Patients & Families
Denali is pursuing new treatments engineered to cross the blood-brain barrier to improve the lives of people impacted by neurodegenerative diseases.
We are unified on a singular purpose to discover and develop therapeutics to defeat degeneration.
Watch this video to learn about our Transport Vehicle (TV) technology that enables delivery of potential treatments across the blood-brain barrier to the brain.
The human brain contains over 400 miles of blood vessels.
These blood vessels are lined by closely linked cells to form the blood-brain barrier, which protects the brain from viruses, bacteria and other harmful elements.
At the same time, this barrier also prevents the delivery of targeted medicines which could treat conditions that cause damage inside the brain.
However, natural pathways at the blood-brain barrier do exist to transport essential nutrients such as glucose and iron across this barrier, to maintain a healthy brain.
Iron, for example, can be carried across the barrier by binding to transferrin receptors, which are like cars that transfer iron from the blood to the brain.
What if we could harness this method to transport medicines too?
Using this natural iron transport process, Denali Therapeutics has engineered a novel transport vehicle that attaches to the transferrin receptor to deliver medicines to where they are needed in the brain and throughout the body.
This unique transport vehicle technology can be used to carry a wide range of medicines, such as enzymes, antibodies and other molecules, and has the potential to address a broad range of diseases, making the development of advanced treatments possible.
Current Clinical Trials
Before a new treatment can be made available to the general public, multiple studies – referred to as clinical trials – must be conducted to evaluate the safety and effectiveness of the investigational treatment.
Denali and our research partners are conducting clinical trials in the following disease areas. Learn more about these ongoing studies by visiting ClinicalTrials.gov.
PARKINSON’S DISEASE
Our investigational LRRK2 inhibitor treatment BIIB122/DNL151 aims to slow disease progression and treat the underlying cause of Parkinson's disease. Learn more about our investigational treatment and plans for future late-stage trials by visiting EngageParkinson's.
HUNTER SYNDROME
We are advancing therapies that aim to address the cognitive, behavioral, and physical aspects of MPS II and other MPS disorders. Learn more about our investigational brain-penetrant enzyme replacement treatment DNL310 for Hunter syndrome by visiting EngageHunter.
AMYOTROPHIC LATERAL SCLEROSIS (ALS)
We are focused on developing potential treatments for ALS that aim to slow disease progression, including advancing clinical trials of our investigational eIF2B activator treatment DNL343. Visit ClinicalTrials.gov to learn more about this study.
The Engage sites are intended for United States audiences only. These investigational treatments are not approved by any Health Authority.
Ongoing research in neurodegenerative diseases
We have a broad pipeline of early-stage programs also targeting neurodegenerative diseases including Alzheimer's, Frontotemporal Dementia (FTD) and Lysosomal storage disorders. Visit our pipeline to learn more.
Our commitment to you
We listen to people living with neurodegenerative diseases. We engage individuals, families, caregivers and advocacy groups in our work as we strive to develop impactful solutions that address your needs.
Contact our patient advocacy team to learn more at patients@dnli.com
OUR EXPANDED ACCESS POLICY (EAP) ON INVESTIGATIONAL DRUGS
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 in a clinical study. Denali Therapeutics understands that not all patients are able to participate in a clinical study, and other treatment options may have been exhausted. In those cases, the individual's treating physician may choose to request access to an investigational medicine outside of a clinical study via what is termed Expanded Access (also referred to as Compassionate Use). Currently, however, participation in clinical studies is the only way to gain access to Denali's investigational medicines.
While we recognize the disappointment this may bring, at Denali, we are doing all we can to complete clinical studies that are both robust and expeditious to secure approval and access for all patients in need. As more clinical data on the safety and efficacy of these investigational medicines become available, we will review and potentially revise our Expanded Access policy.
If you have questions, please speak with your treating physician, or contact Denali directly at patients@dnli.com.