Silence Therapeutics advanced divesiran in polycythemia vera after positive Phase II results from SANRECO (NCT05499013). The siRNA therapy met the primary endpoint, delivering an 88% response rate versus 19% in placebo-controlled groups and maintaining hematocrit control without frequent phlebotomies. Abcuro raised $66 million to progress ulviprubart for inclusion body myositis, a debilitating autoimmune muscle disorder with no approved US therapies. The funding will support a registrational clinical study following encouraging signals from a prior Phase II/III MUSCLE analysis in a subgroup. In stem cell transplantation, ACCESS trial findings expanded donor options for blood cancer patients with difficult matching. The approach pairs younger donors with substantial genetic mismatches with post-transplant cyclophosphamide to preserve transplant viability and improve outcomes—potentially widening access to curative therapies.