Denali Therapeutics Announces Publication In 'Science Translational Medicine' Demonstrating The Potential Of The OTV Platform To Achieve Broad Biodistribution Of Antisense Oligonucleotides In The CNS And Skeletal And Cardiac Muscle Following IV Administration

Denali Therapeutics Inc. (NASDAQ:DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative and lysosomal storage diseases, today announced publication of nonclinical data in the August 14, 2024 issue of Science Translational Medicine (link) demonstrating the ability of the Oligonucleotide Transport Vehicle (OTV) platform to achieve broad biodistribution of antisense oligonucleotides (ASOs) in the central nervous system (CNS) and skeletal and cardiac muscle following intravenous (IV) administration.

Oligonucleotides, such as ASOs, are designed to modify gene expression and hold promise as therapeutics for neurological disorders. A major challenge in their development, however, is that oligonucleotides are unable to cross the BBB on their own. Currently, oligonucleotides must be delivered directly to the CNS through invasive routes such as intrathecal delivery and still may not distribute uniformly throughout the brain where treatment is needed. Denali invented the Transport Vehicle (TV) platform to overcome the challenge of the BBB and deliver multiple types of therapeutic cargo across the BBB, including antibodies, enzymes, and other proteins. The TV platform has been clinically validated and three TV-enabled programs are currently in clinical development.