Re­genxBio ral­ly fades fast af­ter biotech re­ports set­backs, hos­pi­tal­iza­tion in gene ther­a­py study

Re­genxBio $RGNX has some ex­plain­ing to do to­day.

In its quar­ter­ly re­port the gene ther­a­py com­pa­ny re­port­ed that it ex­pe­ri­enced sev­er­al set­backs dur­ing its Phase I/II study of RGX-501, one of 4 pipeline pro­grams at the biotech that’s be­ing de­vel­oped for ho­mozy­gous fa­mil­ial hy­per­c­ho­les­terolemia (HoFH) in a tri­al un­der­way at the Uni­ver­si­ty of Penn­syl­va­nia. In ad­di­tion to re­port­ing ad­verse events, the in­ter­im analy­sis failed to pro­duce ev­i­dence of LDL re­duc­tion in one co­hort — and may not be able to in the oth­er.

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