Compounds and methods for modulating ube3a-ats

A technology of oligomeric compounds, substitutes, applied in the field of compounds and methods for modulating UBE3A-ATS, which can solve the problems of lack of acceptable options, unknowns, damages, etc. for the treatment of neurogenetic disorders

Pending Publication Date: 2021-11-30
IONIS PHARMA INC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

However, the exact mechanism by which paternal UBE3A expression is not silenced remains unknown and topoisomerase inhibitors are fraught with safety concerns as they are known to be non-...

Method used

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  • Compounds and methods for modulating ube3a-ats
  • Compounds and methods for modulating ube3a-ats
  • Compounds and methods for modulating ube3a-ats

Examples

Experimental program
Comparison scheme
Effect test

Embodiment approach 1

[0071] Embodiment 1. An oligomeric compound comprises a modified oligonucleotide consisting of 12 to 30 connected nucleosides, wherein the nucleobase sequence of the modified oligonucleotide is the same as that of UBE3A-ATS RNA Portions of equal length are at least 90% complementary, and wherein said modified oligonucleotide comprises at least one modification selected from modified sugar moieties and modified internucleoside linkages.

Embodiment approach 2

[0072] Embodiment 2. An oligomeric compound comprising 12 to 30 linked nucleosides comprising at least 12, 13, Modified oligonucleotides of a nucleobase sequence of 14, 15, 16, 17, 18, 19 or 20 contiguous nucleobases.

Embodiment approach 3

[0073] Embodiment 3. An oligomeric compound comprising 12 to 30 linked nucleosides comprising at least 12, 13, 14, 15 A modified oligonucleotide of a nucleobase sequence of 1, 16, 17 or 18 contiguous nucleobases.

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PUM

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Abstract

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A) in a cell or subject, and in certain instances increasing the expression of paternal UBE3A and the amount of UBE3A protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurogenetic disorder. Such symptoms and hallmarks include developmental delays, ataxia, speech impairment, sleep problems, seizures, and EEG abnormalities. Such neurogenetic disorders include Angelman Syndrome.

Description

[0001] sequence listing [0002] This application is filed with a sequence listing in electronic format. The sequence listing is provided as a document titled BIOL0349WOSEQ_ST25.tst, which was created on March 16, 2020, and is 1.87MB in size. The information in the electronic format of the Sequence Listing is incorporated herein by reference in its entirety. technical field [0003] Provides for reducing the amount or activity of an endogenous antisense transcript UBE3A-ATS of ubiquitin protein ligase E3A (UBE3A) in a cell or a subject and in certain instances increasing the parent UBE3A in a cell or a subject Compounds, methods and pharmaceutical compositions for the expression and amount of UBE3A protein. Such compounds, methods and pharmaceutical compositions are useful for ameliorating at least one symptom or marker of a neurogenetic disorder. Such symptoms and signs include developmental delays, movement disorders, speech disturbances, sleep problems, seizures, and EEG...

Claims

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Application Information

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IPC IPC(8): C12N15/113
CPCC12N15/113C12N2310/113C12N2310/315C12N2310/3341C12N2310/341C12N2310/346C12N2320/11C12N2310/321C12N2310/3525C12N2310/343C12N2310/111C12N2310/323C07H21/00
Inventor 苏珊·M·弗赖尔H-H·布维P·杰法尔-内贾德F·里戈
Owner IONIS PHARMA INC
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