Gene ther­a­py flunks PhII/III study, but for­mer Sarep­ta part­ner sees a path for­ward — if it can find the cash

The de­vel­op­ment path for Lyso­gene’s gene ther­a­py for MPS II­IA has been a rocky one. Af­ter the FDA slapped a par­tial clin­i­cal hold on a Phase II/III study, a pa­tient al­ready dosed in the tri­al died, al­though it was deemed un­re­lat­ed to treat­ment. Then ear­li­er this year, Sarep­ta pulled out of their three-year part­ner­ship due to dis­agree­ments on who will han­dle com­mer­cial sup­ply.

And now, Lyso­gene re­port­ed the tri­al has failed its pri­ma­ry end­point.

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