Industry Insights: Base editing nears its first BLA as AAV safety comes under scrutiny

This month, Beam Therapeutics reported positive topline pivotal data for the base-edited autologous cell therapy risto-cel in sickle cell disease, supporting what would be the first Biologics License Application for a base editing therapy [1]. CAR-T therapy in autoimmune disease continued to generate data, with Cabaletta, Fate, and Bristol Myers Squibb reporting one-year and interim results across myositis, lupus, systemic sclerosis, and multiple sclerosis [2–4]. On the regulatory side, the US FDA set out a framework for individualized genome editing products while CMS plans to broaden its access model [10], and the agency opened an investigation into insertional oncogenesis linked to onasemnogene abeparvovec [14].