Statements (63)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gene therapy
|
| gptkbp:administrativeDivision |
single dose
|
| gptkbp:analyzes |
filed_with_FDA
|
| gptkbp:clinicalTrials |
gptkb:Spark_Therapeutics
Phase 3 approximately 2 years curative treatment monitored closely Phase 1 and Phase 2 positive outcomes reported multiple sites globally adaptive design used factor IX levels involved in awareness represents significant progress |
| gptkbp:collaboratedWith |
biopharmaceutical companies
|
| gptkbp:collaborations |
various academic institutions
|
| gptkbp:commissionDate |
anticipated 2024
|
| gptkbp:community_service |
bleeding frequency reduction
|
| gptkbp:communityEngagement |
active with stakeholders
|
| gptkbp:communityPartnerships |
formed for distribution.
|
| gptkbp:contraindication |
mild to moderate
|
| gptkbp:currentStatus |
conducted successfully
|
| gptkbp:customerFeedback |
generally positive
|
| gptkbp:dataUsage |
established for trials
|
| gptkbp:developedBy |
gptkb:Spark_Therapeutics
|
| gptkbp:dosageForm |
one-time administration
|
| gptkbp:economicImpact |
aims to improve quality of life
potential cost savings |
| gptkbp:exportMarkets |
planned after approval
|
| gptkbp:firstClaim |
reduces bleeding episodes
|
| gptkbp:firstMatch |
yes
|
| gptkbp:hasPopulation |
estimated thousands
|
| gptkbp:hasPrograms |
established for guidance
|
| gptkbp:healthcare |
age 12 and older
ongoing in trials |
| gptkbp:historicalResearch |
exploring long-term effects
|
| https://www.w3.org/2000/01/rdf-schema#label |
SPK-9001
|
| gptkbp:innovation |
advanced therapy medicinal product
|
| gptkbp:isResponsibleFor |
increased FIX activity
|
| gptkbp:lengthOverall |
ongoing
|
| gptkbp:market |
high
|
| gptkbp:patentStatus |
patented
|
| gptkbp:regulatoryBody |
gptkb:FDA
|
| gptkbp:regulatoryCompliance |
under review
navigated successfully |
| gptkbp:research |
public and private sources
|
| gptkbp:research_areas |
rare diseases
|
| gptkbp:researchField |
gene therapy innovations
|
| gptkbp:researchFocus |
hemophilia_treatment
|
| gptkbp:researchInterest |
ongoing with universities
multiple peer-reviewed articles obtained for trials secured for further studies |
| gptkbp:route |
intravenous infusion
|
| gptkbp:safetyFeatures |
generally well tolerated
|
| gptkbp:supplyChain |
managed_by_Spark_Therapeutics
|
| gptkbp:targets |
hemophilia B
patients with hemophilia B |
| gptkbp:ticketingSystem |
gptkb:Adeno-associated_virus_(AAV)_vector
|
| gptkbp:triggerType |
gene replacement therapy
provides functional copy of the FIX gene |
| gptkbp:year |
2015
|