CA3173647A1 - Opa1 antisense oligomers for treatment of conditions and diseases - Google Patents

Opa1 antisense oligomers for treatment of conditions and diseases Download PDF

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Publication number
CA3173647A1
CA3173647A1 CA3173647A CA3173647A CA3173647A1 CA 3173647 A1 CA3173647 A1 CA 3173647A1 CA 3173647 A CA3173647 A CA 3173647A CA 3173647 A CA3173647 A CA 3173647A CA 3173647 A1 CA3173647 A1 CA 3173647A1
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Prior art keywords
fold
nucleotides
mrna
exon
targeted portion
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Pending
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CA3173647A
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English (en)
French (fr)
Inventor
Isabel AZNAREZ
Aditya VENKATESH
Gene Liau
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Stoke Therapeutics Inc
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Stoke Therapeutics Inc
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Publication of CA3173647A1 publication Critical patent/CA3173647A1/en
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
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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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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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)
    • C12Y306/05005—Dynamin GTPase (3.6.5.5)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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    • C12N2320/00—Applications; Uses
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    • C12N2320/33—Alteration of splicing
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    • 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

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  • Ophthalmology & Optometry (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
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CA3173647A 2020-05-11 2021-04-30 Opa1 antisense oligomers for treatment of conditions and diseases Pending CA3173647A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063023013P 2020-05-11 2020-05-11
US63/023,013 2020-05-11
US202063112458P 2020-11-11 2020-11-11
US63/112,458 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
CA3173647A1 true CA3173647A1 (en) 2021-11-18

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ID=78526029

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CA3173647A Pending CA3173647A1 (en) 2020-05-11 2021-04-30 Opa1 antisense oligomers for treatment of conditions and diseases

Country Status (11)

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

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
AU2021354152A1 (en) * 2020-10-02 2023-06-01 PYC Therapeutics Limited Treatment of optic atrophy
AU2023212479A1 (en) * 2022-01-31 2024-08-01 PYC Therapeutics Limited Method of treatment for optic atrophy
KR20240165951A (ko) * 2022-03-23 2024-11-25 피와이씨 테라퓨틱스 리미티드 녹내장 치료 방법
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
EP4612295A1 (en) * 2022-10-31 2025-09-10 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
EP4720297A2 (en) * 2023-05-31 2026-04-08 Stoke Therapeutics, Inc. Opa1 antisense oligomers for treatment of conditions and diseases

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