Ho­mol­o­gy joins Bio­Marin in halt­ing gene ther­a­py stud­ies for rare meta­bol­ic dis­ease

It was a tough week for com­pa­nies, pa­tients and fam­i­lies hop­ing a gene ther­a­py for the rare meta­bol­ic dis­ease phenylke­tonuria is on its way.

On Fri­day af­ter­noon, af­ter Bio­Marin had al­ready an­nounced its pro­gram would be on hold for at least “sev­er­al quar­ters,” Ho­mol­o­gy Med­i­cines dis­closed that the FDA had al­so halt­ed tri­als for its phenylke­tonuria gene ther­a­py.

The tim­ing part­ly re­flects the greater scruti­ny gene ther­a­py has come un­der at the FDA over the last year. But de­spite sim­i­lar­i­ties be­tween the two pro­grams, the agency’s con­cerns ap­pear to be un­re­lat­ed, re­flect­ing dis­tinct safe­ty con­cerns with the tech­nol­o­gy.

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