Properties (61)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gene editing therapy
|
| gptkbp:administrativeDivision |
single dose
|
| gptkbp:analyzes |
ongoing during trials
filed_with_FDA |
| gptkbp:clinicalTrials |
gptkb:United_States
Phase 1 highly anticipated aims to modify genes aims to provide new options awaited by stakeholders focused on safety and efficacy listed in clinical trial databases monitored post-treatment required for participation in trials |
| gptkbp:collaborations |
with pharmaceutical companies
multiple research institutions |
| gptkbp:community_service |
defined for trials
|
| gptkbp:contraindication |
reported during trials
|
| gptkbp:currentStatus |
conducted prior to human trials
|
| gptkbp:customerFeedback |
collected during trials
|
| gptkbp:dataUsage |
established for oversight
|
| gptkbp:developedBy |
gptkb:Intellia_Therapeutics
|
| gptkbp:dosageForm |
determined in trials
|
| gptkbp:environmentalInitiatives |
included in study design
|
| gptkbp:financialPerformance |
protected by patents
presented at relevant events |
| gptkbp:firstClaim |
reducing TTR protein levels
|
| gptkbp:firstMatch |
2021
|
| gptkbp:hasPopulation |
adults with TTR amyloidosis
|
| gptkbp:hasPrograms |
consulted for research direction
|
| gptkbp:healthcare |
engaged for awareness
ongoing for trials |
| gptkbp:historicalResearch |
with academic institutions
planned based on trial outcomes |
| https://www.w3.org/2000/01/rdf-schema#label |
NTLA-3001
|
| gptkbp:impact |
gene correction
|
| gptkbp:isResponsibleFor |
to be evaluated in trials
|
| gptkbp:lengthOverall |
planned after treatment
|
| gptkbp:market |
significant for rare diseases
|
| gptkbp:patentStatus |
patented
|
| gptkbp:publications |
in peer-reviewed journals
|
| gptkbp:purpose |
treatment of genetic disorders
|
| gptkbp:regulatoryCompliance |
under investigation
sought after trials |
| gptkbp:research |
secured from investors
|
| gptkbp:research_areas |
rare diseases
|
| gptkbp:researchFocus |
genetic diseases
|
| gptkbp:researchInterest |
results published in scientific journals
with biotech firms adhered to in trials applied for funding may be conducted potentially transformative for patients |
| gptkbp:route |
intravenous injection
|
| gptkbp:safetyFeatures |
monitored in clinical trials
|
| gptkbp:scientificName |
blood samples collected
|
| gptkbp:sideEffect |
monitored during trials
|
| gptkbp:status |
investigational
|
| gptkbp:targets |
liver cells
Transthyretin amyloidosis |
| gptkbp:triggerType |
gptkb:CRISPR/Cas9_technology
|