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MX2022014151A - Oligomeros antisentido de opa1 para tratamiento de afecciones y enfermedades. - Google Patents

Oligomeros antisentido de opa1 para tratamiento de afecciones y enfermedades.

Info

Publication number
MX2022014151A
MX2022014151A MX2022014151A MX2022014151A MX2022014151A MX 2022014151 A MX2022014151 A MX 2022014151A MX 2022014151 A MX2022014151 A MX 2022014151A MX 2022014151 A MX2022014151 A MX 2022014151A MX 2022014151 A MX2022014151 A MX 2022014151A
Authority
MX
Mexico
Prior art keywords
opa1
diseases
treatment
conditions
antisense oligomers
Prior art date
Application number
MX2022014151A
Other languages
English (en)
Inventor
Isabel Aznarez
Gene Liau
Aditya Venkatesh
Original Assignee
Stoke 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 Stoke Therapeutics Inc filed Critical Stoke Therapeutics Inc
Publication of MX2022014151A publication Critical patent/MX2022014151A/es

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    • 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
    • C12N15/11—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
    • C12N15/1137—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 against enzymes
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    • C12N15/09—Recombinant DNA-technology
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    • 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
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    • C12Y306/05—Hydrolases acting on acid anhydrides (3.6) acting on GTP; involved in cellular and subcellular movement (3.6.5)
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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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
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Abstract

Los eventos de escisión y empalme alternativo en genes pueden dar lugar a transcriptos de mRNA no productivos que, a su vez, pueden dar lugar a una expresión proteica aberrante y agentes terapéuticos que pueden dirigirse a eventos de escisión y empalme alternativo en genes pueden modular el nivel de expresión de las proteínas funcionales en pacientes y/o inhibir la expresión proteica aberrante. Dichos agentes terapéuticos pueden utilizarse para tratar una afección o enfermedad causada por la deficiencia de proteínas y/o el déficit de la función mitocondrial.
MX2022014151A 2020-05-11 2021-04-30 Oligomeros antisentido de opa1 para tratamiento de afecciones y enfermedades. MX2022014151A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063023013P 2020-05-11 2020-05-11
US202063112458P 2020-11-11 2020-11-11
PCT/US2021/030254 WO2021231107A1 (en) 2020-05-11 2021-04-30 Opa1 antisense oligomers for treatment of conditions and diseases

Publications (1)

Publication Number Publication Date
MX2022014151A true MX2022014151A (es) 2022-11-30

Family

ID=78526029

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MX2022014151A MX2022014151A (es) 2020-05-11 2021-04-30 Oligomeros antisentido de opa1 para tratamiento de afecciones y enfermedades.

Country Status (12)

Country Link
US (3) US12338437B2 (es)
EP (1) EP4150092A4 (es)
JP (1) JP2023525799A (es)
KR (1) KR20230022409A (es)
CN (1) CN115867657A (es)
AU (1) AU2021270720A1 (es)
BR (1) BR112022022889A2 (es)
CA (1) CA3173647A1 (es)
IL (1) IL298063A (es)
MX (1) MX2022014151A (es)
TW (1) TWI901675B (es)
WO (1) WO2021231107A1 (es)

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EP4222265A4 (en) * 2020-10-02 2025-07-16 Pyc Therapeutics Ltd TREATMENT OF OPTIC ATROPHY
US20250002917A1 (en) * 2022-01-31 2025-01-02 PYC Therapeutics Limited Method of treatment for optic atrophy
IL315133A (en) * 2022-03-23 2024-10-01 Pyc Therapeutics Ltd Methods of treating glaucoma
EP4594505A1 (en) * 2022-09-30 2025-08-06 Centre National de la Recherche Scientifique Therapy by trans-splicing of opa1 pre-messenger rnas for the treatment of diseases associated with opa1 gene mutations
AU2023374390A1 (en) * 2022-10-31 2025-05-29 Stoke Therapeutics, Inc. Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
WO2024221049A1 (en) * 2023-04-28 2024-10-31 PYC Therapeutics Limited Agents and method of treatment for optic conditions
AU2024282117A1 (en) * 2023-05-31 2026-01-08 Stoke Therapeutics, Inc. Opa1 antisense oligomers for treatment of conditions and diseases

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