FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene ther­a­py pi­o­neer James Wil­son spear­head­ed an­i­mal stud­ies demon­strat­ing the po­ten­tial of new treat­ments in­ject­ed di­rect­ly in­to the brain, look­ing to jump­start a once-and-done fix for an ex­tra­or­di­nar­i­ly rare dis­ease called GM1 gan­gliosi­do­sis in in­fants. His team at the Uni­ver­si­ty of Penn­syl­va­nia pub­lished their work on mon­keys and hand­ed it over to Pas­sage Bio, a Wil­son-in­spired start­up build­ing a pipeline of gene ther­a­pies — with an IND for PBGM01 to lead the way.

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