Generation Bio Hits 52-Week Low After Preclinical Data From Hemophilia A Program

Generation Bio Co GBIO provided an update on factor VIII expression from a series of mouse and companion non-human primate (NHP) studies in hemophilia A and an update on its development non-viral genetic medicine platform. 

• In mouse studies, rapid enzymatic synthesis (RES)-derived ceDNA delivered by ctLNP generated peak mean human factor VIII expression of 205% of normal at 2.0 mg/kg. 
• This is compared to peak mean human factor VIII expression of 23% of normal at 2.0 mg/kg using ceDNA produced by the Company's prior Sf9-based manufacturing process. 
• Though the increased potency was attributed to the RES-produced ceDNA and ctLNP production process innovations, the Company also observed lower variability of factor VIII expression and tolerability within and between mouse studies. 
• The Company says that additional optimization is needed to translate the improvement in potency and reduction in observed variability and support the nomination of a development candidate for the hemophilia A program.
• The Company expects to announce updates from its pipeline programs in 2022 and provide timing for its first IND submission in the future.
• Price Action: GBIO shares are down 54.70% at $6.30 during the market session on the last check Tuesday.