This segment focuses on the development of leucine-rich repeat kinase 2 (LRRK2) inhibitors for the treatment of Parkinson's disease. Research and development activities include the ongoing Phase 1 and Phase 2 clinical trials of BIIB122/DNL151, a small molecule inhibitor designed to slow the progression of Parkinson's. The program utilizes advanced drug discovery techniques and clinical trial methodologies to assess the safety and efficacy of LRRK2 inhibitors. The goal is to provide a disease-modifying therapy for patients with Parkinson's disease, potentially improving motor function and quality of life. This segment benefits from collaborations with Biogen and leverages Denali's expertise in blood-brain barrier penetration to deliver effective treatments. Future opportunities include expanding the program to other LRRK2-related disorders and exploring combination therapies.

This segment is dedicated to developing and commercializing Tividenofusp Alfa for the treatment of Hunter syndrome (MPS II), a rare lysosomal storage disease. The research and development efforts involve Phase I/II and later-stage clinical trials to evaluate the safety and efficacy of Tividenofusp Alfa, a potential disease-modifying therapy. The program utilizes advanced protein engineering and delivery technologies to enhance the therapeutic effect. The primary goal is to improve the lives of patients by addressing the underlying cause of the disease and reducing the severity of symptoms. This segment has achieved significant milestones, including FDA Breakthrough Therapy designation and BLA rolling submission. Future opportunities include expanding the use of Tividenofusp Alfa to other lysosomal storage diseases and exploring combination therapies.

This segment focuses on developing therapies for Amyotrophic Lateral Sclerosis (ALS), a progressive neurodegenerative disease. Research and development activities include clinical trials for DNL343 and AR443820/DNL788, targeting different pathways involved in ALS. The program utilizes advanced drug discovery and clinical trial methodologies to assess the safety and efficacy of potential treatments. The goal is to provide effective therapies to slow disease progression and improve the quality of life for ALS patients. This segment is actively enrolling patients in late-stage programs and is working to establish partnerships to accelerate the development of new treatments. Future opportunities include exploring combination therapies and expanding the program to other neurodegenerative diseases.