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MX2022012524A - Adeno-asociado para administracion terapeutica al sistema nervioso central. - Google Patents

Adeno-asociado para administracion terapeutica al sistema nervioso central.

Info

Publication number
MX2022012524A
MX2022012524A MX2022012524A MX2022012524A MX2022012524A MX 2022012524 A MX2022012524 A MX 2022012524A MX 2022012524 A MX2022012524 A MX 2022012524A MX 2022012524 A MX2022012524 A MX 2022012524A MX 2022012524 A MX2022012524 A MX 2022012524A
Authority
MX
Mexico
Prior art keywords
nervous system
central nervous
adeno
disease
therapeutic delivery
Prior art date
Application number
MX2022012524A
Other languages
English (en)
Inventor
Karen Kozarsky
R Scott Mcivor
Lalitha R Belur
Original Assignee
Univ Minnesota
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=57320277&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=MX2022012524(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Univ Minnesota filed Critical Univ Minnesota
Publication of MX2022012524A publication Critical patent/MX2022012524A/es

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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/66—Phosphorus compounds
    • A61K31/675—Phosphorus compounds having nitrogen as a ring hetero atom, e.g. pyridoxal phosphate
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K48/0083—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 administration regime
    • 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
    • A61K9/0043—Nose
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6806—Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Analytical Chemistry (AREA)
  • Otolaryngology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Un método para prevenir, inhibir o tratar uno o más síntomas asociados con enfermedad del sistema nervioso central por administración intranasal, intratecal, intracerebrovascular o intravenosa de un rAAV que codifica un producto génico asociado con la enfermedad, por ejemplo, un mamífero en el cual el producto génico está ausente o presente a un nivel reducido con respecto a un mamífero sin la enfermedad, en una cantidad eficaz, por ejemplo, para proveer corrección cruzada.
MX2022012524A 2015-05-15 2017-11-10 Adeno-asociado para administracion terapeutica al sistema nervioso central. MX2022012524A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562162174P 2015-05-15 2015-05-15
US201562252055P 2015-11-06 2015-11-06
US201662301980P 2016-03-01 2016-03-01
US201662331156P 2016-05-03 2016-05-03

Publications (1)

Publication Number Publication Date
MX2022012524A true MX2022012524A (es) 2022-11-16

Family

ID=57320277

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2017014443A MX2017014443A (es) 2015-05-15 2016-05-13 Adeno-asociado para administracion terapeutica al sistema nervioso central.
MX2022012524A MX2022012524A (es) 2015-05-15 2017-11-10 Adeno-asociado para administracion terapeutica al sistema nervioso central.

Family Applications Before (1)

Application Number Title Priority Date Filing Date
MX2017014443A MX2017014443A (es) 2015-05-15 2016-05-13 Adeno-asociado para administracion terapeutica al sistema nervioso central.

Country Status (23)

Country Link
US (5) US20180289839A1 (es)
EP (2) EP4000631A1 (es)
JP (4) JP2018515615A (es)
KR (3) KR20250150167A (es)
CN (1) CN108367055A (es)
AU (2) AU2016263119B2 (es)
BR (1) BR112017024519A2 (es)
CA (1) CA2986252A1 (es)
CY (1) CY1125229T1 (es)
DK (1) DK3294323T3 (es)
ES (1) ES2901766T3 (es)
HK (1) HK1256341A1 (es)
HR (1) HRP20220050T1 (es)
HU (1) HUE057795T2 (es)
LT (1) LT3294323T (es)
MX (2) MX2017014443A (es)
PL (1) PL3294323T3 (es)
PT (1) PT3294323T (es)
RS (1) RS63927B1 (es)
RU (1) RU2751952C2 (es)
SI (1) SI3294323T1 (es)
SM (1) SMT202200022T1 (es)
WO (1) WO2016187017A1 (es)

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BR112015028605A8 (pt) * 2013-05-15 2019-12-24 Univ Minnesota uso de uma composição e uso de um imunossupressor e uma composição
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
RS63927B1 (sr) * 2015-05-15 2023-02-28 Regenxbio Inc Povezani adenovirus za terapijsku isporuku u centralni nervni sistem
JP7360241B2 (ja) 2016-02-03 2023-10-12 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症ｉ型を治療するための遺伝子治療
SG11201808176TA (en) 2016-04-15 2018-10-30 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
EP3541946A1 (en) * 2016-11-15 2019-09-25 Regents Of The University Of Minnesota Method for improving neurological function in mpsi and mpsii and other neurological disorders
BR112019021569A2 (pt) 2017-04-14 2020-05-12 Regenxbio Inc. Precursor de iduronato-2-sulfatase humana recombinante glicosilada (ids), método para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo ii (mps ii)
CN110709090A (zh) * 2017-04-27 2020-01-17 益缪索夫特公司 用于在体内递送治疗剂的b细胞及其剂量
CA3068328A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
CA3076036A1 (en) 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
MX2020003965A (es) 2017-10-03 2020-10-05 Prevail Therapeutics Inc Terapias genicas para los trastornos lisosomales.
CA3078501A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
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JP7384797B2 (ja) * 2017-11-30 2023-11-21 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症ｉｉｉｂ型のための遺伝子療法
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MX2021012184A (es) 2019-04-10 2022-01-24 Prevail Therapeutics Inc Terapias genicas para trastornos lisosomales.
US12338451B2 (en) 2019-05-03 2025-06-24 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
KR20220035107A (ko) * 2019-06-10 2022-03-21 호몰로지 메디슨, 인크. Arsa 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법
IL292372A (en) * 2019-10-23 2022-06-01 Univ Pennsylvania Compositions for drg-specific reduction of transgene expression
IL293684A (en) 2019-12-10 2022-08-01 Takeda Pharmaceuticals Co Adeno associated virus vectors for the treatment of hunter disease
US20230346979A1 (en) * 2020-08-10 2023-11-02 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disorders
WO2022035903A2 (en) * 2020-08-10 2022-02-17 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
WO2022121860A1 (en) * 2020-12-07 2022-06-16 East China University Of Science And Technology Methods and kits for inducing immunotolerance to gene delivering target vehicle
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WO2022212616A1 (en) * 2021-04-01 2022-10-06 The University Of North Carolina At Chapel Hill Aav-ids vectors for treatment of mucopolysaccharidosis ii

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RS63927B1 (sr) * 2015-05-15 2023-02-28 Regenxbio Inc Povezani adenovirus za terapijsku isporuku u centralni nervni sistem
EP3541946A1 (en) * 2016-11-15 2019-09-25 Regents Of The University Of Minnesota Method for improving neurological function in mpsi and mpsii and other neurological disorders
WO2022173605A2 (en) * 2021-02-10 2022-08-18 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)

Also Published As

Publication number Publication date
DK3294323T3 (da) 2022-01-10
HK1256341A1 (zh) 2019-09-20
RU2017143640A3 (es) 2019-11-22
EP3294323A1 (en) 2018-03-21
SI3294323T1 (sl) 2022-06-30
SMT202200022T1 (it) 2022-03-21
PT3294323T (pt) 2022-01-06
KR102406615B1 (ko) 2022-06-08
CY1125229T1 (el) 2023-06-09
US20220211875A1 (en) 2022-07-07
HUE057795T2 (hu) 2022-06-28
JP2018515615A (ja) 2018-06-14
ES2901766T3 (es) 2022-03-23
EP4000631A1 (en) 2022-05-25
US20230226225A1 (en) 2023-07-20
MX2017014443A (es) 2018-08-01
JP2024147704A (ja) 2024-10-16
PL3294323T3 (pl) 2022-06-20
WO2016187017A1 (en) 2016-11-24
RU2751952C2 (ru) 2021-07-21
AU2021266344B2 (en) 2024-08-22
RU2020125073A (ru) 2020-08-27
KR20220082100A (ko) 2022-06-16
US20180289839A1 (en) 2018-10-11
EP3294323B1 (en) 2021-10-13
CA2986252A1 (en) 2016-11-24
HK1252871A1 (en) 2019-06-06
LT3294323T (lt) 2022-02-25
JP2022137261A (ja) 2022-09-21
AU2016263119B2 (en) 2021-08-12
KR20250150167A (ko) 2025-10-17
EP3294323A4 (en) 2019-01-16
RU2017143640A (ru) 2019-06-17
RS63927B1 (sr) 2023-02-28
HRP20220050T1 (hr) 2022-10-14
BR112017024519A2 (en) 2018-07-24
AU2021266344A1 (en) 2021-12-09
CN108367055A (zh) 2018-08-03
AU2016263119A1 (en) 2018-01-18
KR20180118508A (ko) 2018-10-31
US20210369871A1 (en) 2021-12-02
US20180071373A1 (en) 2018-03-15
JP2021167332A (ja) 2021-10-21

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