technology
AAV vectors
Adeno-associated viral vectors — recombinant single-stranded DNA vehicles (serotypes AAV9, rh74) for in vivo gene delivery with high tropism and low immunogenicity.
Where it's used
In-vivo gene therapy: AAV and lentiviral vectors
One-time, potentially curative genetic medicines delivered by engineered adeno-associated and lentiviral vectors — eight FDA-approved in-vivo products since 2017, from Luxturna to Beqvez, now a multi-billion-dollar clinical reality alongside honest commercial retrenchments.
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.