Adeno-associated viral vectors (AAV)

Vectors derived from the Adeno-Associated Virus (AAV) are highly efficient and broadly used tools for in vitro and in vivo gene delivery. In the past few years, AAV have emerged as one of the most effective vehicles for gene therapy due to their ability to transduce a wide variety of mammalian cell types, and their favorable safety profile as AAV is considered non-pathogenic in humans.

The Bertarelli Platform has developed various approaches to improve the yield and the quality of AAV vector production. We can produce a broad range of AAV serotypes to target the CNS (brain and spinal cord), as well as peripheral organs. We can produce most natural serotypes as well as engineered capsids.