The FDA has put a hold on a clinical study of a rare disease gene therapy BMN-307 from Biomarin Pharmaceutical Inc. after several mice developed liver tumors following a high dose in a preclinical trial.
Jnana Therapeutics Inc. closed a $50 million series B round and took the covers off its lead program, a small-molecule inhibitor of the solute carrier transporter SLC6A19, which it is prepping for clinical trials in phenylketonuria (PKU).
Sometimes highly impactful serendipitous discoveries are made when performing genetic loss-of-function studies that were initially focused on putative tumor suppressors or other hypotheses.
