GBIO - Generation Bio plummets 53% on preclinical studies for hemophilia A; hits 52-week low

Shares of Generation Bio (GBIO -53.9%) hit a 52-week low after investors were not impressed with pre-clinical data on its closed-ended DNA (ceDNA) technology to treat hemophilia A. ceDNA is delivered via a novel, cell-targeted lipid nanoparticle. Results showed that in mice, ceDNA generated peak mean human factor VIII expression of 205% of normal at 2.0 mg/kg. "Additional optimization is needed to translate the improvement in potency and reduction in variability observed in mice to [non-human primates], and to support nomination of a development candidate for the company’s hemophilia A program," the company said. Generation Bio said it would provide further pipeline updates in 2022, including timing for an IND submission. Read why Seeking Alpha contributor Numenor Capital called Generation Bio "a stock to monitor for its potential to disrupt the viral gene therapy market."

For further details see:

Generation Bio plummets 53% on preclinical studies for hemophilia A; hits 52-week low