Denali Therapeutics Inc.

The company focuses on discovering, developing, and delivering biotherapeutic medicines for serious diseases, including those affecting the brain. Historically, the blood-brain barrier has hindered the development of central nervous system medicines by preventing sufficient drug delivery to the brain. The company has developed and validated a proprietary technology called the TransportVehicle™ ("TV") to overcome this challenge. The TV has a modular design that allows large molecules, such as enzymes, oligonucleotides, and antibodies, to cross the blood-brain barrier and reach all body tissues, including the brain, after systemic administration. This technology aims to extend the success of large molecule biotherapeutics, which have had limited impact on central nervous system diseases, to neurodegenerative diseases, lysosomal storage disorders, and other serious conditions. The company is building a portfolio of therapeutic candidates through its TV franchises: Enzyme TV ("ETV"), Oligonucleotide TV ("OTV"), and Antibody TV ("ATV"). These franchises are advancing programs for rare diseases like lysosomal storage diseases, and common diseases such as Alzheimer's disease and Parkinson's disease. Its most advanced TV-enabled program is tividenofusp alfa (DNL310, ETV:IDS) for the potential treatment of mucopolysaccharidosis II ("MPS II", or Hunter syndrome). The biologics license application ("BLA") for tividenofusp alfa is under priority review for accelerated approval by the U.S. Food and Drug Administration ("FDA"), with a Prescription Drug User Fee Act (PDUFA) target action date of April 5, 2026. The company has established commercial readiness in anticipation of this date.