Ryan Watts, Denali CEO

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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