|
gptkbp:instance_of
|
gptkb:gene_therapy
|
|
gptkbp:benefits
|
reduce TTR protein levels
|
|
gptkbp:class
|
genetic medicine
|
|
gptkbp:clinical_trial
|
gptkb:2021
gptkb:Intellia_Therapeutics
gptkb:United_States
ongoing
Phase 1
pending
open-label study
|
|
gptkbp:clinical_trial_phase1_results
|
not yet published
|
|
gptkbp:collaboration
|
gptkb:Regeneron_Pharmaceuticals
|
|
gptkbp:collaborations
|
academic partnerships
|
|
gptkbp:developed_by
|
gptkb:Intellia_Therapeutics
|
|
gptkbp:disease_mechanism
|
misfolded TTR protein
|
|
gptkbp:duration
|
not specified
|
|
gptkbp:eligibility
|
specific genetic criteria
|
|
gptkbp:feedback
|
to be collected
|
|
gptkbp:funding
|
gptkb:venture_capital
|
|
gptkbp:future_prospects
|
expanding indications
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
NTLA-2001
|
|
gptkbp:innovation
|
first-in-class therapy
|
|
gptkbp:investment
|
high
global
unknown long-term effects
|
|
gptkbp:is_effective_against
|
under investigation
|
|
gptkbp:is_monitored_by
|
ongoing
|
|
gptkbp:is_tested_for
|
TTR levels
|
|
gptkbp:mechanism_of_action
|
gptkb:CRISPR/_Cas9_technology
gene knockout
|
|
gptkbp:patient_population
|
adults with TTR amyloidosis
|
|
gptkbp:provides_guidance_on
|
limited to clinical trials
|
|
gptkbp:publications
|
forthcoming
|
|
gptkbp:regulatory_body
|
gptkb:FDA
|
|
gptkbp:regulatory_compliance
|
not approved
|
|
gptkbp:related_products
|
filed
|
|
gptkbp:research
|
gptkb:biotechnology
completed
|
|
gptkbp:research_areas
|
rare diseases
in vivo gene editing
|
|
gptkbp:research_focus
|
genetic disorders
|
|
gptkbp:research_institutes
|
various academic centers
|
|
gptkbp:research_output
|
potentially transformative
|
|
gptkbp:route_of_administration
|
intravenous
one-time treatment
|
|
gptkbp:safety_features
|
under evaluation
|
|
gptkbp:scientific_goals
|
curative treatment
|
|
gptkbp:service_frequency
|
single dose
|
|
gptkbp:shipping_options
|
lipid nanoparticles
|
|
gptkbp:status
|
investigational
|
|
gptkbp:target_gene
|
TTR
|
|
gptkbp:targets
|
gptkb:Transthyretin_amyloidosis
|
|
gptkbp:treatment
|
gene editing
to be determined
experimental therapy
hospital setting
to be evaluated
|
|
gptkbp:bfsParent
|
gptkb:Intellia_Therapeutics
|
|
gptkbp:bfsLayer
|
4
|