ES3035262T3 - A nucleic acid comprising ids gene sequence and a tandem repeat of apolipoprotein e gene sequence - Google Patents

A nucleic acid comprising ids gene sequence and a tandem repeat of apolipoprotein e gene sequence

Info

Publication number
ES3035262T3
ES3035262T3 ES21187825T ES21187825T ES3035262T3 ES 3035262 T3 ES3035262 T3 ES 3035262T3 ES 21187825 T ES21187825 T ES 21187825T ES 21187825 T ES21187825 T ES 21187825T ES 3035262 T3 ES3035262 T3 ES 3035262T3
Authority
ES
Spain
Prior art keywords
ids
sequence
nucleic acid
apoell
mps
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
ES21187825T
Other languages
English (en)
Spanish (es)
Inventor
Brian Bigger
Hélène Gleitz
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Manchester
Original Assignee
University of Manchester
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 University of Manchester filed Critical University of Manchester
Application granted granted Critical
Publication of ES3035262T3 publication Critical patent/ES3035262T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine 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
    • C07K14/775—Apolipopeptides
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • C12N5/12—Fused cells, e.g. hybridomas
    • C12N5/16—Animal cells
    • 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
    • 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
    • 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
    • C12N2510/00—Genetically modified cells

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Developmental Biology & Embryology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
ES21187825T 2017-02-07 2018-02-07 A nucleic acid comprising ids gene sequence and a tandem repeat of apolipoprotein e gene sequence Active ES3035262T3 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
GBGB1701968.8A GB201701968D0 (en) 2017-02-07 2017-02-07 Gene therapy

Publications (1)

Publication Number Publication Date
ES3035262T3 true ES3035262T3 (en) 2025-09-01

Family

ID=58462471

Family Applications (2)

Application Number Title Priority Date Filing Date
ES21187825T Active ES3035262T3 (en) 2017-02-07 2018-02-07 A nucleic acid comprising ids gene sequence and a tandem repeat of apolipoprotein e gene sequence
ES18705462T Active ES2895263T3 (es) 2017-02-07 2018-02-07 Un ácido nucleico que comprende la secuencia de genes IDS y una repetición en tándem de la secuencia de genes de apolipoproteína E

Family Applications After (1)

Application Number Title Priority Date Filing Date
ES18705462T Active ES2895263T3 (es) 2017-02-07 2018-02-07 Un ácido nucleico que comprende la secuencia de genes IDS y una repetición en tándem de la secuencia de genes de apolipoproteína E

Country Status (6)

Country Link
US (2) US11701390B2 (enExample)
EP (2) EP3960188B1 (enExample)
JP (1) JP7237364B2 (enExample)
ES (2) ES3035262T3 (enExample)
GB (1) GB201701968D0 (enExample)
WO (1) WO2018146473A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201701968D0 (en) * 2017-02-07 2017-03-22 Univ Manchester Gene therapy
BR112020005271A2 (pt) 2017-10-02 2020-09-15 Denali Therapeutics Inc. proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica
WO2024096738A1 (en) 2022-11-01 2024-05-10 Erasmus University Medical Center Rotterdam Gene therapy constructs for metabolic disorders

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003286870A1 (en) 2003-06-05 2005-01-04 Salk Institute For Biological Studies Targeting polypeptides to the central nervous system
EP1694345A1 (en) 2003-12-04 2006-08-30 Regents Of The University Of Minnesota Compositions and methods for the treatment of lysosomal storage disorders
US10227387B2 (en) 2011-05-18 2019-03-12 Children's Hospital Medical Center Targeted delivery of proteins across the blood-brain barrier
US20140335163A1 (en) 2011-12-01 2014-11-13 Angiochem Inc. Targeted iduronate-2-sulfatase compounds
JP2017534640A (ja) * 2014-11-10 2017-11-24 アレクシオン ファーマシューティカルズ， インコーポレイテッド アルファ−ｌ−イズロニダーゼ、イズロン酸−２−スルファターゼおよびアルファガラクトシダーゼａの治療用組成物ならびにそれらの使用方法
GB201701968D0 (en) * 2017-02-07 2017-03-22 Univ Manchester Gene therapy

Also Published As

Publication number Publication date
GB201701968D0 (en) 2017-03-22
US11701390B2 (en) 2023-07-18
WO2018146473A1 (en) 2018-08-16
EP3579849B1 (en) 2021-09-08
EP3579849A1 (en) 2019-12-18
JP2020506714A (ja) 2020-03-05
JP7237364B2 (ja) 2023-03-13
EP3960188A1 (en) 2022-03-02
US20200000854A1 (en) 2020-01-02
EP3960188B1 (en) 2025-04-02
ES2895263T3 (es) 2022-02-18
US20230390340A1 (en) 2023-12-07

Similar Documents

Publication Publication Date Title
Gleitz et al. Brain‐targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms
US20230390340A1 (en) Gene therapy
ES2790834T3 (es) Vectores virales adenoasociados para el tratamiento de mucopolisacaridosis
Biffi et al. Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells
JP7037574B2 (ja) 脊髄性筋萎縮症の治療に有用な組成物
ES2615180T3 (es) Métodos y composiciones para el tratamiento de enfermedades cerebrales
ES2805355T3 (es) Composiciones para tratar MPSI
ES2859661T3 (es) Vectores de virus adenoasociados para el tratamiento de mucopolisacaridosis
ES2823948T3 (es) Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosómico
US20210147480A1 (en) Viral vector for the targeted transfer of genes in the brain and spinal cord
ES2745470T3 (es) Partícula de AAV2 que comprende una proteína de la cápside de AAV2 y un vector que comprende un ácido nucleico que codifica una tripeptidilo peptidasa 1 (TPP1) para el tratamiento de lipofuscinosis ceroide infantil tardía (LINCL) en un mamífero no roedor mediante inyección intraventricular o administración icv
US20230181683A1 (en) Peptides having specificity for the lungs
Kalburgi et al. Recent gene therapy advancements for neurological diseases
TW201919676A (zh) 用於治療黏多醣病ｉｉ型之基因治療
US12599636B2 (en) Treatment of Krabbe disease with umbilical cord blood transplantion (UCBT) and increased galactocerebrosidase (GALC) expression
JP2025087702A (ja) ヒト神経細胞又はグリア細胞により生産された組換えヒトイズロン酸－２－スルファターゼ（ｉｄｓ）によるムコ多糖症ｉｉ型の治療
US20240325570A1 (en) Treating diseases and improving nucleic acid delivery
JP2025538864A (ja) 代謝障害の遺伝子治療構築物
US20250228907A1 (en) Compositions and methods for treating metachromatic leukodystrophy disease and related disorders
De Ponti Exploring early therapeutic approaches in a Mucopolysaccharidosis type I (MPS I) mouse model
CN118909979A (zh) 一种重组腺相关病毒AAV-Lec及其制备方法和应用
Papanikolaou et al. Novel Therapies for Heart Failure: The Gene and Cell Methods
Liang AAV-Nrf2 Mediated Rescue of Oxidative Stress in Mouse Models
Deficient et al. GENETIC AND METABOLIC DISEASES GENE & CELL THERAPY II
BR112017025892B1 (pt) Vetores de vírus adeno-associados para o tratamento de mucopolissacaridose