Ken Mills, Regenxbio CEO

Re­genxbio to seek ac­cel­er­at­ed ap­proval of Hunter syn­drome gene ther­a­py us­ing bio­mark­er da­ta

A Hunter syn­drome gene ther­a­py de­vel­oped by Re­genxbio sig­nif­i­cant­ly cut lev­els of a key bio­mark­er as­so­ci­at­ed with the dis­ease, ac­cord­ing to new re­sults of a clin­i­cal tri­al shared Wednes­day. The Rockville, MD biotech plans to file for ac­cel­er­at­ed ap­proval based on the bio­mark­er da­ta lat­er this year.

In 10 pa­tients who re­ceived the piv­otal dose of the gene ther­a­py RGX-121, lev­els of a com­po­nent (D2S6) of he­paran sul­fate fell 86% in the cere­brospinal flu­id at week 16. Af­ter that drop, pa­tients stayed “at a near nor­mal or nor­mal range,” lead in­ves­ti­ga­tor Paul Har­matz of the Uni­ver­si­ty of Cal­i­for­nia, San Fran­cis­co told End­points News.

Endpoints News

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