The Rise of Gene Therapies in Neurological Diseases

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.