The FDA approved Waskyra gene therapy for Wiskott-Aldrich syndrome, a rare immune disorder.

The FDA approved the gene therapy Waskyra for Wiskott-Aldrich syndrome, while ianalumab showed promise in reducing bleeding and depleting B cells in immune thrombocytopenia, and bezuclastinib demonstrated strong biomarker reduction in non-advanced systemic mastocytosis. These advances were highlighted at the ASH 2025 meeting and other recent conferences, reflecting progress in targeted treatments for rare and complex disorders.