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8 studies found for:    "spark therapeutics"
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Rank Status Study
1 Recruiting A Gene Transfer Study for Hemophilia A
Condition: Hemophilia A
Intervention: Genetic: SPK-8011
2 Recruiting Safety and Dose Escalation Study of AAV2-hCHM in Subjects With CHM (Choroideremia) Gene Mutations
Conditions: Choroideremia;   CHM (Choroideremia) Gene Mutations
Intervention: Biological: AAV2-hCHM
3 Recruiting A Gene Therapy Study for Hemophilia B
Condition: Hemophilia B
Intervention: Genetic: SPK-9001
4 Terminated Hemophilia B Gene Therapy With AAV8 Vector
Condition: Hemophilia B
Intervention: Biological: AAV8-hFIX19
5 Active, not recruiting Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
Condition: Leber Congenital Amaurosis
Intervention: Biological: AAV2-hRPE65v2
6 Active, not recruiting Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
Conditions: Inherited Retinal Dystrophy Due to RPE65 Mutations;   Leber Congenital Amaurosis
Intervention: Biological: AAV2-hRPE65v2
7 Active, not recruiting Safety Study in Subjects With Leber Congenital Amaurosis
Condition: Leber Congenital Amaurosis
Intervention: Biological: AAV2-hRPE65v2
8 Active, not recruiting LTFU for Gene Transfer Subjects With Hemophilia B
Condition: Hemophilia B
Intervention:

Study has passed its completion date and status has not been verified in more than two years.