|
gptkbp:instanceOf
|
gene therapy
|
|
gptkbp:administrativeDivision
|
single dose
hospital setting
|
|
gptkbp:analyzes
|
yes
filed
|
|
gptkbp:clinicalTrials
|
gptkb:Intellia_Therapeutics
gptkb:United_States
ongoing
Phase 1
pending
required
to be determined
anticipated
recruited
open-label
CRISPR-based therapy
|
|
gptkbp:collaborations
|
multiple research institutions
|
|
gptkbp:collection
|
ongoing
|
|
gptkbp:currentStatus
|
completed
|
|
gptkbp:developedBy
|
gptkb:Intellia_Therapeutics
|
|
gptkbp:discontinued
|
granted
|
|
gptkbp:diseaseResistance
|
amyloid deposition
|
|
gptkbp:firstClaim
|
reduce TTR protein levels
|
|
gptkbp:firstMatch
|
2021
|
|
gptkbp:funding
|
venture capital
|
|
gptkbp:hasPopulation
|
adults
|
|
gptkbp:healthcare
|
required
specific criteria
|
|
gptkbp:highestPoint
|
safety and efficacy
|
|
gptkbp:historicalResearch
|
exploration of long-term effects
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
NTLA-2001
|
|
gptkbp:impact
|
experimental
gene editing
gene editing therapy
|
|
gptkbp:market
|
high
|
|
gptkbp:patentStatus
|
patented
|
|
gptkbp:regulatoryBody
|
gptkb:FDA
|
|
gptkbp:regulatoryCompliance
|
not yet approved
|
|
gptkbp:research
|
philanthropic organizations
|
|
gptkbp:research_areas
|
rare diseases
|
|
gptkbp:researchFocus
|
genetic disorders
|
|
gptkbp:researchInterest
|
academic institutions
peer-reviewed journals
potentially significant
obtained
evaluate safety and efficacy
|
|
gptkbp:route
|
intravenous
|
|
gptkbp:safetyFeatures
|
under evaluation
|
|
gptkbp:scientificName
|
blood
|
|
gptkbp:sideEffect
|
unknown
|
|
gptkbp:status
|
investigational
|
|
gptkbp:targets
|
Transthyretin amyloidosis
TTR
|
|
gptkbp:ticketingSystem
|
lipid nanoparticles
|
|
gptkbp:triggerType
|
gptkb:CRISPR/Cas9_technology
|