AveXis
E564274
AveXis is a biotechnology company best known for developing the gene therapy onasemnogene abeparvovec (Zolgensma) for spinal muscular atrophy.
All labels observed (1)
| Label | Occurrences |
|---|---|
| AveXis canonical | 1 |
How this entity was disambiguated
This entity first appeared as the object of triple T6004714 — 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: AveXis Context triple: [onasemnogene abeparvovec, developedBy, AveXis]
-
A.
onasemnogene abeparvovec
Onasemnogene abeparvovec is a gene therapy medication that delivers a functional copy of the SMN1 gene to treat infants and young children with certain forms of spinal muscular atrophy.
-
B.
Spinraza
Spinraza is a prescription medication used to treat spinal muscular atrophy (SMA) by modifying SMN2 gene expression to increase production of survival motor neuron (SMN) protein.
-
C.
Evrysdi
Evrysdi is an oral prescription medication used to treat spinal muscular atrophy (SMA) by increasing production of the survival motor neuron (SMN) protein.
-
D.
inotersen
Inotersen is an antisense oligonucleotide drug used to treat hereditary transthyretin-mediated amyloidosis by reducing the production of transthyretin protein.
-
E.
Alexion Pharmaceuticals
Alexion Pharmaceuticals is a biopharmaceutical company specializing in the development of therapies for rare and severe diseases, particularly in the field of complement biology.
- 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: AveXis Target entity description: AveXis is a biotechnology company best known for developing the gene therapy onasemnogene abeparvovec (Zolgensma) for spinal muscular atrophy.
-
A.
onasemnogene abeparvovec
Onasemnogene abeparvovec is a gene therapy medication that delivers a functional copy of the SMN1 gene to treat infants and young children with certain forms of spinal muscular atrophy.
-
B.
Spinraza
Spinraza is a prescription medication used to treat spinal muscular atrophy (SMA) by modifying SMN2 gene expression to increase production of survival motor neuron (SMN) protein.
-
C.
Evrysdi
Evrysdi is an oral prescription medication used to treat spinal muscular atrophy (SMA) by increasing production of the survival motor neuron (SMN) protein.
-
D.
inotersen
Inotersen is an antisense oligonucleotide drug used to treat hereditary transthyretin-mediated amyloidosis by reducing the production of transthyretin protein.
-
E.
Alexion Pharmaceuticals
Alexion Pharmaceuticals is a biopharmaceutical company specializing in the development of therapies for rare and severe diseases, particularly in the field of complement biology.
- F. None of above. chosen
Statements (44)
| Predicate | Object |
|---|---|
| instanceOf |
biotechnology company
ⓘ
gene therapy ⓘ |
| acquiredBy | Novartis NERFINISHED ⓘ |
| acquisitionDate | 2018 ⓘ |
| acquisitionValue | 8770000000 USD ⓘ |
| approvalDate |
2019
ⓘ
2019 ⓘ |
| approvedBy |
U.S. Food and Drug Administration
NERFINISHED
ⓘ
U.S. Food and Drug Administration NERFINISHED ⓘ |
| changedNameTo | Novartis Gene Therapies NERFINISHED ⓘ |
| chiefExecutiveOfficer | Sean Nolan NERFINISHED ⓘ |
| clinicalTrialPhase | Phase 3 (for SMA gene therapy prior to approval) ⓘ |
| collaboratedWith | Nationwide Children’s Hospital NERFINISHED ⓘ |
| country |
United States of America
ⓘ
surface form:
United States
|
| developed |
Zolgensma
NERFINISHED
ⓘ
onasemnogene abeparvovec NERFINISHED ⓘ |
| focusesOn |
neuromuscular disorders
ⓘ
rare diseases ⓘ spinal muscular atrophy ⓘ |
| formerName | Biolife Cell Bank NERFINISHED ⓘ |
| foundedBy |
Brian Kaspar
NERFINISHED
ⓘ
John Carbona NERFINISHED ⓘ Sean Nolan NERFINISHED ⓘ |
| headquartersLocation | Bannockburn, Illinois NERFINISHED ⓘ |
| indication |
spinal muscular atrophy
ⓘ
spinal muscular atrophy ⓘ |
| industry |
biotechnology
ⓘ
gene therapy ⓘ |
| listedOn | NASDAQ ⓘ |
| parentOrganization |
Novartis
NERFINISHED
ⓘ
Novartis NERFINISHED ⓘ |
| product |
Zolgensma
NERFINISHED
ⓘ
onasemnogene abeparvovec NERFINISHED ⓘ |
| receivedApprovalFor | onasemnogene abeparvovec NERFINISHED ⓘ |
| regulatoryApproval | U.S. Food and Drug Administration NERFINISHED ⓘ |
| researchFocus |
central nervous system disorders
ⓘ
genetic neuromuscular diseases ⓘ |
| specializesIn | AAV-based gene therapies ⓘ |
| status | subsidiary of Novartis ⓘ |
| technology | adeno-associated virus vector ⓘ |
| therapeuticArea | gene replacement therapy ⓘ |
| tickerSymbol | AVXS NERFINISHED ⓘ |
| tradeName | Zolgensma NERFINISHED ⓘ |
| usesVector | AAV9 NERFINISHED ⓘ |
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: AveXis Description of subject: AveXis is a biotechnology company best known for developing the gene therapy onasemnogene abeparvovec (Zolgensma) for spinal muscular atrophy.
Referenced by (1)
Full triples — surface form annotated when it differs from this entity's canonical label.