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MX2018007234A - Vectores virales adenoasociados utiles en el tratamiento de atrofia muscular espinal. - Google Patents

Vectores virales adenoasociados utiles en el tratamiento de atrofia muscular espinal.

Info

Publication number
MX2018007234A
MX2018007234A MX2018007234A MX2018007234A MX2018007234A MX 2018007234 A MX2018007234 A MX 2018007234A MX 2018007234 A MX2018007234 A MX 2018007234A MX 2018007234 A MX2018007234 A MX 2018007234A MX 2018007234 A MX2018007234 A MX 2018007234A
Authority
MX
Mexico
Prior art keywords
spinal muscular
associated viral
adeno
treatment
viral vectors
Prior art date
Application number
MX2018007234A
Other languages
English (en)
Inventor
James M Wilson
Hinderer Christain
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 MX2018007234A publication Critical patent/MX2018007234A/es

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
    • 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
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (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)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (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)

Abstract

Se proporcionan composiciones y métodos útiles en el tratamiento de atrofia muscular espinal; las composiciones comprenden un vector viral combinante adenoasociado que contiene una cápside AAV, por ejemplo, cápside AAVrh.10 y secuencias de ácidos nucleicos que codifican una proteína SMN funcional; los métodos involucran administrar estas composiciones a seres humanos que lo necesiten.
MX2018007234A 2015-12-14 2016-12-14 Vectores virales adenoasociados utiles en el tratamiento de atrofia muscular espinal. MX2018007234A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562267012P 2015-12-14 2015-12-14
PCT/US2016/066669 WO2017106354A1 (en) 2015-12-14 2016-12-14 Adeno-associated viral vectors useful in treatment of spinal muscular atropy

Publications (1)

Publication Number Publication Date
MX2018007234A true MX2018007234A (es) 2018-11-09

Family

ID=57737990

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018007234A MX2018007234A (es) 2015-12-14 2016-12-14 Vectores virales adenoasociados utiles en el tratamiento de atrofia muscular espinal.

Country Status (13)

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

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG10202100632WA (en) * 2016-07-26 2021-03-30 Univ Cornell Gene therapy for the treatment of aldehyde dehydrogenase deficiency
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
AU2018227440B2 (en) 2017-02-28 2024-06-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clade f vector and uses therefor
KR20200042935A (ko) * 2017-08-25 2020-04-24 오비드 테라퓨틱스 인크. 재조합 아데노-관련된 벡터들
EP3837374A4 (en) * 2018-08-15 2022-06-08 Biogen MA Inc. COMBINATION THERAPY FOR SPINAL MUSCLE ATROPHY
CA3122319A1 (en) * 2018-12-21 2020-06-25 Genethon Expression cassettes for gene therapy vectors
JP7689110B2 (ja) * 2019-08-15 2025-06-05 バイオジェン・エムエイ・インコーポレイテッド 脊髄性筋萎縮症のための併用療法
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
WO2021219762A1 (en) * 2020-04-28 2021-11-04 Genethon Use of a synthetic aav capsid for gene therapy of muscle and central nervous system disorders
BR112022022212A2 (pt) 2020-05-12 2022-12-13 Univ Pennsylvania Composições para redução específica de drg de expressão de transgene
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
BR112023015177A2 (pt) * 2021-01-29 2023-11-14 Biocad Joint Stock Co Ácido nucleico compreendendo smn1 e mir-23a para tratamento da atrofia muscular espinhal, cassete de expressão, vetor de expressão, vírus recombinante, composição farmacêutica, uso e método relacionados
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
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
WO2023086822A2 (en) * 2021-11-09 2023-05-19 Asimov Inc. Stable production systems for aav vector production
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
KR102717399B1 (ko) * 2022-05-10 2024-10-16 서울대학교산학협력단 인간 smn1 단백질 변이체 및 이의 용도
WO2024165839A1 (en) * 2023-02-06 2024-08-15 Royal Holloway And Bedford New College Transgenes

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2292779T3 (en) * 2003-09-30 2017-02-27 Univ Pennsylvania ADENOASS-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME AND APPLICATIONS THEREOF
WO2013190059A1 (en) * 2012-06-21 2013-12-27 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
CA2911105A1 (en) * 2013-05-01 2014-11-06 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
EP4036241A1 (en) 2013-10-22 2022-08-03 Translate Bio, Inc. Cns delivery of mrna and uses thereof
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
FI3872085T3 (fi) * 2015-07-30 2023-04-19 Massachusetts Eye & Ear Infirmary Esimuotoiset virussekvenssit ja niiden käytöt

Also Published As

Publication number Publication date
MA44119A (fr) 2018-10-31
AU2016370630B2 (en) 2023-04-13
WO2017106354A1 (en) 2017-06-22
BR112018011975A2 (pt) 2018-12-11
IL259877A (en) 2018-07-31
AU2016370630A1 (en) 2018-06-28
US20220265861A1 (en) 2022-08-25
JP7082050B2 (ja) 2022-06-07
CN109072254A (zh) 2018-12-21
JP2018537984A (ja) 2018-12-27
ZA201803956B (en) 2019-04-24
KR20180086266A (ko) 2018-07-30
EP3394270A1 (en) 2018-10-31
US20180353624A1 (en) 2018-12-13
CA3008280A1 (en) 2017-06-22

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