Denali Therapeutics, Inc. is a clinical-stage biotechnology company focused on the discovery and development of life-transforming treatments for neurodegenerative diseases. Headquartered in South San Francisco, California, the company applies cutting-edge science to address central nervous system disorders such as Parkinson’s disease, Alzheimer’s disease, amyotrophic lateral sclerosis (ALS) and frontotemporal dementia. By targeting underlying disease mechanisms, Denali aims to develop therapies that can alter the natural history of progressive neurologic conditions.
The company’s proprietary Transport Vehicle platform is designed to deliver therapeutic molecules across the blood-brain barrier, a longstanding obstacle in neurology drug development. Denali engineers these vehicles to carry enzymes, antibodies and small molecules directly into the brain, where they can engage targets implicated in lysosomal dysfunction, protein aggregation and neuroinflammation. In addition to its Transport Vehicle technology, Denali leverages small-molecule kinase inhibitors, biologics and gene therapy approaches to address diverse molecular pathways.
Denali has advanced multiple candidates into human testing. Its LRRK2 inhibitor program is being evaluated for slowing progression in Parkinson’s disease, while a progranulin gene therapy is in clinical trials for frontotemporal dementia caused by progranulin deficiency. Other pipeline highlights include an anti-TNF agent for Alzheimer’s disease and an autophagy enhancer for ALS. The company’s robust preclinical portfolio further explores targets such as TDP-43 proteinopathy and glucocerebrosidase modulators, underscoring its commitment to addressing both common and rare neurodegenerative disorders.
Founded in 2015 by a multidisciplinary team of industry and academic scientists, Denali has established research sites in the United States and the United Kingdom to support discovery, translational science and clinical development. The leadership team, led by veteran biotech executive Steve Paul as Chairman and Chief Executive Officer, brings deep expertise in neuroscience, clinical research and regulatory strategy. Through strategic collaborations and its in-house capabilities, Denali continues to build a foundation for delivering novel therapies to patients worldwide.
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