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)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (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)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (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 (enExample)
EP (1) EP2839014B1 (enExample)
JP (1) JP6342886B2 (enExample)
KR (1) KR102063483B1 (enExample)
CN (1) CN104487579B (enExample)
AU (1) AU2013249202B2 (enExample)
BR (2) BR112014025985A2 (enExample)
CA (1) CA2870736C (enExample)
CO (1) CO7200251A2 (enExample)
DK (1) DK2839014T3 (enExample)
ES (1) ES2862912T3 (enExample)
HU (1) HUE054087T2 (enExample)
IL (1) IL235102B (enExample)
IN (1) IN2014DN08812A (enExample)
MX (1) MX359518B (enExample)
MY (1) MY172457A (enExample)
NZ (1) NZ701693A (enExample)
PE (1) PE20150163A1 (enExample)
PH (1) PH12014502347A1 (enExample)
PL (1) PL2839014T3 (enExample)
PT (1) PT2839014T (enExample)
RU (1) RU2683497C2 (enExample)
SG (1) SG11201406776TA (enExample)
SI (1) SI2839014T1 (enExample)
WO (1) WO2013158879A1 (enExample)
ZA (1) ZA201407582B (enExample)

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 (132)

* 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
EP3492596A1 (en) 2007-04-09 2019-06-05 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
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
AU2012249540B2 (en) 2011-04-29 2017-07-13 Selecta Biosciences, Inc. Tolerogenic synthetic nanocarriers to reduce immune responses to therapeutic proteins
KR102063483B1 (ko) 2012-04-18 2020-01-08 더 칠드런스 호스피탈 오브 필라델피아 Ａａｖ 캡시드 변이체를 이용한 고효율 유전자 전달을 위한 조성물 및 방법
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
EP2991646B1 (en) 2013-05-03 2020-10-07 Selecta Biosciences, Inc. Methods and compositions for enhancing cd4+ regulatory t cells
CA2907799A1 (en) * 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
MX2016001044A (es) * 2013-07-22 2016-09-09 The Children's Hospital Of Philadelphia Aav variante y composiciones, metodos y usos para transferencia genica a celulas, organos y tejidos.
US20160369298A1 (en) 2013-09-26 2016-12-22 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
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
EP3189147A1 (en) 2014-09-07 2017-07-12 Selecta Biosciences, Inc. Methods and compositions for attenuating gene expression modulating anti-viral transfer vector immune responses
AU2015335923B2 (en) 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
SG10202007103TA (en) 2014-11-05 2020-09-29 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
CN112375760A (zh) 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
KR20230169197A (ko) 2014-11-14 2023-12-15 보이저 테라퓨틱스, 인크. 근위축성 측삭 경화증(als)을 치료하는 조성물 및 방법
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
US20180030096A1 (en) 2015-02-03 2018-02-01 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
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
CN108138159A (zh) 2015-06-23 2018-06-08 费城儿童医院 经修饰的因子ix、以及用于基因转移到细胞、器官和组织的组合物、方法和用途
US10745447B2 (en) 2015-09-28 2020-08-18 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
WO2017070476A2 (en) 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
MX2018005353A (es) 2015-10-28 2018-08-14 Univ Pennsylvania Administracion intratecal de vectores virales adenop-asociados para terapia genica.
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
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
BR112018071180A2 (pt) 2016-04-16 2019-02-12 University Of Florida Research Foundation, Incorporated métodos de reforço potência biológica de baculovirus system-produzido vírus adeno-associado recombinante
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
CN116333057A (zh) 2016-05-13 2023-06-27 4D分子治疗有限公司 腺相关病毒变体衣壳和其使用方法
CN117904112A (zh) 2016-05-18 2024-04-19 沃雅戈治疗公司 调节性多核苷酸
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
KR20240172773A (ko) * 2016-08-15 2024-12-10 젠자임 코포레이션 Aav의 검출 방법
EP3506817A4 (en) 2016-08-30 2020-07-22 The Regents of The University of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
AU2017341849B2 (en) * 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
WO2018129268A1 (en) 2017-01-07 2018-07-12 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
EP3592389B1 (en) 2017-03-11 2025-05-07 Cartesian Therapeutics, 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
CN117801075A (zh) 2017-03-15 2024-04-02 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
PL3601367T3 (pl) 2017-03-30 2025-03-31 The University Of Queensland Cząsteczki chimeryczne i ich zastosowania
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
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
MY207281A (en) 2017-06-07 2025-02-13 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
KR102766238B1 (ko) 2017-06-30 2025-02-13 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. 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
PT3684423T (pt) 2017-09-20 2023-06-09 4D Molecular Therapeutics Inc Cápsides variantes de vírus adeno-associado e métodos de utilização das mesmas
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
US20190142974A1 (en) 2017-10-13 2019-05-16 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
AU2018352236B2 (en) 2017-10-16 2025-04-10 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
BR112020010674A2 (pt) 2017-11-27 2020-11-10 4D Molecular Therapeutics Inc. capsídeos de variante de vírus adeno-associado e uso para inibir a angiogênese
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
BR112020015798A2 (pt) 2018-02-01 2021-03-09 Homology Medicines, Inc. Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas
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
EP3758682A4 (en) 2018-02-26 2021-12-15 Antolrx, Inc. TOLEROGENIC LIPOSOMES AND THEIR PROCEDURES FOR USE
BR112020017348A2 (pt) * 2018-02-27 2020-12-29 The Trustees Of The University Of Pennsylvania Vetores de vírus adenoassociados (aav), vetores de aav tendo desamidação de capsídeos reduzida e usos dos mesmos
KR102834868B1 (ko) * 2018-03-16 2025-07-17 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 캡시드 변형에 의한 증가하는 조직 특정 유전자 전달
MX2020010464A (es) 2018-04-03 2021-01-29 Vectores de virus que evitan anticuerpos.
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
SG11202009451VA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
KR20210015770A (ko) 2018-04-05 2021-02-10 제네똥 감소된 간 향성을 갖는 AAV9과 AAVrh74의 하이브리드 재조합 아데노-관련 바이러스 세로타입
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
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
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
MY207355A (en) * 2018-05-16 2025-02-21 Spark Therapeutics Inc Codon-optimized acid lpha-glucosidase expression cassettes and methods of using same
CN112654370A (zh) 2018-07-16 2021-04-13 西莱克塔生物科技公司 Mma构建体和载体的方法和组合物
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
SG11202103151RA (en) 2018-09-28 2021-04-29 Voyager Therapeutics Inc Frataxin expression constructs having engineered promoters and methods of use thereof
CN112955557A (zh) 2018-10-01 2021-06-11 奥特吉尼克斯制药公司 用于治疗丙酸血症的基因疗法
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
WO2020082046A2 (en) 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for expressing factor ix
WO2020163743A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
EP3920974A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
SG11202108044YA (en) 2019-02-25 2021-09-29 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
US20220154211A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
SG11202110146VA (en) 2019-03-21 2021-10-28 Stridebio Inc Recombinant adeno-associated virus vectors
CA3138525A1 (en) 2019-04-28 2020-11-05 Selecta Biosciences, Inc. Methods for treatment of subjects with preexisting immunity to viral transfer vectors
MX2021014566A (es) 2019-05-28 2022-03-22 Selecta Biosciences Inc Métodos y composiciones para la respuesta inmune atenuada anti-vector de transferencia viral.
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
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
IL292620A (en) * 2019-10-30 2022-07-01 Decibel Therapeutics Inc Compounds and methods for use in dual vector autoperlin arrays for the treatment of structural hearing loss
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
KR20220140537A (ko) 2020-02-14 2022-10-18 울트라제닉스 파마수티컬 인코포레이티드 Cdkl5 결핍 장애를 치료하기 위한 유전자 요법
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
JP2023520402A (ja) 2020-03-31 2023-05-17 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 プロピオン酸血症を処置するための遺伝子治療
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
TW202227632A (zh) 2020-08-19 2022-07-16 美商史崔德生物公司 用於治療雷特症候群之腺相關病毒載體
IL302046A (en) 2020-10-15 2023-06-01 Hoffmann La Roche Nucleic acid structures for va RNA transcription
MX2023004178A (es) 2020-10-15 2023-05-03 Hoffmann La Roche Construcciones de acido nucleico para la activacion simultanea de genes.
CA3207247A1 (en) 2021-01-05 2022-07-14 Selecta Biosciences, Inc. Viral vector dosing protocols
MX2023012509A (es) * 2021-04-23 2024-01-03 Univ Florida Vectores aavrh74 para la terapia génica de distrofias musculares.
US20250019721A1 (en) 2021-08-11 2025-01-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
PE20241995A1 (es) 2021-08-20 2024-09-27 Biocad Joint Stock Co Metodo para obtener una capside de virus adenoasociado modificada
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
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
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
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
CN119137263A (zh) 2022-03-07 2024-12-13 奥特吉尼克斯制药公司 改进的分批aav产生系统和方法
WO2023172624A1 (en) 2022-03-09 2023-09-14 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
CN118974274A (zh) 2022-04-13 2024-11-15 豪夫迈·罗氏有限公司 用于确定aav基因组的方法
AU2023269646A1 (en) * 2022-05-11 2024-12-05 The Children's Hospital Of Philadelphia Adeno-associated viral vectors for targeting deep brain structures
CN119256220A (zh) 2022-05-23 2025-01-03 豪夫迈·罗氏有限公司 用于区分aav颗粒血清型和aav颗粒荷载状态的基于拉曼的方法
CA3258165A1 (en) 2022-06-03 2023-12-07 F. Hoffmann-La Roche Ag PROCESS FOR THE PRODUCTION OF RECOMBINED ADENOVIRUS-ASSOCIATED VIRUS (AAV) PARTICLES
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
AU2023321906A1 (en) 2022-08-11 2025-03-27 Cartesian Therapeutics, Inc. Compositions and methods related to immunoglobulin proteases and fusions thereof
CA3264415A1 (en) 2022-08-15 2024-02-22 F. Hoffmann-La Roche Ag PREVENTION OR MITIGATION OF ADVERSE EFFECTS ASSOCIATED WITH RECOMBINATING VIRAL VECTORS
KR20250067819A (ko) 2022-09-12 2025-05-15 에프. 호프만-라 로슈 아게 핵산 포함 및 핵산 비포함 aav 입자를 분리하는 방법
IL322001A (en) 2023-02-07 2025-09-01 Hoffmann La Roche Method for detecting antibodies to anti-AAV particles
KR20250162543A (ko) 2023-03-21 2025-11-18 에프. 호프만-라 로슈 아게 재조합 aav 입자 제제 생산 방법
CN120936723A (zh) 2023-04-13 2025-11-11 豪夫迈·罗氏有限公司 改进的重组多腺苷酸化信号序列及其用途
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
WO2025252480A1 (en) 2024-06-07 2025-12-11 F. Hoffmann-La Roche Ag Method for purifying plasmid dna

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
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
EP2018421B1 (en) 2006-04-28 2012-12-19 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
KR102063483B1 (ko) 2012-04-18 2020-01-08 더 칠드런스 호스피탈 오브 필라델피아 Ａａｖ 캡시드 변이체를 이용한 고효율 유전자 전달을 위한 조성물 및 방법

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

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
CN111235158A (zh) 用于表达重组人β-珠蛋白的病毒载体及其应用
RU2761879C1 (ru) Вакцина на основе AAV5 для индукции специфического иммунитета к вирусу SARS-CoV-2 и/или профилактики коронавирусной инфекции, вызванной SARS-CoV-2
RU2760301C1 (ru) Вакцина на основе AAV5 для индукции специфического иммунитета к вирусу SARS-CoV-2 и/или профилактики коронавирусной инфекции, вызванной SARS-CoV-2
CN117247973B (zh) 一种用于遗传性凝血因子缺乏病治疗的核酸构建体及其用途
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