BPN-15477

Biological Activity

BPN-15477 is a potent SMC (splicing modulator compound) that restores correct splicing of ELP1 (Elongator complex protein 1) exon 20. BPN-15477 corrects splicing of the ELP1 transcript, significantly increases the level of functional protein in vivo in all tissues, including brain. BPN-15477 can be used for frontotemporal dementia research[1].

References

[1]. Gao D, et al. A deep learning approach to identify gene targets of a therapeutic for human splicing disorders. Nat Commun. 2021 Jun 7;12(1):3332.