de ornitina transcarbamilasa humana (hotch) modificada genéticamente y su uso en el tratamiento de deficiencia de la otc. (divisional solicitud 201602235)

Family Applications Before (1)

Application Number Title Priority Date Filing Date
CL2016002235A CL2016002235A1 (es) 2014-03-09 2016-09-06 Composiciones útiles en el tratamiento de deficiencia de ornitina-transcarbamilasa (otc)

Country Status (22)

Country Link
US (6) US10167454B2 (es)
EP (2) EP3116900B1 (es)
JP (2) JP6822841B2 (es)
KR (1) KR102390075B1 (es)
AU (2) AU2015229750A1 (es)
BR (2) BR112016020688B1 (es)
CA (1) CA2939950C (es)
CL (2) CL2016002235A1 (es)
CY (1) CY1123378T1 (es)
DK (1) DK3116900T3 (es)
ES (1) ES2821938T3 (es)
HR (1) HRP20201544T1 (es)
HU (1) HUE051311T2 (es)
IL (2) IL247329B (es)
LT (1) LT3116900T (es)
MX (1) MX377804B (es)
PL (1) PL3116900T3 (es)
PT (1) PT3116900T (es)
RS (1) RS60902B1 (es)
SI (1) SI3116900T1 (es)
SM (1) SMT202000531T1 (es)
WO (2) WO2015138348A1 (es)

Families Citing this family (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117205331A (zh) 2011-04-29 2023-12-12 西莱克塔生物科技公司 用于降低抗体应答的致耐受性合成纳米载体
CN110639024A (zh) 2013-05-03 2020-01-03 西莱克塔生物科技公司 用于增强cd4+调节性t细胞的方法和组合物
CA3160394C (en) 2013-07-30 2023-11-07 Genevant Sciences Gmbh Block copolymers and their conjugates or complexes with oligonucleotides
US10167454B2 (en) 2014-03-09 2019-01-01 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of ornithine transcarbamylase (OTC) deficiency
BR112017001601A2 (pt) 2014-09-07 2017-11-21 Selecta Biosciences Inc métodos e composições para atenuar as respostas imunes do vetor de transferência antiviral de modulação da expressão genética
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
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
US11584780B2 (en) 2016-07-26 2023-02-21 Biomarin Pharmaceutical Inc. Adeno-associated virus capsid proteins
JP2019524162A (ja) 2016-08-18 2019-09-05 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 モジュラーＡＡＶ送達システムによるＣＲＩＳＰＲ−Ｃａｓゲノム編集
EP3538136A1 (en) * 2016-11-10 2019-09-18 Translate Bio, Inc. Subcutaneous delivery of messenger rna
KR102738752B1 (ko) * 2016-12-30 2024-12-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 페닐케톤뇨증을 치료하기 위한 유전자 치료
US11684584B2 (en) 2016-12-30 2023-06-27 Genevant Sciences Gmbh 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
US11938197B2 (en) 2017-01-10 2024-03-26 The Sydney Children's Hospitals Network (Randwick And Westmead (Incorporating The Royal Alexandra Hospital For Children) Polynucleotides and vectors for the expression of transgenes
MX2019010757A (es) 2017-03-11 2020-01-20 Selecta Biosciences Inc Métodos y composiciones relacionados con el tratamiento combinado con antiinflamatorios y nanoportadores sintéticos que comprenden un inmunosupresor.
CN114875051A (zh) 2017-05-31 2022-08-09 北卡罗来纳大学教堂山分校 优化的人凝血因子ix基因表达盒及其应用
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
BR112020002394A2 (pt) 2017-08-09 2020-07-28 Bioverativ Therapeutics Inc. moléculas de ácidos nucleicos e usos das mesmas
EP3714048B1 (en) 2017-11-22 2025-04-09 ModernaTX, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders
EP3790627A2 (en) 2018-05-09 2021-03-17 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
US12319929B2 (en) 2018-05-15 2025-06-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
WO2020018583A1 (en) 2018-07-16 2020-01-23 Selecta Biosciences, Inc. Methods and compositions of otc constructs and vectors
AU2019304569B2 (en) 2018-07-17 2023-07-06 Helixmith Co., Ltd 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
CN121160803A (zh) 2018-09-28 2025-12-19 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
WO2020118115A1 (en) * 2018-12-06 2020-06-11 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
KR20210130158A (ko) 2019-01-31 2021-10-29 오레곤 헬스 앤드 사이언스 유니버시티 Aav 캡시드의 전사 의존적 유도 진화를 사용하는 방법
WO2021178510A1 (en) 2020-03-03 2021-09-10 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
JP2023515692A (ja) * 2020-03-04 2023-04-13 ポセイダ セラピューティクス，インコーポレイティド 代謝性肝障害の治療のための組成物と方法
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
WO2021231579A1 (en) 2020-05-12 2021-11-18 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
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
JP2024534124A (ja) * 2021-08-23 2024-09-18 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 逆位末端反復配列によるクローズドエンドｄｎａの作製
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
CA3242279A1 (en) * 2022-01-21 2023-07-27 Lili Wang Methods for treatment of ornithine transcarbamylase (otc) deficiency
WO2024081592A2 (en) * 2022-10-11 2024-04-18 Pacific Northwest Research Institute Constructs relating 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
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
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
EP0950111A1 (en) 1996-09-06 1999-10-20 The Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US20020037867A1 (en) 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
EP1696036B1 (en) 1996-09-06 2010-04-21 The Trustees of The University of Pennsylvania Use of recombinant adeno-associated virus in the manufacture of a medicament for gene therapy via muscle cells
EP0932694A2 (en) 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav4 vector and uses thereof
NZ336185A (en) 1996-12-05 2001-02-23 Introgene Bv Genetic modification of primate hemopoietic repopulating stem cells
AU6846698A (en) 1997-04-01 1998-10-22 Glaxo Group Limited Method of nucleic acid amplification
DE69824859T2 (de) 1997-04-14 2005-08-04 Cell Genesys, Inc., Foster City Methoden zur erhöhung der effizienz rekombinanter aav produkte
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
AU9774998A (en) 1997-09-19 1999-04-12 Trustees Of The University Of Pennsylvania, The 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
WO1999015685A1 (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
JP2002506652A (ja) 1998-03-20 2002-03-05 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を含まない製造のための組成物及び方法
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
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
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
ES2288037T3 (es) 1998-11-05 2007-12-16 The Trustees Of The University Of Pennsylvania Secuencia de acido nucleico del serotipo 1 de adenovirus asociado, vectores y celulas huesped que las contienen.
EP1135468B1 (en) 1998-11-10 2010-01-06 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
CA2375098A1 (en) 1999-06-02 2000-12-14 Trustees Of The University Of Pennsylvania Compositions and methods useful for production of recombinant viruses which require helper viruses
EP2369002A1 (en) 1999-08-09 2011-09-28 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
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
AU1071701A (en) 1999-09-29 2001-04-30 Trustees Of The University Of Pennsylvania, The Methods for rapid peg-modification of viral vectors, compositions for enhanced gene transduction, compositions with enhanced physical stability, and uses therefor
EP1222299A1 (en) 1999-10-01 2002-07-17 Genovo, Incorporated Production of recombinant aav using adenovirus comprising aav rep/cap genes
US7115391B1 (en) 1999-10-01 2006-10-03 Genovo, Inc. Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
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
AU1796801A (en) 1999-12-03 2001-06-12 Trustees Of The University Of Pennsylvania, The Compositions and methods for increasing packaging and yields 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
CA2406743A1 (en) 2000-04-28 2001-11-08 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
AU2001289177A1 (en) 2000-08-30 2002-03-13 Haplogen, Llc Method for determining alleles
ATE403013T1 (de) 2001-05-18 2008-08-15 Wisconsin Alumni Res Found Verfahren zur synthese von dna-sequenzen die photolabile linker verwenden
US20070015145A1 (en) 2001-08-14 2007-01-18 Clifford Woolf Nucleic acid and amino acid sequences involved in pain
EP1442125A4 (en) * 2001-10-04 2004-12-08 Carlos Estuard Aguilar-Cordova CHIMERAL VIRUS VECTORS FOR GENE THERAPY
CA2406745C (en) 2001-11-13 2006-01-10 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
CA2469785C (en) 2001-12-17 2014-04-22 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
DK1463805T3 (en) 2001-12-17 2015-01-19 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 9, vectors containing these as well as their uses
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
EP3211085B1 (en) 2003-09-30 2021-03-31 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
JP5702519B2 (ja) 2005-04-07 2015-04-15 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 Ａａｖベクターの機能を上げる方法
WO2006132118A1 (ja) 2005-06-09 2006-12-14 Matsushita Electric Industrial Co., Ltd. 振幅誤差補償装置及び直交度誤差補償装置
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
ES2434169T3 (es) 2006-11-08 2013-12-13 Veritas Bio, Llc Entrega in vivo de ARN bicatenario a una célula objetivo
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
EP4154903A1 (en) 2008-04-22 2023-03-29 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
CA2724105C (en) 2008-05-13 2017-09-05 University Of Washington Diblock copolymers and polynucleotide complexes thereof for delivery into cells
CA2742880A1 (en) 2008-11-06 2010-05-14 University Of Washington Multiblock copolymers
US8822213B2 (en) 2008-11-06 2014-09-02 University Of Washington Bispecific intracellular delivery vehicles
WO2010053572A2 (en) 2008-11-07 2010-05-14 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
MX2012001075A (es) 2009-07-24 2012-05-22 Immune Design Corp Vectores lentivirales pseudotipificados con una glicoproteina de envoltura de virus sindbis.
EP2281579A1 (en) 2009-08-05 2011-02-09 BioNTech AG Vaccine composition comprising 5'-Cap modified RNA
MX342858B (es) 2010-03-29 2016-10-13 The Trustees Of The Univ Of Pennsylvania * Sistema de ablacion transgenica inducida farmacologicamente.
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
EP2708660B1 (en) 2011-05-12 2017-09-20 Hitachi Construction Machinery Co., Ltd. Construction machine
CN103906527B (zh) 2011-06-08 2020-07-10 川斯勒佰尔公司 Mrna递送的脂质纳米颗粒组合物和方法
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
EP2771319B1 (en) 2011-10-27 2022-03-02 Massachusetts Institute of Technology Amino acid derivatives 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
CN104411338A (zh) * 2012-04-02 2015-03-11 现代治疗公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
JP2015513912A (ja) * 2012-04-02 2015-05-18 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． タンパク質の産生のための修飾ポリヌクレオチド
JP6208750B2 (ja) 2012-06-08 2017-10-04 エスリス ゲーエムベーハーｅｔｈｒｉｓ ＧｍｂＨ メッセンジャーｒｎａの肺送達
CA3160394C (en) * 2013-07-30 2023-11-07 Genevant Sciences Gmbh Block copolymers and their conjugates or complexes with oligonucleotides
US10167454B2 (en) * 2014-03-09 2019-01-01 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of ornithine transcarbamylase (OTC) deficiency

Also Published As

Publication number Publication date
DK3116900T3 (da) 2020-09-28
CA2939950A1 (en) 2015-09-17
CA2939950C (en) 2023-08-22
US20180135030A1 (en) 2018-05-17
BR112016020688B1 (pt) 2024-01-30
EP3116900B1 (en) 2020-07-08
WO2015138348A1 (en) 2015-09-17
JP6920500B2 (ja) 2021-08-18
WO2015138357A2 (en) 2015-09-17
US20190071651A1 (en) 2019-03-07
IL275799B2 (en) 2023-02-01
US10626382B2 (en) 2020-04-21
US11732246B2 (en) 2023-08-22
AU2020201190A1 (en) 2020-03-05
CY1123378T1 (el) 2021-12-31
NZ724165A (en) 2024-04-26
CL2016002235A1 (es) 2017-06-23
US20170051259A1 (en) 2017-02-23
SMT202000531T1 (it) 2020-11-10
HUE051311T2 (hu) 2021-03-01
IL247329A0 (en) 2016-09-29
SI3116900T1 (sl) 2021-02-26
RS60902B1 (sr) 2020-11-30
ES2821938T3 (es) 2021-04-28
IL275799A (en) 2020-08-31
MX377804B (es) 2025-03-11
US20200377866A1 (en) 2020-12-03
BR122023023004A2 (pt) 2023-12-26
US9890365B2 (en) 2018-02-13
KR102390075B1 (ko) 2022-04-26
MX2016011686A (es) 2016-11-07
LT3116900T (lt) 2020-12-10
US20170021037A1 (en) 2017-01-26
KR20160124229A (ko) 2016-10-26
AU2015229750A1 (en) 2016-09-15
EP3116900A1 (en) 2017-01-18
JP2020114235A (ja) 2020-07-30
US10167454B2 (en) 2019-01-01
US10781430B2 (en) 2020-09-22
PT3116900T (pt) 2020-10-08
HRP20201544T1 (hr) 2021-02-19
NZ761706A (en) 2024-04-26
AU2020201190B2 (en) 2022-04-07
WO2015138357A3 (en) 2015-12-10
US20230416700A1 (en) 2023-12-28
PL3116900T3 (pl) 2021-03-08
EP3778627A1 (en) 2021-02-17
IL247329B (en) 2020-07-30
IL275799B (en) 2022-10-01
BR112016020688A2 (pt) 2017-10-03
JP6822841B2 (ja) 2021-01-27
JP2017512466A (ja) 2017-05-25

Similar Documents

Publication Publication Date Title
CL2019002280A1 (es) Vectores virales, adenovirales y composiciones a base de una secuencia de ornitina transcarbamilasa humana (hotch) modificada genéticamente y su uso en el tratamiento de deficiencia de la otc. (divisional solicitud 201602235)
CL2019002474A1 (es) Vector clado f de virus adeno-asociado (vaa) y usos relacionados.
MX2016010649A (es) Vector de virus adeno-asociado.
JP2017538401A5 (es)
FI3872085T3 (fi) Esimuotoiset virussekvenssit ja niiden käytöt
EA201692359A1 (ru) Композиция на основе нового штамма prrsv или его белков, продукт, их применение, вирус prrsv, выделенная нк и рекомбинантный вектор экспрессии для получения этого вируса
RU2016149206A (ru) Аденоассоциированные вирусные векторы для лечения лизосомных болезней накопления
JP2015096070A5 (es)
NZ603348A (en) Simian adenovirus nucleic acid- and amino acid-sequences, vectors containing same, and uses thereof
MX2020000221A (es) Secuencias de aminoacidos y de acidos nucleicos de adenovirus de grandes simios no humanos, vectores que contienen las mismas y usos de las mismas.
JP2018520997A5 (es)
EA201690545A1 (ru) Онколитические аденовирусы, несущие гетерологичные гены
WO2013151670A3 (en) Modified polynucleotides for the production of nuclear proteins
HRP20231183T1 (hr) Optimizirani gen faktora viii
RU2015135890A (ru) Композиция вакцины
JP2014517694A5 (es)
AR065075A1 (es) Vectores para la expresion multiple de genes
JP2018531624A5 (es)
JP2017524380A5 (ja) 腫瘍血管遮断剤であるポリペプチド、遺伝子、発現ベクター、腫瘍血管遮断剤の組成物、及び腫瘍血管遮断剤であるポリペプチドの調製方法
BR112021017853A2 (pt) Vetores de dna não virais e usos dos mesmos para expressar terapêuticas de fviii
RU2015112625A (ru) Пептид, применение пептида для лечения сердечно-сосудистых заболеваний, нуклеотидная последовательность, вектор экспрессии, хозяйская клетка, фармацевтическая композиция
NZ706884A (en) Fc gamma receptor iib variants
PE20221254A1 (es) Proteina vp1 modificada aislada de la capside del virus adeno-asociado de serotipo 5 (aav5), capside y vector basado en la misma
EA201890274A1 (ru) Векторная система из аденовируса 9 птиц (fadv-9) и связанные способы
EA201790296A1 (ru) Вакцина в виде рекомбинантного вектора на основе птичьего аденовируса серотипа 9