An­oth­er gene ther­a­py com­pa­ny launch­es with VC cash and a CRISPR tech to tar­get tox­ic RNA

A San Diego-based biotech has at­tract­ed sub­stan­tial ven­ture sup­port for its clin­i­cal for­ay in­to gene ther­a­pies that can tar­get tox­ic brands of RNA — sparked by DNA mu­ta­tions — that trig­ger ge­net­ic dis­eases. And they’ve wooed a well-known West Coast en­tre­pre­neur to the helm.

The biotech is called Lo­cana, which is work­ing on the sci­en­tif­ic ex­plo­rations done by UC San Diego pro­fes­sor Gene Yeo. Yeo’s pub­lished work in­cludes lab ex­per­i­ments that used CRISPR Cas9 tech to mod­i­fy repet­i­tive RNA —an ap­proach with im­pli­ca­tions for some tough dis­eases like my­oton­ic dy­s­tro­phy type 1 and 2, Hunt­ing­ton’s dis­ease, and C9orf72-linked amy­otroph­ic lat­er­al scle­ro­sis. That work was pub­lished in Cell.

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