PE20191261A1 - Composiciones utiles para el tratamiento de atrofia muscular espinal - Google Patents

Composiciones utiles para el tratamiento de atrofia muscular espinal

Info

Publication number
PE20191261A1
PE20191261A1 PE2019001774A PE2019001774A PE20191261A1 PE 20191261 A1 PE20191261 A1 PE 20191261A1 PE 2019001774 A PE2019001774 A PE 2019001774A PE 2019001774 A PE2019001774 A PE 2019001774A PE 20191261 A1 PE20191261 A1 PE 20191261A1
Authority
PE
Peru
Prior art keywords
amino acids
seq
aavhu68
nucleotide sequence
treatment
Prior art date
Application number
PE2019001774A
Other languages
English (en)
Inventor
James M Wilson
Christian Hinderer
Nathan Katz
Qiang Wang
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 PE20191261A1 publication Critical patent/PE20191261A1/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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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

Abstract

La presente invencion se refiere a un vector viral recombinante adenoasociado (rAAV) con una capside AAVhu68 y un casete de expresion, el cual comprende secuencias de acido nucleico que codifican una proteina de supervivencia de las neuronas motoras (SMN) funcional y secuencias de control de expresion que dirigen la expresion de las secuencias SMN en una celula huesped. La capside AAVhu68 comprende: una poblacion heterogenea de proteinas vp1 producidas a partir de la secuencia de nucleotidos SEC ID NO: 7 (aminoacidos 1 a 736) o SEC ID NO:8 (aminoacidos 1 a 736); una poblacion heterologa de proteinas vp2 AAVhu68 producidas a partir de la secuencia de nucleotidos SEC ID NO: 8 (aminoacidos 138 a 736) o SEC ID NO: 7 (aminoacidos 412 a 2211); una poblacion heterogenea de proteinas vp3 AAVhu68 producidas a partir de la secuencia de nucleotidos SEC ID NO: 8 (aminoacidos 203 a 736) o SEC ID NO: 7 (aminoacidos 607 a 2211). Dicho vector es util en el tratamiento de atrofia muscular espinal. Tambien se refiere a una composicion farmaceutica.
PE2019001774A 2017-02-28 2018-02-27 Composiciones utiles para el tratamiento de atrofia muscular espinal PE20191261A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762464756P 2017-02-28 2017-02-28
US201762515902P 2017-06-06 2017-06-06
US201862618437P 2018-01-17 2018-01-17

Publications (1)

Publication Number Publication Date
PE20191261A1 true PE20191261A1 (es) 2019-09-18

Family

ID=61800665

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2019001774A PE20191261A1 (es) 2017-02-28 2018-02-27 Composiciones utiles para el tratamiento de atrofia muscular espinal

Country Status (18)

Country Link
US (2) US11578341B2 (es)
EP (1) EP3589739A2 (es)
JP (2) JP7037574B2 (es)
KR (1) KR20190123752A (es)
CN (1) CN110741089A (es)
AU (1) AU2018227442A1 (es)
BR (1) BR112019016444A2 (es)
CA (1) CA3055619A1 (es)
CL (1) CL2019002473A1 (es)
CO (1) CO2019009522A2 (es)
IL (1) IL268519A (es)
JO (1) JOP20190200A1 (es)
MA (1) MA47688A (es)
MX (1) MX2019010276A (es)
PE (1) PE20191261A1 (es)
PH (1) PH12019550149A1 (es)
SG (2) SG11201907474UA (es)
WO (1) WO2018160585A2 (es)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3528852A4 (en) 2016-10-20 2020-06-03 Sangamo Therapeutics, Inc. METHODS AND COMPOSITIONS FOR THE TREATMENT OF FABRY'S DISEASE
AU2018227440A1 (en) 2017-02-28 2019-08-29 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clade f vector and uses therefor
KR20210006327A (ko) * 2018-02-27 2021-01-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 캡시드 탈아미드화가 감소된 신규 아데노-연관 바이러스(aav) 벡터 및 이의 용도
CN113646005A (zh) * 2018-12-21 2021-11-12 宾夕法尼亚州大学信托人 用于drg特异性降低转基因表达的组合物
SG11202107325TA (en) * 2019-01-04 2021-08-30 Sangamo Therapeutics Inc Methods and compositions for the treatment of fabry disease
WO2021183469A1 (en) * 2020-03-09 2021-09-16 Amprion, Inc. Cerebrospinal fluid assay control solution
BR112022022212A2 (pt) 2020-05-12 2022-12-13 Univ Pennsylvania Composições para redução específica de drg de expressão de transgene
CA3209779A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
CN112852882A (zh) * 2021-02-04 2021-05-28 中吉智药(南京)生物技术有限公司 一种杆状病毒感染昆虫细胞生产aav基因药物的系统及方法
WO2023061499A1 (en) * 2021-10-15 2023-04-20 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating spinal muscular atrophy
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
CN114177346B (zh) * 2021-12-24 2023-05-26 中国人民解放军军事科学院军事医学研究院 一种止血组合物及止血贴与其应用

Family Cites Families (149)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4816567A (en) 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
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
US7101993B1 (en) 1990-01-11 2006-09-05 Isis Pharmaceuticals, Inc. Oligonucleotides containing 2′-O-modified purines
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
EP0682717A4 (en) 1993-02-05 1997-04-23 Laporte Group Australia ANTI-FOAMING COMPOSITE FOR DAIRY.
PL310327A1 (en) 1993-02-12 1995-12-11 Univ Leland Stanford Junior Adjustable transcription of target genes and other biological processes
US6972193B1 (en) 1993-02-12 2005-12-06 Board Of Trustees Of Leland Stanford Junior University Regulated transcription of targeted genes and other biological events
US20020173474A1 (en) 1993-02-12 2002-11-21 President And Fellows Of Harvard College Methods & materials involving dimerization-mediated regulation of biological events
US5830462A (en) 1993-02-12 1998-11-03 President & Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
US5869337A (en) 1993-02-12 1999-02-09 President And Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
US6140120A (en) 1993-02-12 2000-10-31 Board Of Trustees Of Leland Stanford Jr. University Regulated transcription of targeted genes and other biological events
US5834266A (en) 1993-02-12 1998-11-10 President & Fellows Of Harvard College Regulated apoptosis
US6150137A (en) 1994-05-27 2000-11-21 Ariad Pharmaceuticals, Inc. Immunosuppressant target proteins
US6476200B1 (en) 1994-06-27 2002-11-05 The Johns Hopkins University Mammalian proteins that bind to FKBP12 in a rapamycin-dependent fashion
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
CA2197793C (en) 1994-08-18 2007-04-24 Dennis A. Holt New multimerizing agents
US6133456A (en) 1994-08-18 2000-10-17 Ariad Gene Therapeutics, Inc. Synthetic multimerizing agents
US6150527A (en) 1994-08-18 2000-11-21 Ariad Pharmaceuticals, Inc. Synthetic multimerizing agents
US5786464C1 (en) 1994-09-19 2012-04-24 Gen Hospital Corp Overexpression of mammalian and viral proteins
US6326166B1 (en) 1995-12-29 2001-12-04 Massachusetts Institute Of Technology Chimeric DNA-binding proteins
EP0805819B1 (en) 1994-12-29 2012-02-08 Massachusetts Institute Of Technology Chimeric dna-binding proteins
US6166197A (en) 1995-03-06 2000-12-26 Isis Pharmaceuticals, Inc. Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5811524A (en) 1995-06-07 1998-09-22 Idec Pharmaceuticals Corporation Neutralizing high affinity human monoclonal antibodies specific to RSV F-protein and methods for their manufacture and therapeutic use thereof
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
WO1996041865A1 (en) 1995-06-07 1996-12-27 Ariad Gene Therapeutics, Inc. Rapamcycin-based regulation of biological events
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
JP4936345B2 (ja) 1996-02-28 2012-05-23 アリアド・ファーマシューティカルズ・インコーポレイテッド イムノフィリン由来ドメインとのキメラタンパク質用の多量体化剤としてのラパマイシンの合成誘導体
US6723531B2 (en) 1996-04-05 2004-04-20 The Salk Institute For Biological Studies Method for modulating expression of exogenous genes in mammalian systems, and products related thereto
WO1998002441A2 (en) 1996-07-12 1998-01-22 Ariad Pharmaceuticals, Inc. Non immunosuppressive antifungal rapalogs
ES2563643T3 (es) 1997-04-01 2016-03-15 Illumina Cambridge Limited Método de secuenciación de ácido nucleico
AU728220B2 (en) 1997-04-14 2001-01-04 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
US6015709A (en) 1997-08-26 2000-01-18 Ariad Pharmaceuticals, Inc. Transcriptional activators, and compositions and uses related thereto
JP2003524368A (ja) 1997-08-26 2003-08-19 アリアド ジーン セラピューティクス インコーポレイテッド ニ量化ドメイン、三量化ドメインまたは四量化ドメインおよび補足的非相同転写活性化ドメイン、転写抑制ドメイン、ｄｎａ結合ドメインまたはリガンド結合ドメインを含む融合蛋白
US6479653B1 (en) 1997-08-26 2002-11-12 Ariad Gene Therapeutics, Inc. Compositions and method for regulation of transcription
IL134643A0 (en) 1997-08-27 2001-04-30 Ariad Gene Therapeutics Inc Chimeric transcriptional activators and compositions and uses related thereto
JP2002508971A (ja) 1998-01-15 2002-03-26 アリアド・ジーン・セラピューティクス・インコーポレーテッド 多量体キメラ蛋白質を使用する生物学的イベントの調節
US6984635B1 (en) 1998-02-13 2006-01-10 Board Of Trustees Of The Leland Stanford Jr. University Dimerizing agents, their production and use
JP2002503667A (ja) 1998-02-13 2002-02-05 プレジデント・アンド・フェローズ・オブ・ハーバード・カレッジ 新規な二量体化剤、その製造および使用
US6333318B1 (en) 1998-05-14 2001-12-25 The Salk Institute For Biological Studies Formulations useful for modulating expression of exogenous genes in mammalian systems, and products related thereto
EP1080218A1 (en) 1998-05-27 2001-03-07 University of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient
US6258603B1 (en) 1998-06-17 2001-07-10 Rohm And Haas Company Ligands for modulating the expression of exogenous genes via an ecdysone receptor complex
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US7109317B1 (en) 1998-11-06 2006-09-19 President And Fellows Of Harvard College FK506-based regulation of biological events
CA2348382C (en) 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Chimeric parvovirus vectors and methods of making and administering the same
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
US7067526B1 (en) 1999-08-24 2006-06-27 Ariad Gene Therapeutics, Inc. 28-epirapalogs
ES2219388T3 (es) 1999-08-24 2004-12-01 Ariad Gene Therapeutics, Inc. 28-epi-rapalogos.
US6780639B1 (en) 1999-08-24 2004-08-24 Universite Libre De Bruxelles Antibiotic inducible/repressible genetic construct for gene therapy or gene immunization
JP3961737B2 (ja) 2000-02-29 2007-08-22 株式会社小糸製作所 車両用灯具およびその製造方法
US20040033600A1 (en) 2001-03-21 2004-02-19 Palli Subba Reddy Ecdysone receptor-based inducible gene expression system
DE60122685T2 (de) 2000-03-22 2007-08-23 Rheogene Holdings Inc. Induziertes genexpressionssystem basierend auf ecdysonrezeptoren
WO2001091803A2 (en) 2000-06-01 2001-12-06 University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
US8105825B2 (en) 2000-10-03 2012-01-31 Intrexon Corporation Multiple inducible gene regulation system
DK1456346T3 (da) 2001-02-20 2012-05-29 Intrexon Corp Nyt inducerbart genekspressionssystem baseret på ecdysonreceptor/invertebrat-retinoid-x-receptor
AU2002248500B2 (en) 2001-02-20 2007-12-13 Intrexon Corporation Novel substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system
EP1572862B1 (en) 2001-02-20 2012-08-01 Intrexon Corporation Chimeric retinoid x receptors and their use in a novel ecdysone receptor-based inducible gene expression system
WO2002066612A2 (en) 2001-02-20 2002-08-29 Rheogene, Inc. Novel substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system
IL158487A0 (en) 2001-05-18 2004-05-12 Wisconsin Alumni Res Found Method for the synthesis of dna sequences
NZ578982A (en) 2001-11-13 2011-03-31 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
ES2717377T3 (es) 2001-12-17 2019-06-20 Univ Pennsylvania Secuencias de serotipo 8 de virus adeno-asociado (AAV), vectores que las contienen y usos de las mismas
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
US8005620B2 (en) 2003-08-01 2011-08-23 Dna Twopointo Inc. Systems and methods for biopolymer engineering
WO2005013090A2 (en) 2003-08-01 2005-02-10 Dna Twopointo Inc. Systems and methods for biopolymer engineering
EP3910063A1 (en) 2003-09-30 2021-11-17 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
US20060246079A1 (en) 2003-11-14 2006-11-02 Morrow Phillip R Neutralizing human antibodies to anthrax toxin
US7935510B2 (en) 2004-04-30 2011-05-03 Intrexon Corporation Mutant receptors and their use in a nuclear receptor-based inducible gene expression system
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
EP2359865B1 (en) 2005-04-07 2013-10-02 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
PL2420256T3 (pl) 2005-05-02 2016-12-30 Terapia genowa dla zaburzeń neurometabolicznych
US7456683B2 (en) 2005-06-09 2008-11-25 Panasonic Corporation Amplitude error compensating device and quadrature skew error compensating device
DK3308788T3 (da) 2005-06-23 2019-01-02 Biogen Ma Inc Sammensætninger og fremgangsmåder til modulering af smn2-splejsning
US8877187B2 (en) 2005-07-25 2014-11-04 Avianax, Llc Therapeutic antibodies for treatment and prophylaxis of transmittable viral diseases
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
HUE044182T2 (hu) 2006-06-07 2019-10-28 Genzyme Corp Génterápia amiotrófiás laterális szklerózis és más gerincvelõ-rendellenességek kezelésére
WO2008042420A2 (en) 2006-10-03 2008-04-10 Genzyme Corporation Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
JP2010520748A (ja) 2007-02-20 2010-06-17 アナプティスバイオ インコーポレイティッド 体細胞超変異系
EP2125886A2 (en) 2007-03-13 2009-12-02 Humabs LLC Antibodies against h5n1 strains of influenza a virus
WO2008124934A1 (en) 2007-04-17 2008-10-23 National Research Council Of Canada Constructs for enhancement of gene expression in muscle
US7879326B2 (en) 2007-06-15 2011-02-01 The Board Of Trustees Of The Leland Stanford Junior University Human neutralizing monoclonal antibodies to H5N1 influenza A virus
KR20100097691A (ko) 2007-11-12 2010-09-03 테라클론 사이언시스, 아이엔씨. 인플루엔자의 치료 및 진단을 위한 조성물 및 방법
NZ586611A (en) 2007-12-06 2012-09-28 Dana Farber Cancer Inst Inc Antibodies against influenza virus and methods of use thereof
ITTO20080204A1 (it) 2008-03-17 2009-09-18 Pomona Biotechnologies Llc Anticorpi monoclonali atti a reagire con una pluralita di sottotipi del virus influenzale a
EP2698380A1 (en) 2008-03-28 2014-02-19 Sea Lane Biotechnologies, LLC Neutralizing molecules to viral antigens
ITTO20080398A1 (it) 2008-05-27 2009-11-28 Pomona Biotechnologies Llc Anticorpi monoclonali aventi proprieta' di cross-neutralizzazione omosubtipica per virus influenzali di tipo a sottotipo h1
EP2296700A2 (en) 2008-06-03 2011-03-23 Vaxin, Inc. Intranasal administration of receptor-binding ligands or genes encoding such ligands as a therapeutic regimen for mitigating infections caused by respiratory pathogens
AU2009275226B2 (en) 2008-07-25 2015-05-14 Institute For Research In Biomedicine Neutralizing anti-influenza a virus antibodies and uses thereof
US8871207B2 (en) 2008-07-25 2014-10-28 Humabs, LLC Neutralizing anti-influenza A virus antibodies and uses thereof
CA3006395C (en) 2008-11-07 2022-05-31 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
US8211631B2 (en) 2008-12-18 2012-07-03 Wisconsin Alumni Research Foundation In vitro model of spinal muscular atrophy
JP5879256B2 (ja) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
KR101790354B1 (ko) 2009-05-11 2017-10-25 얀센 백신스 앤드 프리벤션 비.브이. 인플루엔자 바이러스 ｈ3ｎ2를 중화시킬 수 있는 인간 결합 분자 및 그것의 용도
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
IT1395961B1 (it) 2009-06-01 2012-11-02 Pomona Biotechnologies Llc Anticorpi monoclonali come medicamento per il trattamento terapeutico e/o profilattico delle infezioni da virus influenzale a (h1n1) di origine suina (s-oiv)
EP3643783A1 (en) 2009-06-17 2020-04-29 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
WO2010151673A1 (en) 2009-06-25 2010-12-29 Medimmune, Llc Swine influenza hemagglutinin variants
US8598331B2 (en) 2009-09-28 2013-12-03 The University Of British Columbia CLDN5 mini-promoters
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
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2678433B1 (en) 2011-02-22 2017-05-03 California Institute of Technology Delivery of proteins using adeno-associated virus (aav) vectors
WO2012145572A1 (en) 2011-04-20 2012-10-26 The Trustees Of The University Of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
US20140127749A1 (en) 2011-04-21 2014-05-08 Arizona Borad Of Regents, A Body Corporate Of The State Of Arizona Acting For And On Behalf Of Arizo Methods of protein production and compositions thereof
KR20200084048A (ko) 2011-06-08 2020-07-09 샤이어 휴먼 지네틱 테라피즈 인크. Mrna 전달을 위한 지질 나노입자 조성물 및 방법
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
MY166282A (en) 2011-07-14 2018-06-25 Crucell Holland Bv Human binding molecules capable of neutralizing influenza a viruses of phylogenetic group 1 and phylogenetic group 2 and influenza b viruses
EP2814841A4 (en) 2012-01-31 2015-06-17 Univ Osaka HUMAN MONOCLONAL ANTIBODIES FOR WIDE PROTECTION AGAINST INFLUENZA B VIRUS AND METHOD OF USE THEREOF
WO2013132007A1 (en) 2012-03-08 2013-09-12 Crucell Holland B.V. Human binding molecules capable of binding to and neutralizing influenza b viruses and uses thereof
JP2015516143A (ja) * 2012-04-02 2015-06-08 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． ヒト疾患に関連するタンパク質の産生のための修飾ポリヌクレオチド
WO2013155222A2 (en) 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
PL2839014T3 (pl) 2012-04-18 2021-08-23 The Children's Hospital Of Philadelphia Kompozycja i sposoby na wysoko wydajny transfer genu za pomocą kapsydu wariantów aav
WO2013182683A1 (en) 2012-06-08 2013-12-12 Ethris Gmbh Pulmonary delivery of messenger rna
EP3536795A3 (en) 2012-06-21 2019-12-11 Association Institut de Myologie Widespread gene expression
DK2880053T3 (da) 2012-08-01 2020-05-11 Ikaika Therapeutics Llc Lindring af vævsskade og fibrose via anti-LTBP4-antistof
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
AU2013388083B2 (en) * 2013-05-01 2019-08-22 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
EP3024497B1 (en) 2013-07-26 2021-01-13 University of Iowa Research Foundation Methods and compositions for treating brain diseases
JP6893785B2 (ja) * 2013-10-22 2021-06-23 トランスレイト バイオ， インコーポレイテッド ｍＲＮＡのＣＮＳ送達及びその使用方法
US20160243260A1 (en) 2013-10-24 2016-08-25 Uniqure Ip B.V. Treatment of neurological diseases using adeno-associated virus (AAV) comprising AAV-5 capsid proteins
US20170183396A1 (en) 2014-02-19 2017-06-29 Jody Berry Ebola monoclonal antibodies
WO2015164757A1 (en) 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
CA2946392A1 (en) 2014-04-25 2015-10-29 James M. Wilson Ldlr variants and their use in compositions for reducing cholesterol levels
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
CN114231563A (zh) 2014-05-02 2022-03-25 建新公司 用于视网膜及cns基因疗法的aav载体
MA44179B1 (fr) 2014-05-13 2020-10-28 Univ Pennsylvania Compositions comprenant un virus adeno-associe exprimant des constructions a double anticorps et leurs utilisations
DK3198018T3 (da) 2014-09-24 2021-03-01 Hope City Vektorvarianter af adenoassocieret virus til højeffektiv genomredigering og fremgangsmåder dertil
JP2017531642A (ja) 2014-10-03 2017-10-26 マサチューセッツ インスティテュート オブ テクノロジー エボラ糖タンパク質に結合する抗体およびその使用
CN107207611B (zh) 2015-02-05 2021-11-09 扬森疫苗与预防公司 针对流感血球凝集素的结合分子及其用途
CN108135994B (zh) 2015-05-13 2022-01-25 宾夕法尼亚州大学信托人 Aav-介导的抗-流感抗体的表达及其使用方法
CN108779167A (zh) 2015-10-28 2018-11-09 宾夕法尼亚州大学信托人 鞘内施用腺伴随病毒载体用于基因治疗
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
EP4215605A1 (en) 2015-12-11 2023-07-26 The Trustees of The University of Pennsylvania Scalable purification method for aav8
EP3992283A1 (en) 2015-12-11 2022-05-04 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
WO2017160360A2 (en) 2015-12-11 2017-09-21 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
WO2017106326A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Aav-anti pcsk9 antibody constructs and uses thereof
WO2017106244A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Compositions and methods for regulatable antibody expression
MA44119A (fr) 2015-12-14 2018-10-31 Univ Pennsylvania Vecteurs viraux adéno-associés à utiliser dans le traitement de l'amyotrophie spinale
MA43968A (fr) 2016-02-03 2018-12-12 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
KR102425289B1 (ko) 2016-08-15 2022-07-27 젠자임 코포레이션 Aav의 검출 방법
US20190240328A1 (en) 2016-09-24 2019-08-08 The Trustees Of The University Of Pennsylvania Novel humanized anti-ebola antibodies useful in preventing ebola infections
JP2020510430A (ja) 2017-02-28 2020-04-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Ａａｖベクターに基づくインフルエンザワクチン
AU2018227440A1 (en) 2017-02-28 2019-08-29 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clade f vector and uses therefor

Also Published As

Publication number Publication date
SG10201912495SA (en) 2020-02-27
AU2018227442A1 (en) 2019-08-29
CO2019009522A2 (es) 2019-09-18
BR112019016444A2 (pt) 2020-04-07
US20200056205A1 (en) 2020-02-20
US11578341B2 (en) 2023-02-14
WO2018160585A2 (en) 2018-09-07
WO2018160585A3 (en) 2018-10-25
EP3589739A2 (en) 2020-01-08
KR20190123752A (ko) 2019-11-01
CN110741089A (zh) 2020-01-31
MX2019010276A (es) 2019-10-15
IL268519A (en) 2019-09-26
US20230265459A1 (en) 2023-08-24
CL2019002473A1 (es) 2019-11-29
MA47688A (fr) 2020-01-08
JP2022078208A (ja) 2022-05-24
JP7037574B2 (ja) 2022-03-16
PH12019550149A1 (en) 2020-06-08
JOP20190200A1 (ar) 2019-08-27
JP2020510428A (ja) 2020-04-09
CA3055619A1 (en) 2018-09-07
SG11201907474UA (en) 2019-09-27

Similar Documents

Publication Publication Date Title
PE20191261A1 (es) Composiciones utiles para el tratamiento de atrofia muscular espinal
PE20190401A1 (es) Novedosas proteinas de la capside del virus adenoasociado
CO2020004738A2 (es) Variantes de cápsides de virus adenoasociados y métodos de uso de estas
PE20160188A1 (es) Variantes aav y composiciones, metodos y usos para transferencia de gen a celulas, organos y tejidos
PE20190129A1 (es) Capsides variantes de virus adenoasociados y metodos de uso de estas
PE20210915A1 (es) Capsides variantes de virus adenoasociados y su uso para inhibir la angiogenesis
EA201991911A1 (ru) Способы и композиции для переноса генов по сосудистой сети
PE20231949A1 (es) VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
MX2020008933A (es) Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos.
MX2020008932A (es) Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos.
PE20220930A1 (es) Composiciones utiles en el tratamiento de leucodistrofia metacromatica
BR112021021792A2 (pt) Composições úteis para tratamento de doença de pompe
PE20090483A1 (es) Vectores para la expresion multiple de genes
BR112022004027A2 (pt) Métodos para tratar doenças neovasculares oculares usando variantes de aav2 que codificam aflibercepte
AR124119A2 (es) Partículas virales modificadas y usos de estas
PE20221254A1 (es) Proteina vp1 modificada aislada de la capside del virus adeno-asociado de serotipo 5 (aav5), capside y vector basado en la misma
CO2023009633A2 (es) Efecto sinérgico de smn1 y mir-23a en el tratamiento de la atrofia muscular espinal
JP2020533968A5 (es)
MX2022000551A (es) Proteínas de la cápside de aav modificadas para el tratamiento de una enfermedad artrítica.
UY38881A (es) Métodos de tratamiento de enfermedades neovasculares oculares utilizando variantes de aav2 que codifican aflibercept
JP2017500030A5 (es)
EA201600428A1 (ru) Способ стимуляции репаративного ангиогенеза и регенерации соединительной ткани, при ее повреждении, методом генной терапии с использованием видоспецифичных генов белковых факторов vegf и fgf2, в медицине, и генетическая конструкция для реализации заявленного способа
RU2021124726A (ru) Выделенный модифицированный белок VPI капсида аденоассоциированного вируса 9 серотипа (AAV9), капсид и вектор на его основе
RU2020136195A (ru) РЕКОМБИНАНТНЫЙ АДЕНОАССОЦИИРОВАННЫЙ ВИРУС, ЯВЛЯЮЩИЙСЯ ГИБРИДОМ СЕРОТИПОВ AAV9 И AAVrh74, С ПОНИЖЕННЫМ ТРОПИЗМОМ К ПЕЧЕНОЧНОЙ ТКАНИ
EA202092046A1 (ru) Новые векторы на основе аденоассоциированного вируса (aav), векторы aav, со сниженным дезамидированием капсида, а также их применения