BR112022021635A2 - Distrofinas miniaturizadas tendo domínios de fusão de espectrina e usos das mesmas - Google Patents

Distrofinas miniaturizadas tendo domínios de fusão de espectrina e usos das mesmas

Info

Publication number
BR112022021635A2
BR112022021635A2 BR112022021635A BR112022021635A BR112022021635A2 BR 112022021635 A2 BR112022021635 A2 BR 112022021635A2 BR 112022021635 A BR112022021635 A BR 112022021635A BR 112022021635 A BR112022021635 A BR 112022021635A BR 112022021635 A2 BR112022021635 A2 BR 112022021635A2
Authority
BR
Brazil
Prior art keywords
miniaturized
spectrin
fusion domains
dystrophins
miniaturized dystrophins
Prior art date
Application number
BR112022021635A
Other languages
English (en)
Inventor
Banks Glen
Harry Davis Jonathan
Charles Levesque Paul
Original Assignee
Bristol Myers Squibb Co
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 Bristol Myers Squibb Co filed Critical Bristol Myers Squibb Co
Publication of BR112022021635A2 publication Critical patent/BR112022021635A2/pt

Links

Classifications

    • 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
    • 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
    • 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
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Abstract

DISTROFINAS MINIATURIZADAS TENDO DOMÍNIOS DE FUSÃO DE ESPECTRINA E USOS DAS MESMAS. São descritos aqui moléculas de ácido nucleico, polipeptídeos, células, vetores e composições farmacêuticas relacionando uma distrofina miniaturizada. Métodos de produção e método de uso terapêutico da disfrofina miniaturizada são também descritos
BR112022021635A 2020-04-29 2021-04-28 Distrofinas miniaturizadas tendo domínios de fusão de espectrina e usos das mesmas BR112022021635A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063017148P 2020-04-29 2020-04-29
PCT/US2021/029514 WO2021222332A1 (en) 2020-04-29 2021-04-28 Miniaturized dystrophins having spectrin fusion domains and uses thereof

Publications (1)

Publication Number Publication Date
BR112022021635A2 true BR112022021635A2 (pt) 2022-12-06

Family

ID=75977820

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022021635A BR112022021635A2 (pt) 2020-04-29 2021-04-28 Distrofinas miniaturizadas tendo domínios de fusão de espectrina e usos das mesmas

Country Status (13)

Country Link
US (2) US11535868B2 (pt)
EP (1) EP4143216A1 (pt)
JP (1) JP2023524713A (pt)
KR (1) KR20230003557A (pt)
CN (1) CN115485291A (pt)
AR (1) AR121954A1 (pt)
AU (1) AU2021265103A1 (pt)
BR (1) BR112022021635A2 (pt)
CA (1) CA3174312A1 (pt)
IL (1) IL297753A (pt)
MX (1) MX2022013477A (pt)
TW (1) TW202206447A (pt)
WO (1) WO2021222332A1 (pt)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE102020131374B4 (de) 2020-11-26 2024-02-08 Dionex Softron Gmbh Fluoreszenzdetektion

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Also Published As

Publication number Publication date
AR121954A1 (es) 2022-07-27
CA3174312A1 (en) 2021-11-04
AU2021265103A1 (en) 2023-01-19
JP2023524713A (ja) 2023-06-13
US20230407331A1 (en) 2023-12-21
MX2022013477A (es) 2022-11-30
US11535868B2 (en) 2022-12-27
CN115485291A (zh) 2022-12-16
KR20230003557A (ko) 2023-01-06
WO2021222332A1 (en) 2021-11-04
TW202206447A (zh) 2022-02-16
IL297753A (en) 2022-12-01
EP4143216A1 (en) 2023-03-08
US20210340195A1 (en) 2021-11-04

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B11A Dismissal acc. art.33 of ipl - examination not requested within 36 months of filing