Thiomorpholino antisense oligonucleotides for treating PTP1B-related diseases

JP2025539260A5Pending Publication Date: 2026-10-07THE REGENTS OF THE UNIVERSITY OF COLORADO +1
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Patent Information

Application Number
JP2025527696
Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
Priority Date
2022-11-14
Filing Date
2023-11-13
Publication Date
2026-10-07

AI Technical Summary

Technical Problem

Current chemical approaches for splice switching, such as phosphorodiamidate morpholino oligomers (PMOs), 2'-O-methyl (2'-OMe), and 2'-O-methoxyethyl (2'-MOE) oligomers, face limitations including toxicity, rapid excretion, difficulty in synthesis, and inability to form complexes with transfection reagents, hindering their therapeutic efficacy.

Method used

Development of thiomorpholino (TMO) antisense oligonucleotides (ASOs) that induce exon skipping in the PTPN1 gene transcript, specifically targeting exon 2, to inhibit the expression of protein tyrosine phosphatase-1B (PTP1B) protein, thereby reducing functional PTP1B production.

Benefits of technology

The TMO ASOs effectively reduce the production of functional PTP1B protein, addressing insulin resistance, leptin resistance, obesity, Rett syndrome, and cancer by altering pre-mRNA splicing to produce truncated or nonsense proteins, thus restoring normal metabolic and signaling pathways.

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Abstract

The present invention relates to antisense oligonucleotides (ASOs) used to treat, prevent, or mitigate the progression of conditions such as type 2 diabetes mellitus (T2DM) and insulin resistance, leptin resistance and obesity, Rett syndrome, and cancer. Specifically, thiomorpholino-containing ASOs target the protein tyrosine phosphatase non-receptor type 1 (PTPN1) gene transcript during RNA processing and induce exon skipping (including exon 2), thereby inhibiting the expression of protein tyrosine phosphatase-1B (PTP1B) protein.
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