Take­da, Sarep­ta dou­ble down on a biotech part­ner's plat­form to en­gi­neer bet­ter AAV cap­sids by map­ping out their struc­ture

When Ar­avind Asokan and Mavis Ag­band­je-McKen­na first pulled to­geth­er their re­spec­tive re­search at the Uni­ver­si­ty of North Car­oli­na-Chapel Hill and the Uni­ver­si­ty of Flori­da on en­gi­neer­ing ade­no-as­so­ci­at­ed virus vec­tors and start­ed Stride­Bio in 2015, the FDA was still two years away from an even­tu­al ground­break­ing first ap­proval for an AAV gene ther­a­py.

But even then — and more­so since — pre­clin­i­cal and clin­i­cal stud­ies had ex­posed the glar­ing room for im­prove­ment in the way genes are de­liv­ered.

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