FDA slams clin­i­cal hold on Log­icBio's gene edit­ing drug af­ter clot­ting cas­es in child pa­tients

An­oth­er day, an­oth­er AAV-de­liv­ered gene ther­a­py run­ning in­to road­blocks with the FDA.

The agency has placed a clin­i­cal hold on Log­icBio’s AAV-de­liv­ered gene edit­ing ther­a­py LB-001 af­ter two ado­les­cent pa­tients in an ear­ly tri­al for the drug were di­ag­nosed with throm­bot­ic mi­croan­giopa­thy (TMA), a clin­i­cal syn­drome de­fined by the for­ma­tion of tiny clots in blood ves­sels, the com­pa­ny said Wednes­day.

The biotech is try­ing LB-001 in a Phase I/II tri­al dubbed SUN­RISE to treat chil­dren with the rare meta­bol­ic dis­ease methyl­malonic acidemia, which re­sults in pa­tients not be­ing able to break down cer­tain fats and pro­teins.

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