JP7361037B2 - ライソゾーム病の遺伝子治療 - Google Patents

ライソゾーム病の遺伝子治療 Download PDF

Info

Publication number
JP7361037B2
JP7361037B2 JP2020540683A JP2020540683A JP7361037B2 JP 7361037 B2 JP7361037 B2 JP 7361037B2 JP 2020540683 A JP2020540683 A JP 2020540683A JP 2020540683 A JP2020540683 A JP 2020540683A JP 7361037 B2 JP7361037 B2 JP 7361037B2
Authority
JP
Japan
Prior art keywords
raav
itr
nucleic acid
seq
vector
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
JP2020540683A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020537544A (ja
JP2020537544A5 (enExample
Inventor
アベリオビック，アーサ
ヘックマン，ローラ
ライン，エルベ
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.)
Prevail Therapeutics Inc
Original Assignee
Prevail Therapeutics Inc
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 Prevail Therapeutics Inc filed Critical Prevail Therapeutics Inc
Publication of JP2020537544A publication Critical patent/JP2020537544A/ja
Publication of JP2020537544A5 publication Critical patent/JP2020537544A5/ja
Priority to JP2022007736A priority Critical patent/JP7336730B2/ja
Priority to JP2022007752A priority patent/JP7389828B2/ja
Application granted granted Critical
Publication of JP7361037B2 publication Critical patent/JP7361037B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • 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
    • 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
    • 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
    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A61K39/23—Parvoviridae, e.g. feline panleukopenia virus
    • 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
    • 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
    • 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
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • C07K14/475—Growth factors; Growth regulators
    • 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
    • C07K14/575—Hormones
    • C07K14/61—Growth hormone [GH], i.e. somatotropin
    • 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
    • C07K14/70503—Immunoglobulin superfamily
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/20—Animals treated with compounds which are neither proteins nor nucleic acids
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2710/14011—Baculoviridae
    • C12N2710/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vectore
    • 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
    • C12N2750/14022—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
    • C12N2750/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14043—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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Mycology (AREA)
  • Endocrinology (AREA)
  • Dermatology (AREA)
  • Hospice & Palliative Care (AREA)
JP2020540683A 2017-10-03 2018-10-03 ライソゾーム病の遺伝子治療 Active JP7361037B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022007736A JP7336730B2 (ja) 2017-10-03 2022-01-21 ライソゾーム病の遺伝子治療
JP2022007752A JP7389828B2 (ja) 2017-10-03 2022-01-21 ライソゾーム病の遺伝子治療

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201762567311P 2017-10-03 2017-10-03
US201762567296P 2017-10-03 2017-10-03
US201762567310P 2017-10-03 2017-10-03
US201762567319P 2017-10-03 2017-10-03
US201762567301P 2017-10-03 2017-10-03
US62/567,310 2017-10-03
US62/567,301 2017-10-03
US62/567,319 2017-10-03
US62/567,296 2017-10-03
US62/567,311 2017-10-03
PCT/US2018/054227 WO2019070894A1 (en) 2017-10-03 2018-10-03 GENE THERAPIES FOR LYSOSOMIAL DISORDERS

Related Child Applications (2)

Application Number Title Priority Date Filing Date
JP2022007736A Division JP7336730B2 (ja) 2017-10-03 2022-01-21 ライソゾーム病の遺伝子治療
JP2022007752A Division JP7389828B2 (ja) 2017-10-03 2022-01-21 ライソゾーム病の遺伝子治療

Publications (3)

Publication Number Publication Date
JP2020537544A JP2020537544A (ja) 2020-12-24
JP2020537544A5 JP2020537544A5 (enExample) 2021-11-11
JP7361037B2 true JP7361037B2 (ja) 2023-10-13

Family

ID=65994362

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2020540683A Active JP7361037B2 (ja) 2017-10-03 2018-10-03 ライソゾーム病の遺伝子治療
JP2022007752A Active JP7389828B2 (ja) 2017-10-03 2022-01-21 ライソゾーム病の遺伝子治療
JP2022007736A Active JP7336730B2 (ja) 2017-10-03 2022-01-21 ライソゾーム病の遺伝子治療

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2022007752A Active JP7389828B2 (ja) 2017-10-03 2022-01-21 ライソゾーム病の遺伝子治療
JP2022007736A Active JP7336730B2 (ja) 2017-10-03 2022-01-21 ライソゾーム病の遺伝子治療

Country Status (11)

Country Link
US (5) US11993790B2 (enExample)
EP (1) EP3692158A4 (enExample)
JP (3) JP7361037B2 (enExample)
KR (3) KR102700963B1 (enExample)
CN (3) CN111492061A (enExample)
AU (3) AU2018346105C1 (enExample)
BR (1) BR112020006661A2 (enExample)
CA (1) CA3078464A1 (enExample)
IL (3) IL273771B1 (enExample)
MX (3) MX2020003965A (enExample)
WO (1) WO2019070894A1 (enExample)

Families Citing this family (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JOP20190248A1 (ar) 2017-04-21 2019-10-20 Amgen Inc بروتينات ربط مولد ضد trem2 واستخداماته
HRP20230675T1 (hr) 2017-08-03 2023-09-29 Alector Llc Anti-trem2 protutijela i postupci njihove uporabe
BR112020006661A2 (pt) 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
CN112501208A (zh) 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
US11802294B2 (en) 2017-10-03 2023-10-31 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
FI3850089T3 (fi) 2019-02-04 2024-02-14 Freeline Therapeutics Ltd Polynukleotidejä
EP3927381A1 (en) 2019-02-22 2021-12-29 The Trustees of The University of Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
MX2021010266A (es) * 2019-02-26 2021-09-23 Univ Pennsylvania Composiciones utiles en el tratamiento de la enfermedad de krabbe.
WO2020210713A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
DK3953377T3 (da) * 2019-04-10 2025-12-01 Prevail Therapeutics Inc Genterapier til lysosomale lidelser
GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector
CN115715327A (zh) * 2019-10-22 2023-02-24 应用遗传科技公司 用于治疗颗粒蛋白前体相关的神经退行性疾病或病症的腺伴随病毒(aav)系统
EP4069315A4 (en) * 2019-12-05 2024-03-20 The Trustees Of Columbia University In The City Of New York Stabilization of retromer for the treatment of alzheimer's disease and other neurodegenerative disorders
WO2021163681A2 (en) * 2020-02-14 2021-08-19 The Trustees Of Columbia University In The City Of New York Combination of retromer pharmacological chaperones and exogenous retromer for the treatment of alzheimer's disease and other neurodegenerative diseases and disorders
TW202208622A (zh) * 2020-05-12 2022-03-01 賓州大學委員會 用於治療克拉培氏病之組成物
US20230285595A1 (en) * 2020-07-23 2023-09-14 The University Of North Carolina At Chapel Hill Optimized slc13a5 genes and expression cassettes and their use
CA3190309A1 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
CA3189801A1 (en) 2020-07-29 2022-02-03 Freeline Therapeutics Limited Polypeptide
US20230304036A1 (en) * 2020-08-03 2023-09-28 Prevail Therapeutics, Inc. Aav vectors encoding parkin and uses thereof
KR20230066360A (ko) * 2020-08-10 2023-05-15 프리베일 테라퓨틱스, 인크. 신경퇴행성 장애를 위한 유전자 요법
JP2023537903A (ja) 2020-08-10 2023-09-06 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害に対する遺伝子療法
JP2023545462A (ja) 2020-10-14 2023-10-30 デナリ セラピューティクス インコーポレイテッド 前頭側頭型認知症を治療及びモニタリングするための方法
KR20230088393A (ko) 2020-10-15 2023-06-19 프리베일 테라퓨틱스, 인크. 재조합 아데노 연관 바이러스 조성물 및 이의 제조 방법
JP2023545835A (ja) 2020-10-15 2023-10-31 プリベイル セラピューティクス，インコーポレーテッド 遺伝子療法製剤の効力を測定するためのアッセイ
CA3205351A1 (en) * 2021-02-01 2022-08-04 James M. Wilson Compositions and methods for treatment of niemann pick type a disease
US20240325506A1 (en) * 2021-06-23 2024-10-03 Avrobio, Inc. Compositions and methods for treatment of gaucher disease
US20250049955A1 (en) 2021-11-17 2025-02-13 Voyager Therapeutics, Inc. Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
JP1727698S (ja) * 2021-12-20 2022-10-19 ゴルフボールケース
CN119487188A (zh) * 2022-04-19 2025-02-18 上海天泽云泰生物医药有限公司 用于治疗神经退行性障碍的重组aav载体
US20240226332A9 (en) 2022-10-04 2024-07-11 Eli Lilly And Company Gene therapy for trem2-associated diseases and disorders
CN118715316B (zh) * 2022-10-08 2025-09-19 凌意(杭州)生物科技有限公司 用于治疗GCase缺乏相关疾病的多核苷酸
GB202216168D0 (en) 2022-10-31 2022-12-14 UCB Biopharma SRL Route of administration
AU2023427408A1 (en) * 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024189094A1 (en) 2023-03-14 2024-09-19 UCB Biopharma SRL Gene therapy
WO2025039622A1 (zh) * 2023-08-23 2025-02-27 上海金珂博生物技术有限公司 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途
CN118086341B (zh) * 2024-04-25 2024-08-02 上海凌医生物科技有限公司 在肝脏中高表达人源葡萄糖脑苷脂酶基因的表达框

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004516016A (ja) 2000-09-18 2004-06-03 ジェンザイム・コーポレイション ハイブリッドユビキチンプロモーターを含む発現ベクター
WO2009120978A2 (en) 2008-03-27 2009-10-01 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of bdnf and compositions therfor
JP2009538631A (ja) 2006-05-30 2009-11-12 メイヨ・ファウンデーション・フォー・メディカル・エデュケーション・アンド・リサーチ 認知症の検出および治療
JP2015516143A (ja) 2012-04-02 2015-06-08 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ヒト疾患に関連するタンパク質の産生のための修飾ポリヌクレオチド
JP2016503405A (ja) 2012-11-05 2016-02-04 ジェンザイム・コーポレーション タンパク質症を処置するための組成物および方法
WO2016081927A2 (en) 2014-11-21 2016-05-26 University Of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors

Family Cites Families (80)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS6182035A (ja) * 1984-09-27 1986-04-25 Toshiba Corp 制振装置
US5879680A (en) 1987-12-23 1999-03-09 The United States Of America As Represented By The Department Of Health And Human Services Cloned DNA for synthesizing unique glucocerebrosidase
US5490559A (en) * 1994-07-20 1996-02-13 Dinulescu; Horia A. Heat exchanger with finned partition walls
US20020068285A1 (en) * 1996-01-11 2002-06-06 Frudakis Tony N. Compositions and methods for the therapy and diagnosis of breast cancer
EP0933997A4 (en) 1996-09-06 2000-08-16 Chiron Corp METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS
EP1112090A2 (en) 1998-09-09 2001-07-04 Myelos Corporation Method of stimulating prosaposin receptor activity
WO2000023587A2 (en) 1998-10-16 2000-04-27 Introgene B.V. Gene therapy of alzheimer's disease by delivery of an encoded apolipoprotein e
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
US6696272B1 (en) 1999-06-02 2004-02-24 Hsc Research & Development Limited Partnership Products and methods for gaucher disease therapy
DE19937800B4 (de) * 1999-08-10 2005-06-16 Gea Energietechnik Gmbh Anlage zur Kondensation von Dampf
CA2405870A1 (en) 2000-04-06 2001-10-18 Kos Pharmaceuticals, Inc. Compounds and methods for lowering cholesterol levels without inducing hypertriglyceridemia
GB0009887D0 (en) 2000-04-20 2000-06-07 Btg Int Ltd Cytotoxic agents
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
US7232670B2 (en) 2001-09-28 2007-06-19 St. Jude Children's Research Hospital Targeting proteins to cells expressing mannose receptors via expression in insect cells
US20030133924A1 (en) 2001-12-21 2003-07-17 Novazyme Pharmaceuticals, Inc. Highly phosphorylated acid beta-glucocerebrosidase and methods of treating gaucher's disease
US20060292117A1 (en) 2002-04-17 2006-12-28 Loiler Scott A Improved rAAv vectors
US6691742B1 (en) * 2003-04-25 2004-02-17 Thomas L. Cooper Method and apparatus for supporting a pipe
PL1620133T3 (pl) 2003-05-01 2016-05-31 Genzyme Corp Terapia genowa dla zaburzeń neurometabolicznych
ATE414113T1 (de) 2003-11-21 2008-11-15 Basf Se Polyalkylenoxidgruppen und quartäre stickstoffatome enthaltende copolymere
CN101443046A (zh) * 2004-06-23 2009-05-27 建新公司 治疗多囊病的方法和组合物
WO2006036465A2 (en) 2004-09-03 2006-04-06 University Of Florida Compositions and methods for treating cystic fibrosis
EP1799826B1 (en) 2004-09-29 2009-08-12 Children's Memorial Hospital siRNA-MEDIATED GENE SILENCING OF ALPHA SYNUCLEIN
CN104771402A (zh) 2005-06-08 2015-07-15 阿米库斯治疗学公司 溶酶体酶编码基因突变相关的cns紊乱的治疗
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
DK2037948T3 (en) 2006-05-30 2016-08-01 Mayo Foundation Detection and treatment of dementia
ES2596885T3 (es) * 2006-06-07 2017-01-12 Genzyme Corporation Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la medula espinal
DE102006029773B3 (de) * 2006-06-27 2007-07-12 Gea Energietechnik Gmbh Verfahren zur Errichtung einer Kondensationsanlage
CN101583621A (zh) * 2006-07-20 2009-11-18 诺华有限公司 治疗、诊断或检测癌症的amigo-2抑制剂
EP2145014B1 (en) 2007-04-05 2012-12-12 The J. David Gladstone Institutes Agents that reduce neuronal overexcitation
WO2008143797A1 (en) 2007-05-11 2008-11-27 Genzyme Corporation Methods of producing a secreted protein
HUE027278T2 (en) 2007-05-16 2016-11-28 Brigham & Womens Hospital Inc Treatment of synucleinopathies
EP3666284A1 (en) * 2007-06-22 2020-06-17 Children's Medical Center, Corp. Methods and uses thereof of a fragment of saposin a
WO2009079399A2 (en) 2007-12-14 2009-06-25 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
ES2596360T3 (es) 2008-01-16 2017-01-09 Neurodyn Life Sciences Inc. Progranulina para su uso en el tratamiento de enfermedad de Parkinson o enfermedad de Alzheimer
RS67077B1 (sr) 2009-05-02 2025-08-29 Genzyme Corp Genska terapija za neurodegenerativne poremećaje
US9255266B2 (en) 2009-05-06 2016-02-09 Rutgers, The State University Of New Jersey RNA targeting in alpha-synucleinopathies
CN102858985A (zh) * 2009-07-24 2013-01-02 西格马-奥尔德里奇有限责任公司 基因组编辑方法
EP3366695B1 (en) * 2009-12-17 2021-07-07 Children's Medical Center Corporation Saposin-a derived peptides and uses thereof
EP3514232A1 (en) 2010-04-23 2019-07-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
US9290759B2 (en) 2010-08-25 2016-03-22 The Trustees Of Columbia University In The City Of New York Optimized miRNA constructs
WO2012027713A2 (en) 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
CN107828820B (zh) 2010-10-27 2022-06-07 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
EP2640407A4 (en) 2010-11-16 2014-07-09 Denis G Kay PROCESS FOR INCREASING THE EXPRESSION AND ACTIVITY OF NEPRILYSIN
US8697627B2 (en) 2011-05-09 2014-04-15 Eip Pharma, Llc Compositions and methods for treating alzheimer's disease
ES2786078T3 (es) 2012-05-18 2020-10-08 Univ Iowa Res Found Métodos y composiciones para tratar depósitos amiloides
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
EP2983707B1 (en) 2013-04-08 2019-06-12 University of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
RU2692251C2 (ru) 2013-05-15 2019-06-24 Риджентс Оф Зэ Юниверсити Оф Миннесота Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему
US9347107B2 (en) 2013-05-23 2016-05-24 Norman Z Lai Vector containing multiple nucleotide sequences for the expression of enzymes
JP2016523980A (ja) 2013-07-11 2016-08-12 ザ トラスティーズ オブ コロンビア ユニバーシティ イン ザ シティ オブ ニューヨーク タウ発現を抑制するマイクロｒｎａ
US9574184B2 (en) 2013-09-25 2017-02-21 Children's Hospital Medical Center Lysosomal protein targeting sequence and therapeutic applications of same
SG11201700901SA (en) * 2014-08-08 2017-03-30 Alector Llc Anti-trem2 antibodies and methods of use thereof
EP3193942B1 (en) 2014-08-11 2020-03-25 Shire Human Genetic Therapies, Inc. Lysosomal targeting and uses thereof
US10724096B2 (en) * 2014-09-05 2020-07-28 Population Bio, Inc. Methods and compositions for inhibiting and treating neurological conditions
ES2714130T3 (es) 2015-02-02 2019-05-27 Procter & Gamble Composición detergente
WO2016151523A1 (en) 2015-03-25 2016-09-29 Università Degli Studi Di Trento Rna interference mediated therapy for neurodegenerative diseases
US20170035860A1 (en) 2015-04-02 2017-02-09 Alexander C. Flynn Compositions and methods for treatment of neurogenerative diseases
US11020443B2 (en) * 2015-04-23 2021-06-01 University Of Massachusetts Modulation of AAV vector transgene expression
AU2016256895B2 (en) 2015-05-07 2022-05-26 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
EP3091087A1 (en) 2015-05-08 2016-11-09 Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol Method for in vitro diagnosis of synucleinopathies using alpha-synuclein gene transcripts
WO2016187017A1 (en) 2015-05-15 2016-11-24 Mcivor R Scott Adeno-associated for therapeutic delivery to central nervous system
WO2017058561A1 (en) 2015-10-02 2017-04-06 The Procter & Gamble Company Cleaning pad, cleaning implement, and a method of improving shine of a hard surface using the cleaning pad or cleaning implement with a cleaning composition
WO2017075338A2 (en) 2015-10-29 2017-05-04 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
CN108348621A (zh) 2015-11-05 2018-07-31 竹治疗股份有限公司 用于基因治疗的经修饰的弗里德赖希共济失调基因及载体
US11826433B2 (en) 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
IL300254B2 (en) 2016-02-05 2025-06-01 Univ Emory Administration of single stranded or self-complementary adeno-associated virus 9 by injection into the cerebrospinal fluid to target gene therapy to the central nervous system
US11066456B2 (en) 2016-02-25 2021-07-20 Washington University Compositions comprising TREM2 and methods of use thereof
CA3016314A1 (en) 2016-03-02 2017-09-08 Julianne REIDERS Therapy for frontotemporal dementia
EP3228690B1 (en) * 2016-04-08 2020-05-13 The Procter and Gamble Company Automatic dishwashing cleaning composition
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US11802294B2 (en) 2017-10-03 2023-10-31 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CN112501208A (zh) 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
BR112020006661A2 (pt) 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
BR112020008033A2 (pt) 2017-10-23 2020-10-27 Prevail Therapeutics, Inc. terapias gênicas para doença neurodegenerativa
US20200283800A1 (en) 2017-10-23 2020-09-10 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative diseases
JP7616995B2 (ja) 2018-11-28 2025-01-17 プリベイル セラピューティクス，インコーポレーテッド 神経変性疾患のための遺伝子治療
US20220211871A1 (en) 2019-04-10 2022-07-07 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
DK3953377T3 (da) * 2019-04-10 2025-12-01 Prevail Therapeutics Inc Genterapier til lysosomale lidelser
WO2020210713A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CA3134841A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004516016A (ja) 2000-09-18 2004-06-03 ジェンザイム・コーポレイション ハイブリッドユビキチンプロモーターを含む発現ベクター
JP2009538631A (ja) 2006-05-30 2009-11-12 メイヨ・ファウンデーション・フォー・メディカル・エデュケーション・アンド・リサーチ 認知症の検出および治療
WO2009120978A2 (en) 2008-03-27 2009-10-01 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of bdnf and compositions therfor
JP2015516143A (ja) 2012-04-02 2015-06-08 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ヒト疾患に関連するタンパク質の産生のための修飾ポリヌクレオチド
JP2016503405A (ja) 2012-11-05 2016-02-04 ジェンザイム・コーポレーション タンパク質症を処置するための組成物および方法
WO2016081927A2 (en) 2014-11-21 2016-05-26 University Of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
HUMAN GENE THERAPY METHODS，2017年10月01日，Vol. 28, No. 5，pp. 277-289
J Virol.，2005年，Vol. 79, No. 17，pp. 11082-11094
Molecular Brain，2014年，7:17，URL<http://www.molecularbrain.com/content/7/1/17>, 検索日: 2023-01-19
World J Exp Med.，2015年，Vol. 5, Issue 1，pp. 11-20
植物由来脂肪酸不飽和化酵素を哺乳類細胞において高発現させるためのコドン使用頻度の最適化，Mem. School. B. O. S. T. Kinki University，2008年，No. 22，pp.33-41

Also Published As

Publication number Publication date
IL273771B1 (en) 2025-10-01
AU2018346105A2 (en) 2020-06-25
US20200276335A1 (en) 2020-09-03
AU2018346105B2 (en) 2023-06-15
US20230287358A1 (en) 2023-09-14
EP3692158A1 (en) 2020-08-12
AU2020260485B2 (en) 2023-07-06
AU2020260491A1 (en) 2020-11-26
JP7389828B2 (ja) 2023-11-30
AU2018346105C1 (en) 2023-10-12
BR112020006661A2 (pt) 2020-10-13
EP3692158A4 (en) 2021-08-04
AU2020260491B2 (en) 2023-06-29
IL279931A (en) 2021-03-01
AU2018346105A1 (en) 2020-04-30
KR102700963B1 (ko) 2024-09-02
MX2020011746A (es) 2021-01-08
MX2020011747A (es) 2021-01-08
CN111492061A (zh) 2020-08-04
AU2020260485A1 (en) 2020-11-26
KR20220015500A (ko) 2022-02-08
US10689625B2 (en) 2020-06-23
US20200308554A1 (en) 2020-10-01
JP2020537544A (ja) 2020-12-24
KR20200074132A (ko) 2020-06-24
JP7336730B2 (ja) 2023-09-01
US11661585B2 (en) 2023-05-30
JP2022068158A (ja) 2022-05-09
US20200071680A1 (en) 2020-03-05
KR102709597B1 (ko) 2024-09-26
US11655460B2 (en) 2023-05-23
JP2022068157A (ja) 2022-05-09
KR20220015499A (ko) 2022-02-08
CA3078464A1 (en) 2019-04-11
IL273771A (en) 2020-05-31
CN112553210B (zh) 2024-09-24
WO2019070894A1 (en) 2019-04-11
IL279930A (en) 2021-03-01
US11993790B2 (en) 2024-05-28
US20200282080A1 (en) 2020-09-10
CN112481269A (zh) 2021-03-12
MX2020003965A (es) 2020-10-05
CN112553210A (zh) 2021-03-26
KR102697811B1 (ko) 2024-08-22

Similar Documents

Publication Publication Date Title
JP7389828B2 (ja) ライソゾーム病の遺伝子治療
JP7254815B2 (ja) ライソゾーム病の遺伝子治療
US20250011783A1 (en) Gene therapy for neurodegenerative disorders
CA3134841A1 (en) Gene therapies for lysosomal disorders
US20220211871A1 (en) Gene therapies for lysosomal disorders
JP2022526823A (ja) リソソーム障害のための遺伝子療法
JP2022527015A (ja) リソソーム障害のための遺伝子治療

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211001

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20211001

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220909

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20221209

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230317

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230616

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20230831

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20231002

R150 Certificate of patent or registration of utility model

Ref document number: 7361037

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

RD02 Notification of acceptance of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: R3D02

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: R3D04