Novartis has entered an exclusive licensing agreement with PTC Therapeutics to develop PTC518, a novel small molecule therapy targeting the mutated Huntingtin protein responsible for Huntington’s disease. This partnership aims to address the unmet medical need for disease-modifying treatments, as current therapies only manage symptoms. PTC518 is currently in Phase II trials, with the potential to become the first approved therapy to alter the disease's progression, marking a significant advancement in neurodegenerative disease research.
