MA46863A - Administration intrathécale de virus adéno-associé recombinant codant pour la protéine 2 de liaison méthyl-cpg - Google Patents

Administration intrathécale de virus adéno-associé recombinant codant pour la protéine 2 de liaison méthyl-cpg

Info

Publication number
MA46863A
MA46863A MA046863A MA46863A MA46863A MA 46863 A MA46863 A MA 46863A MA 046863 A MA046863 A MA 046863A MA 46863 A MA46863 A MA 46863A MA 46863 A MA46863 A MA 46863A
Authority
MA
Morocco
Prior art keywords
cpg
methyl
binding protein
associated virus
recombinant adeno
Prior art date
Application number
MA046863A
Other languages
English (en)
Inventor
Kevin Foust
Brian K Kaspar
Original Assignee
Kevin Foust
Nationwide Childrens Hospital 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
Application filed by Kevin Foust, Nationwide Childrens Hospital Inc filed Critical Kevin Foust
Publication of MA46863A publication Critical patent/MA46863A/fr

Links

Classifications

    • 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
    • A61K48/005—Medicinal 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • 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
    • A61K48/0075—Medicinal 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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • 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)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Neurosurgery (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • General Engineering & Computer Science (AREA)
  • Dermatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Mycology (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)
MA046863A 2016-11-17 2017-11-17 Administration intrathécale de virus adéno-associé recombinant codant pour la protéine 2 de liaison méthyl-cpg MA46863A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201662423618P 2016-11-17 2016-11-17

Publications (1)

Publication Number Publication Date
MA46863A true MA46863A (fr) 2019-09-25

Family

ID=62145776

Family Applications (1)

Application Number Title Priority Date Filing Date
MA046863A MA46863A (fr) 2016-11-17 2017-11-17 Administration intrathécale de virus adéno-associé recombinant codant pour la protéine 2 de liaison méthyl-cpg

Country Status (15)

Country Link
US (2) US11583564B2 (fr)
EP (1) EP3541429A4 (fr)
JP (3) JP7106534B2 (fr)
KR (1) KR102641846B1 (fr)
CN (1) CN110325219B (fr)
AU (2) AU2017362491B2 (fr)
BR (1) BR112019009834B1 (fr)
CA (1) CA3044291A1 (fr)
CL (1) CL2019001354A1 (fr)
IL (1) IL266678A (fr)
MA (1) MA46863A (fr)
MX (1) MX2019005874A (fr)
SA (1) SA519401806B1 (fr)
SG (1) SG10202105090WA (fr)
WO (1) WO2018094251A1 (fr)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2017362491B2 (en) * 2016-11-17 2023-02-02 Nationwide Children's Hospital Inc. Intrathecal delivery of recombinant Adeno-associated virus encoding Methyl-CpG binding protein 2
JP2022519596A (ja) * 2019-02-04 2022-03-24 リサーチ・インスティチュート・アット・ネーションワイド・チルドレンズ・ホスピタル Ｃｌｎ３ポリヌクレオチドのアデノ随伴ウイルス送達
CA3158131A1 (fr) * 2019-10-18 2021-04-22 Nicolas Sebastien Wein Materiaux et procedes pour le traitement de troubles associes au gene irf2bpl
WO2022221424A1 (fr) 2021-04-13 2022-10-20 Research Institute At Nationwide Children's Hospital Virus adéno-associé recombinant codant pour la protéine 2 de liaison à la méthyl-cpg pour traiter le syndrome de pitt hopkins par administration intrathécale

Family Cites Families (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
DK0733103T3 (da) 1993-11-09 2004-07-12 Univ Johns Hopkins Fremstilling af höje titre af rekombinante AAV vektorer
EP0728214B1 (fr) 1993-11-09 2004-07-28 Medical College Of Ohio Lignees cellulaires stables aptes a exprimer le gene de replication du virus adeno-associe
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
RU2273645C9 (ru) 1994-08-17 2006-11-27 Дзе Рокефеллер Юниверсити Полипептид ожирения (ов)(варианты), его аналог (варианты) и слитый белок (варианты), изолированная молекула нуклеиновой кислоты, молекула днк, рекомбинантный вектор клонирования, рекомбинантный вектор экспрессии, фармацевтическая композиция, моноклональное и поликлональное антитело
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
JPH10511264A (ja) 1994-12-06 1998-11-04 ターゲティッド ジェネティックス コーポレイション 高力価組換えａａｖベクターの生成のためのパッケージング細胞株
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
CA2625279A1 (fr) 1995-08-30 1997-03-06 Genzyme Corporation Purification d'adenovirus et de virus adeno-associe (aav) par voie chromatographique
CA2230758A1 (fr) 1995-09-08 1997-03-13 Genzyme Corporation Vecteurs aav ameliores pour la therapie genique
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
AU723497C (en) 1996-09-06 2001-10-11 Trustees Of The University Of Pennsylvania, The Method for recombinant adeno-associated virus-directed gene therapy
CA2830694C (fr) 1997-09-05 2018-02-27 Genzyme Corporation Procedes de generation de preparations de vecteurs de aav recombinants dont le titre est eleve et qui sont exemptes de virus assistant
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
GB9916702D0 (en) 1999-07-16 1999-09-15 Cantab Pharma Res Virus vectors and preparations and their use
JP2004514407A (ja) 2000-04-28 2004-05-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
EP3339430A1 (fr) 2001-12-17 2018-06-27 The Trustees of The University of Pennsylvania Séquences de sérotype 9 de virus adéno associé, vecteurs les contenant et leurs utilisations
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
CA2554380C (fr) * 2004-02-17 2015-05-19 The Hospital For Sick Children Gene mecp2e1
EP3632472A1 (fr) * 2006-10-03 2020-04-08 Genzyme Corporation Thérapie génique pour la sclérose latérale amyotrophique et autres troubles de la moelle épinière
JP5635912B2 (ja) 2008-01-15 2014-12-03 アッヴィ・インコーポレイテッド 改善された哺乳動物発現ベクター及びその使用
US20090246768A1 (en) * 2008-02-15 2009-10-01 Sawalha Amr H Predicting and Diagnosing Patients With Autoimmune Disease
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
US9415121B2 (en) * 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
WO2015141521A1 (fr) 2014-03-21 2015-09-24 株式会社日立国際電気 Appareil de traitement de substrat, procédé de fabrication de dispositif semi-conducteur et support d'enregistrement
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
CA2967468A1 (fr) * 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Therapie genique pour la maladie de steinert juvenile
AU2017362491B2 (en) * 2016-11-17 2023-02-02 Nationwide Children's Hospital Inc. Intrathecal delivery of recombinant Adeno-associated virus encoding Methyl-CpG binding protein 2
EA201992882A1 (ru) * 2017-06-06 2020-05-25 Юниверсити Оф Массачусеттс САМОРЕГУЛИРУЕМЫЕ ВЕКТОРЫ НА ОСНОВЕ AAV ДЛЯ БЕЗОПАСНОЙ ЭКСПРЕССИИ MeCP2 ПРИ СИНДРОМЕ PETTA

Also Published As

Publication number Publication date
US11583564B2 (en) 2023-02-21
JP2024045289A (ja) 2024-04-02
US20200179467A1 (en) 2020-06-11
EP3541429A1 (fr) 2019-09-25
EP3541429A4 (fr) 2020-08-12
CL2019001354A1 (es) 2019-09-13
AU2017362491A1 (en) 2019-06-06
AU2017362491B2 (en) 2023-02-02
CA3044291A1 (fr) 2018-05-24
US20230321164A1 (en) 2023-10-12
WO2018094251A1 (fr) 2018-05-24
JP7106534B2 (ja) 2022-07-26
JP2020503265A (ja) 2020-01-30
SA519401806B1 (ar) 2023-03-08
BR112019009834B1 (pt) 2022-05-17
RU2019118300A3 (fr) 2021-01-11
CN110325219A (zh) 2019-10-11
CN110325219B (zh) 2024-03-26
SG10202105090WA (en) 2021-06-29
AU2023202598A1 (en) 2023-07-20
KR102641846B1 (ko) 2024-02-27
BR112019009834A2 (pt) 2019-10-08
MX2019005874A (es) 2020-02-07
IL266678A (en) 2019-07-31
RU2019118300A (ru) 2020-12-17
JP2022137210A (ja) 2022-09-21
KR20190126047A (ko) 2019-11-08

Similar Documents

Publication Publication Date Title
CL2020002043A1 (es) Métodos y composiciones para la entrega de proteínas terapeuticas.
MA49270A (fr) Récepteurs de liaison à l'antigène améliorés
MA46863A (fr) Administration intrathécale de virus adéno-associé recombinant codant pour la protéine 2 de liaison méthyl-cpg
MA52245A (fr) Promédicaments d'antagonistes de c5ar bicycliques fusionnés
MA47386A (fr) Protéines de liaison icos
MA45481A (fr) Utilisation d'exosomes pour le traitement de maladies
DK3559022T3 (da) Virulenssvækket bakteriebaseret proteinlevering
EA201591661A1 (ru) Пептиды и композиции для лечения повреждений суставов
BR112017008416A2 (pt) ?produtos terapêuticos à base de lantionina sintetase c-like 2?.
DK3197472T3 (da) Rekombinante phe-frie proteiner til anvendelse i behandlingen af phenylketonuri
MA43987A (fr) Détermination de la pureté de protéine multimère
IL269853A (en) Proteins for the treatment of epithelial barrier activity disorders
MA44074A (fr) Immunoglobulines conjuguées à la lysine c-terminale
MA43049A (fr) Anticorps de liaison à un récepteur cannabinoïde 1 humain (cb1)
EA201790336A1 (ru) Антитела к ангиопоэтинподобному белку 4 и способы применения
MA42497A (fr) Procédés et compositions pour la régulation sélective de l'expression protéique
ES2970116T3 (es) Composición farmacéutica para tratar la sarcopenia agonista del receptor del péptido de tipo 1 similar al glucagón
MA53551A (fr) Dosage nocturne chronique de lasmiditan pour la prévention de la migraine
MA42828A (fr) Plate-forme d'échafaudage à noeud de cystine
MA45227A (fr) Peptides pour le traitement de l'ostéoarthrite
EP3526321A4 (fr) Utilisation d'agents tonifiants pour augmenter le rendement de virus adéno-associés recombinés
MA45159A (fr) Polypeptides d'hémagglutinine d'influenza modifiés
SG11201703624PA (en) Compounds for the diagnosis or treatment of disorders associated with protein or peptide oligomers
MA52148A (fr) Peptides chimères pour administration d'antisens
MA51688A (fr) Protéines prohémostatiques pour le traitement de saignement