Statements (23)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gene editing therapy
|
| gptkbp:administeredBy |
humans
|
| gptkbp:clinicalTrialPhase |
Phase 1
|
| gptkbp:collaboratesWith |
gptkb:Regeneron_Pharmaceuticals
|
| gptkbp:countryOfTrial |
gptkb:New_Zealand
gptkb:United_Kingdom gptkb:United_States |
| gptkbp:deliveredBy |
lipid nanoparticle
|
| gptkbp:developedBy |
gptkb:Intellia_Therapeutics
|
| gptkbp:effect |
reduces TTR protein levels
|
| gptkbp:firstInHumanTrial |
2021
|
| https://www.w3.org/2000/01/rdf-schema#label |
NTLA-2001
|
| gptkbp:indication |
gptkb:hereditary_transthyretin_amyloidosis
gptkb:wild-type_transthyretin_amyloidosis |
| gptkbp:mechanismOfAction |
CRISPR-Cas9 gene editing
|
| gptkbp:notable_for |
first in vivo CRISPR therapy in humans
|
| gptkbp:routeOfAdministration |
intravenous
|
| gptkbp:status |
investigational
|
| gptkbp:target |
gptkb:transthyretin_amyloidosis
|
| gptkbp:targetGene |
gptkb:TTR
|
| gptkbp:trialIdentifier |
gptkb:NCT04601051
|
| gptkbp:bfsParent |
gptkb:Intellia_Therapeutics
|
| gptkbp:bfsLayer |
5
|