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branaplam
- Development stage
- Discontinued
- Lead developer
- Novartis
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Branaplam is a small molecule pyridazine derivative developed by Novartis as an orally administered splicing modulator. Its primary mechanism of action is to interact with SMN2 pre-mRNA, enhancing exon 7 inclusion and thereby increasing the production of functional survival motor neuron (SMN) protein. This mechanism targets the underlying genetic deficiency in spinal muscular atrophy (SMA), particularly type 1 SMA[1][3][7]. Branaplam was also investigated for Huntington's disease, where it reduces mutant huntingtin protein levels by promoting the inclusion of a frameshift-inducing exon in HTT mRNA, leading to decreased production of toxic protein[6][8]. Clinical development for SMA reached phase II trials, while development for Huntington's disease was discontinued due to safety concerns[3][7].
- Other names
- branaplam
02
Targets
U1 snRNP (U1 small nuclear ribonucleoprotein)
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