HUE060720T2 - Készítmények és eljárások génterápiás vektorok transzdukciójának növelésére vagy fokozására és immunoglobulinok eltávolítására vagy csökkentésére - Google Patents

Készítmények és eljárások génterápiás vektorok transzdukciójának növelésére vagy fokozására és immunoglobulinok eltávolítására vagy csökkentésére

Info

Publication number
HUE060720T2
HUE060720T2 HUE19740384A HUE19740384A HUE060720T2 HU E060720 T2 HUE060720 T2 HU E060720T2 HU E19740384 A HUE19740384 A HU E19740384A HU E19740384 A HUE19740384 A HU E19740384A HU E060720 T2 HUE060720 T2 HU E060720T2
Authority
HU
Hungary
Prior art keywords
immunoglobulins
compositions
increasing
reducing
methods
Prior art date
Application number
HUE19740384A
Other languages
English (en)
Hungarian (hu)
Inventor
Sebastien Lacroix-Desmazes
Federico Mingozzi
Jordan Dimitrov
Christian Leborgne
Sean Armour
Original Assignee
Inst Nat Sante Rech Med
Genethon
Univ Sorbonne
Univ Paris Cite
Spark Therapeutics Inc
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=67303476&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HUE060720(T2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Inst Nat Sante Rech Med, Genethon, Univ Sorbonne, Univ Paris Cite, Spark Therapeutics Inc filed Critical Inst Nat Sante Rech Med
Publication of HUE060720T2 publication Critical patent/HUE060720T2/hu

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4873Cysteine endopeptidases (3.4.22), e.g. stem bromelain, papain, ficin, cathepsin H
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/06Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies from serum
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/861Adenoviral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/52Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from bacteria or Archaea
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/58Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from fungi
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6472Cysteine endopeptidases (3.4.22)
    • C12N9/6475Interleukin 1-beta convertase-like enzymes (3.4.22.10; 3.4.22.36; 3.4.22.63)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/22Cysteine endopeptidases (3.4.22)
    • C12Y304/22038Cathepsin K (3.4.22.38)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53Immunoassay; Biospecific binding assay; Materials therefor
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/22Cysteine endopeptidases (3.4.22)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/22Cysteine endopeptidases (3.4.22)
    • C12Y304/2201Streptopain (3.4.22.10)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6854Immunoglobulins

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Urology & Nephrology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Food Science & Technology (AREA)
  • Analytical Chemistry (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Mycology (AREA)
HUE19740384A 2018-07-17 2019-07-17 Készítmények és eljárások génterápiás vektorok transzdukciójának növelésére vagy fokozására és immunoglobulinok eltávolítására vagy csökkentésére HUE060720T2 (hu)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP18305971 2018-07-17
US201862768731P 2018-11-16 2018-11-16

Publications (1)

Publication Number Publication Date
HUE060720T2 true HUE060720T2 (hu) 2023-04-28

Family

ID=67303476

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE19740384A HUE060720T2 (hu) 2018-07-17 2019-07-17 Készítmények és eljárások génterápiás vektorok transzdukciójának növelésére vagy fokozására és immunoglobulinok eltávolítására vagy csökkentésére

Country Status (28)

Country Link
US (3) US20210228738A1 (https=)
EP (2) EP4154904A1 (https=)
JP (2) JP2021529821A (https=)
KR (1) KR20210042308A (https=)
CN (2) CN112689515A (https=)
AU (2) AU2019306121B2 (https=)
BR (1) BR112021000807A8 (https=)
CA (1) CA3106368A1 (https=)
CL (1) CL2021000112A1 (https=)
CO (1) CO2021000326A2 (https=)
DK (1) DK3768304T3 (https=)
ES (1) ES2935970T3 (https=)
FI (1) FI3768304T3 (https=)
HR (1) HRP20221542T1 (https=)
HU (1) HUE060720T2 (https=)
IL (1) IL280099A (https=)
LT (1) LT3768304T (https=)
MX (1) MX2021000580A (https=)
PE (1) PE20211859A1 (https=)
PH (1) PH12021550115A1 (https=)
PL (1) PL3768304T3 (https=)
RS (1) RS63878B1 (https=)
SG (1) SG11202100306VA (https=)
SI (1) SI3768304T1 (https=)
SM (1) SMT202300005T1 (https=)
UA (1) UA130604C2 (https=)
WO (1) WO2020016318A1 (https=)
ZA (1) ZA202100202B (https=)

Families Citing this family (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4219547A3 (en) 2008-09-15 2023-10-18 uniQure biopharma B.V. Factor ix polypeptide mutant, its uses and a method for its production
GB201502305D0 (en) * 2015-02-12 2015-04-01 Hansa Medical Ab Protein
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
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
US20210363191A1 (en) 2018-04-03 2021-11-25 Stridebio, Inc. Antibody-evading virus vectors
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
US20200165608A1 (en) * 2018-11-23 2020-05-28 Florida State University Research Foundation, Inc. Inhibition of vascular endothelial cell-mediated phagocytic processes for treatment of demyelinating conditions
AU2020215682B2 (en) * 2019-01-28 2025-12-18 Duke University Compositions and methods for evading humoral immunity
CN114732822B (zh) * 2019-03-13 2023-06-30 中国人民解放军军事科学院军事医学研究院 氨基葡萄糖及其衍生物作为抗病毒药物的应用
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
AR118928A1 (es) * 2019-05-14 2021-11-10 Biomarin Pharm Inc Métodos de redosificación de vectores de terapia génica
US12544437B2 (en) 2019-10-04 2026-02-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant AAV
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
US11180540B2 (en) 2019-12-06 2021-11-23 Regeneron Pharmaceuticals, Inc. Anti-VEGF protein compositions and methods for producing the same
KR20220133969A (ko) 2020-01-28 2022-10-05 프리라인 테라퓨틱스 리미티드 바이러스 벡터에 대한 중화 항체 역가를 결정하기 위한 개선된 분석법
MX2022009982A (es) 2020-02-14 2022-09-12 Ultragenyx Pharmaceutical Inc Terapia genica para el tratamiento de transtorno por deficiencia de cdkl5.
JP2023525034A (ja) 2020-05-08 2023-06-14 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Vegfトラップおよびミニトラップならびに眼障害およびがんの治療方法
CN111621506B (zh) * 2020-05-09 2021-07-30 华中农业大学 牛支原体分泌蛋白MbovP0145及其应用
GB202007431D0 (en) * 2020-05-19 2020-07-01 Hansa Biopharma AB Cysteine protease
EP4162951A4 (en) * 2020-06-05 2024-07-10 Shanghai Bao Pharmaceuticals Co., Ltd. PHARMACEUTICAL COMPOSITION OF ENZYMES AND VIRUSES AND ASSOCIATED APPLICATION
JP2023532219A (ja) * 2020-06-18 2023-07-27 上海宝済薬業有限公司 免疫グロブリン分解酵素IdeEの突然変異体
RU2020122141A (ru) * 2020-07-03 2022-01-04 Максим Петрович Никитин Способ и композиция на основе генотерапевтического агента и антитела, связывающегося с клетками крови
WO2022026632A2 (en) * 2020-07-29 2022-02-03 University Of Florida Research Foundation, Incorporated Improved aav-mediated x-linked retinoschisis therapies
EP4200408A1 (en) 2020-08-19 2023-06-28 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of rett syndrome
AU2021350732A1 (en) * 2020-09-28 2023-05-11 Scout Bio, Inc. Composition and uses thereof
AU2021358957A1 (en) 2020-10-07 2023-06-08 AskBio Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21
EP4304662A4 (en) * 2021-03-08 2025-06-18 The Regents of the University of California Lentivirus protection via fc overexpression
WO2022192193A1 (en) * 2021-03-09 2022-09-15 The Board Of Regents Of The University Of Texas System Methods and compositions for mutagenesis screening in mammalian cells
JP2024515626A (ja) 2021-04-16 2024-04-10 アスクレピオス バイオファーマシューティカル, インコーポレイテッド 血液脳関門を横断し低減された液性応答を惹起する合理的ポリプロイドaavビリオン
WO2023280175A1 (en) * 2021-07-06 2023-01-12 National Institute Of Biological Sciences, Beijing Methods for treating complex i deficiencies or cancers by modulating gro3p biosynthesis
WO2023019168A1 (en) 2021-08-11 2023-02-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
EP4349365A1 (en) * 2022-10-07 2024-04-10 Hansa Biopharma AB Co-treatment for gene therapy
CN115558021B (zh) * 2022-12-01 2023-03-17 山东大学 一种转录因子、重组细胞及其在制备肿瘤治疗药物的应用
CN116425873B (zh) * 2023-06-02 2023-09-05 苏州百道医疗科技有限公司 一种抗ck6重组兔单克隆抗体及其应用
CN117126270B (zh) * 2023-10-25 2024-02-13 首都儿科研究所 一种2型人博卡病毒型别特异性抗体及其应用
CN118217443B (zh) * 2024-01-15 2026-04-03 国科温州研究院(温州生物材料与工程研究所) 一种人发角蛋白止血海绵及其制备方法与应用
WO2025155923A1 (en) 2024-01-17 2025-07-24 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with the transferrin receptor
WO2025217174A1 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with carbonic anhydrase iv
WO2025217163A2 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Novel aav capsids binding to human cd59
WO2025256977A1 (en) 2024-06-13 2025-12-18 Bayer Aktiengesellschaft Novel igm and igg cleaving enzymes
WO2025256978A1 (en) 2024-06-13 2025-12-18 Bayer Aktiengesellschaft Variants of igm and igg cleaving enzymes
WO2026041758A1 (en) * 2024-08-22 2026-02-26 Genethon Modified aav capsid with enhanced transduction efficiency
GB202414654D0 (en) 2024-10-04 2024-11-20 Hansa Biopharma AB Regimen for vector-based therapies

Family Cites Families (52)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US414A (en) 1837-09-28 Moetise-latch foe
US941A (en) 1838-09-22 Machine for sawing shingles and staves
US5173A (en) 1847-06-26 Machinery for
US5139A (en) 1847-06-05 Cajsfkles
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
WO1990005183A1 (en) 1988-10-31 1990-05-17 Immunex Corporation Interleukin-4 receptors
AU651596B2 (en) 1990-06-05 1994-07-28 Immunex Corporation Type II interleukin-1 receptors
WO1992001070A1 (en) 1990-07-09 1992-01-23 The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce High efficiency packaging of mutant adeno-associated virus using amber suppressions
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
JP3534749B2 (ja) 1991-08-20 2004-06-07 アメリカ合衆国 アデノウイルスが介在する胃腸管への遺伝子の輸送
AU7676894A (en) 1993-08-27 1995-03-21 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Convection-enhanced drug delivery
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US6074849A (en) 1995-07-19 2000-06-13 Genetics Institute, Inc. Polynucleotides encoding human CTLA-8 related proteins
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6569645B2 (en) 1999-05-14 2003-05-27 Genentech, Inc. IL-17 homologous polypeptides and therapeutic uses thereof
US6579520B2 (en) 1998-05-15 2003-06-17 Genentech, Inc. IL-17 related mammalian cytokine polypeptides (IL-17E)
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
NZ592917A (en) 2003-09-15 2012-12-21 Protiva Biotherapeutics Inc Stable polyethyleneglycol (PEG) dialkyloxypropyl (DAA) lipid conjugates
US7745651B2 (en) 2004-06-07 2010-06-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
JP5427409B2 (ja) * 2005-06-09 2014-02-26 ハンサ メディカル アクチボラゲット 自己免疫疾患及び移植片拒絶反応を治療するためのIdeSプロテイナーゼ(化膿連鎖球菌由来)の使用
EP3192788A1 (en) 2006-10-03 2017-07-19 Arbutus Biopharma Corporation Lipid containing formulations
US7649243B2 (en) 2006-11-06 2010-01-19 International Business Machines Corporation Semiconductor structures incorporating multiple crystallographic planes and methods for fabrication thereof
GB0624874D0 (en) 2006-12-13 2007-01-24 Hansa Medical Ab Treatment
EP2219587A4 (en) 2007-11-14 2012-11-21 Univ California STEROL-MODIFIED AMPHIPHILE LIPIDES
CN104119242B (zh) 2008-10-09 2017-07-07 泰米拉制药公司 改善的氨基脂质和递送核酸的方法
HUE037082T2 (hu) 2008-11-10 2018-08-28 Arbutus Biopharma Corp Új lipidek és készítmények terápiás hatóanyagok szállítására
WO2010054384A1 (en) 2008-11-10 2010-05-14 Alnylam Pharmaceuticals, Inc. Lipids and compositions for the delivery of therapeutics
US9018187B2 (en) 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
WO2011141705A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
GB201103780D0 (en) 2011-03-04 2011-04-20 Hansa Medical Ab Treatment for ige-mediated disease
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
US9126966B2 (en) 2011-08-31 2015-09-08 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use thereof
GB201115841D0 (en) 2011-09-13 2011-10-26 Genovis Ab Protein and method
GB201201314D0 (en) * 2012-01-26 2012-03-07 Isis Innovation Composition
SG11201405157PA (en) 2012-02-24 2014-10-30 Protiva Biotherapeutics Inc Trialkyl cationic lipids and methods of use thereof
AU2013249202B2 (en) 2012-04-18 2018-08-09 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using AAV capsid variants
TWI775096B (zh) 2012-05-15 2022-08-21 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
EP3605090A1 (en) 2012-05-23 2020-02-05 F. Hoffmann-La Roche AG Selection method for therapeutic agents
HK1222673A1 (zh) 2013-07-12 2017-07-07 The Children's Hospital Of Philadelphia Aav载体和用於抗aav(腺相关病毒)中和抗体的检测
KR20240090694A (ko) 2013-07-22 2024-06-21 더 칠드런스 호스피탈 오브 필라델피아 변종 aav 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
GB201413240D0 (en) 2014-07-25 2014-09-10 Hansa Medical Ab Method
GB201502305D0 (en) * 2015-02-12 2015-04-01 Hansa Medical Ab Protein
GB201502306D0 (en) 2015-02-12 2015-04-01 Hansa Medical Ab Protein
AU2017207736A1 (en) * 2016-01-11 2018-07-12 Rubius Therapeutics, Inc. Compositions and methods related to multimodal therapeutic cell systems for immune indications
ES2877754T3 (es) 2016-02-04 2021-11-17 Genovis Ab Nuevas proteasas estreptocócicas
WO2018093868A1 (en) 2016-11-16 2018-05-24 University Of Florida Research Foundation, Inc. Immunoglobulin proteases, compositions, and uses thereof
WO2018109207A1 (en) 2016-12-16 2018-06-21 Uniqure Ip B.V. Immunoadsorption
WO2018158397A1 (en) 2017-03-02 2018-09-07 Genethon Method for removing anti-aav antibodies from a blood-derived composition
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

Also Published As

Publication number Publication date
BR112021000807A8 (pt) 2022-12-06
US20210246469A1 (en) 2021-08-12
PE20211859A1 (es) 2021-09-21
HRP20221542T1 (hr) 2023-02-17
PL3768304T3 (pl) 2023-03-13
DK3768304T3 (da) 2023-01-09
EP3768304B1 (en) 2022-10-12
JP2021529821A (ja) 2021-11-04
FI3768304T3 (fi) 2023-01-13
UA130604C2 (uk) 2026-04-01
US20230142731A1 (en) 2023-05-11
CN119345338A (zh) 2025-01-24
MX2021000580A (es) 2021-07-15
ES2935970T3 (es) 2023-03-13
BR112021000807A2 (pt) 2021-05-11
AU2019306121B2 (en) 2025-08-28
ZA202100202B (en) 2022-01-26
IL280099A (en) 2021-03-01
SMT202300005T1 (it) 2023-03-17
RS63878B1 (sr) 2023-02-28
WO2020016318A1 (en) 2020-01-23
CN112689515A (zh) 2021-04-20
EP3768304A1 (en) 2021-01-27
SI3768304T1 (sl) 2023-02-28
CL2021000112A1 (es) 2021-09-03
CO2021000326A2 (es) 2021-04-19
EP4154904A1 (en) 2023-03-29
LT3768304T (lt) 2023-03-27
PH12021550115A1 (en) 2021-10-04
SG11202100306VA (en) 2021-02-25
CA3106368A1 (en) 2020-01-23
JP2024075765A (ja) 2024-06-04
US20210228738A1 (en) 2021-07-29
AU2025217293A1 (en) 2025-09-04
AU2019306121A1 (en) 2021-01-28
KR20210042308A (ko) 2021-04-19

Similar Documents

Publication Publication Date Title
HUE060720T2 (hu) Készítmények és eljárások génterápiás vektorok transzdukciójának növelésére vagy fokozására és immunoglobulinok eltávolítására vagy csökkentésére
IL291933A (en) Oligonucleotide compositions and methods of use thereof
IL281277A (en) Preparations and methods for treating viral infections
IL289970A (en) Compositions and methods for treating autoimmune disorders
EP3955891C0 (en) ADENO-ASSOCIATED VIRUS VECTOR FORMULATIONS AND METHODS
HUE058755T2 (hu) Anti-tigit antigénkötõ proteinek és felhasználásuk módszerei
EP3294165A4 (en) METHOD FOR IMPLANTING A BONE SCREW
HUE055458T2 (hu) Kompozíciók és módszerek 5'-Cap RNS-ek szintézisére
IL290792B1 (en) Preparations and methods for treating viral infections
EP3442561A4 (en) ADENO ASSOCIATED VIRUS VECTOR ADMINISTRATION OF MICRORNA-29 AND MICRO-DYSTROPHIN FOR TREATING MUSCLE DYSTROPHY
EP3600434A4 (en) TREATMENT OF PULMONAL HYPERTENSION
SG11202103033WA (en) Compositions and methods for the treatment of presbyopia
EP3864159A4 (en) Compositions and methods for preparing viral vectors
MA47611A (fr) Compositionset méthodes de transduction tumorale
IL291192A (en) Compositions and methods of treating lupus nephritis
EP3506930A4 (en) METHODS AND VECTORS FOR THE TREATMENT OF CNS DISORDERS
IT201700058843A1 (it) Ventola per forni e metodo di realizzazione di detta ventola
PL3886799T3 (pl) Kompozycje do leczenia utraty włosów
PL3573620T3 (pl) Kompozycje do leczenia nadciśnienia
DK3810128T3 (da) Sammensætninger til behandling og/eller forebyggelse af proteinaggregeringssygdomme
DK3710590T3 (da) Virale vektorer omfattende rdh12-kodende områder og fremgangsmåder til behandling af nethindedystrofier
DK3618883T3 (da) Smøredygtige hydrofile belægninger og fremgangsmåder til dannelse heraf
ZA201908276B (en) Charge-tagged nucleotides and methods of use thereof
HUE051948T2 (hu) Protokollok és eljárások sclerosis multiplex kezelésére ofatumumab alkalmazásával
IT201700104446A1 (it) Compositions, uses and methods for treatment of infertility and subfertility