Novartis Takes Another Shot at Alpha-Synuclein With $2.2B Arrowhead CNS Deal

Novartis has entered into a global license and collaboration deal with Arrowhead Pharmaceuticals worth up to $2.2 billion, including a $200 million upfront payment, for exclusive rights to develop and commercialize ARO-SNCA, an experimental RNA interference (RNAi) therapy targeting alpha-synuclein for neurodegenerative diseases such as Parkinson's1 2 3 4 5.

ARO-SNCA is designed to silence the gene encoding alpha-synuclein, a protein whose misfolding and accumulation are closely linked to the pathology of Parkinson's disease and related synucleinopathies1 2 4.

The therapy leverages Arrowhead's Targeted RNAi Molecule (TRiM) platform technology, which enables subcutaneous administration and aims for targeted delivery to deep regions of the central nervous system, an area that has historically been challenging for genetic medicines to reach1 2 4.

Novartis previously pursued alpha-synuclein as a target in Parkinson’s disease, but earlier programs failed clinically; this new deal demonstrates renewed commitment to RNA-based approaches in neurodegeneration1 2 5.

Preclinical data cited by Arrowhead show that ARO-SNCA achieves delivery and activity in the central nervous system, supporting advancement into clinical trials1 2 4.