MX359518B - Composición y métodos para una transferencia génica altamente eficiente utilizando variantes de cápsides de aav. - Google Patents

Composición y métodos para una transferencia génica altamente eficiente utilizando variantes de cápsides de aav.

Info

Publication number
MX359518B
MX359518B MX2014012680A MX2014012680A MX359518B MX 359518 B MX359518 B MX 359518B MX 2014012680 A MX2014012680 A MX 2014012680A MX 2014012680 A MX2014012680 A MX 2014012680A MX 359518 B MX359518 B MX 359518B
Authority
MX
Mexico
Prior art keywords
methods
composition
gene transfer
highly efficient
aav capsid
Prior art date
Application number
MX2014012680A
Other languages
English (en)
Spanish (es)
Other versions
MX2014012680A (es
Inventor
A High Katherine
Mingozzi Federico
N Yazicioglu Mustafa
Anguela Xavier
Original Assignee
Childrens Hospital 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 Childrens Hospital Philadelphia filed Critical Childrens Hospital Philadelphia
Publication of MX2014012680A publication Critical patent/MX2014012680A/es
Publication of MX359518B publication Critical patent/MX359518B/es

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    • 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

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  • 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)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Communicable Diseases (AREA)
  • Diabetes (AREA)
  • Hematology (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)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
MX2014012680A 2012-04-18 2013-04-18 Composición y métodos para una transferencia génica altamente eficiente utilizando variantes de cápsides de aav. MX359518B (es)

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 (2)

Publication Number Publication Date
MX2014012680A MX2014012680A (es) 2015-05-11
MX359518B true MX359518B (es) 2018-10-01

Family

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Family Applications (1)

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MX2014012680A MX359518B (es) 2012-04-18 2013-04-18 Composición y métodos para una transferencia génica altamente eficiente utilizando variantes de cápsides de 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) BR122021020419B1 (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) PH12014502347B1 (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)

Families Citing this family (133)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104293835B (zh) * 2005-04-07 2017-07-04 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
DK2191001T3 (en) 2007-04-09 2016-09-19 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
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
MX374963B (es) 2011-04-29 2025-03-06 Selecta Biosciences Inc Nanoportadores sintéticos tolerogénicos para reducir las respuestas de anticuerpos.
SG11201406776TA (en) 2012-04-18 2015-03-30 Philadelphia Children Hospital 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
BR112015027288B1 (pt) 2013-05-03 2022-03-15 Selecta Biosciences, Inc Composição que compreende um inibidor de mtor ligado a nanocarreadores sintéticos poliméricos
CN105247044B (zh) * 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
HUE047996T2 (hu) * 2013-07-22 2020-05-28 Childrens Hospital Philadelphia AAV variáns és készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez
EP3049527A4 (en) 2013-09-26 2017-08-16 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
EP3113787B1 (en) 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
AU2015311707B2 (en) 2014-09-07 2021-12-09 Selecta Biosciences, Inc. Methods and compositions for attenuating gene expression modulating anti-viral transfer vector immune responses
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
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
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
IL284949B2 (en) 2014-11-14 2024-06-01 Voyager Therapeutics Inc Modulatory polynucleotides
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
WO2016126857A1 (en) 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
US10648000B2 (en) 2015-02-16 2020-05-12 University Of Florida Research Foundation, Incorporated rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes
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
IL256517B2 (en) 2015-06-23 2024-06-01 Childrens Hospital Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
AU2016332821B2 (en) 2015-09-28 2022-02-17 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
CA3003435A1 (en) 2015-10-28 2017-05-04 The Trustees Of The University Of Pennsylvania Intrathecal administration of adeno-associated-viral vectors for gene therapy
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
WO2017096162A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
KR20250065734A (ko) 2016-04-16 2025-05-13 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 바큘로바이러스 시스템-생산된 재조합 아데노-연관 바이러스의 생물학적 효력을 증진시키는 방법
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
RU2725286C2 (ru) 2016-05-13 2020-06-30 4Д Молекьюлар Терапьютикс Инк. Варианты капсидов аденоассоциированного вируса и способы их применения
AU2017268382B2 (en) 2016-05-18 2023-09-28 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
EP3458588B1 (en) 2016-05-18 2025-10-29 Voyager Therapeutics, Inc. Modulatory polynucleotides
US11698377B2 (en) * 2016-08-15 2023-07-11 Genzyme Corporation Methods for detecting 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
KR20190075964A (ko) * 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 Aav 캡시드 설계
KR20190104194A (ko) 2017-01-07 2019-09-06 셀렉타 바이오사이언시즈, 인크. 합성 나노담체에 커플링된 면역억제제의 패턴화된 투여
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
EP3610023B1 (en) 2017-03-15 2023-10-04 The University of North Carolina at Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
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
MX2019011599A (es) 2017-03-30 2019-12-19 Univ Queensland Moleculas quimericas y usos de las mismas.
CN111108198A (zh) 2017-05-05 2020-05-05 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
SG11201911603SA (en) 2017-06-07 2020-01-30 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
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
WO2019040994A1 (en) 2017-09-01 2019-03-07 The Australian National University IMMUNOREGULATOR MOLECULES AND USES THEREOF
MX2020003176A (es) 2017-09-20 2020-08-20 4D Molecular Therapeutics Inc Variantes de capsides de virus adenoasociados y metodos de uso de estas.
EP3687582A4 (en) 2017-09-29 2021-07-14 Voyager Therapeutics, Inc. RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION
AU2018347583A1 (en) 2017-10-13 2020-05-21 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector IgM responses
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
CN111479924B (zh) 2017-10-16 2024-06-14 沃雅戈治疗公司 肌萎缩性侧索硬化症(als)的治疗
CA3083472A1 (en) 2017-11-27 2019-05-31 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis
EP3728568A4 (en) 2017-12-19 2021-10-06 The University of North Carolina at Chapel Hill METHODS AND COMPOSITIONS FOR DELIVERING VIRAL VECTORS BY THE BLOOD-BRAIN BARRIER
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
PE20212076A1 (es) 2018-02-01 2021-10-26 Homology Medicines Inc Composiciones de virus adeno-asociado para restaurar la funcion del gen pah y metodos de uso de las mismas
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
IL276957B2 (en) 2018-02-26 2023-12-01 Antolrx Inc Tolerogenic liposomes and methods of using them
IL276859B2 (en) * 2018-02-27 2025-12-01 Univ Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having 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
JP7425738B2 (ja) 2018-04-03 2024-01-31 ギンコ バイオワークス インコーポレイテッド 眼組織を標的とするウイルスベクター
CN112272672A (zh) 2018-04-03 2021-01-26 斯特里迪比奥公司 抗体逃避性病毒载体
WO2019195444A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
EP3775173A1 (en) 2018-04-05 2021-02-17 Genethon Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism
EP3784697A4 (en) * 2018-04-27 2022-07-06 Spark Therapeutics, Inc. GMO AAV CAPSIDS WITH INCREASED TROPISM AND AAV VECTORS CONTAINING THE GMO CAPSIDS, AND METHODS FOR THEIR MANUFACTURE AND USE
US12233136B2 (en) 2018-04-27 2025-02-25 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
MA52631A (fr) 2018-05-15 2021-03-24 Voyager Therapeutics Inc Compositions et méthodes pour le traitement de la maladie de parkinson
JP2021523718A (ja) * 2018-05-16 2021-09-09 スパーク セラピューティクス インコーポレイテッドＳｐａｒｋ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｉｎｃ． コドン最適化された酸性α−グルコシダーゼ発現カセット及びそれを使用する方法
JP2021530571A (ja) 2018-07-16 2021-11-11 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. Ｍｍａコンストラクトおよびベクターの方法および組成物
US20200038462A1 (en) 2018-07-16 2020-02-06 Selecta Biosciences, Inc. Methods and compositions of otc constructs and vectors
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
JP2022513318A (ja) 2018-10-01 2022-02-07 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
US12214023B2 (en) 2018-10-18 2025-02-04 Intellia Therapeutics, Inc. Compositions and methods for expressing factor IX
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
EP3920974A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS
JP2022521025A (ja) 2019-02-25 2022-04-04 ノバルティス アーゲー Ｂｉｅｔｔｉクリスタリン網膜症を治療するための組成物及び方法
WO2020174369A2 (en) 2019-02-25 2020-09-03 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
CA3133453A1 (en) 2019-03-21 2020-09-24 Daniel Mccoy 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
EP3976802A1 (en) 2019-05-28 2022-04-06 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
EP4038194A1 (en) 2019-10-04 2022-08-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
JP7665609B2 (ja) * 2019-10-30 2025-04-21 デシベル セラピューティクス インコーポレイテッド オトフェリン二重ベクター系及びそれを含む感音難聴を治療するための組成物
US20230204595A1 (en) * 2020-01-03 2023-06-29 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
BR112022015921A2 (pt) 2020-02-14 2022-10-04 Ultragenyx Pharmaceutical Inc Terapia gênica para tratar o transtorno de deficiência de cdkl5
JP2023518415A (ja) 2020-03-19 2023-05-01 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においてｃａｐおよびｒｅｐ配列の逆パッケージングを低減するための組成物および方法
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
KR20230068444A (ko) 2020-08-19 2023-05-17 사렙타 쎄러퓨틱스 인코퍼레이티드 레트 증후군의 치료를 위한 아데노 관련 바이러스 벡터
AR123777A1 (es) 2020-10-15 2023-01-11 Hoffmann La Roche Constructos de ácido nucleico para transcripción de arn va
IL302045A (en) 2020-10-15 2023-06-01 Hoffmann La Roche Nucleic acid constructs for simultaneous gene activation
AU2022206197A1 (en) 2021-01-05 2023-07-13 Selecta Biosciences, Inc. Viral vector dosing protocols
AU2022262407A1 (en) * 2021-04-23 2023-10-26 University Of Florida Research Foundation, Incorporated Aavrh74 vectors for gene therapy of muscular dystrophies
JP2024531138A (ja) 2021-08-11 2024-08-29 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 筋ジストロフィーを処置するための組成物および方法
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
EP4389880A1 (en) 2021-08-20 2024-06-26 Joint Stock Company "Biocad" Method of obtaining a modified adeno-associated virus capsid
US20230086710A1 (en) * 2021-09-14 2023-03-23 California Institute Of Technology Aav capsid variants
JP2024539646A (ja) 2021-10-12 2024-10-29 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 ウイルスベクター投薬プロトコル
WO2023064367A1 (en) 2021-10-12 2023-04-20 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
EP4429662A1 (en) 2021-11-14 2024-09-18 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
JP2025509328A (ja) 2022-03-07 2025-04-11 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 改良されたバッチａａｖ産生システムおよび方法
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
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
AR129500A1 (es) 2022-06-03 2024-09-04 Hoffmann La Roche Método para producir partículas de aav recombinante
AR129895A1 (es) 2022-07-14 2024-10-09 Hoffmann La Roche Método para producir partículas de aav recombinantes
CA3264659A1 (en) 2022-08-11 2024-02-15 Cartesian Therapeutics, Inc. COMPOSITIONS AND PROCESSES ASSOCIATED WITH IMMUNOGLOBULIN PROTEASES AND FUSION THEREOF
WO2024038002A1 (en) 2022-08-15 2024-02-22 F. Hoffmann-La Roche Ag Prevention or mitigation of adverse effects related to recombinant viral vectors
CA3264505A1 (en) 2022-09-12 2024-03-21 F. Hoffmann-La Roche Ag METHOD FOR SEPARING SOLID AND EMPTY VAA PARTICLES
AU2024216670A1 (en) 2023-02-07 2025-07-31 F. Hoffmann-La Roche Ag Method for the detection of anti-aav particle antibodies
WO2024194280A1 (en) 2023-03-21 2024-09-26 F. Hoffmann-La Roche Ag Method for the production of recombinant aav particle preparations
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
US20260022401A1 (en) 2024-07-22 2026-01-22 Hoffmann-La Roche Inc. Novel AAV rep ORFs and Rep polypeptides

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
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
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
CN104293835B (zh) 2005-04-07 2017-07-04 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
WO2007127264A2 (en) 2006-04-28 2007-11-08 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.
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
SG11201406776TA (en) 2012-04-18 2015-03-30 Philadelphia Children Hospital Composition and methods for highly efficient gene transfer using aav capsid variants

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