ES2784639T8 - Uso combinado de un vector que codifica un receptor modificado y su agonista exógeno en el tratamiento de convulsiones - Google Patents

Uso combinado de un vector que codifica un receptor modificado y su agonista exógeno en el tratamiento de convulsiones

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Publication number
ES2784639T8
ES2784639T8 ES15709326T ES15709326T ES2784639T8 ES 2784639 T8 ES2784639 T8 ES 2784639T8 ES 15709326 T ES15709326 T ES 15709326T ES 15709326 T ES15709326 T ES 15709326T ES 2784639 T8 ES2784639 T8 ES 2784639T8
Authority
ES
Spain
Prior art keywords
seizures
treatment
combined use
vector encoding
modified receptor
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
ES15709326T
Other languages
English (en)
Other versions
ES2784639T3 (es
Inventor
Dennis Kaetzel
Matthew Charles Walker
Stephanie Schorge
Dimitri Michael Kullmann
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.)
UCL Business Ltd
Original Assignee
UCL Business Ltd
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 UCL Business Ltd filed Critical UCL Business Ltd
Application granted granted Critical
Publication of ES2784639T3 publication Critical patent/ES2784639T3/es
Publication of ES2784639T8 publication Critical patent/ES2784639T8/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
    • 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
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1787—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/55—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/55—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
    • A61K31/551—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole having two nitrogen atoms, e.g. dilazep
    • A61K31/5513—1,4-Benzodiazepines, e.g. diazepam or clozapine
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70571—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • 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
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/16043—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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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
    • 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)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Neurology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Neurosurgery (AREA)
  • Pain & Pain Management (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
ES15709326T 2014-03-13 2015-03-06 Uso combinado de un vector que codifica un receptor modificado y su agonista exógeno en el tratamiento de convulsiones Active ES2784639T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB201404470A GB201404470D0 (en) 2014-03-13 2014-03-13 Therapeutic methods and materials
PCT/GB2015/050657 WO2015136247A1 (en) 2014-03-13 2015-03-06 Combined use of a vector encoding a modified receptor and its exogenous agonist in the treatment of seizures

Publications (2)

Publication Number Publication Date
ES2784639T3 ES2784639T3 (es) 2020-09-29
ES2784639T8 true ES2784639T8 (es) 2020-10-06

Family

ID=50634693

Family Applications (1)

Application Number Title Priority Date Filing Date
ES15709326T Active ES2784639T3 (es) 2014-03-13 2015-03-06 Uso combinado de un vector que codifica un receptor modificado y su agonista exógeno en el tratamiento de convulsiones

Country Status (6)

Country Link
US (2) US10391145B2 (es)
EP (1) EP3116508B1 (es)
JP (1) JP6590828B2 (es)
ES (1) ES2784639T3 (es)
GB (1) GB201404470D0 (es)
WO (1) WO2015136247A1 (es)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20200108514A (ko) * 2015-09-17 2020-09-21 코다 바이오테라퓨틱스 인코포레이티드 신경 장애를 치료하기 위한 조성물 및 방법
US20190083573A1 (en) * 2016-01-29 2019-03-21 The Board Of Trustees Of The Leland Stanford Junior University Designer receptors for modulating pain and methods of use thereof
JP6968331B2 (ja) * 2016-03-09 2021-11-17 アサフ，ファディ ニューロン性疾患の治療におけるニューロン調節のためのｄｒｅａｄｄの使用
US11542315B2 (en) 2016-08-31 2023-01-03 Rutgers, The State University Of New Jersey Methods and compositions for treating diseases and disorders of the nervous system
US20190060400A1 (en) * 2017-03-15 2019-02-28 Ovid Therapeutics Inc. Use of designer receptors exclusively activated by designer drugs and ultrasound in the treatment of seizure disorders
GB201709551D0 (en) 2017-06-15 2017-08-02 Ucl Business Plc Expression vectors comprising engineered genes
MX2020005470A (es) 2017-11-27 2020-11-09 Coda Biotherapeutics Inc Composiciones y metodos para enfermedades neurologicas.
WO2019126473A1 (en) * 2017-12-20 2019-06-27 Ovid Therapeutics Inc. USE OF hM4Di IN THE TREATMENT OF SEIZURE DISORDERS
JP6926357B2 (ja) * 2018-06-21 2021-08-25 国立研究開発法人量子科学技術研究開発機構 人工受容体に結合する新規化合物、人工受容体のイメージング方法、アゴニストまたはアンタゴニスト、治療薬、コンパニオン診断薬、神経細胞のイメージング方法
US20220152222A1 (en) * 2019-02-27 2022-05-19 Pathways Neuro Pharma, Inc. Gene Therapy for Addiction Disorders
CN114144425A (zh) * 2019-05-24 2022-03-04 布里格姆妇女医院 用于阿尔茨海默症的基因治疗
CN112640847B (zh) * 2020-12-30 2022-12-13 重庆医科大学附属第一医院 一种内源性癫痫发作动物模型及其构建方法
JP2024519329A (ja) 2021-05-12 2024-05-10 ユーシーエル ビジネス リミテッド 合成受容体
WO2023278295A1 (en) 2021-06-29 2023-01-05 The Broad Institute, Inc. Compositions and methods for ameliorating anterodorsal thalamus hyperexcitability
WO2023131682A1 (en) 2022-01-06 2023-07-13 Ucl Business Ltd Endogenous gene regulation to treat neurological disorders and diseases

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2430897A (en) * 1996-03-26 1997-10-17 Regents Of The University Of California, The Selective target cell activation by expression of a g protein-coupled receptor activated superiorly by synthetic ligand
US6214620B1 (en) 1998-09-29 2001-04-10 The Johns Hopkins University Inducible genetic suppression of cellular excitability
WO2016161124A1 (en) 2015-03-31 2016-10-06 New York University Compositions and method for reducing seizures

Also Published As

Publication number Publication date
JP2017509625A (ja) 2017-04-06
WO2015136247A1 (en) 2015-09-17
US10391145B2 (en) 2019-08-27
GB201404470D0 (en) 2014-04-30
EP3116508B1 (en) 2020-01-22
US10525103B2 (en) 2020-01-07
JP6590828B2 (ja) 2019-10-16
EP3116508A1 (en) 2017-01-18
US20190134155A1 (en) 2019-05-09
US20160375097A1 (en) 2016-12-29
ES2784639T3 (es) 2020-09-29

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