Statements (56)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gptkb:Company
|
| gptkbp:administrativeDivision |
single dose
|
| gptkbp:analyzes |
submitted
|
| gptkbp:clinicalTrials |
gptkb:United_States
not available pending secured not published Phase 1/2 NCT04216820 |
| gptkbp:collaborations |
multiple research institutions
|
| gptkbp:community_service |
safety and efficacy
factor VIII levels |
| gptkbp:communityPartnerships |
biotechnology companies
|
| gptkbp:contraindication |
under monitoring
|
| gptkbp:currentStatus |
completed
|
| gptkbp:developedBy |
gptkb:Sangamo_Therapeutics
|
| gptkbp:diseaseResistance |
gene replacement therapy
|
| gptkbp:evaluates |
under investigation
|
| gptkbp:firstClaim |
reduce bleeding episodes
|
| gptkbp:firstMatch |
2020
|
| gptkbp:hasPopulation |
hemophilia A patients
|
| gptkbp:healthcare |
ongoing
engaged specific genetic mutations |
| gptkbp:historicalResearch |
expanding patient access
|
| https://www.w3.org/2000/01/rdf-schema#label |
SPK-7001
|
| gptkbp:impact |
gene therapy
long-term |
| gptkbp:is_expected_to |
long-term expression of factor VIII
|
| gptkbp:market |
high
|
| gptkbp:operationalRole |
adults
|
| gptkbp:patentStatus |
patented
|
| gptkbp:regulatoryCompliance |
not approved
|
| gptkbp:research |
public and private sources
ongoing studies |
| gptkbp:research_areas |
rare diseases
novel gene therapy approach |
| gptkbp:researchFocus |
hemophilia_treatment
|
| gptkbp:researchInterest |
improve quality of life
ongoing academic institutions peer-reviewed journals regulatory hurdles obtained potential to change treatment landscape |
| gptkbp:route |
intravenous
|
| gptkbp:safetyFeatures |
under evaluation
|
| gptkbp:sponsor |
gptkb:Sangamo_Therapeutics
|
| gptkbp:status |
investigational
|
| gptkbp:targets |
gptkb:F8_gene
hemophilia A patients with hemophilia A |
| gptkbp:ticketingSystem |
AAV_vector
|
| gptkbp:triggerType |
gene editing
|
| gptkbp:type |
gene therapy
|