RU2683497C2 - Композиция и способы высокоэффективного переноса генов с помощью вариантов капсида aav - Google Patents

Композиция и способы высокоэффективного переноса генов с помощью вариантов капсида aav Download PDF

Info

Publication number
RU2683497C2
RU2683497C2 RU2014146159A RU2014146159A RU2683497C2 RU 2683497 C2 RU2683497 C2 RU 2683497C2 RU 2014146159 A RU2014146159 A RU 2014146159A RU 2014146159 A RU2014146159 A RU 2014146159A RU 2683497 C2 RU2683497 C2 RU 2683497C2
Authority
RU
Russia
Prior art keywords
nucleic acid
aav
acid sequence
heterologous nucleic
vector
Prior art date
Application number
RU2014146159A
Other languages
English (en)
Russian (ru)
Other versions
RU2014146159A (ru
Inventor
Мустафа Н. ЯЗИСИОГЛУ
Федерико МИНГОЗЗИ
Хавьер АНГЕЛА
Кэтрин А. ХАЙ
Original Assignee
Дзе Чилдрен'З Хоспитал Оф Филадельфия
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 Дзе Чилдрен'З Хоспитал Оф Филадельфия filed Critical Дзе Чилдрен'З Хоспитал Оф Филадельфия
Publication of RU2014146159A publication Critical patent/RU2014146159A/ru
Application granted granted Critical
Publication of RU2683497C2 publication Critical patent/RU2683497C2/ru

Links

Images

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)
  • Hematology (AREA)
  • Oncology (AREA)
  • Diabetes (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)
RU2014146159A 2012-04-18 2013-04-18 Композиция и способы высокоэффективного переноса генов с помощью вариантов капсида aav RU2683497C2 (ru)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201261635273P 2012-04-18 2012-04-18
US61/635,273 2012-04-18
US201361794995P 2013-03-15 2013-03-15
US61/794,995 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 (2)

Publication Number Publication Date
RU2014146159A RU2014146159A (ru) 2016-06-10
RU2683497C2 true RU2683497C2 (ru) 2019-03-28

Family

ID=49384070

Family Applications (1)

Application Number Title Priority Date Filing Date
RU2014146159A RU2683497C2 (ru) 2012-04-18 2013-04-18 Композиция и способы высокоэффективного переноса генов с помощью вариантов капсида aav

Country Status (26)

Country Link
US (2) US9909142B2 (forum.php)
EP (1) EP2839014B1 (forum.php)
JP (1) JP6342886B2 (forum.php)
KR (1) KR102063483B1 (forum.php)
CN (1) CN104487579B (forum.php)
AU (1) AU2013249202B2 (forum.php)
BR (2) BR112014025985A2 (forum.php)
CA (1) CA2870736C (forum.php)
CO (1) CO7200251A2 (forum.php)
DK (1) DK2839014T3 (forum.php)
ES (1) ES2862912T3 (forum.php)
HU (1) HUE054087T2 (forum.php)
IL (1) IL235102B (forum.php)
IN (1) IN2014DN08812A (forum.php)
MX (1) MX359518B (forum.php)
MY (1) MY172457A (forum.php)
NZ (1) NZ701693A (forum.php)
PE (1) PE20150163A1 (forum.php)
PH (1) PH12014502347B1 (forum.php)
PL (1) PL2839014T3 (forum.php)
PT (1) PT2839014T (forum.php)
RU (1) RU2683497C2 (forum.php)
SG (1) SG11201406776TA (forum.php)
SI (1) SI2839014T1 (forum.php)
WO (1) WO2013158879A1 (forum.php)
ZA (1) ZA201407582B (forum.php)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2777404C1 (ru) * 2022-05-06 2022-08-03 федеральное государственное бюджетное учреждение "Национальный исследовательский центр эпидемиологии и микробиологии имени почетного академика Н.Ф. Гамалеи" Министерства здравоохранения Российской Федерации Экспрессионный вектор на основе аденоассоциированного вируса и способ его применения для экстренной профилактики и профилактики заболеваний, вызываемых вирусом SARS-CoV-2 (варианты)

Families Citing this family (128)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117363655A (zh) * 2005-04-07 2024-01-09 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
DK2623605T3 (en) 2007-04-09 2019-03-25 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
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
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
BR112013027514B1 (pt) 2011-04-29 2021-09-28 Selecta Biosciences, Inc. Composição compreendendo nanotransportadores sintéticos tolerogênicos, sua forma de dosagem e seu uso
WO2013158879A1 (en) 2012-04-18 2013-10-24 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
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
IL319892A (en) * 2013-07-22 2025-05-01 Childrens Hospital Philadelphia Modified AAV and compositions, methods and uses for gene delivery to cells, organs and tissues
AU2014324717B2 (en) 2013-09-26 2020-10-08 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
CA2941640A1 (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
IL292577A (en) 2014-09-07 2022-06-01 Selecta Biosciences Inc Methods and preparations for attenuating immune responses of gene editing in an antiviral transfer system
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
CN112553229A (zh) 2014-11-05 2021-03-26 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
IL292999A (en) 2014-11-14 2022-07-01 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN112410339A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
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
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
WO2016134337A1 (en) * 2015-02-19 2016-08-25 University Of Florida Research Foundation, Inc. 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
DK3313991T3 (da) 2015-06-23 2024-09-09 Childrens Hospital Philadelphia Modificeret faktor ix og sammensætninger, fremgangsmåder og anvendelser til genoverførsel til celler, organer og væv
DK3356390T3 (da) 2015-09-28 2021-04-12 Univ North Carolina Chapel Hill Fremgangsmåder og sammensætninger til virusvektorer, der undgår antistoffer
WO2017070476A2 (en) 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
HK1257801A1 (zh) 2015-10-28 2019-11-01 宾夕法尼亚州大学信托人 用於基因治療的腺伴隨病毒載體的鞘內給藥
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
CA3006569A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
JP6975980B2 (ja) * 2016-04-16 2021-12-01 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド バキュロウイルス系で産生される組換えアデノ随伴ウイルスの生物学的力価を増強する方法
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A 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
AU2017268382B2 (en) 2016-05-18 2023-09-28 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
ES2863674T3 (es) * 2016-08-15 2021-10-11 Genzyme Corp Métodos para detectar AAV
US11298041B2 (en) 2016-08-30 2022-04-12 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
JP7577291B6 (ja) 2016-10-13 2024-11-26 ユニバーシティ オブ マサチューセッツ Ａａｖカプシド設計
WO2018129268A1 (en) 2017-01-07 2018-07-12 Selecta Biosciences, Inc. Patterned dosing of immunosuppressants coupled to synthetic nanocarriers
JP7081767B2 (ja) * 2017-01-30 2022-06-07 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
CA3055936A1 (en) 2017-03-11 2018-09-20 Selecta Biosciences, Inc. Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
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
CN110770346B (zh) 2017-03-15 2024-01-12 北卡罗来纳-查佩尔山大学 多倍体腺相关病毒载体及其制备和使用方法
SG11201908280SA (en) 2017-03-30 2019-10-30 Univ Queensland "chimeric molecules and uses thereof"
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN119491003A (zh) 2017-05-05 2025-02-21 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
BR112019025792A2 (pt) 2017-06-07 2020-07-07 Spark Therapeutics, Inc. agentes de aperfeiçoamento para transfecção de células melhoradas e/ou produção de vetor raav
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3053154A1 (en) 2017-06-30 2019-01-03 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
AU2018302016A1 (en) 2017-07-17 2020-02-06 The Regents Of The University Of California Trajectory array guide system
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
EP4400174A3 (en) 2017-09-01 2024-10-23 The Francis Crick Institute Limited Immunoregulatory molecules and uses therefor
NZ763018A (en) 2017-09-20 2023-02-24 4D Molecular Therapeutics Inc Adeno-associated virus variant capsids and methods of use thereof
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.
CN111542336A (zh) 2017-10-13 2020-08-14 西莱克塔生物科技公司 用于减弱抗病毒转移载体igm应答的方法和组合物
CA3077426A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
US20200237799A1 (en) 2017-10-16 2020-07-30 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
US20190256867A1 (en) 2018-02-01 2019-08-22 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
CN112041437A (zh) 2018-02-19 2020-12-04 同源药物公司 用于恢复f8基因功能的腺相关病毒组合物和其使用方法
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
IL276859B1 (en) * 2018-02-27 2025-08-01 Univ Pennsylvania Novel adeno-associated virus (AAV) vectors, AAV vectors with reduced capsid deamidation and uses therefor
CN111819286B (zh) * 2018-03-16 2024-05-24 国家儿童医院研究所 通过衣壳修饰增加组织特异性的基因递送
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
KR20210006357A (ko) 2018-04-03 2021-01-18 스트라이드바이오 인코포레이티드 항체-회피 바이러스 벡터
WO2019195444A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
CN112004925B (zh) 2018-04-05 2024-09-27 吉尼松公司 具有降低的肝向性的AAV9和AAVrh74的杂合重组腺相关病毒血清型
CA3098198A1 (en) 2018-04-27 2019-10-31 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
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
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
PE20210337A1 (es) * 2018-05-16 2021-02-19 Spark Therapeutics Inc CASETES DE EXPRESION DE alfa-GLUCOSIDASA DE ACIDO OPTIMIZADO CON CODON Y METODOS DE USO DEL MISMO
KR20210034015A (ko) 2018-07-16 2021-03-29 셀렉타 바이오사이언시즈, 인크. Mma 구축물 및 벡터의 방법 및 조성물
JP2021531282A (ja) 2018-07-16 2021-11-18 セレクタ バイオサイエンシーズ インコーポレーテッドSelecta Biosciences, Inc. Ｏｔｃコンストラクトおよびベクターの方法および組成物
JP7528066B2 (ja) 2018-09-28 2024-08-05 ボイジャー セラピューティクス インコーポレイテッド 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法
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
AU2019360270B2 (en) 2018-10-18 2025-08-07 Intellia Therapeutics, Inc. Compositions and methods for expressing factor IX.
CN114072179B (zh) 2019-02-08 2024-11-01 分贝治疗公司 肌球蛋白15启动子及其用途
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
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
CN113677801A (zh) 2019-02-25 2021-11-19 诺华股份有限公司 治疗bietti晶体营养不良的组合物和方法
WO2020191300A1 (en) 2019-03-21 2020-09-24 Stridebio, Inc. Recombinant adeno-associated virus vectors
KR20220004121A (ko) 2019-04-28 2022-01-11 셀렉타 바이오사이언시즈, 인크. 바이러스 전달 벡터에 대한 기존의 면역을 갖는 대상체의 치료 방법
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
JP2022550435A (ja) 2019-10-04 2022-12-01 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 組換えａａｖの改善された治療的使用のための方法
CA3157700A1 (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
KR20220140537A (ko) 2020-02-14 2022-10-18 울트라제닉스 파마수티컬 인코포레이티드 Cdkl5 결핍 장애를 치료하기 위한 유전자 요법
WO2021188892A1 (en) 2020-03-19 2021-09-23 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
EP4127189A1 (en) 2020-03-31 2023-02-08 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
US20230304038A1 (en) 2020-08-19 2023-09-28 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of rett syndrome
HRP20250766T1 (hr) 2020-10-15 2025-08-15 F. Hoffmann - La Roche Ag Konstrukti nukleinske kiseline za transkripciju va rna
CA3197726A1 (en) 2020-10-15 2022-04-21 Simon Auslaender Nucleic acid constructs for simultaneous gene activation
EP4274571A1 (en) 2021-01-05 2023-11-15 Selecta Biosciences, Inc. Viral vector dosing protocols
BR112023021495A2 (pt) * 2021-04-23 2023-12-19 Univ Florida Vetores aavrh74 para terapia gênica de distrofias musculares
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
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
IL310949A (en) 2021-08-20 2024-04-01 Biocad Joint Stock Co Method for producing modified adeno-associated virus capsid
US20230086710A1 (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
WO2023064367A1 (en) 2021-10-12 2023-04-20 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
JP2024545594A (ja) 2021-11-14 2024-12-10 カーティザン セラピューティクス，インコーポレーテッド ウイルスベクターでの複数回投薬
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
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
KR20250031153A (ko) * 2022-05-11 2025-03-06 더 칠드런스 호스피탈 오브 필라델피아 뇌 심부 구조를 표적화하기 위한 아데노-관련 바이러스 벡터
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
JP2025519228A (ja) 2022-06-03 2025-06-24 エフ． ホフマン－ラ ロシュ アーゲー 組換えａａｖ粒子を生成するための方法
CN119546320A (zh) 2022-07-14 2025-02-28 豪夫迈·罗氏有限公司 用于生产重组aav颗粒的方法
US20240148841A1 (en) 2022-08-11 2024-05-09 Selecta Biosciences Inc. Compositions and methods related to immunoglobulin proteases and fusions thereof
IL317385A (en) 2022-08-15 2025-02-01 Hoffmann La Roche Preventing or reducing side effects associated with recombinant viral vectors
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

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6127525A (en) * 1995-02-21 2000-10-03 Cornell Research Foundation, Inc. Chimeric adenoviral coat protein and methods of using same
RU2335542C2 (ru) * 2003-06-13 2008-10-10 Джэпэн Эз Репрезентед Бай Президент Оф Нэшнл Сентер Фор Джериэтрикс Энд Джеронтолоджи Аденоассоциированный вирусный вектор для лечения болезни альцгеймера, его применение для получения терапевтических средств, а также способ лечения болезни альцгеймера с помощью данного вектора
US20090197338A1 (en) * 2005-04-07 2009-08-06 The Trustees Of Teh University Of Pennsylvania Method of Increasing the Function of an AAV Vector
US7629322B2 (en) * 2003-06-11 2009-12-08 Kleinschmidt Juergon AAV vector for gene therapy
WO2011126808A2 (en) * 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system

Family Cites Families (8)

* 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
AU722624B2 (en) 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
WO2007127264A2 (en) 2006-04-28 2007-11-08 The Trustees Of The University Of Pennsylvania Scalable production method for aav
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
WO2013158879A1 (en) 2012-04-18 2013-10-24 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6127525A (en) * 1995-02-21 2000-10-03 Cornell Research Foundation, Inc. Chimeric adenoviral coat protein and methods of using same
US7629322B2 (en) * 2003-06-11 2009-12-08 Kleinschmidt Juergon AAV vector for gene therapy
RU2335542C2 (ru) * 2003-06-13 2008-10-10 Джэпэн Эз Репрезентед Бай Президент Оф Нэшнл Сентер Фор Джериэтрикс Энд Джеронтолоджи Аденоассоциированный вирусный вектор для лечения болезни альцгеймера, его применение для получения терапевтических средств, а также способ лечения болезни альцгеймера с помощью данного вектора
US20090197338A1 (en) * 2005-04-07 2009-08-06 The Trustees Of Teh University Of Pennsylvania Method of Increasing the Function of an AAV Vector
WO2011126808A2 (en) * 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2842439C2 (ru) * 2019-05-24 2025-06-26 Регенерон Фармасьютикалз, Инк. Модифицированные вирусные частицы и пути их применения
RU2777404C1 (ru) * 2022-05-06 2022-08-03 федеральное государственное бюджетное учреждение "Национальный исследовательский центр эпидемиологии и микробиологии имени почетного академика Н.Ф. Гамалеи" Министерства здравоохранения Российской Федерации Экспрессионный вектор на основе аденоассоциированного вируса и способ его применения для экстренной профилактики и профилактики заболеваний, вызываемых вирусом SARS-CoV-2 (варианты)

Also Published As

Publication number Publication date
IL235102A0 (en) 2014-12-31
EP2839014A4 (en) 2015-12-30
CN104487579A (zh) 2015-04-01
NZ701693A (en) 2017-02-24
CO7200251A2 (es) 2015-02-27
MX2014012680A (es) 2015-05-11
HUE054087T2 (hu) 2021-09-28
SG11201406776TA (en) 2015-03-30
JP6342886B2 (ja) 2018-06-13
CN104487579B (zh) 2022-01-18
MX359518B (es) 2018-10-01
ZA201407582B (en) 2021-05-26
PT2839014T (pt) 2021-03-19
US9909142B2 (en) 2018-03-06
KR102063483B1 (ko) 2020-01-08
PH12014502347A1 (en) 2014-12-22
BR122021020419B1 (pt) 2023-01-17
IN2014DN08812A (forum.php) 2015-05-22
EP2839014A1 (en) 2015-02-25
HK1207663A1 (en) 2016-02-05
CA2870736C (en) 2021-11-02
US20180245098A1 (en) 2018-08-30
AU2013249202B2 (en) 2018-08-09
ES2862912T3 (es) 2021-10-08
AU2013249202A1 (en) 2014-11-06
SI2839014T1 (sl) 2021-05-31
WO2013158879A1 (en) 2013-10-24
DK2839014T3 (da) 2021-03-08
CA2870736A1 (en) 2013-10-24
JP2015514427A (ja) 2015-05-21
PL2839014T3 (pl) 2021-08-23
US20150065562A1 (en) 2015-03-05
PE20150163A1 (es) 2015-02-23
MY172457A (en) 2019-11-26
EP2839014B1 (en) 2020-12-16
US11279950B2 (en) 2022-03-22
KR20150004859A (ko) 2015-01-13
PH12014502347B1 (en) 2014-12-22
RU2014146159A (ru) 2016-06-10
BR112014025985A2 (pt) 2017-07-11
IL235102B (en) 2019-08-29

Similar Documents

Publication Publication Date Title
RU2683497C2 (ru) Композиция и способы высокоэффективного переноса генов с помощью вариантов капсида aav
EP2675484B1 (en) Improved aav8 vector with enhanced functional activity and methods of use thereof
KR102234672B1 (ko) 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
KR102516647B1 (ko) 고 형질도입 효율 raav 벡터, 조성물 및 사용 방법
AU6626500A (en) Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
CN116685329B (zh) 核酸构建体及其用于治疗脊髓性肌肉萎缩症的用途
RU2761879C1 (ru) Вакцина на основе AAV5 для индукции специфического иммунитета к вирусу SARS-CoV-2 и/или профилактики коронавирусной инфекции, вызванной SARS-CoV-2
CN111235158A (zh) 用于表达重组人β-珠蛋白的病毒载体及其应用
RU2760301C1 (ru) Вакцина на основе AAV5 для индукции специфического иммунитета к вирусу SARS-CoV-2 и/или профилактики коронавирусной инфекции, вызванной SARS-CoV-2
RU2742837C1 (ru) Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение
JP2023552535A (ja) 異種導入遺伝子の発現を制御するためのオーファンモチーフ（ｏｒｐｈａｎ ｍｏｔｉｆ）とＣｐＧ密度との組み合わせの使用
US20250084129A1 (en) Aav5 capsid variants
HK1207663B (en) Composition and methods for highly efficient gene transfer using aav capsid variants
EA050137B1 (ru) ВАКЦИНА НА ОСНОВЕ AAV5 ПРОТИВ SARS-CoV-2