Key points from article :
Arrowhead Pharmaceuticals has struck a major deal worth up to $2 billion with Novartis to advance its experimental therapy for neurodegenerative diseases. The partnership centers on ARO-SNCA, a preclinical RNA interference (RNAi) treatment that targets the alpha-synuclein protein, a key player in the progression of Parkinson’s disease and related conditions. The therapy uses Arrowhead’s proprietary TRiM platform, which allows RNAi medicines to be delivered via simple subcutaneous injections and reach deep brain regions that are typically hard to access.
The goal of ARO-SNCA is to silence the gene responsible for producing alpha-synuclein, thereby reducing levels of the protein that accumulates in the brains of patients with Parkinson’s and other synucleinopathies. By limiting this buildup, researchers hope to slow or even alter the course of these debilitating diseases. Arrowhead’s TRiM technology has already shown promising preclinical results in animal models, where durable protein knockdown was observed across the central nervous system.
For Novartis, the collaboration expands its neuroscience pipeline and underscores a growing commitment to RNA-based medicines for neurodegeneration. The company believes Arrowhead’s delivery platform has strong potential to unlock the therapeutic benefits of RNA medicines in brain diseases that have long resisted treatment. Arrowhead, meanwhile, secures a $200 million upfront payment once regulatory approvals are cleared in 2025, with the potential for up to $2 billion in milestone payments and royalties if the program succeeds commercially.
Under the agreement, Arrowhead will complete the preclinical work needed to file for clinical testing, while Novartis will take over the heavy lifting on trials, manufacturing, and commercialization. If successful, this partnership could mark a breakthrough in RNA medicine for Parkinson’s and related disorders, providing hope for new ways to treat conditions that currently lack disease-modifying therapies.
