In the recent years, the level of interest in Gene Therapy from both industry and academia has been unparalleled. CNS disorders in particular have gained a lot of attention with a considerable emphasis on neurological targets; and this is especially visible when analysing several high-profile deals that occurred in 2018. The biggest stand-out deal was struck by Novartis, buying AveXis for $8.7 billion giving the Basel based biopharma company access to what could potentially be the first one-time only treatment for Spinal Muscular Atrophy (SMA). On top of this, AveXis will also give Novartis access to a promising platform technology based around a non-replicating adeno-associated virus (AAV) capsid that allows penetratration of the blood brain barrier.
The Rise of Gene Therapies in Neurological Diseases