Compositions and methods for splicing modulation of UNC13A
Dual-target antisense oligonucleotides targeting UNC13A pre-mRNA regions inhibit cryptic exon inclusion, restoring UNC13A expression and providing a potential treatment for ALS and FTD.
Patent Information
- Authority / Receiving Office
- JP · JP
- Patent Type
- Applications
- Current Assignee / Owner
- TAKEDA PHARMA CO LTD
- Filing Date
- 2026-03-13
- Publication Date
- 2026-07-07
AI Technical Summary
Current treatments for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are ineffective, as they do not address the underlying issue of UNC13A protein expression reduction due to cryptic exon inclusion in mRNA, which is caused by TDP-43 dysfunction.
The use of dual-target antisense oligonucleotides that inhibit the inclusion of cryptic exons in UNC13A mRNA and restore UNC13A expression by targeting specific regions within the UNC13A pre-mRNA, specifically intron 20, to modulate splicing and enhance protein production.
This approach effectively inhibits the inclusion of cryptic exons in UNC13A mRNA, thereby restoring UNC13A protein expression and potentially treating ALS and FTD by addressing the root cause of these diseases.
Smart Images
Figure 2026113503000001 
Figure 2026113503000002 
Figure 2026113503000003