|
gptkbp:instanceOf
|
drug
|
|
gptkbp:administrativeDivision
|
single dose
IV infusion
|
|
gptkbp:analyzes
|
active
filed
|
|
gptkbp:clinicalTrials
|
gptkb:United_States
2020-01-01
2023-01-01
ongoing
pending
anticipated.
Phase 1/2
not yet published
open-label
|
|
gptkbp:collaborations
|
various research institutions
|
|
gptkbp:community_service
|
defined
|
|
gptkbp:contraindication
|
monitored
|
|
gptkbp:currentStatus
|
completed
|
|
gptkbp:dataUsage
|
established
|
|
gptkbp:developedBy
|
gptkb:Sangamo_Therapeutics
|
|
gptkbp:dosageForm
|
single administration
|
|
gptkbp:firstClaim
|
long-term factor VIII expression
|
|
gptkbp:future_plans
|
clinical trial
|
|
gptkbp:hasPopulation
|
adults with hemophilia A
|
|
gptkbp:healthcare
|
ongoing
specific to hemophilia A
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
SPK-1001
|
|
gptkbp:is_expected_to
|
reduction in bleeding episodes
|
|
gptkbp:lengthOverall
|
planned
|
|
gptkbp:mandates
|
treatment of hemophilia A
|
|
gptkbp:market
|
high
|
|
gptkbp:patentStatus
|
patented
|
|
gptkbp:regulatoryBody
|
included
|
|
gptkbp:regulatoryCompliance
|
ongoing
not approved
|
|
gptkbp:research
|
secured
|
|
gptkbp:research_areas
|
hematology
|
|
gptkbp:researchField
|
genetic medicine
|
|
gptkbp:researchInterest
|
gptkb:Sangamo_Therapeutics
ongoing
in progress
obtained
|
|
gptkbp:route
|
intravenous
|
|
gptkbp:safetyFeatures
|
under investigation
|
|
gptkbp:status
|
investigational
|
|
gptkbp:targets
|
hemophilia A
FVIII gene
|
|
gptkbp:ticketingSystem
|
AAV_vector
|
|
gptkbp:triggerType
|
gene therapy
gene editing
|