HUE063755T2 - Adenoasszociált vírusok variánsai és alkalmazási eljárások - Google Patents

Adenoasszociált vírusok variánsai és alkalmazási eljárások

Info

Publication number
HUE063755T2
HUE063755T2 HUE17734575A HUE17734575A HUE063755T2 HU E063755 T2 HUE063755 T2 HU E063755T2 HU E17734575 A HUE17734575 A HU E17734575A HU E17734575 A HUE17734575 A HU E17734575A HU E063755 T2 HUE063755 T2 HU E063755T2
Authority
HU
Hungary
Prior art keywords
methods
associated viruses
variant adeno
adeno
variant
Prior art date
Application number
HUE17734575A
Other languages
English (en)
Inventor
Joshua Dudman
Adam Hantman
Loren Looger
Kimberly Ritola
David Schaffer
Dougal Gowanlock Robinson Tervo
Sarada Viswanathan
Bum-Yeol Hwang
Alla Karpova
Original Assignee
Hughes Howard Med Inst
Univ California
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 Hughes Howard Med Inst, Univ California filed Critical Hughes Howard Med Inst
Publication of HUE063755T2 publication Critical patent/HUE063755T2/hu

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01DNA viruses
    • C07K14/015Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02Muscle relaxants, e.g. for tetanus or cramps
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/02Drugs for disorders of the nervous system for peripheral neuropathies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/18Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C40COMBINATORIAL TECHNOLOGY
    • C40BCOMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B30/00Methods of screening libraries
    • C40B30/06Methods of screening libraries by measuring effects on living organisms, tissues or cells
    • CCHEMISTRY; METALLURGY
    • C40COMBINATORIAL TECHNOLOGY
    • C40BCOMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B40/00Libraries per se, e.g. arrays, mixtures
    • C40B40/04Libraries containing only organic compounds
    • C40B40/06Libraries containing nucleotides or polynucleotides, or derivatives thereof
    • C40B40/08Libraries containing RNA or DNA which encodes proteins, e.g. gene libraries
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
HUE17734575A 2016-06-15 2017-06-15 Adenoasszociált vírusok variánsai és alkalmazási eljárások HUE063755T2 (hu)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662350361P 2016-06-15 2016-06-15
US201662404585P 2016-10-05 2016-10-05

Publications (1)

Publication Number Publication Date
HUE063755T2 true HUE063755T2 (hu) 2024-01-28

Family

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HUE17734575A HUE063755T2 (hu) 2016-06-15 2017-06-15 Adenoasszociált vírusok variánsai és alkalmazási eljárások

Country Status (27)

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US (2) US10961282B2 (hu)
EP (2) EP4268852A3 (hu)
JP (2) JP7094277B2 (hu)
KR (2) KR102522661B1 (hu)
CN (2) CN116003532A (hu)
AU (2) AU2017286652B2 (hu)
BR (1) BR112018076090A2 (hu)
CA (1) CA3028113A1 (hu)
DK (1) DK3472183T3 (hu)
EA (1) EA201990033A1 (hu)
ES (1) ES2962611T3 (hu)
FI (1) FI3472183T3 (hu)
HR (1) HRP20231403T1 (hu)
HU (1) HUE063755T2 (hu)
IL (2) IL263719B2 (hu)
LT (1) LT3472183T (hu)
MA (1) MA44546B1 (hu)
MX (1) MX2018015770A (hu)
MY (1) MY190221A (hu)
PH (1) PH12018502664A1 (hu)
PL (1) PL3472183T3 (hu)
PT (1) PT3472183T (hu)
RS (1) RS64769B1 (hu)
SG (1) SG11201811189RA (hu)
SI (1) SI3472183T1 (hu)
WO (1) WO2017218842A1 (hu)
ZA (1) ZA201900278B (hu)

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JP6600624B2 (ja) 2013-05-31 2019-10-30 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア アデノ随伴ウイルス変異体及びその使用方法
UA124343C2 (uk) 2016-05-13 2021-09-01 4Д Молекьюлар Терапьютікс Інк. Капсиди аденоасоційованого вірусу і спосіб його використання
WO2018022905A2 (en) 2016-07-29 2018-02-01 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
CA3059995A1 (en) 2017-08-28 2019-03-07 The Regents Of The University Of California Adeno-associated virus capsid variants and methods of use thereof
CN111601884A (zh) * 2017-10-16 2020-08-28 维格内罗有限责任公司 Aav载体
KR20210052450A (ko) * 2018-07-31 2021-05-10 코넬 유니버시티 기관 기능을 제어하기 위한 유전자 요법 방법
WO2020068990A1 (en) 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
WO2021022208A1 (en) * 2019-08-01 2021-02-04 Kaplitt Michael G Targeted gene therapy to treat neurological diseases
CN111825772B (zh) * 2020-07-30 2023-10-20 中国科学院精密测量科学与技术创新研究院 具有变异衣壳蛋白的腺相关病毒及其应用
CN116390773A (zh) 2020-08-06 2023-07-04 应用医学研究基金会 用于通过基因疗法治疗诸如阿尔茨海默病的Tau蛋白病的病毒颗粒
EP4263575A1 (en) * 2020-12-16 2023-10-25 Children's Medical Research Institute Adeno-associated virus capsids and vectors
AU2021399882A1 (en) * 2020-12-16 2023-07-06 Children's Medical Research Institute Aav capsids and vectors
CN117730154A (zh) 2021-06-17 2024-03-19 梅里特斯英国第二有限公司 Aav制造方法
IL310018A (en) 2021-07-14 2024-03-01 Meiragtx Uk Ii Ltd KCNV2 gene therapy
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WO2023288184A2 (en) * 2021-07-14 2023-01-19 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for treating chronic pain and for retrograde transduction of neurons
WO2023131811A2 (en) 2021-12-15 2023-07-13 Meiragtx Uk Ii Limited Polycistronic expression of gut peptides
TW202346600A (zh) * 2022-03-30 2023-12-01 高等教育聯邦系統 匹茲堡大學 用於將核酸遞送至視網膜神經節細胞和/或視網膜色素上皮細胞的腺相關病毒載體
WO2024056902A2 (en) 2022-09-16 2024-03-21 Christopher Shaw Compositions and methods for treating neurological diseases
WO2024079665A1 (en) 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Nucleic acid regulatory elements for constitutive gene expression and methods of use
WO2024079655A1 (en) 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Chromatography methods for purification of aav capsids
WO2024079661A1 (en) 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Atp7b gene therapy
WO2024079662A1 (en) 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Upf1 expression constructs
WO2024079657A1 (en) 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Formulations for aav gene therapy

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WO2018022905A2 (en) * 2016-07-29 2018-02-01 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof

Also Published As

Publication number Publication date
EP3472183A1 (en) 2019-04-24
AU2017286652A1 (en) 2019-01-24
MA44546B1 (fr) 2021-03-31
DK3472183T3 (da) 2023-11-06
WO2017218842A1 (en) 2017-12-21
AU2021225247A1 (en) 2021-09-30
EP3472183B1 (en) 2023-08-09
HRP20231403T1 (hr) 2024-02-16
IL263719B1 (en) 2023-05-01
JP7094277B2 (ja) 2022-07-01
ZA201900278B (en) 2022-04-28
AU2021225247B2 (en) 2023-02-16
MX2018015770A (es) 2019-08-29
LT3472183T (lt) 2023-09-25
PL3472183T3 (pl) 2024-02-12
ES2962611T3 (es) 2024-03-20
JP2022126759A (ja) 2022-08-30
US11939355B2 (en) 2024-03-26
CN116003532A (zh) 2023-04-25
KR20190039930A (ko) 2019-04-16
US20210230231A1 (en) 2021-07-29
PT3472183T (pt) 2023-11-16
SG11201811189RA (en) 2019-01-30
KR20230054754A (ko) 2023-04-25
MA44546A1 (fr) 2019-07-31
JP2019518793A (ja) 2019-07-04
EA201990033A1 (ru) 2019-06-28
US10961282B2 (en) 2021-03-30
RS64769B1 (sr) 2023-11-30
CN109641939B (zh) 2022-11-08
PH12018502664A1 (en) 2019-10-07
EP4268852A3 (en) 2024-02-07
IL263719A (en) 2019-01-31
CA3028113A1 (en) 2017-12-21
EP4268852A2 (en) 2023-11-01
MY190221A (en) 2022-04-06
IL300981A (en) 2023-04-01
US20190300579A1 (en) 2019-10-03
SI3472183T1 (sl) 2023-11-30
KR102522661B1 (ko) 2023-04-18
BR112018076090A2 (pt) 2019-03-26
IL263719B2 (en) 2023-09-01
FI3472183T3 (fi) 2023-11-03
CN109641939A (zh) 2019-04-16
AU2017286652B2 (en) 2021-10-07

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