Denali unveil proof-of-concept data for brain delivery tech in rare disease, with eyes on much bigger indications
Nearly a year after unveiling their brain delivery technology in Science Translational Medicine, Denali has delivered promising new results from a trial that used it on patients with a rare genetic disease and could point down the road to studies and treatments in larger indications.
Denali studied five patients with Hunter syndrome, a condition where patients are missing a key enzyme involved in the body’s storage and disposal system. Consequently, they accumulate massive levels of several damaging molecules, with severe consequences for muscular and cognitive development.
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