BR112023024078A2 - Produção de vetores de aav recombinantes para o tratamento de distrofia muscular - Google Patents

Produção de vetores de aav recombinantes para o tratamento de distrofia muscular

Info

Publication number
BR112023024078A2
BR112023024078A2 BR112023024078A BR112023024078A BR112023024078A2 BR 112023024078 A2 BR112023024078 A2 BR 112023024078A2 BR 112023024078 A BR112023024078 A BR 112023024078A BR 112023024078 A BR112023024078 A BR 112023024078A BR 112023024078 A2 BR112023024078 A2 BR 112023024078A2
Authority
BR
Brazil
Prior art keywords
muscular dystrophy
treatment
production
aav vectors
recombinant aav
Prior art date
Application number
BR112023024078A
Other languages
English (en)
Inventor
Louise Rodino-Klapac
Maroof Alam
Original Assignee
Sarepta 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 Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Publication of BR112023024078A2 publication Critical patent/BR112023024078A2/pt

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
    • 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
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • 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
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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

produção de vetores de aav recombinantes para o tratamento de distrofia muscular. a presente revelação fornece vetores de terapia gênica, por exemplo, vírus adenoassociado recombinante (raav), produzido em células de mamíferos aderentes cultivadas em condições de suspensão, para expressão de um gene de microdistrofina humana. a presente revelação também fornece composições e métodos de utilização desses raav para tratar distrofia muscular como, por exemplo, distrofia muscular de duchenne.
BR112023024078A 2021-05-17 2022-05-13 Produção de vetores de aav recombinantes para o tratamento de distrofia muscular BR112023024078A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163189676P 2021-05-17 2021-05-17
US202163209733P 2021-06-11 2021-06-11
US202163243944P 2021-09-14 2021-09-14
US202163253998P 2021-10-08 2021-10-08
PCT/US2022/029328 WO2022245675A1 (en) 2021-05-17 2022-05-13 Production of recombinant aav vectors for treating muscular dystrophy

Publications (1)

Publication Number Publication Date
BR112023024078A2 true BR112023024078A2 (pt) 2024-01-30

Family

ID=82483227

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112023024078A BR112023024078A2 (pt) 2021-05-17 2022-05-13 Produção de vetores de aav recombinantes para o tratamento de distrofia muscular

Country Status (8)

Country Link
EP (1) EP4341413A1 (pt)
KR (1) KR20240021765A (pt)
AU (1) AU2022279062A1 (pt)
BR (1) BR112023024078A2 (pt)
CA (1) CA3218350A1 (pt)
CO (1) CO2023017400A2 (pt)
IL (1) IL308431A (pt)
WO (1) WO2022245675A1 (pt)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024064913A1 (en) * 2022-09-23 2024-03-28 Sarepta Therapeutics, Inc. Recombinant aav vectors for treating muscular dystrophy

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PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
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JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
BR112020025995A2 (pt) 2018-06-18 2021-03-23 Research Institute At Nationwide Children's Hospital administração de microdistrofina músculo-específica por vetor de vírus adeno-associado para tratar a distrofia muscular
SG11202104295UA (en) * 2018-11-14 2021-06-29 Regenxbio Inc Gene therapy for neuronal ceroid lipofuscinoses
CA3127629A1 (en) * 2019-01-25 2020-07-30 Biogen Ma Inc. Seed culture process for aav production

Also Published As

Publication number Publication date
CA3218350A1 (en) 2022-11-24
AU2022279062A1 (en) 2024-01-04
CO2023017400A2 (es) 2024-01-15
WO2022245675A1 (en) 2022-11-24
EP4341413A1 (en) 2024-03-27
IL308431A (en) 2024-01-01
KR20240021765A (ko) 2024-02-19

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