SI2839014T1 - Sestavek in postopki za zelo učinkovit prenos genov z uporabo variant kapside AAV-JA - Google Patents

Sestavek in postopki za zelo učinkovit prenos genov z uporabo variant kapside AAV-JA

Info

Publication number
SI2839014T1
SI2839014T1 SI201331862T SI201331862T SI2839014T1 SI 2839014 T1 SI2839014 T1 SI 2839014T1 SI 201331862 T SI201331862 T SI 201331862T SI 201331862 T SI201331862 T SI 201331862T SI 2839014 T1 SI2839014 T1 SI 2839014T1
Authority
SI
Slovenia
Prior art keywords
composition
methods
gene transfer
highly efficient
aav capsid
Prior art date
Application number
SI201331862T
Other languages
English (en)
Slovenian (sl)
Inventor
Mustafa N. Yazicioglu
Federico Mingozzi
Xavier Anguela
Katherine A. High
Original Assignee
The Children's Hospital Of Philadelphia
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by The Children's Hospital Of Philadelphia filed Critical The Children's Hospital Of Philadelphia
Publication of SI2839014T1 publication Critical patent/SI2839014T1/sl

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (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)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
SI201331862T 2012-04-18 2013-04-18 Sestavek in postopki za zelo učinkovit prenos genov z uporabo variant kapside AAV-JA SI2839014T1 (sl)

Applications Claiming Priority (4)

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
EP13778449.2A EP2839014B1 (en) 2012-04-18 2013-04-18 Composition and methods for highly efficient gene transfer using aav capsid variants

Publications (1)

Publication Number Publication Date
SI2839014T1 true SI2839014T1 (sl) 2021-05-31

Family

ID=49384070

Family Applications (1)

Application Number Title Priority Date Filing Date
SI201331862T SI2839014T1 (sl) 2012-04-18 2013-04-18 Sestavek in postopki za zelo učinkovit prenos genov z uporabo variant kapside AAV-JA

Country Status (26)

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

Families Citing this family (134)

* 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
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
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
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US9265815B2 (en) 2011-04-29 2016-02-23 Selecta Biosciences, Inc. Tolerogenic synthetic nanocarriers
IN2014DN08812A (show.php) 2012-04-18 2015-05-22 Philadelphia Children Hospital
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
CN111760025A (zh) 2013-05-03 2020-10-13 西莱克塔生物科技公司 用于诱导免疫耐受的具有特定药效学有效持续期之免疫抑制剂与抗原的递送
WO2014194132A1 (en) * 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
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
EP3633041A3 (en) 2013-09-26 2020-07-29 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
ES2768763T3 (es) 2014-03-04 2020-06-23 Univ Florida Vectores rAAV mejorados y métodos para la transducción de fotorreceptores y células EPR
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
WO2016037162A1 (en) 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector immune responses
RU2738421C2 (ru) 2014-10-21 2020-12-14 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
US10335466B2 (en) 2014-11-05 2019-07-02 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
SG11201703281RA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
JP6863891B2 (ja) 2014-11-14 2021-04-21 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 調節性ポリヌクレオチド
HK1245326A1 (zh) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
WO2016126857A1 (en) 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
WO2016133917A1 (en) 2015-02-16 2016-08-25 University Of Florida Research Foundation Raav vector compositions, methods for targeting vascular endothelial cells and use in treatment of type i diabetes
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
PT3313991T (pt) 2015-06-23 2024-09-27 Childrens Hospital Philadelphia Fator ix modificado e composições, métodos e utilizações para transferência de genes para células, órgãos e tecidos
IL299901B2 (en) 2015-09-28 2024-12-01 Univ Florida Methods and compositions for antibody-evading virus vectors
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated 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
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
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
MX2018013463A (es) 2016-05-13 2019-07-04 4D Molecular Therapeutics Inc Capsides variantes de virus adenoasociados y metodos de uso de las mismas.
JP7220080B2 (ja) 2016-05-18 2023-02-09 ボイジャー セラピューティクス インコーポレイテッド ハンチントン病治療組成物及び方法
EP3458588B1 (en) 2016-05-18 2025-10-29 Voyager Therapeutics, Inc. Modulatory polynucleotides
CN110168080B (zh) 2016-08-15 2024-05-31 建新公司 检测aav的方法
JP2019531787A (ja) 2016-08-30 2019-11-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
EP3526333A4 (en) * 2016-10-13 2020-07-29 University of Massachusetts AAV CAPSIDE DESIGNS
CA3049384A1 (en) 2017-01-07 2018-07-12 Selecta Biosciences, Inc. Patterned dosing of immunosuppressants coupled to synthetic nanocarriers
KR20190111966A (ko) * 2017-01-30 2019-10-02 각꼬호우징 닛뽄 이까다이가꾸 아데노 관련 바이러스(aav) 캡시드 단백질의 돌연변이체
WO2018156654A1 (en) * 2017-02-21 2018-08-30 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
KR20250011715A (ko) 2017-03-11 2025-01-21 셀렉타 바이오사이언시즈, 인크. 항염증제, 및 면역억제제를 포함하는 합성 나노담체를 사용한 조합 치료와 관련된 방법 및 조성물
IL268891B2 (en) 2017-03-15 2024-09-01 Univ North Carolina 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
NZ757178A (en) 2017-03-30 2026-01-30 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)
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
JP7801093B2 (ja) 2017-06-07 2026-01-16 スパーク セラピューティクス インコーポレイテッド 細胞トランスフェクション及び／又はｒＡＡＶベクター産生の改善のための増強剤
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
AU2018290954B2 (en) 2017-06-30 2023-07-13 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
US20210228738A1 (en) 2017-07-17 2021-07-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
EP3654860A1 (en) 2017-07-17 2020-05-27 Voyager Therapeutics, Inc. Trajectory array guide system
EP4400174A3 (en) 2017-09-01 2024-10-23 The Francis Crick Institute Limited Immunoregulatory molecules and uses therefor
CA3076348A1 (en) 2017-09-20 2019-03-28 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
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
EP3694543A1 (en) 2017-10-13 2020-08-19 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
KR102793696B1 (ko) 2017-11-27 2025-04-09 4디 몰레큘러 테라퓨틱스 아이엔씨. 아데노-관련 바이러스 변이체 캡시드 및 혈관 신생을 억제하기 위한 용도
CN111727243A (zh) 2017-12-19 2020-09-29 北卡罗来纳-查佩尔山大学 用于跨越血-脑屏障递送病毒载体的方法和组合物
KR20200127170A (ko) 2018-02-01 2020-11-10 호몰로지 메디슨, 인크. Pah 유전자 기능 복원을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
WO2019161365A1 (en) 2018-02-19 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
EP3758682A4 (en) 2018-02-26 2021-12-15 Antolrx, Inc. TOLEROGENIC LIPOSOMES AND THEIR PROCEDURES FOR USE
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
IL277270B2 (en) * 2018-03-16 2025-05-01 Res Inst Nationwide Childrens Hospital Increasing tissue-specific gene delivery through capsid modification
WO2019195444A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
US12116384B2 (en) 2018-04-03 2024-10-15 Ginkgo Bioworks, Inc. Virus vectors for targeting ophthalmic tissues
KR20210006357A (ko) 2018-04-03 2021-01-18 스트라이드바이오 인코포레이티드 항체-회피 바이러스 벡터
US12037362B2 (en) 2018-04-05 2024-07-16 Genethon Hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced liver tropism
US12233136B2 (en) 2018-04-27 2025-02-25 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
WO2019210267A2 (en) * 2018-04-27 2019-10-31 Spark Therapeutics, Inc. Engineered aav capsids with increased tropism and aav vectors comprising the engineered capsids and methods of making and using same
TW202012628A (zh) 2018-05-15 2020-04-01 美商航海家醫療公司 用於帕金森氏症治療的組成物及方法
KR20210021310A (ko) * 2018-05-16 2021-02-25 스파크 테라퓨틱스, 인코포레이티드 코돈-최적화된 산 알파-글루코시다제 발현 카세트 및 이를 사용하는 방법
MX2021000637A (es) 2018-07-16 2021-06-23 Selecta Biosciences Inc Métodos y composiciones de vectores y constructos ornitina transcarbamilasa (otc).
US20200038463A1 (en) 2018-07-16 2020-02-06 Selecta Biosciences, Inc. Methods and compositions of mma constructs and vectors
AU2019346655B2 (en) 2018-09-28 2025-03-13 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
KR20210082460A (ko) 2018-10-01 2021-07-05 울트라제닉스 파마수티컬 인코포레이티드 프로피온산혈증을 치료하기 위한 유전자 요법
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
CN113260701A (zh) 2018-10-18 2021-08-13 英特利亚治疗股份有限公司 用于表达因子ix的组合物和方法
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
AU2020218547A1 (en) 2019-02-08 2021-08-26 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
US20220154210A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
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
EP3962537A1 (en) 2019-04-28 2022-03-09 Selecta Biosciences, Inc. Methods for treatment of subjects with preexisting immunity to viral transfer vectors
WO2020243261A1 (en) 2019-05-28 2020-12-03 Selecta Biosciences, Inc. Methods and compositions for attenuated anti-viral transfer vector immune response
WO2020247625A1 (en) 2019-06-04 2020-12-10 Selecta Biosciences, Inc. Formulations and doses of pegylated uricase
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
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ，インコーポレイテッド ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター
BR112022008214A2 (pt) * 2019-10-30 2022-07-12 Decibel Therapeutics Inc Composições e métodos para tratar perda auditiva neurossensorial usando sistemas de vetor duplo de otoferlina
CA3222852A1 (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
WO2021163322A1 (en) 2020-02-14 2021-08-19 Ultragenyx Pharmaceutical Inc. Gene therapy for treating cdkl5 deficiency disorder
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
WO2021202532A1 (en) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
BR112023003145A2 (pt) 2020-08-19 2023-05-09 Sarepta Therapeutics Inc Vetores de vírus adenoassociado para tratamento da síndrome de rett
EP4229205B1 (en) 2020-10-15 2025-05-14 F. Hoffmann-La Roche AG Nucleic acid constructs for va rna transcription
WO2022079082A1 (en) 2020-10-15 2022-04-21 F. Hoffmann-La Roche Ag Nucleic acid constructs for simultaneous gene activation
CA3207247A1 (en) 2021-01-05 2022-07-14 Selecta Biosciences, Inc. Viral vector dosing protocols
WO2022226289A2 (en) * 2021-04-23 2022-10-27 University Of Florida Research Foundation, Incorporated Aavrh74 vectors for gene therapy of muscular dystrophies
US20250019721A1 (en) 2021-08-11 2025-01-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
AR126839A1 (es) 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta
MA64631B1 (fr) 2021-08-20 2024-12-31 Joint Stock Company "Biocad" Procédé de production de capside modifié de virus adéno-associé
AR126840A1 (es) 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en esta
WO2023044306A1 (en) * 2021-09-14 2023-03-23 California Institute Of Technology Aav capsid variants
US20230141563A1 (en) 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
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
CA3237037A1 (en) 2021-11-14 2023-05-19 Cartesian Therapeutics, Inc. Multiple dosing with viral vectors
CN116554278A (zh) * 2022-01-30 2023-08-08 上海玮美基因科技有限责任公司 变异型腺相关病毒及其在疾病治疗中的应用
EP4490278A1 (en) 2022-03-07 2025-01-15 Ultragenyx Pharmaceutical Inc. Modified batch aav production systems and methods
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
EP4529614A1 (en) 2022-05-23 2025-04-02 F. Hoffmann-La Roche AG Raman-based method for the differentiation of aav particle serotype and aav particle loading status
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
WO2024036324A1 (en) 2022-08-11 2024-02-15 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
CN119816351A (zh) 2022-09-12 2025-04-11 豪夫迈·罗氏有限公司 用于分离完整的aav颗粒和空aav颗粒的方法
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
KR20250162543A (ko) 2023-03-21 2025-11-18 에프. 호프만-라 로슈 아게 재조합 aav 입자 제제 생산 방법
EP4695406A1 (en) 2023-04-13 2026-02-18 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
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
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
JP4888876B2 (ja) 2003-06-13 2012-02-29 田平 武 アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター
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
BR112012024934A2 (pt) 2010-03-29 2016-12-06 Univ Pennsylvania sistemas de ablação de transgene induzida farmacologicamente
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
IN2014DN08812A (show.php) 2012-04-18 2015-05-22 Philadelphia Children Hospital

Also Published As

Publication number Publication date
EP2839014A1 (en) 2015-02-25
US9909142B2 (en) 2018-03-06
ES2862912T3 (es) 2021-10-08
IN2014DN08812A (show.php) 2015-05-22
PL2839014T3 (pl) 2021-08-23
JP6342886B2 (ja) 2018-06-13
MY172457A (en) 2019-11-26
US11279950B2 (en) 2022-03-22
KR20150004859A (ko) 2015-01-13
RU2683497C2 (ru) 2019-03-28
HUE054087T2 (hu) 2021-09-28
DK2839014T3 (da) 2021-03-08
KR102063483B1 (ko) 2020-01-08
EP2839014B1 (en) 2020-12-16
AU2013249202B2 (en) 2018-08-09
JP2015514427A (ja) 2015-05-21
ZA201407582B (en) 2021-05-26
CO7200251A2 (es) 2015-02-27
NZ701693A (en) 2017-02-24
BR122021020419B1 (pt) 2023-01-17
BR112014025985A2 (pt) 2017-07-11
IL235102A0 (en) 2014-12-31
MX359518B (es) 2018-10-01
CA2870736C (en) 2021-11-02
US20150065562A1 (en) 2015-03-05
RU2014146159A (ru) 2016-06-10
PE20150163A1 (es) 2015-02-23
CN104487579B (zh) 2022-01-18
MX2014012680A (es) 2015-05-11
IL235102B (en) 2019-08-29
PH12014502347B1 (en) 2014-12-22
CA2870736A1 (en) 2013-10-24
EP2839014A4 (en) 2015-12-30
AU2013249202A1 (en) 2014-11-06
PH12014502347A1 (en) 2014-12-22
PT2839014T (pt) 2021-03-19
SG11201406776TA (en) 2015-03-30
US20180245098A1 (en) 2018-08-30
HK1207663A1 (en) 2016-02-05
WO2013158879A1 (en) 2013-10-24
CN104487579A (zh) 2015-04-01

Similar Documents

Publication Publication Date Title
IL235102B (en) Compositions and methods for efficient gene transfer using aav caspid variants
EP2922959A4 (en) TAL-MEDIATED TRANSFER DNA INTRODUCTION
SG10201601110VA (en) Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
DK2922554T3 (en) Terminalt modificeret rna
IL231983B (en) Dna containing tricyclo-phosphorothioate
ZA201409228B (en) Compositions and methods for modulating smn gene family expression
ZA201400563B (en) Axmi270 toxin gene and methods for its use
PL2539472T3 (pl) Szybka PCR do genotypowania STR
IL239480B (en) Improved transfer solution
EP2934572A4 (en) FORMULATIONS AND METHODS FOR STABILIZING PCR REAGENTS
ZA201309516B (en) Protein matrix vaccine compositions including polycations
SG11201404852WA (en) Oligonucleotides for modulating gene expression and uses thereof
ZA201307363B (en) Axmi115 variant insecticidal gene and methods for its use
GB201206455D0 (en) Gene expression
ZA201303410B (en) Viral polymerase inhibitors
GB201214645D0 (en) Algal genome modification
IL235676A0 (en) Preparations and methods for regulating smn family gene expression
IL233445A0 (en) Methods and preparations for gene transfer