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.
All labels observed (1)
| Label | Occurrences |
|---|---|
| branaplam canonical | 1 |
How this entity was disambiguated
This entity first appeared as the object of triple T6065086 — resolving that mention is where its identity was fixed. The disambiguator weighed these candidate entities and picked the highlighted one (or “None”, minting a new entity). This is how homonymy is resolved: the same surface form can point to different entities.
NED1
Entity disambiguation (via context triple)
gpt-5-mini-2025-08-07
Target entity: branaplam Context triple: [SMN2, targetOfDrug, branaplam]
-
A.
Brn
Brn is the station code used to identify Baarn railway station in the Netherlands’ rail network.
-
B.
Brakni
Brakni is the surname of Rachida Brakni, a French actress and director known for her work in film, television, and theater.
-
C.
Brenz
The Brenz is a river in southern Germany that flows through Baden-Württemberg and Bavaria before joining the Danube.
-
D.
BPN
BPN is the National Rail station code for Blackpool North railway station, a primary rail terminus serving the seaside town of Blackpool in Lancashire, England.
-
E.
Brupass
Brupass is an integrated public transport ticketing system that allows seamless travel across multiple operators and modes within the Brussels metropolitan area.
- F. None of above. chosen
- G. Unsure - the case is ambiguous/there is not enough information to decide.
NED2
Entity disambiguation (via description)
gpt-5-mini-2025-08-07
Target entity: branaplam Target entity description: 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.
-
A.
Brn
Brn is the station code used to identify Baarn railway station in the Netherlands’ rail network.
-
B.
Brakni
Brakni is the surname of Rachida Brakni, a French actress and director known for her work in film, television, and theater.
-
C.
Brenz
The Brenz is a river in southern Germany that flows through Baden-Württemberg and Bavaria before joining the Danube.
-
D.
BPN
BPN is the National Rail station code for Blackpool North railway station, a primary rail terminus serving the seaside town of Blackpool in Lancashire, England.
-
E.
Brupass
Brupass is an integrated public transport ticketing system that allows seamless travel across multiple operators and modes within the Brussels metropolitan area.
- F. None of above. chosen
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 ⓘ |
How these facts were elicited
The pipeline generated the facts above by prompting gpt-5.1 with this entity's name + description and the instruction below.
Instruction
You are a knowledge base construction expert. Given a subject entity and a description of it, return factual statements that you know for the subject as a JSON list of dictionaries(triples), where keys must be "subject", "predicate" and "object". The number of facts may be very high, between 25 to 50 or more, for very popular subjects. For less popular subjects, the number of facts can be very low, like 5 or 10. # Requirements - If you don't know the subject at all, return an empty list. - If the subject is not a named entity, return an empty list. - Include at least one triple where predicate is "instanceOf". - Do not get too wordy. - Separate several objects into multiple triples with one object.
Input
Subject: branaplam Description of subject: 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.
Referenced by (1)
Full triples — surface form annotated when it differs from this entity's canonical label.