The FDA has placed Logicbio Therapeutics Inc.’s phase I/II clinical trial of LB-001, an investigational AAV genome-editing therapy for treating pediatric patients with methylmalonic acidemia (MMA), on a clinical hold. So far, four patients have been dosed in the study and two have had serious adverse events related to the candidate, the company’s lead asset.

A pivotal trial of Omeros Corp.'s narsoplimab, an investigational therapy for an occasional but serious complication of hematopoietic stem cell transplant (HSCT), found that just one dose was enough to deliver a beneficial effect in more than half the study's high-risk participants, meeting the study's primary endpoint.