Branaplam is a highly potent, selective and orally active SMN2 splicing modulator. Branaplam increases the amount of functional SMN protein produced by the SMN2 gene through modifying its splicing.

Branaplam (LMI 070) is a highly potent, selective and orally active small molecule SMN2 splicing modulator.

Branaplam (LMI070; NVS-SM1) hydrochloride is a selective, orally active SMN2 splicing modulator that acts on SMN (EC50: 20 nM). branaplam hydrochloride inhibits hERG (IC50: 6.3 μM). In a mouse model of severe spinal muscular atrophy (SMA), Branaplam hydrochloride increased full-length SMN protein and improved survival.

mHTT-IN-2 (compound 27) is a potent inhibitor of mutant huntingtin protein (mHTT), with an EC50 value of 0.066 μM. It specifically modulates the splicing of HTT RNA exons [49-50], acting as a splicing regulator for the HTT gene. Demonstrating inhibitory effects both in vitro and in vivo, mHTT-IN-2 has been tested in human HD stem cells and mouse BACHD models. Additionally, it is applicable in the investigation of branaplam-related peripheral neuropathy [1].