Shots:

• The P-III GENEr8-1 study results involves assessing of valoctocogene roxaparvovec (6e13 vg/kg) in 20 patients with hemophilia A
• The P-III GENEr8-1 study results: @23-26eks. 8 patients achieved Factor VIII levels of 40 IU/dL- mean ABR (1.5); reduction in median annualized Factor VIII usage (84%); reduction in mean FVIII usage (94%); mean & median Factor VIII level using the CS assay (36 IU/dL & 33 IU/dL)
• Valoctocogene roxaparvovec is gene therapy developed utilizing an AAV-factor VIII vector and has received FDA’s BT designation- EMA’s PRIME designation for hemophilia A with its expected FDA & EU regulatory submission in Q3’19

Ref: BioMarin | Image: BioMarin