The stock of Generation Bio was momentarily stopped on Tuesday morning following the presentation of mouse data that hampered the company’s hunt for a potential haemophilia A target to take to the clinic. In a Securities and Exchange Commission filing, the biotech, which went public in June 2020 with an IPO that raised $230 million, stated that data from early preclinical mouse trials did not translate to nonhuman primates.
Although the details are sparse, Generation earlier study in mice models revealed that their candidate had a peak mean human factor VIII expression of 205 percent of the normal level. Factor VIII is an important blood clotting protein and a biomarker for haemophilia sufferers. To reduce bleeding episodes, new gene therapies are attempting to address shortage of that protein.
Generation had stated that it would choose its clinical candidates throughout 2020, with IND-enabling trials scheduled for current year. Human testing applications were planned to be submitted to the FDA in 2022. That deadline will be pushed back—a long time. The business now aims to deliver updates on its pipeline programme in 2022, with IND submission deadlines to be determined “in the future.”
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