Neurodegenerative diseases are one of the largest medical challenges of our time. Denali Therapeutics is dedicated to defeating neurodegenerative and lysosomal storage diseases through rigorous therapeutic discovery and development.
Learn about Denali's Frontotemporal dementia (FTD) program. Frontotemporal dementia (FTD) associated with a mutation in the GRN (granulin) gene, referred to as FTD-GRN, is primarily caused by genetic factors.
Denali’s scientific approach to Sanfilippo syndrome Type A. Denali is evaluating DNL126 in a clinical study. This treatment is proposed to work by using Enzyme Transport Vehicle (ETV) technology to carry SGSH across the blood-brain barrier (BBB) and to other parts of the body.
Success is accelerated by experience, collaboration and leadership. Denali’s Board of Directors combines expertise across industry, academia, drug development and the investment community, unified by the mission to improve the lives of patients.
Denali Therapeutics believes that the goal of making life-changing medicines available to the patient community is achieved through well-designed clinical studies followed by regulatory approval. The best way to access a medicine which has not yet received regulatory marketing approval, i.e. an investigational medicine, is through participation ...
Denali has reported a greater than 90% mean reduction in CSF heparan sulfate at week 24 of treatment, remaining unchanged at week 104. Directly correlating the biomarker with clinical benefit is especially difficult in neuronal MPS patients who are not treated until they show obvious symptoms, Kakkis added.
Delivering therapeutics across the BBB has been a major obstacle to successful drug development in neurodegeneration, and is critical to enabling effective treatments.