NZ701693A - Composition and methods for highly efficient gene transfer using aav capsid variants - Google Patents

Composition and methods for highly efficient gene transfer using aav capsid variants

Info

Publication number
NZ701693A
NZ701693A NZ701693A NZ70169313A NZ701693A NZ 701693 A NZ701693 A NZ 701693A NZ 701693 A NZ701693 A NZ 701693A NZ 70169313 A NZ70169313 A NZ 70169313A NZ 701693 A NZ701693 A NZ 701693A
Authority
NZ
New Zealand
Prior art keywords
methods
aav
composition
gene transfer
highly efficient
Prior art date
Application number
NZ701693A
Other languages
English (en)
Inventor
Mustafa N Yazicioglu
Federico Mingozzi
Xavier Anguela
Katherine A High
Original Assignee
The Children’S Hospital Of Philadelphia
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 The Children’S Hospital Of Philadelphia filed Critical The Children’S Hospital Of Philadelphia
Publication of NZ701693A publication Critical patent/NZ701693A/en

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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • 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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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

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)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
  • Hematology (AREA)
  • Communicable Diseases (AREA)
  • Diabetes (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
NZ701693A 2012-04-18 2013-04-18 Composition and methods for highly efficient gene transfer using aav capsid variants NZ701693A (en)

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
NZ701693A true NZ701693A (en) 2017-02-24

Family

ID=49384070

Family Applications (1)

Application Number Title Priority Date Filing Date
NZ701693A NZ701693A (en) 2012-04-18 2013-04-18 Composition and methods for highly efficient gene transfer using aav capsid variants

Country Status (26)

Country Link
US (2) US9909142B2 (enEXAMPLES)
EP (1) EP2839014B1 (enEXAMPLES)
JP (1) JP6342886B2 (enEXAMPLES)
KR (1) KR102063483B1 (enEXAMPLES)
CN (1) CN104487579B (enEXAMPLES)
AU (1) AU2013249202B2 (enEXAMPLES)
BR (2) BR122021020419B1 (enEXAMPLES)
CA (1) CA2870736C (enEXAMPLES)
CO (1) CO7200251A2 (enEXAMPLES)
DK (1) DK2839014T3 (enEXAMPLES)
ES (1) ES2862912T3 (enEXAMPLES)
HU (1) HUE054087T2 (enEXAMPLES)
IL (1) IL235102B (enEXAMPLES)
IN (1) IN2014DN08812A (enEXAMPLES)
MX (1) MX359518B (enEXAMPLES)
MY (1) MY172457A (enEXAMPLES)
NZ (1) NZ701693A (enEXAMPLES)
PE (1) PE20150163A1 (enEXAMPLES)
PH (1) PH12014502347B1 (enEXAMPLES)
PL (1) PL2839014T3 (enEXAMPLES)
PT (1) PT2839014T (enEXAMPLES)
RU (1) RU2683497C2 (enEXAMPLES)
SG (1) SG11201406776TA (enEXAMPLES)
SI (1) SI2839014T1 (enEXAMPLES)
WO (1) WO2013158879A1 (enEXAMPLES)
ZA (1) ZA201407582B (enEXAMPLES)

Families Citing this family (128)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101203613B (zh) * 2005-04-07 2012-12-12 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
CA2720097C (en) 2007-04-09 2016-07-19 University Of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
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
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
IL283728B2 (en) 2011-04-29 2024-01-01 Selecta Biosciences Inc Tolerogenic synthetic nanocarriers to reduce antibody responses
PE20150163A1 (es) 2012-04-18 2015-02-23 Philadelphia Children Hospital Composicion y metodos para una transferencia de genes altamente eficiente usando variantes capside aav
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
ES2897508T3 (es) * 2013-05-31 2022-03-01 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
MY175477A (en) * 2013-07-22 2020-06-30 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
CA2930549A1 (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
WO2015134643A1 (en) * 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
WO2016037165A1 (en) 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Methods and compositions for attenuating exon skipping anti-viral transfer vector immune responses
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts 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
RU2020108189A (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (als)
CA2975583A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
HK1245326A1 (zh) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
WO2016126857A1 (en) 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
WO2016133917A1 (en) 2015-02-16 2016-08-25 University Of Florida Research Foundation 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
SI3313991T1 (sl) 2015-06-23 2024-11-29 The Children's Hospital Of Philadelphia Modificirani faktor ix ter sestavki, postopki in uporabe za prenos genov v celice, organe in tkiva
PT3356390T (pt) 2015-09-28 2021-04-21 Univ Florida Métodos e composições para vetores virais que se evadem a anticorpos
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
BR112018008407A2 (pt) 2015-10-28 2018-11-27 Univ Pennsylvania administração intratecal de vetores virais adenoassociados para terapia genética
PE20231949A1 (es) 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
EP3384035A4 (en) 2015-12-02 2019-08-07 Voyager Therapeutics, Inc. ASSAYS FOR THE DETECTION OF AAV-NEUTRALIZING ANTIBODIES
US11384364B2 (en) * 2016-04-16 2022-07-12 University Of Florida Research Foundation, Incorporated Methods of enhancing biological potency of baculovirus system-produced recombinant adeno-associated virus
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
BR112018072849B1 (pt) 2016-05-13 2020-12-22 4D Molecular Therapeutics Inc. proteína de capsídeo variante de vírus adeno-associado, ácido nucleico isolado, vírion de raav infeccioso e respectivo uso e composição farmacêutica
KR20220108216A (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
CN117904112A (zh) 2016-05-18 2024-04-19 沃雅戈治疗公司 调节性多核苷酸
CN118688363A (zh) * 2016-08-15 2024-09-24 建新公司 检测aav的方法
WO2018044933A1 (en) 2016-08-30 2018-03-08 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
CN110462037A (zh) * 2017-01-30 2019-11-15 学校法人日本医科大学 腺相关病毒(aav)衣壳蛋白的突变体
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
AU2018236123B2 (en) 2017-03-11 2024-04-18 Selecta Biosciences, Inc. Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
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
JP7132934B2 (ja) 2017-03-15 2022-09-07 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル ポリプロイド性アデノ随伴ウイルスベクターならびにその作製および使用の方法
ES3001108T3 (es) 2017-03-30 2025-03-04 Univ Queensland Moléculas quiméricas y usos de las mismas
CN110913866A (zh) 2017-05-05 2020-03-24 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
EP3619308A4 (en) 2017-05-05 2021-01-27 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT FOR HUNTINGTON'S MORBUS
CA3066358A1 (en) 2017-06-07 2018-12-13 Spark Therapeutics, Inc. Enhancing agents for improved cell transfection and/or raav vector production
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3645052A4 (en) 2017-06-30 2021-07-28 Regents of the University of California ADENO-ASSOCIATED VIRUS VIRIONS WITH ASSOCIATED CAPSIDE VARIANTS AND METHODS OF USE
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
EP3654860A1 (en) 2017-07-17 2020-05-27 Voyager Therapeutics, Inc. Trajectory array guide system
WO2019040994A1 (en) 2017-09-01 2019-03-07 The Australian National University IMMUNOREGULATOR MOLECULES AND USES THEREOF
BR112020005436B1 (pt) 2017-09-20 2022-08-02 4D Molecular Therapeutics Inc Proteína do capsídeo de variante do vírus adenoassociado, virion do aav recombinante (raav) infeccioso, composições, composições farmacêuticas e usos de virion de raav ou de composições farmacêuticas
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
KR20200086670A (ko) 2017-10-13 2020-07-17 셀렉타 바이오사이언시즈, 인크. 항바이러스 전달 벡터 IgM 반응을 약화시키기 위한 방법 및 조성물
AU2018352236B2 (en) 2017-10-16 2025-04-10 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
FI3717636T3 (fi) 2017-11-27 2023-06-01 4D Molecular Therapeutics Inc Adenoassosioidun viruksen kapsidivariantteja ja käyttö angiogeneesin estämisessä
EP3728568A4 (en) 2017-12-19 2021-10-06 The University of North Carolina at Chapel Hill METHODS AND COMPOSITIONS FOR THE ADMINISTRATION OF VIRAL VECTORS ACROSS THE HAEMATOENCEPHALIC BARRIER
CA3090226A1 (en) 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
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
AU2019225251A1 (en) 2018-02-26 2020-10-15 AnTolRx, Inc. Tolerogenic liposomes and methods of use thereof
IL276859B1 (en) * 2018-02-27 2025-08-01 Univ Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
BR112020018354A2 (pt) * 2018-03-16 2020-12-29 Research Institute At Nationwide Children's Hospital Aumento de fornecimento gênico específico a tecido através de modificação de capsídeo
WO2019195449A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
MX2020010466A (es) 2018-04-03 2021-01-08 Vectores de virus que evitan anticuerpos.
WO2019193119A1 (en) 2018-04-05 2019-10-10 Genethon HYBRID RECOMBINANT ADENO-ASSOCIATED VIRUS SEROTYPE BETWEEN AAV9 AND AAVrh74 WITH REDUCED LIVER TROPISM
CN119410636A (zh) 2018-04-27 2025-02-11 分贝治疗公司 肌球蛋白15启动子及其用途
EP3784697A4 (en) * 2018-04-27 2022-07-06 Spark Therapeutics, Inc. Engineered aav capsids with increased tropism and aav vectors comprising the engineered capsids and methods of making and using same
MX2020012077A (es) 2018-05-15 2021-03-09 Voyager Therapeutics Inc Composiciones y metodos para el tratamiento de la enfermedad de parkinson.
CA3100213A1 (en) * 2018-05-16 2019-11-21 Spark Therapeutics, Inc. Codon-optimized acid .alpha.-glucosidase expression cassettes and methods of using same
MX2021000638A (es) 2018-07-16 2021-06-23 Selecta Biosciences Inc Métodos y composiciones de construcciones y vectores de acidemia metilmalónica.
CN112771070A (zh) 2018-07-16 2021-05-07 西莱克塔生物科技公司 Otc构建体和载体的方法和组合物
KR20210068068A (ko) 2018-09-28 2021-06-08 보이저 테라퓨틱스, 인크. 조작된 프로모터를 갖는 프라탁신 발현 구축물 및 그의 사용 방법
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
JP7578590B2 (ja) 2018-10-18 2024-11-06 インテリア セラピューティクス，インコーポレーテッド 第ｉｘ因子を発現するための組成物及び方法
AU2020218547A1 (en) 2019-02-08 2021-08-26 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
BR112021016501A2 (pt) 2019-02-25 2021-10-26 Novartis Ag Composições e métodos para tratar distrofia cristalina de bietti
CN113474461A (zh) 2019-02-25 2021-10-01 诺华股份有限公司 治疗bietti晶体营养不良的组合物和方法
MX2021011468A (es) 2019-03-21 2021-12-15 Vectores de virus adenoasociados recombinantes.
MX2021013163A (es) 2019-04-28 2022-02-21 Selecta Biosciences Inc Métodos para el tratamiento de sujetos con inmunidad preexistente a vectores de transferencia viral.
CN114206396A (zh) 2019-05-28 2022-03-18 西莱克塔生物科技公司 用于减弱抗病毒转移载体免疫应答的方法和组合物
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
EP4038194A1 (en) 2019-10-04 2022-08-10 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
WO2021138381A1 (en) 2020-01-03 2021-07-08 Sarepta Therapeutics, Inc. Methods for analyzing aav capsid proteins
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
CN115151648A (zh) 2020-02-14 2022-10-04 阿尔特拉吉尼克斯制药公司 用于治疗cdkl5缺陷障碍的基因疗法
US20230235353A1 (en) 2020-03-19 2023-07-27 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
WO2021202532A1 (en) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
US12104163B2 (en) 2020-08-19 2024-10-01 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
IL302046A (en) 2020-10-15 2023-06-01 Hoffmann La Roche Nucleic acid constructs for va rna transcription
AR123776A1 (es) 2020-10-15 2023-01-11 Hoffmann La Roche Construcciones de ácido nucleico para la activación simultánea de genes
CA3207247A1 (en) 2021-01-05 2022-07-14 Selecta Biosciences, Inc. Viral vector dosing protocols
TW202304954A (zh) * 2021-04-23 2023-02-01 美商佛羅里達大學研究基金會公司 用於肌肉萎縮症之基因療法的aavrh74載體
US20250019721A1 (en) 2021-08-11 2025-01-16 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
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
CR20240137A (es) 2021-08-20 2024-07-29 Biocad Joint Stock Co Método para obtener una cápside de virus adenoasociado modificada
US20230086710A1 (en) * 2021-09-14 2023-03-23 California Institute Of Technology Aav capsid variants
US20230141563A1 (en) 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
WO2023064350A1 (en) 2021-10-12 2023-04-20 Selecta Biosciences, Inc. Viral vector dosing protocols
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
AU2023232781A1 (en) 2022-03-07 2024-09-26 Ultragenyx Pharmaceutical Inc. Modified batch aav production systems and methods
WO2023172624A1 (en) 2022-03-09 2023-09-14 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
JP2025516117A (ja) 2022-04-13 2025-05-27 エフ． ホフマン－ラ ロシュ アーゲー Ａａｖゲノムを決定するための方法
WO2023220287A1 (en) * 2022-05-11 2023-11-16 The Children's Hospital Of Philadelphia Adeno-associated viral vectors for targeting deep brain structures
EP4529614A1 (en) 2022-05-23 2025-04-02 F. Hoffmann-La Roche AG Raman-based method for the differentiation of aav particle serotype and aav particle loading status
CN119301260A (zh) 2022-06-03 2025-01-10 豪夫迈·罗氏有限公司 用于生产重组aav颗粒的方法
EP4554604A1 (en) 2022-07-14 2025-05-21 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
TW202416993A (zh) 2022-08-15 2024-05-01 瑞士商赫孚孟拉羅股份公司 預防或減輕與重組病毒載體相關的不良反應
IL318766A (en) 2022-09-12 2025-04-01 Hoffmann La Roche Method for separating full and empty AAV particles
CN120641755A (zh) 2023-02-07 2025-09-12 豪夫迈·罗氏有限公司 用于检测抗aav颗粒抗体的方法
WO2024194280A1 (en) 2023-03-21 2024-09-26 F. Hoffmann-La Roche Ag Method for the production of recombinant aav particle preparations
WO2024213596A1 (en) 2023-04-13 2024-10-17 F. Hoffmann-La Roche Ag Improved recombinant polyadenylation signal sequences and use thereof
WO2025057182A1 (en) * 2023-09-15 2025-03-20 Indian Institute Of Technology Kanpur Modified aav vectors for enhanced gene therapy in hemophilia b
WO2025082618A1 (en) 2023-10-20 2025-04-24 Fuse Vectors Aps In vitro parvovirus capsid manufacturing
WO2025168663A1 (en) 2024-02-09 2025-08-14 F. Hoffmann-La Roche Ag Method for producing recombinant adeno-associated viral 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
CA2264482A1 (en) 1996-09-06 1998-03-12 The 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
CN101203613B (zh) 2005-04-07 2012-12-12 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
US9198984B2 (en) 2006-04-28 2015-12-01 The Trustees Of The University Of Pennsylvania Scalable production method for AAV
MX342858B (es) * 2010-03-29 2016-10-13 The Trustees Of The Univ Of Pennsylvania * Sistema de ablacion transgenica inducida farmacologicamente.
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
PE20150163A1 (es) 2012-04-18 2015-02-23 Philadelphia Children Hospital Composicion y metodos para una transferencia de genes altamente eficiente usando variantes capside aav

Also Published As

Publication number Publication date
WO2013158879A1 (en) 2013-10-24
IL235102A0 (en) 2014-12-31
PL2839014T3 (pl) 2021-08-23
CA2870736A1 (en) 2013-10-24
IN2014DN08812A (enEXAMPLES) 2015-05-22
US20180245098A1 (en) 2018-08-30
JP6342886B2 (ja) 2018-06-13
US11279950B2 (en) 2022-03-22
EP2839014A4 (en) 2015-12-30
CN104487579B (zh) 2022-01-18
RU2683497C2 (ru) 2019-03-28
EP2839014B1 (en) 2020-12-16
CA2870736C (en) 2021-11-02
RU2014146159A (ru) 2016-06-10
US20150065562A1 (en) 2015-03-05
HK1207663A1 (en) 2016-02-05
AU2013249202A1 (en) 2014-11-06
US9909142B2 (en) 2018-03-06
MX2014012680A (es) 2015-05-11
SG11201406776TA (en) 2015-03-30
JP2015514427A (ja) 2015-05-21
MY172457A (en) 2019-11-26
SI2839014T1 (sl) 2021-05-31
PH12014502347A1 (en) 2014-12-22
BR122021020419B1 (pt) 2023-01-17
MX359518B (es) 2018-10-01
CN104487579A (zh) 2015-04-01
DK2839014T3 (da) 2021-03-08
BR112014025985A2 (pt) 2017-07-11
IL235102B (en) 2019-08-29
CO7200251A2 (es) 2015-02-27
PT2839014T (pt) 2021-03-19
EP2839014A1 (en) 2015-02-25
PH12014502347B1 (en) 2014-12-22
AU2013249202B2 (en) 2018-08-09
HUE054087T2 (hu) 2021-09-28
ES2862912T3 (es) 2021-10-08
KR20150004859A (ko) 2015-01-13
PE20150163A1 (es) 2015-02-23
KR102063483B1 (ko) 2020-01-08
ZA201407582B (en) 2021-05-26

Similar Documents

Publication Publication Date Title
NZ701693A (en) Composition and methods for highly efficient gene transfer using aav capsid variants
PH12020551744A1 (en) Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
EA201591992A1 (ru) Эффективная доставка больших генов посредством двойных aav векторов
MX2022006652A (es) Proteinas de capside modificadas para un suministro mejorado de vectores de parvovirus.
MX2018005490A (es) Variantes de factor viii reducido en cpg, composiciones, metodos y usos para el tratamiento de trastornos de hemostasia.
WO2011133890A8 (en) Cns targeting aav vectors and methods of use thereof
MX367100B (es) Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
WO2012112578A3 (en) Improved aav8 vector with enhanced functional activity and methods of use thereof
EA201001491A1 (ru) Репликационно-дефектные вакцины и вакцинные векторы против флавивирусов
WO2010138263A3 (en) Novel aav 's and uses thereof
MX2014015002A (es) Polipeptidos que tienen actividad de transgalactosilacion.
EP3845552A3 (en) High-transduction-efficiency raav vectors, compositions, and methods of use
MX344972B (es) Oligonucleotidos inmunoestimulantes.
PH12015502126A1 (en) Human pac1 antibodies
PH12015500771A1 (en) Novel fc gamma receptor iib variants
WO2011098778A3 (en) Peptides for vaccines against birch allergy
WO2012090207A3 (en) Par1 and par2 c-tail peptides and peptide mimetics
HK1206385A1 (zh) 具有改变的末端的成批重组腺病毒
MY205057A (en) Varian aav and compositions, methods and uses for gene transfer to cells, organs and tissues
TN2013000171A1 (en) Cxcr2 binding polypeptides

Legal Events

Date Code Title Description
PSEA Patent sealed
RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 18 APR 2019 BY CPA GLOBAL

Effective date: 20180302

S38C Proceedings under section 38 (amendment of specification with leave of commissioner): specification amended
S38C Proceedings under section 38 (amendment of specification with leave of commissioner): specification amended
RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 18 APR 2020 BY CPA GLOBAL

Effective date: 20190307

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 18 APR 2021 BY CPA GLOBAL

Effective date: 20200306

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 18 APR 2022 BY CPA GLOBAL

Effective date: 20210305

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 18 APR 2023 BY CPA GLOBAL

Effective date: 20220304

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 18 APR 2024 BY CPA GLOBAL

Effective date: 20230303

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 18 APR 2025 BY ABHISHEK KUMAR - CPA-PTO PAYMENTS

Effective date: 20240307

RENW Renewal (renewal fees accepted)

Free format text: PATENT RENEWED FOR 1 YEAR UNTIL 18 APR 2026 BY ABHISHEK KUMAR - CPA-PTO PAYMENTS

Effective date: 20250307