4 studies found for:    weill AND adeno associated virus 2 | Open Studies
Show Display Options
Rank Status Study
1 Recruiting AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis
Conditions: Late Infantile Neuronal Ceroid Lipofuscinosis;   Batten Disease
Intervention: Biological: AAVrh.10CUCLN2
2 Recruiting Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis
Conditions: Batten Disease;   Late-Infantile Neuronal Ceroid Lipofuscinosis
Interventions: Genetic: AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies;   Genetic: AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies
3 Recruiting A Gene Transfer Study for Hemophilia A
Condition: Hemophilia A
Intervention: Genetic: SPK-8011
4 Recruiting A Gene Therapy Study for Hemophilia B
Condition: Hemophilia B
Intervention: Genetic: SPK-9001

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