technology

Gene therapy

Delivery of a therapeutic gene sequence in vivo or ex vivo using viral (AAV, lentivirus) or non-viral (LNP) vectors to correct genetic disease.

Where it's used

Personalized & regenerative medicine
Gene and cell therapy platforms for rare monogenic and oncological diseases: AAV vectors, lentiviral HSC editing, ex vivo CRISPR (Casgevy) and in vivo CRISPR (LNP delivery) now deliver durable one-time treatments approved by FDA and EMA.
regenerative-personalized 7 min