A T-LNP built on novel ionizable lipids plus a delivery device forms a safer non-viral dosing scheme, replacing part of the viral-vector delivery load.

• LNP delivery can replace 66% of the AAV delivery load
• Lower AAV dose, less immune activation, higher safety margin
• Novel ionizable lipids with T-LNP formulation and delivery device
• Level-1 lipid targeting plus level-2 ligand targeting

Extension directions: sickle cell disease (SCD), transfusion-dependent β-thalassaemia (TDT) and further liver-targeted gene editing programmes.

Novel lipids deliver passive spleen targeting, with ligand decoration adding active cell targeting — engineering T cells directly in vivo.

• Dual targeting for higher target-tissue expression
• Lower dose, higher safety margin
• LNP delivery: less immune activation, lower cytokine-storm risk
• Extending the boundary of cancer therapy

Ex vivo CAR-T manufacturing is complex and expensive; in vivo delivery simplifies the process and raises efficiency.