MA44119A - Vecteurs viraux adéno-associés à utiliser dans le traitement de l'amyotrophie spinale - Google Patents

Vecteurs viraux adéno-associés à utiliser dans le traitement de l'amyotrophie spinale

Info

Publication number
MA44119A
MA44119A MA044119A MA44119A MA44119A MA 44119 A MA44119 A MA 44119A MA 044119 A MA044119 A MA 044119A MA 44119 A MA44119 A MA 44119A MA 44119 A MA44119 A MA 44119A
Authority
MA
Morocco
Prior art keywords
adeno
treatment
viral vectors
associated viral
spinal amyotrophy
Prior art date
Application number
MA044119A
Other languages
English (en)
Inventor
Christain Hinderer
James M Wilson
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of MA44119A publication Critical patent/MA44119A/fr

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • 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
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
MA044119A 2015-12-14 2016-12-14 Vecteurs viraux adéno-associés à utiliser dans le traitement de l'amyotrophie spinale MA44119A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201562267012P 2015-12-14 2015-12-14

Publications (1)

Publication Number Publication Date
MA44119A true MA44119A (fr) 2018-10-31

Family

ID=57737990

Family Applications (1)

Application Number Title Priority Date Filing Date
MA044119A MA44119A (fr) 2015-12-14 2016-12-14 Vecteurs viraux adéno-associés à utiliser dans le traitement de l'amyotrophie spinale

Country Status (13)

Country Link
US (2) US20180353624A1 (fr)
EP (1) EP3394270A1 (fr)
JP (1) JP7082050B2 (fr)
KR (1) KR20180086266A (fr)
CN (1) CN109072254A (fr)
AU (1) AU2016370630B2 (fr)
BR (1) BR112018011975A2 (fr)
CA (1) CA3008280A1 (fr)
IL (1) IL259877A (fr)
MA (1) MA44119A (fr)
MX (1) MX2018007234A (fr)
WO (1) WO2017106354A1 (fr)
ZA (1) ZA201803956B (fr)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102621134B1 (ko) * 2016-07-26 2024-01-04 코넬 유니버시티 알데히드 데히드로게나제 결핍의 치료를 위한 유전자 요법
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
SI3589730T1 (sl) 2017-02-28 2024-04-30 The Trustees Of The University Of Pennsylvania Adeno-povezan virus (AAV) clade F vektor in njihova uporaba
WO2019040586A1 (fr) * 2017-08-25 2019-02-28 Ovid Therapeutics Inc. Vecteurs adéno-associés recombinants
US20210308281A1 (en) * 2018-08-15 2021-10-07 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
CN113474459A (zh) * 2018-12-21 2021-10-01 吉尼松公司 用于基因疗法载体的表达盒
US20220280548A1 (en) * 2019-08-15 2022-09-08 Biogen Ma Inc. Combination therapy for spinal muscular atrophy
CN112011571A (zh) * 2020-04-26 2020-12-01 辉大(上海)生物科技有限公司 一种用于治疗脊髓性肌萎缩的基因治疗药物
US20230173102A1 (en) * 2020-04-28 2023-06-08 Genethon Use of a Synthetic AAV Capsid for Gene Therapy of Muscle and Central Nervous System Disorders
EP4162059A1 (fr) 2020-05-12 2023-04-12 The Trustees of The University of Pennsylvania Compositions pour la réduction spécifique à des drg d'expression transgénique
RU2742837C1 (ru) * 2020-06-02 2021-02-11 Общество С Ограниченной Ответственностью "Анабион" Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение
CN113755524B (zh) * 2020-06-02 2023-11-03 舒泰神（北京）生物制药股份有限公司 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途
TW202246501A (zh) * 2021-01-29 2022-12-01 俄羅斯聯邦商亞那拜恩有限公司 ＳＭＮ１和ｍｉＲ－２３ａ在治療脊髓性肌萎縮中的協同作用
EP4284335A1 (fr) 2021-02-01 2023-12-06 RegenxBio Inc. Thérapie génique de céroïdes-lipofuscinoses neuronales
CN112852882A (zh) * 2021-02-04 2021-05-28 中吉智药(南京)生物技术有限公司 一种杆状病毒感染昆虫细胞生产aav基因药物的系统及方法
KR20240101620A9 (ko) * 2021-11-09 2025-12-10 아시모브 인코포레이티드 Aav 벡터 생산을 위한 안정한 생산 시스템
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023219394A1 (fr) * 2022-05-10 2023-11-16 서울대학교산학협력단 Variant de protéine smn1 humaine et son utilisation
WO2024165839A1 (fr) * 2023-02-06 2024-08-15 Royal Holloway And Bedford New College Transgènes

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3211085T3 (da) * 2003-09-30 2021-06-21 Univ Pennsylvania Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf
WO2013190059A1 (fr) * 2012-06-21 2013-12-27 Association Institut De Myologie Délivrance généralisée dans un gène de vecteurs de traitement génique
US10821154B2 (en) * 2013-05-01 2020-11-03 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
CN105658800A (zh) 2013-10-22 2016-06-08 夏尔人类遗传性治疗公司 Mrna的cns递送及其用途
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
CA2994160C (fr) * 2015-07-30 2021-08-10 Massachusetts Eye And Ear Infirmary Sequences virales ancestrales et leurs utilisations

Also Published As

Publication number Publication date
KR20180086266A (ko) 2018-07-30
JP2018537984A (ja) 2018-12-27
US20220265861A1 (en) 2022-08-25
AU2016370630A1 (en) 2018-06-28
US20180353624A1 (en) 2018-12-13
JP7082050B2 (ja) 2022-06-07
ZA201803956B (en) 2019-04-24
WO2017106354A1 (fr) 2017-06-22
MX2018007234A (es) 2018-11-09
AU2016370630B2 (en) 2023-04-13
CA3008280A1 (fr) 2017-06-22
EP3394270A1 (fr) 2018-10-31
CN109072254A (zh) 2018-12-21
BR112018011975A2 (pt) 2018-12-11
IL259877A (en) 2018-07-31

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