MX2019005266A - Composiciones, metodos y usos de transferencia genetica para tratar enfermedades neurodegenerativas. - Google Patents

Composiciones, metodos y usos de transferencia genetica para tratar enfermedades neurodegenerativas.

Info

Publication number
MX2019005266A
MX2019005266A MX2019005266A MX2019005266A MX2019005266A MX 2019005266 A MX2019005266 A MX 2019005266A MX 2019005266 A MX2019005266 A MX 2019005266A MX 2019005266 A MX2019005266 A MX 2019005266A MX 2019005266 A MX2019005266 A MX 2019005266A
Authority
MX
Mexico
Prior art keywords
methods
gene transfer
neurodegenerative diseases
treating neurodegenerative
transfer compositions
Prior art date
Application number
MX2019005266A
Other languages
English (en)
Spanish (es)
Inventor
L Davidson Beverly
Hong Chen Yong
TECEDOR Luis
Original Assignee
The Children´S Hospital Of Philadelphia
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 The Children´S Hospital Of Philadelphia filed Critical The Children´S Hospital Of Philadelphia
Publication of MX2019005266A publication Critical patent/MX2019005266A/es

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    • 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
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/365—Lactones
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7008—Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
    • 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
    • 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
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/12—Cyclic peptides, e.g. bacitracins; Polymyxins; Gramicidins S, C; Tyrocidins A, B or C
    • A61K38/13—Cyclosporins
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4813—Exopeptidases (3.4.11. to 3.4.19)
    • 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
    • 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
    • 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
    • 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
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (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)
MX2019005266A 2016-11-04 2017-11-03 Composiciones, metodos y usos de transferencia genetica para tratar enfermedades neurodegenerativas. MX2019005266A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662418033P 2016-11-04 2016-11-04
PCT/US2017/059986 WO2018085688A1 (en) 2016-11-04 2017-11-03 Gene transfer compositions, methods and uses for treating neurodegenerative diseases

Publications (1)

Publication Number Publication Date
MX2019005266A true MX2019005266A (es) 2019-09-27

Family

ID=62075988

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2019005266A MX2019005266A (es) 2016-11-04 2017-11-03 Composiciones, metodos y usos de transferencia genetica para tratar enfermedades neurodegenerativas.

Country Status (9)

Country Link
US (1) US20190269797A1 (cg-RX-API-DMAC7.html)
EP (1) EP3534892A4 (cg-RX-API-DMAC7.html)
JP (2) JP2019537576A (cg-RX-API-DMAC7.html)
CN (1) CN110198712A (cg-RX-API-DMAC7.html)
AU (1) AU2017355502B2 (cg-RX-API-DMAC7.html)
BR (1) BR112019009074A2 (cg-RX-API-DMAC7.html)
CA (1) CA3041548A1 (cg-RX-API-DMAC7.html)
MX (1) MX2019005266A (cg-RX-API-DMAC7.html)
WO (1) WO2018085688A1 (cg-RX-API-DMAC7.html)

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US9818239B2 (en) 2015-08-20 2017-11-14 Zendrive, Inc. Method for smartphone-based accident detection
MY198804A (en) 2017-06-07 2023-09-28 Regeneron Pharma Compositions and methods for internalizing enzymes
WO2019079807A1 (en) 2017-10-20 2019-04-25 Zendrive, Inc. METHOD AND SYSTEM FOR VEHICULAR COMMUNICATIONS
BR112020016005A2 (pt) 2018-02-07 2020-12-15 Regeneron Pharmaceuticals, Inc. Método de administração de uma proteína terapêutica ao sistema nervoso central, proteína terapêutica multidomínio, polinucleotídeo, vetor de terapia gênica, e, uso de um nucleotídeo que codifica a proteína terapêutica multidomínio
CN112135624B (zh) 2018-05-17 2024-07-19 瑞泽恩制药公司 抗cd63抗体、缀合物和其用途
WO2020068990A1 (en) 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
IL282808B2 (en) * 2018-11-14 2025-02-01 Regenxbio Inc Gene therapy for neural ceroid lipofuscinosis
JP2022518931A (ja) * 2019-02-01 2022-03-17 スパーク セラピューティクス インコーポレイテッド 遅発型小児性神経セロイドリポフスチン沈着症２型に対するａａｖベクター治療法
JP2023504269A (ja) 2019-12-03 2023-02-02 ゼンドライヴ，インコーポレイテッド ルートのリスク判定方法およびシステム
EP4118219A4 (en) * 2020-03-11 2024-04-17 Shanghai Belief-Delivery Biomed Co., Ltd. Novel use of aspirin compound in increasing nucleic acid expression
WO2025231406A1 (en) * 2024-05-02 2025-11-06 The Children's Hospital Of Philadelphia Methods to increase transduction of ependyma cells in brain

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US20070003518A1 (en) * 2002-04-19 2007-01-04 Atkinson Mark A Raav vector-based compositions and methods for the prevention and treatment of mammalian diseases
US20040258666A1 (en) * 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
KR20070004637A (ko) * 2004-01-22 2007-01-09 가부시키가이샤 디나벡크 겐큐쇼 바이러스 벡터의 제조방법
EP3456331B1 (en) * 2006-02-08 2021-05-26 Genzyme Corporation Gene therapy for niemann-pick disease type a
ES2635726T3 (es) * 2007-06-06 2017-10-04 Genzyme Corporation Terapia génica para enfermedades de almacenamiento lisosómico
PT2691529T (pt) * 2011-03-31 2019-09-27 Univ Iowa Res Found Partícula de aav2 que compreende uma proteína do capsídeo de aav2 e um vetor que compreende um ácido nucleico que codifica uma tripeptidil peptidase 1 (tpp1) para utilização no tratamento da lipofuscinose ceroide infantil tardia (lincl) num mamífero não roedor por injeção intraventricular ou entrega icv
KR102380265B1 (ko) * 2013-07-22 2022-03-29 더 칠드런스 호스피탈 오브 필라델피아 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
EP3024497B1 (en) * 2013-07-26 2021-01-13 University of Iowa Research Foundation Methods and compositions for treating brain diseases
EP3060669B1 (en) * 2013-10-24 2024-12-11 uniQure IP B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
KR20160079891A (ko) * 2013-11-20 2016-07-06 유니버시티 오브 아이오와 리써치 파운데이션 아밀로이드 침착의 치료를 위한 방법 및 조성물
SG11201803218PA (en) * 2015-10-23 2018-05-30 Univ Iowa Res Found Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection
IL270422B2 (en) * 2017-05-11 2025-04-01 Univ Pennsylvania Gene therapy for neuronal ceroid lipofuscinosis

Also Published As

Publication number Publication date
WO2018085688A1 (en) 2018-05-11
EP3534892A4 (en) 2020-05-27
EP3534892A1 (en) 2019-09-11
US20190269797A1 (en) 2019-09-05
RU2019117062A (ru) 2020-12-04
JP2019537576A (ja) 2019-12-26
RU2019117062A3 (cg-RX-API-DMAC7.html) 2021-03-11
CA3041548A1 (en) 2018-05-11
BR112019009074A2 (pt) 2019-07-16
AU2017355502B2 (en) 2023-08-31
JP2023002721A (ja) 2023-01-10
CN110198712A (zh) 2019-09-03
AU2017355502A1 (en) 2019-05-16

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