branaplam
E567204
investigational medicinal product
orally bioavailable compound
small-molecule drug
splicing modulator
Branaplam is an investigational small-molecule splicing modulator originally developed for spinal muscular atrophy that enhances SMN protein production by altering SMN2 pre-mRNA splicing.
Statements (30)
| Predicate | Object |
|---|---|
| instanceOf |
investigational medicinal product
ⓘ
orally bioavailable compound ⓘ small-molecule drug ⓘ splicing modulator ⓘ |
| actsOn | SMN2 pre-mRNA ⓘ |
| administrationForm | oral solution ⓘ |
| affectsBiologicalProcess |
RNA splicing
ⓘ
SMN2 exon inclusion ⓘ |
| affectsGene | SMN2 NERFINISHED ⓘ |
| alsoKnownAs |
LMI070
NERFINISHED
ⓘ
NVS-SM1 NERFINISHED ⓘ |
| developedBy | Novartis NERFINISHED ⓘ |
| developedFor | spinal muscular atrophy ⓘ |
| drugClass |
RNA splicing modulator
ⓘ
SMN2 splicing modifier ⓘ |
| hasClinicalTrialPhase |
Phase 1
ⓘ
Phase 2 ⓘ |
| increases | SMN protein levels ⓘ |
| indication | spinal muscular atrophy type 1 ⓘ |
| intendedEffect | increase functional SMN protein from SMN2 gene ⓘ |
| mechanismOfAction |
enhancement of SMN protein production
ⓘ
modulation of pre-mRNA splicing ⓘ |
| molecularTarget | SMN2 pre-mRNA splicing ⓘ |
| relatedToDisease | spinal muscular atrophy ⓘ |
| routeOfAdministration | oral ⓘ |
| status | investigational ⓘ |
| targetPopulation | pediatric patients with spinal muscular atrophy ⓘ |
| therapeuticArea |
neuromuscular disorders
ⓘ
rare diseases ⓘ |
| therapeuticIntent | treatment of spinal muscular atrophy ⓘ |
Referenced by (1)
Full triples — surface form annotated when it differs from this entity's canonical label.