MX2021006359A - Vectores virales de vaa y sus usos. - Google Patents

Vectores virales de vaa y sus usos.

Info

Publication number
MX2021006359A
MX2021006359A MX2021006359A MX2021006359A MX2021006359A MX 2021006359 A MX2021006359 A MX 2021006359A MX 2021006359 A MX2021006359 A MX 2021006359A MX 2021006359 A MX2021006359 A MX 2021006359A MX 2021006359 A MX2021006359 A MX 2021006359A
Authority
MX
Mexico
Prior art keywords
viral vectors
aav viral
patients
sma
type
Prior art date
Application number
MX2021006359A
Other languages
English (en)
Inventor
Robert Emil Hodge
Brian K Kaspar
James Michael Hatfield
Joseph Balleydier
Allan Arman Kaspar
Douglas Feltner
Matthew Meriggioli
Original Assignee
Novartis Ag
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 Novartis Ag filed Critical Novartis Ag
Publication of MX2021006359A publication Critical patent/MX2021006359A/es

Links

Classifications

    • 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
    • A61K48/0075—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
    • A61K48/0083—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • 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

Abstract

Se divulgan en la presente composiciones que comprenden vectores virales VAA9 y sus métodos de uso para tratar pacientes con AME, por ejemplo, pacientes con atrofia muscular espinal (AME) Tipo II y Tipo III.
MX2021006359A 2018-11-30 2019-11-27 Vectores virales de vaa y sus usos. MX2021006359A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862773894P 2018-11-30 2018-11-30
US201962835242P 2019-04-17 2019-04-17
PCT/US2019/063649 WO2020113034A1 (en) 2018-11-30 2019-11-27 Aav viral vectors and uses thereof

Publications (1)

Publication Number Publication Date
MX2021006359A true MX2021006359A (es) 2021-08-11

Family

ID=69167903

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021006359A MX2021006359A (es) 2018-11-30 2019-11-27 Vectores virales de vaa y sus usos.

Country Status (12)

Country Link
US (1) US20220001028A1 (es)
EP (1) EP3886919A1 (es)
JP (1) JP2022511776A (es)
KR (1) KR20210099025A (es)
CN (1) CN113226380A (es)
AU (1) AU2019389047A1 (es)
BR (1) BR112021009739A2 (es)
CA (1) CA3116630A1 (es)
IL (1) IL282885A (es)
MX (1) MX2021006359A (es)
TW (1) TW202039859A (es)
WO (1) WO2020113034A1 (es)

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Publication number Priority date Publication date Assignee Title
JP2021534154A (ja) * 2018-08-15 2021-12-09 バイオジェン・エムエイ・インコーポレイテッドBiogen MA Inc. 脊髄性筋萎縮症のための併用療法
TW202140791A (zh) * 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
CN112121179A (zh) * 2020-09-15 2020-12-25 山东兴瑞生物科技有限公司 一种组合物及其在治疗脊髓性肌萎缩症中的应用
EP4277915A1 (en) * 2021-01-13 2023-11-22 Dignity Health Modulation of chitinase protein expression
WO2023010133A2 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
AU2022318664A1 (en) 2021-07-30 2024-02-29 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
WO2023015189A2 (en) * 2021-08-03 2023-02-09 The Regents Of The University Of California Adeno-associated viral (aav)-mediated sphingosine-1-phosphate lyase (spl) expression for treating pulmonary fibrosis
CN114276419B (zh) * 2021-12-30 2023-11-17 上海勉亦生物科技有限公司 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用
KR20230152503A (ko) * 2022-04-27 2023-11-03 주식회사 헬릭스미스 척수강 내 투여에 최적화 된 간세포 성장인자 유전자가 도입된 aav 벡터
KR20230159287A (ko) * 2022-05-10 2023-11-21 서울대학교산학협력단 인간 smn1 단백질 변이체 및 이의 용도
WO2023246734A1 (en) 2022-06-21 2023-12-28 Skyline Therapeutics (Shanghai) Co., Ltd. Recombinant aav for the gene therapy of sma disease
WO2024015881A2 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. Compositions, systems, and methods for targeted transcriptional activation

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PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
AU688428B2 (en) 1993-11-09 1998-03-12 Johns Hopkins University, The Generation of high titers of recombinant AAV vectors
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Also Published As

Publication number Publication date
EP3886919A1 (en) 2021-10-06
BR112021009739A2 (pt) 2021-10-19
AU2019389047A1 (en) 2021-05-20
CN113226380A (zh) 2021-08-06
KR20210099025A (ko) 2021-08-11
CA3116630A1 (en) 2020-06-04
JP2022511776A (ja) 2022-02-01
US20220001028A1 (en) 2022-01-06
TW202039859A (zh) 2020-11-01
WO2020113034A1 (en) 2020-06-04
IL282885A (en) 2021-06-30

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