At the recent 39th J.P. Morgan Healthcare Conference, Biomarin Pharmaceutical Inc. popped the lid off top-line results from its ongoing phase III GENEr8-1 study with valoctocogene roxaparvovec – also known as valrox, now commonly called Roctavian. Data, though encouraging, may not have quelled controversy around the prospect.

Imara Inc. reported a mixed bag of phase IIa study data for its lead candidate, IMR-687, for treating sickle cell disease in adults, bringing an end to a study that the company found cumbersome and that eventually stung its stock.