Novartis has signed a global licensing agreement with Arrowhead Pharmaceuticals for the preclinical RNA interference (RNAi) therapy ARO-SNCA, targeting alpha-synuclein in Parkinson’s disease and related disorders. The deal, announced Tuesday and reported by Reuters, includes a $200 million upfront payment to Arrowhead, with up to $2 billion in potential milestones, plus tiered royalties (capped in…
Gain Therapeutics’ supercomputer-driven therapy offers potential Parkinson’s breakthrough
The Bethesda biotech Gain Therapeutics, armed with supercomputer firepower, aims to disrupt Parkinson’s disease treatment. Their GCase regulator, named GT-02287, completely restored motor function in a recent animal study. GCase, short for glucocerebrosidase, targets both lysosomal and mitochondrial dysfunction. In Parkinson’s, the failure of cellular powerhouses (mitochondria) and recycling centers (lysosomes) disrupts essential functions. This…