One of Pfiz­er’s Duchenne gene ther­a­py tri­als put on hold in wake of pa­tient death as high-dose AAV con­cerns still cloud field

A young man with Duchenne mus­cu­lar dy­s­tro­phy has died in Pfiz­er’s Phase Ib tri­al of its mi­ni-dy­s­trophin gene ther­a­py, trig­ger­ing a halt in screen­ing and dos­ing — and a clin­i­cal hold im­posed by the FDA.

Pfiz­er in­formed the Par­ent Project Mus­cu­lar Dy­s­tro­phy of the death in a com­mu­ni­ty let­ter. The pa­tient was par­tic­i­pat­ing in the non-am­bu­la­to­ry co­hort of the tri­al, the com­pa­ny wrote, adding:

At this time, we do not yet have com­plete in­for­ma­tion and are ac­tive­ly work­ing with the tri­al sitein­ves­ti­ga­tor to un­der­stand what hap­pened.

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