MX2023002755A - Composiciones y metodos para el tratamiento de la distrofia muscular de duchenne. - Google Patents

Composiciones y metodos para el tratamiento de la distrofia muscular de duchenne.

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Publication number
MX2023002755A
MX2023002755A MX2023002755A MX2023002755A MX2023002755A MX 2023002755 A MX2023002755 A MX 2023002755A MX 2023002755 A MX2023002755 A MX 2023002755A MX 2023002755 A MX2023002755 A MX 2023002755A MX 2023002755 A MX2023002755 A MX 2023002755A
Authority
MX
Mexico
Prior art keywords
compositions
methods
muscular dystrophy
treatment
duchenne muscular
Prior art date
Application number
MX2023002755A
Other languages
English (en)
Inventor
Eric Anderson
Yurong Xin
Jesper Gromada
Tudor Fulga
Alison Mcvie-Wylie
Gutierrez Giselle Dominguez
Yi-Li Min
Mehmet Fatih Bolukbasi
Original Assignee
Vertex Pharma
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 Vertex Pharma filed Critical Vertex Pharma
Priority claimed from PCT/US2021/049468 external-priority patent/WO2022056000A1/en
Publication of MX2023002755A publication Critical patent/MX2023002755A/es

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
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    • 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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
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    • C07K2319/41—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a Myc-tag
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • 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

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  • General Chemical & Material Sciences (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Abstract

Se abarcan composiciones y métodos para el tratamiento de la distrofia muscular de Duchenne (DMD).
MX2023002755A 2020-09-09 2021-09-08 Composiciones y metodos para el tratamiento de la distrofia muscular de duchenne. MX2023002755A (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063076250P 2020-09-09 2020-09-09
US202163152114P 2021-02-22 2021-02-22
US202163166174P 2021-03-25 2021-03-25
US202163179850P 2021-04-26 2021-04-26
PCT/US2021/049468 WO2022056000A1 (en) 2020-09-09 2021-09-08 Compositions and methods for treatment of duchenne muscular dystrophy

Publications (1)

Publication Number Publication Date
MX2023002755A true MX2023002755A (es) 2023-05-19

Family

ID=85685062

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023002755A MX2023002755A (es) 2020-09-09 2021-09-08 Composiciones y metodos para el tratamiento de la distrofia muscular de duchenne.

Country Status (7)

Country Link
EP (1) EP4211242A1 (es)
JP (1) JP2023540783A (es)
KR (1) KR20230084491A (es)
AU (1) AU2021342057A1 (es)
CA (1) CA3193753A1 (es)
IL (1) IL300949A (es)
MX (1) MX2023002755A (es)

Also Published As

Publication number Publication date
CA3193753A1 (en) 2022-03-17
AU2021342057A1 (en) 2023-04-06
KR20230084491A (ko) 2023-06-13
IL300949A (en) 2023-04-01
JP2023540783A (ja) 2023-09-26
EP4211242A1 (en) 2023-07-19

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