KR20220046513A - 유아 바텐병의 치료를 위한 척수강내 및 정맥내 조합 유전자 요법 - Google Patents

유아 바텐병의 치료를 위한 척수강내 및 정맥내 조합 유전자 요법 Download PDF

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KR20220046513A
KR20220046513A KR1020217038864A KR20217038864A KR20220046513A KR 20220046513 A KR20220046513 A KR 20220046513A KR 1020217038864 A KR1020217038864 A KR 1020217038864A KR 20217038864 A KR20217038864 A KR 20217038864A KR 20220046513 A KR20220046513 A KR 20220046513A
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South Korea
Prior art keywords
polynucleotide
aav
viral particle
cln1
promoter
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KR1020217038864A
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Korean (ko)
Inventor
티모시 제이 밀러
스티븐 제이 그레이
Original Assignee
더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐
아베오나 테라퓨틱스 인코퍼레이티드
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Application filed by 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐, 아베오나 테라퓨틱스 인코퍼레이티드 filed Critical 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐
Publication of KR20220046513A publication Critical patent/KR20220046513A/ko
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    • 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
    • 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
    • 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
    • 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
    • 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
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
    • 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
    • C12N2750/14123—Virus like particles [VLP]
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pain & Pain Management (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (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)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020217038864A 2019-04-29 2020-04-29 유아 바텐병의 치료를 위한 척수강내 및 정맥내 조합 유전자 요법 Withdrawn KR20220046513A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962840360P 2019-04-29 2019-04-29
US62/840,360 2019-04-29
PCT/US2020/030427 WO2020223322A1 (en) 2019-04-29 2020-04-29 Intrathecal and intravenous combination gene therapy for the treatment of infantile batten disease

Publications (1)

Publication Number Publication Date
KR20220046513A true KR20220046513A (ko) 2022-04-14

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KR1020217038864A Withdrawn KR20220046513A (ko) 2019-04-29 2020-04-29 유아 바텐병의 치료를 위한 척수강내 및 정맥내 조합 유전자 요법

Country Status (12)

Country Link
US (1) US20220193268A1 (cg-RX-API-DMAC10.html)
EP (1) EP3963081A4 (cg-RX-API-DMAC10.html)
JP (1) JP7788594B2 (cg-RX-API-DMAC10.html)
KR (1) KR20220046513A (cg-RX-API-DMAC10.html)
CN (1) CN114269935A (cg-RX-API-DMAC10.html)
AU (1) AU2020264438A1 (cg-RX-API-DMAC10.html)
BR (1) BR112021021632A8 (cg-RX-API-DMAC10.html)
CA (1) CA3138274A1 (cg-RX-API-DMAC10.html)
IL (1) IL287608A (cg-RX-API-DMAC10.html)
MX (1) MX2021013275A (cg-RX-API-DMAC10.html)
SG (1) SG11202111908XA (cg-RX-API-DMAC10.html)
WO (1) WO2020223322A1 (cg-RX-API-DMAC10.html)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023303015A1 (en) * 2022-07-06 2025-01-23 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery of cln1 polynucleotide
WO2024015794A2 (en) * 2022-07-12 2024-01-18 The Research Institute At Nationwide Children's Hospital Adeno-associated virus gene therapy products and methods

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2550359B1 (en) * 2010-03-23 2017-08-09 Intrexon Corporation Vectors conditionally expressing therapeutic proteins, host cells comprising the vectors, and uses thereof
JP6754361B2 (ja) * 2014-12-16 2020-09-09 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法
JP2019517274A (ja) * 2016-06-13 2019-06-24 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒルＴｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｎｏｒｔｈ Ｃａｒｏｌｉｎａ Ａｔ Ｃｈａｐｅｌ Ｈｉｌｌ 最適化されたｃｌｎ１遺伝子および発現カセットおよびそれらの使用
WO2019011817A1 (en) * 2017-07-08 2019-01-17 Genethon TREATMENT OF SPINAL AMYOTROPHY
US20210115453A1 (en) 2017-08-31 2021-04-22 Poseida Therapeutics, Inc. Transposon system and methods of use

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Publication number Publication date
BR112021021632A8 (pt) 2022-06-28
US20220193268A1 (en) 2022-06-23
JP2022530264A (ja) 2022-06-28
CA3138274A1 (en) 2020-11-05
JP7788594B2 (ja) 2025-12-19
WO2020223322A1 (en) 2020-11-05
AU2020264438A1 (en) 2021-12-16
BR112021021632A2 (cg-RX-API-DMAC10.html) 2021-12-21
IL287608A (en) 2021-12-01
CN114269935A (zh) 2022-04-01
MX2021013275A (es) 2022-03-17
EP3963081A1 (en) 2022-03-09
SG11202111908XA (en) 2021-11-29
EP3963081A4 (en) 2023-07-26

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