IN2014DN08812A - - Google Patents

Info

Publication number
IN2014DN08812A
IN2014DN08812A IN8812DEN2014A IN2014DN08812A IN 2014DN08812 A IN2014DN08812 A IN 2014DN08812A IN 8812DEN2014 A IN8812DEN2014 A IN 8812DEN2014A IN 2014DN08812 A IN2014DN08812 A IN 2014DN08812A
Authority
IN
India
Prior art keywords
disclosed
methods
gene therapy
mediated gene
improved compositions
Prior art date
Application number
Other languages
English (en)
Inventor
Mustafa N Yazicioglu
Federico Mingozzi
Xavier Anguela
Katherine A High
Original Assignee
Philadelphia Children Hospital
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 Philadelphia Children Hospital filed Critical Philadelphia Children Hospital
Publication of IN2014DN08812A publication Critical patent/IN2014DN08812A/en

Links

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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01DNA viruses
    • C07K14/015Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • 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
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • 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
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Oncology (AREA)
  • Communicable Diseases (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)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
IN8812DEN2014 2012-04-18 2013-04-18 IN2014DN08812A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261635273P 2012-04-18 2012-04-18
US201361794995P 2013-03-15 2013-03-15
PCT/US2013/037170 WO2013158879A1 (en) 2012-04-18 2013-04-18 Composition and methods for highly efficient gene transfer using aav capsid variants

Publications (1)

Publication Number Publication Date
IN2014DN08812A true IN2014DN08812A (ja) 2015-05-22

Family

ID=49384070

Family Applications (1)

Application Number Title Priority Date Filing Date
IN8812DEN2014 IN2014DN08812A (ja) 2012-04-18 2013-04-18

Country Status (27)

Country Link
US (2) US9909142B2 (ja)
EP (1) EP2839014B1 (ja)
JP (1) JP6342886B2 (ja)
KR (1) KR102063483B1 (ja)
CN (1) CN104487579B (ja)
AU (1) AU2013249202B2 (ja)
BR (2) BR112014025985A2 (ja)
CA (1) CA2870736C (ja)
CO (1) CO7200251A2 (ja)
DK (1) DK2839014T3 (ja)
ES (1) ES2862912T3 (ja)
HK (1) HK1207663A1 (ja)
HU (1) HUE054087T2 (ja)
IL (1) IL235102B (ja)
IN (1) IN2014DN08812A (ja)
MX (1) MX359518B (ja)
MY (1) MY172457A (ja)
NZ (1) NZ701693A (ja)
PE (1) PE20150163A1 (ja)
PH (1) PH12014502347A1 (ja)
PL (1) PL2839014T3 (ja)
PT (1) PT2839014T (ja)
RU (1) RU2683497C2 (ja)
SG (1) SG11201406776TA (ja)
SI (1) SI2839014T1 (ja)
WO (1) WO2013158879A1 (ja)
ZA (1) ZA201407582B (ja)

Families Citing this family (103)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2359867T3 (en) * 2005-04-07 2015-01-05 Univ Pennsylvania A method for increasing an AAV vector function
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
DK2191001T3 (en) 2007-04-09 2016-09-19 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
CN107261154A (zh) 2011-04-29 2017-10-20 西莱克塔生物科技公司 致耐受性合成纳米载体
MY172457A (en) 2012-04-18 2019-11-26 Childrens Hospital Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
US11136557B2 (en) * 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
CA3209883A1 (en) * 2013-07-22 2015-01-29 The Children's Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
WO2015048534A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
US10308957B2 (en) 2014-03-04 2019-06-04 University Of Florida Research Foundation, Inc. rAAV vectors and methods for transduction of photoreceptors and RPE cells
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
EP3189138A4 (en) 2014-09-07 2018-04-18 Selecta Biosciences, Inc. Methods and compositions for attenuating gene editing anti-viral transfer vector immune responses
CA2964272A1 (en) 2014-10-21 2016-04-28 Guangping Gao Recombinant aav variants and uses thereof
BR112017009497A2 (pt) 2014-11-05 2018-02-06 Voyager Therapeutics, Inc. polinucleotídeos de aadc para o tratamento da doença de parkinson
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
BR112017010087A2 (pt) 2014-11-14 2018-06-05 Voyager Therapeutics, Inc. composições e métodos para tratar esclerose lateral amiotrófica (ela)
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
WO2016126857A1 (en) 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
US10648000B2 (en) 2015-02-16 2020-05-12 University Of Florida Research Foundation, Incorporated rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes
US20180044698A1 (en) * 2015-02-19 2018-02-15 University Of Florida Research Foundation, Incorporated Site-specific integrating recombinant aav vectors for gene therapy and improved production methods
JP6519080B2 (ja) * 2015-06-22 2019-05-29 ライオン株式会社 繊維製品用抗ウイルス組成物
JP6831779B2 (ja) 2015-06-23 2021-02-17 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia 修飾された第ix因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用
SG10202107733QA (en) 2015-09-28 2021-09-29 Univ North Carolina Chapel Hill Methods and compositions for antibody-evading virus vectors
BR112018008407A2 (pt) 2015-10-28 2018-11-27 Univ Pennsylvania administração intratecal de vetores virais adenoassociados para terapia genética
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
RU2762948C2 (ru) * 2016-04-16 2021-12-24 Юниверсити Оф Флорида Рисерч Фаундэйшн, Инкорпорейтед Способы усиления биологической активности рекомбинантного аденоассоциированного вуруса, продуцируемого бакуловирусной системой
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR THE TREATMENT OF DISEASES
MA44740A (fr) 2016-05-13 2019-02-27 4D Molecular Therapeutics Inc Variantes de capsides de virus adéno-associé et leurs procédés d'utilisation
MX2018014154A (es) 2016-05-18 2019-05-06 Voyager Therapeutics Inc Polinucleotidos moduladores.
RU2764587C2 (ru) 2016-05-18 2022-01-18 Вояджер Терапьютикс, Инк. Способы и композиции для лечения хореи гентингтона
TN2019000047A1 (en) 2016-08-15 2020-07-15 Genzyme Corp Methods for detecting aav
EP3831281A1 (en) 2016-08-30 2021-06-09 The Regents of The University of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
EP3526333A4 (en) * 2016-10-13 2020-07-29 University of Massachusetts AAV CAPSIDE DESIGNS
EP3565572A1 (en) 2017-01-07 2019-11-13 Selecta Biosciences, Inc. Patterned dosing of immunosuppressants coupled to synthetic nanocarriers
WO2018139634A1 (ja) * 2017-01-30 2018-08-02 学校法人日本医科大学 アデノ随伴ウイルス(aav)キャプシドタンパク質の変異体
CA3054131C (en) 2017-02-21 2021-05-25 University Of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
US10550405B2 (en) 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
AU2018234695A1 (en) 2017-03-15 2019-09-12 The University Of North Carolina At Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
AU2018241252A1 (en) 2017-03-30 2019-10-03 The University Of Queensland Chimeric molecules and uses thereof
EP3619308A4 (en) 2017-05-05 2021-01-27 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT FOR HUNTINGTON'S MORBUS
AU2018261790A1 (en) 2017-05-05 2019-11-28 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
US20210228738A1 (en) 2017-07-17 2021-07-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. NETWORK EQUIPMENT TRACK GUIDE SYSTEM
PT3684423T (pt) 2017-09-20 2023-06-09 4D Molecular Therapeutics Inc Cápsides variantes de vírus adeno-associado e métodos de utilização das mesmas
KR20200086670A (ko) 2017-10-13 2020-07-17 셀렉타 바이오사이언시즈, 인크. 항바이러스 전달 벡터 IgM 반응을 약화시키기 위한 방법 및 조성물
EP4124658A3 (en) 2017-10-16 2023-04-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
EP3717636B1 (en) 2017-11-27 2023-03-08 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis
JP7403165B2 (ja) 2017-12-19 2023-12-22 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 血液脳関門を通過してウイルスベクターを送達するための方法および組成物
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
WO2019161365A1 (en) 2018-02-19 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
JP2021515037A (ja) 2018-02-26 2021-06-17 アントルクス,インコーポレーテッド 寛容原性リポソーム及びその使用方法
CA3091806A1 (en) * 2018-02-27 2019-09-06 The Trustees Of The University Of Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
US20210087584A1 (en) * 2018-03-16 2021-03-25 Research Intitute at Nationwide Children's Hospital Increasing tissue specific gene delivery by capsid modification
WO2019195449A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
TN2020000187A1 (en) 2018-04-05 2022-04-04 Univ Devry Val Dessonne Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism
US20210363192A1 (en) * 2018-04-27 2021-11-25 Spark Therapeutics, Inc. Engineered aav capsids with increased tropism and aav vectors comprising the engineered capsids and methods of making and using same
BR112020023393A2 (pt) * 2018-05-16 2021-02-09 Spark Therapeutics, Inc. cassetes de expressão de a-glicosidase ácida otimizados por códon e métodos de uso dos mesmos
CA3106639A1 (en) 2018-07-16 2020-01-23 Selecta Biosciences, Inc. Methods and compositions of otc constructs and vectors
CA3106640A1 (en) 2018-07-16 2020-01-23 Selecta Biosciences, Inc. Methods and compositions of mma constructs and vectors
US11702673B2 (en) 2018-10-18 2023-07-18 University Of Florida Research Foundation, Incorporated Methods of enhancing biological potency of baculovirus system-produced recombinant adeno-associated virus
JP2022520875A (ja) 2019-02-25 2022-04-01 ノバルティス アーゲー Biettiクリスタリン網膜症を治療するための組成物及び方法
CA3130731A1 (en) 2019-02-25 2020-09-03 Friedrich Miescher Institute For Biomedical Research Compositions and methods to treat bietti crystalline dystrophy
MX2021011468A (es) 2019-03-21 2021-12-15 Vectores de virus adenoasociados recombinantes.
US20200360453A1 (en) 2019-04-28 2020-11-19 Selecta Biosciences, Inc. Methods for treatment of subjects with preexisting immunity to viral transfer vectors
CA3141863A1 (en) 2019-05-28 2020-12-03 Selecta Biosciences, Inc. Methods and compositions for attenuated anti-viral transfer vector immune response
RU2751592C2 (ru) 2019-08-22 2021-07-15 Общество С Ограниченной Ответственностью "Анабион" Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
US20220347298A1 (en) 2019-10-04 2022-11-03 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
WO2021076925A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
MX2022008003A (es) * 2020-01-03 2022-10-07 Sarepta Therapeutics Inc Métodos para analizar proteínas de la cápside de aav.
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
BR112022015921A2 (pt) 2020-02-14 2022-10-04 Ultragenyx Pharmaceutical Inc Terapia gênica para tratar o transtorno de deficiência de cdkl5
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
US20230129893A1 (en) 2020-03-31 2023-04-27 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
IL302046A (en) 2020-10-15 2023-06-01 Hoffmann La Roche Nucleic acid structures for va RNA transcription
KR20230085170A (ko) 2020-10-15 2023-06-13 에프. 호프만-라 로슈 아게 동시 유전자 활성화를 위한 핵산 구조체
WO2022150335A1 (en) 2021-01-05 2022-07-14 Selecta Biosciences, Inc. Viral vector dosing protocols
KR20240000542A (ko) * 2021-04-23 2024-01-02 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 근이영양증의 유전자 요법을 위한 AAVrh74 벡터
WO2023019168A1 (en) 2021-08-11 2023-02-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
AR126839A1 (es) 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta
WO2023022631A1 (ru) 2021-08-20 2023-02-23 Акционерное общество "БИОКАД" Способ получения модифицированного капсида аденоассоциированного вируса
AR126840A1 (es) 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en esta
WO2023044306A1 (en) * 2021-09-14 2023-03-23 California Institute Of Technology Aav capsid variants
US20230140196A1 (en) 2021-10-12 2023-05-04 Selecta Biosciences, Inc. Viral vector dosing protocols
US20230141563A1 (en) 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
CA3237037A1 (en) 2021-11-14 2023-05-19 Cartesian Therapeutics, Inc. Multiple dosing with viral vectors
CN116554278A (zh) * 2022-01-30 2023-08-08 上海玮美基因科技有限责任公司 变异型腺相关病毒及其在疾病治疗中的应用
TW202342740A (zh) 2022-03-07 2023-11-01 美商奧崔基尼克斯製藥公司 改良的批量aav生產系統和方法
US20230357437A1 (en) 2022-03-09 2023-11-09 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
WO2023198685A1 (en) 2022-04-13 2023-10-19 F. Hoffmann-La Roche Ag Method for determining aav genomes
WO2023220287A1 (en) * 2022-05-11 2023-11-16 The Children's Hospital Of Philadelphia Adeno-associated viral vectors for targeting deep brain structures
WO2023227438A1 (en) 2022-05-23 2023-11-30 F. Hoffmann-La Roche Ag Raman-based method for the differentiation of aav particle serotype and aav particle loading status
WO2023232922A1 (en) 2022-06-03 2023-12-07 F. Hoffmann-La Roche Ag Method for producing recombinant aav particles
WO2024013239A1 (en) 2022-07-14 2024-01-18 F. Hoffmann-La Roche Ag Method for producing recombinant aav particles
US20240148841A1 (en) 2022-08-11 2024-05-09 Selecta Biosciences Inc. Compositions and methods related to immunoglobulin proteases and fusions thereof
WO2024038002A1 (en) 2022-08-15 2024-02-22 F. Hoffmann-La Roche Ag Prevention or mitigation of adverse effects related to recombinant viral vectors
WO2024056561A1 (en) 2022-09-12 2024-03-21 F. Hoffmann-La Roche Ag Method for separating full and empty aav particles

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA1202962A (en) 1982-10-19 1986-04-08 David P. Clifford Substituted n-phenyl-n'-benzoyl ureas and their use as insecticides and acaricides
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
US4965188A (en) 1986-08-22 1990-10-23 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences using a thermostable enzyme
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US6127525A (en) 1995-02-21 2000-10-03 Cornell Research Foundation, Inc. Chimeric adenoviral coat protein and methods of using same
WO1998010088A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
EP1486567A1 (en) * 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
JP4888876B2 (ja) * 2003-06-13 2012-02-29 田平 武 アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター
DK2359867T3 (en) * 2005-04-07 2015-01-05 Univ Pennsylvania A method for increasing an AAV vector function
US9198984B2 (en) 2006-04-28 2015-12-01 The Trustees Of The University Of Pennsylvania Scalable production method for AAV
CA2793633A1 (en) * 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
MY172457A (en) 2012-04-18 2019-11-26 Childrens Hospital Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants

Also Published As

Publication number Publication date
CO7200251A2 (es) 2015-02-27
CN104487579B (zh) 2022-01-18
KR102063483B1 (ko) 2020-01-08
ZA201407582B (en) 2021-05-26
HUE054087T2 (hu) 2021-09-28
DK2839014T3 (da) 2021-03-08
SI2839014T1 (sl) 2021-05-31
MX2014012680A (es) 2015-05-11
KR20150004859A (ko) 2015-01-13
AU2013249202B2 (en) 2018-08-09
US20150065562A1 (en) 2015-03-05
MX359518B (es) 2018-10-01
PH12014502347B1 (en) 2014-12-22
US20180245098A1 (en) 2018-08-30
BR122021020419B1 (pt) 2023-01-17
IL235102B (en) 2019-08-29
EP2839014A4 (en) 2015-12-30
MY172457A (en) 2019-11-26
JP2015514427A (ja) 2015-05-21
NZ701693A (en) 2017-02-24
EP2839014B1 (en) 2020-12-16
CA2870736C (en) 2021-11-02
JP6342886B2 (ja) 2018-06-13
AU2013249202A1 (en) 2014-11-06
RU2683497C2 (ru) 2019-03-28
PH12014502347A1 (en) 2014-12-22
CN104487579A (zh) 2015-04-01
BR112014025985A2 (pt) 2017-07-11
IL235102A0 (en) 2014-12-31
HK1207663A1 (en) 2016-02-05
SG11201406776TA (en) 2015-03-30
US9909142B2 (en) 2018-03-06
WO2013158879A1 (en) 2013-10-24
US11279950B2 (en) 2022-03-22
RU2014146159A (ru) 2016-06-10
CA2870736A1 (en) 2013-10-24
ES2862912T3 (es) 2021-10-08
EP2839014A1 (en) 2015-02-25
PL2839014T3 (pl) 2021-08-23
PT2839014T (pt) 2021-03-19
PE20150163A1 (es) 2015-02-23

Similar Documents

Publication Publication Date Title
IN2014DN08812A (ja)
PH12019502292A1 (en) Methods and compositions for treatment of a genetic condition
AU2019232913A1 (en) Methods and processes for non-invasive assessment of genetic variations
HK1217214A1 (zh) 用於增强核酸酶介導的基因破壞的方法和組合物
CA2877331C (en) Methods and processes for non-invasive assessment of genetic variations
EP2850189B8 (en) Compositions and methods for modulating gene expression
HK1207111A1 (en) Methods and compositions for controlling gene expression by rna processing rna
EP2850184A4 (en) COMPOSITIONS AND METHOD FOR MODULATING GENE EXPRESSION
EP2850183A4 (en) COMPOSITIONS AND METHOD FOR MODULATING GENE EXPRESSION
HK1208700A1 (en) Compositions and methods for modulating smn gene family expression smn
HUE044907T2 (hu) Módszerek és készítmények célzott genetikai módosításhoz, párosított mutató RNS-ek használatával
EP2855680B8 (en) Compositions and methods for silencing gene expression
ZA201409229B (en) Compositions and methods for modulating utrn expression
EP2904106A4 (en) COMPOSITIONS AND METHODS FOR TARGETING STROMAL CELLS FOR THE TREATMENT OF CANCER
IN2014DN08481A (ja)
MX356525B (es) Vectores de virus asociados con adeno (aav) para uso en terapia de genes de coroideremia.
PL3260558T3 (pl) Nowe kompozycje i sposoby poprawy specyficzności pcr
GB201222934D0 (en) Particle toughening for improving fracture toughness
AU2013225816A8 (en) Compositions and methods for treating Type III Gaucher Disease
MX359769B (es) Metodos para descelularizar huesos.
JO3755B1 (ar) تركيبات تستوستيرون
EP3091999A4 (en) Improved cell compositions and methods for cancer therapy
MX350868B (es) Medicamentos y metodos para tratar cancer.
EP2880166A4 (en) COMPOSITIONS AND METHODS FOR GENETIC CONSTRUCTIONS
IL233445A0 (en) Methods and preparations for gene transfer