News brief­ing: Pfiz­er and Vivet get the OK to start gene ther­a­py tri­al for rare liv­er dis­or­der; Flori­da biotech inks $50M Chi­na deal

Vivet Ther­a­peu­tics and Pfiz­er are one step clos­er to bring­ing a gene ther­a­py for a rare liv­er dis­or­der in­to the clin­ic.

The com­pa­nies an­nounced Wednes­day morn­ing that the FDA has ac­cept­ed its IND ap­pli­ca­tion for a Phase I/II study in the treat­ment of Wil­son’s dis­ease. The study, eval­u­at­ing a pro­gram dubbed VTX-801, is ex­pect­ed to launch ear­ly next year.

VTX-801 is an rAAV-based gene ther­a­py vec­tor de­signed to de­liv­er a pro­tein called ATP7B in the hopes of restor­ing cop­per home­osta­sis, re­vers­ing liv­er pathol­o­gy and re­duc­ing cop­per ac­cu­mu­la­tion in the brain, as it was shown to do in mouse mod­els.

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