Branaplam (development codes LMI070 and NVS-SM1) is a pyridazine derivative that is being studied as an experimental drug.

[1] As a treatment for SMA, branaplam increases the amount of functional survival of motor neuron protein produced by the SMN2 gene through modifying its splicing pattern.

In October 2020, Novartis announced that branaplam reduces the amount of huntingtin protein, which is one of the major therapeutic approaches in Huntington's disease.

[8] Branaplam can be used as a so-called Xon inducer[9] of splicing for control of gene expression.

For example, branaplam was used as an Xon inducer to tightly control Cas9 expression with a modified branaplam-sensitive splicing cassette from the SF3B3 gene.