B/BE/21/BVW8 |
Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene |
Humans |
Non-replicating recombinant vector derived from adeno-associated virus AAV 5 carrying the human RPGR gene |
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 |
MB-CART2019.1 |
A phase I/II safety, dose finding and feasibility trial of MB-CART2019.1 in patients with relapsed or resistant B-NHL |
Humans |
CD19 and CD20 chimeric antigen receptor |