Mi­cro­cap gene ther­a­py play­er says three chil­dren suf­fered se­ri­ous side ef­fects in PhI/II study

A small biotech look­ing to de­vel­op a gene ther­a­py for a rare con­di­tion that can cause col­or or le­gal blind­ness ran in­to some sig­nif­i­cant safe­ty trou­ble in a Phase I/II dose-es­ca­la­tion study.

Ap­plied Ge­net­ic Tech­nolo­gies Cor­po­ra­tion, al­so known as AGTC, re­port­ed Thurs­day af­ter­noon that three of five chil­dren in the study’s high­est dose co­hort ex­pe­ri­enced se­vere in­flam­ma­tion one month af­ter dos­ing. The events were con­cern­ing enough to be cat­e­go­rized as sus­pect­ed un­ex­pect­ed se­ri­ous ad­verse re­ac­tions, or SUSARs.

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