Our Science

Gene therapy is probably the most potent way to tackle genetic diseases, with viral gene transfer vectors and targeted gene editing being at the forefront of the field.

Genespire develops transformative gene therapies for inherited metabolic diseases and for primary immune deficiencies, based on two innovative technology platforms: the “immune-shielded” lentiviral vector platform, for direct liver-targeted in vivo delivery, and the improved gene editing technology, applied ex vivo to T-cells and Hematopoietic Stem Cells.

Our candidate products respond to actual unmet medical needs and are designed to enhance both the safety and efficacy profile of gene therapy.