|
gptkbp:instance_of
|
gptkb:biotechnology
|
|
gptkbp:administration_setting
|
outpatient
|
|
gptkbp:benefits
|
reducing TTR protein levels
|
|
gptkbp:clinical_trial
|
gptkb:2021
gptkb:Intellia_Therapeutics
ongoing
Phase 1
multiple sites
NCT04601051
|
|
gptkbp:clinical_trial_phase1_results
|
data being analyzed
|
|
gptkbp:collaboration
|
gptkb:Regeneron_Pharmaceuticals
|
|
gptkbp:collaborations
|
academic institutions
|
|
gptkbp:developed_by
|
gptkb:Intellia_Therapeutics
|
|
gptkbp:disease_targeted
|
hereditary transthyretin amyloidosis
|
|
gptkbp:duration
|
not applicable
|
|
gptkbp:efficacy_results
|
promising in early trials
|
|
gptkbp:eligibility
|
specific genetic mutation
|
|
gptkbp:funding
|
supported by grants
|
|
gptkbp:has_advisory_board
|
established
|
|
gptkbp:healthcare
|
yes
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
NTLA-2002
|
|
gptkbp:invention
|
patented
|
|
gptkbp:investment
|
gptkb:significant
high
|
|
gptkbp:is_monitored_by
|
required post-treatment
|
|
gptkbp:is_tested_for
|
TTR levels
|
|
gptkbp:market_launch
|
pending approval
|
|
gptkbp:mechanism_of_action
|
gptkb:CRISPR/_Cas9_technology
|
|
gptkbp:objective
|
evaluate safety and efficacy
|
|
gptkbp:patient_population
|
adults
|
|
gptkbp:production_company
|
gptkb:Intellia_Therapeutics
|
|
gptkbp:publication
|
multiple peer-reviewed articles
|
|
gptkbp:recruitment
|
ongoing
|
|
gptkbp:regulatory_body
|
gptkb:FDA
|
|
gptkbp:regulatory_compliance
|
under investigation
|
|
gptkbp:related_products
|
submitted
|
|
gptkbp:research
|
successful in animal models
|
|
gptkbp:research_areas
|
gptkb:gene_therapy
genetic disorders
|
|
gptkbp:research_ethics_approval
|
obtained
|
|
gptkbp:research_focus
|
in vivo gene editing
|
|
gptkbp:research_milestone
|
first patient dosed.
|
|
gptkbp:route_of_administration
|
intravenous infusion
|
|
gptkbp:safety_features
|
under evaluation
|
|
gptkbp:service_frequency
|
single dose
|
|
gptkbp:shipping_options
|
lipid nanoparticles
|
|
gptkbp:side_effect
|
immune response
|
|
gptkbp:status
|
investigational
|
|
gptkbp:target_gene
|
TTR
|
|
gptkbp:targets
|
gptkb:Transthyretin_amyloidosis
|
|
gptkbp:treatment
|
to be determined
one-time treatment
gene correction
CRISPR-mediated gene editing
single intravenous infusion
|
|
gptkbp:bfsParent
|
gptkb:Intellia_Therapeutics
|
|
gptkbp:bfsLayer
|
4
|