|
B/BE/25/BVW3 |
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) with Severe or Moderately Severe Hemophilia B |
Humans |
Recombinant adeno associated viral vector containing the codon-optimised human PADUA factor IX (FIX) gene |
|
Only notified under the "contained use" procedure. Dossier submitted on 02/09/2022. |
A single-arm, open-label, multi-centre, phase I/II study evaluating the safety and clinical activity of QEL-001, an autologous CAR T regulatory cell treatment targeting HLA-A2, in HLA-A2/ A28neg patients that have received an HLA-A2pos liver transplant |
Humans |
BCMA CAR; shRNA CD3ζ; tCD34 |
|
B/BE/19/BVW2 |
A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Residual FVIII Levels Receiving Prophylactic FVIII |
Humans |
Recombinant adeno-associated virus serotype 5 (AAV5) containing a gene cassette expressing the human coagulation factor VIII (FVIII) gene |
