MX2018015770A - Variantes de virus adenoasociados y metodos para usarlos. - Google Patents

Variantes de virus adenoasociados y metodos para usarlos.

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Publication number
MX2018015770A
MX2018015770A MX2018015770A MX2018015770A MX2018015770A MX 2018015770 A MX2018015770 A MX 2018015770A MX 2018015770 A MX2018015770 A MX 2018015770A MX 2018015770 A MX2018015770 A MX 2018015770A MX 2018015770 A MX2018015770 A MX 2018015770A
Authority
MX
Mexico
Prior art keywords
methods
associated viruses
variant adeno
adeno
variant
Prior art date
Application number
MX2018015770A
Other languages
English (en)
Inventor
Schaffer David
Dudman Joshua
Hantman Adam
Looger Loren
Ritola Kimberly
Gowanlock Robinson Tervo Dougal
Viswanathan Sarada
Karpova Alla
Hwang Bum-Yeol
Original Assignee
Hughes Howard Med Inst
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Filing date
Publication date
Application filed by Hughes Howard Med Inst filed Critical Hughes Howard Med Inst
Publication of MX2018015770A publication Critical patent/MX2018015770A/es

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    • 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
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • 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
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B30/00—Methods of screening libraries
    • C40B30/06—Methods of screening libraries by measuring effects on living organisms, tissues or cells
    • C—CHEMISTRY; METALLURGY
    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B40/00—Libraries per se, e.g. arrays, mixtures
    • C40B40/04—Libraries containing only organic compounds
    • C40B40/06—Libraries containing nucleotides or polynucleotides, or derivatives thereof
    • C40B40/08—Libraries containing RNA or DNA which encodes proteins, e.g. gene libraries
    • 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
    • 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
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    • 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
    • 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
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • Y—GENERAL 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
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Abstract

La presente divulgación provee variantes de AAV que exhiben preferencia por el movimiento retrógrado en las neuronas y métodos de uso de dichas variantes.
MX2018015770A 2016-06-15 2017-06-15 Variantes de virus adenoasociados y metodos para usarlos. MX2018015770A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662350361P 2016-06-15 2016-06-15
US201662404585P 2016-10-05 2016-10-05
PCT/US2017/037781 WO2017218842A1 (en) 2016-06-15 2017-06-15 Variant adeno-associated viruses and methods of using

Publications (1)

Publication Number Publication Date
MX2018015770A true MX2018015770A (es) 2019-08-29

Family

ID=59258370

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018015770A MX2018015770A (es) 2016-06-15 2017-06-15 Variantes de virus adenoasociados y metodos para usarlos.

Country Status (27)

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US (2) US10961282B2 (es)
EP (2) EP4268852A3 (es)
JP (2) JP7094277B2 (es)
KR (2) KR20230054754A (es)
CN (2) CN109641939B (es)
AU (2) AU2017286652B2 (es)
BR (1) BR112018076090A2 (es)
CA (1) CA3028113A1 (es)
DK (1) DK3472183T3 (es)
EA (1) EA201990033A1 (es)
ES (1) ES2962611T3 (es)
FI (1) FI3472183T3 (es)
HR (1) HRP20231403T1 (es)
HU (1) HUE063755T2 (es)
IL (2) IL300981A (es)
LT (1) LT3472183T (es)
MA (1) MA44546B1 (es)
MX (1) MX2018015770A (es)
MY (1) MY190221A (es)
PH (1) PH12018502664A1 (es)
PL (1) PL3472183T3 (es)
PT (1) PT3472183T (es)
RS (1) RS64769B1 (es)
SG (1) SG11201811189RA (es)
SI (1) SI3472183T1 (es)
WO (1) WO2017218842A1 (es)
ZA (1) ZA201900278B (es)

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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
EP4206216A1 (en) 2016-05-13 2023-07-05 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
JP7071332B2 (ja) 2016-07-29 2022-05-18 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 変異カプシドを有するアデノ随伴ウイルスビリオン及びその使用方法
AU2018324477A1 (en) 2017-08-28 2019-10-17 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
CA3108324A1 (en) * 2018-07-31 2020-02-06 Cornell University Gene therapy methods to control organ function
KR20210084459A (ko) 2018-09-26 2021-07-07 캘리포니아 인스티튜트 오브 테크놀로지 표적화된 유전자 요법을 위한 아데노-연관 바이러스 조성물
EP4007609A1 (en) * 2019-08-01 2022-06-08 Cornell University 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
JP2023536080A (ja) 2020-08-06 2023-08-23 ファンダシオン パラ ラ インベスティガシオン メディカ アプリカダ 遺伝子治療によるアルツハイマー病などのタウオパチーの治療に使用するためのウイルス粒子
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EP4263575A1 (en) * 2020-12-16 2023-10-25 Children's Medical Research Institute Adeno-associated virus capsids and vectors
WO2022263930A2 (en) 2021-06-17 2022-12-22 Meiragtx Uk Ii Limited Aav manufacturing methods
IL310017A (en) 2021-07-14 2024-03-01 Meiragtx Uk Ii Ltd RETGC gene therapy
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
WO2023285986A1 (en) 2021-07-14 2023-01-19 Meiragtx Uk Ii Limited Kcnv2 gene therapy
WO2023131811A2 (en) 2021-12-15 2023-07-13 Meiragtx Uk Ii Limited Polycistronic expression of gut peptides
WO2023192450A1 (en) * 2022-03-30 2023-10-05 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Adeno-associated virus vectors for nucleic acid delivery to retinal ganglion cells and/or retinal pigment epithelium cells
WO2024056902A2 (en) 2022-09-16 2024-03-21 Christopher Shaw Compositions and methods for treating neurological diseases

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AU2017286652A1 (en) 2019-01-24
EA201990033A1 (ru) 2019-06-28
US11939355B2 (en) 2024-03-26
US20210230231A1 (en) 2021-07-29
WO2017218842A1 (en) 2017-12-21
PT3472183T (pt) 2023-11-16
AU2017286652B2 (en) 2021-10-07
EP3472183B1 (en) 2023-08-09
MA44546A1 (fr) 2019-07-31
CN116003532A (zh) 2023-04-25
EP4268852A2 (en) 2023-11-01
IL263719A (en) 2019-01-31
AU2021225247A1 (en) 2021-09-30
EP3472183A1 (en) 2019-04-24
MA44546B1 (fr) 2021-03-31
IL300981A (en) 2023-04-01
JP2019518793A (ja) 2019-07-04
MY190221A (en) 2022-04-06
HUE063755T2 (hu) 2024-01-28
IL263719B1 (en) 2023-05-01
BR112018076090A2 (pt) 2019-03-26
SI3472183T1 (sl) 2023-11-30
PH12018502664A1 (en) 2019-10-07
KR20230054754A (ko) 2023-04-25
PL3472183T3 (pl) 2024-02-12
DK3472183T3 (da) 2023-11-06
ZA201900278B (en) 2022-04-28
US20190300579A1 (en) 2019-10-03
SG11201811189RA (en) 2019-01-30
KR102522661B1 (ko) 2023-04-18
LT3472183T (lt) 2023-09-25
CN109641939A (zh) 2019-04-16
JP7094277B2 (ja) 2022-07-01
FI3472183T3 (fi) 2023-11-03
ES2962611T3 (es) 2024-03-20
JP2022126759A (ja) 2022-08-30
US10961282B2 (en) 2021-03-30
HRP20231403T1 (hr) 2024-02-16
AU2021225247B2 (en) 2023-02-16
RS64769B1 (sr) 2023-11-30
KR20190039930A (ko) 2019-04-16
EP4268852A3 (en) 2024-02-07
CA3028113A1 (en) 2017-12-21
CN109641939B (zh) 2022-11-08
IL263719B2 (en) 2023-09-01

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