Denali's Investigational Treatment Approach to Hunter syndrome
Brain delivery is a critical unmet need in Hunter syndrome therapy1
Treatments that cross the blood-brain barrier and aim to treat the neurocognitive components of Hunter syndrome, such as developmental delay, disruptive behavior and impaired cognition, remain a critical unmet medical need.
Denali is investigating an innovative approach 1,2,3
Denali has developed a technology called an "Enzyme Transport Vehicle" (ETV). This technology is designed to leverage the brain's own natural iron transport mechanisms to broadly and efficiently deliver large enzymes across the blood-brain barrier to the brain.
Using ETV technology, Denali has developed DNL310, an investigational weekly IV treatment for children living with Hunter syndrome. DNL310 is designed to deliver the iduronate-2-sulfatase enzyme across the blood-brain barrier to the brain. Through this transport process, DNL310 aims to address the behavioral and cognitive aspects of Hunter syndrome while also addressing the physical symptoms of the disease.
Image is for descriptive purposes only. DNL310 is an investigational drug and is not approved by any Health Authority
Visit our corporate website to read recent press releases on our DNL310 program
Learn More About Clinical Trials
Whether you’re interested in active participation or just want to know about research in progress, see our latest clinical trial information here
References: 1. R. J. Desnick, E. H. Schuchman, Enzyme replacement therapy for lysosomal diseases: Lessons from 20 years of experience and remaining challenges. Annu. Rev. Genomics Hum. Genet. 13, 307–335 (2012). 2. Kariolis et al., Sci. Transl. Med. 12, eaay1359 (2020) 27 May 2020 3. Ullman et al., Sci. Transl. Med. 12, eaay1163 (2020) 27 May 2020
