Latest data give Rocket clear path forward in rare CV disease Danon

While an adverse event in the high-dose cohort continues to outline the limits of high volume AAV dosing, Rocket believes that higher-than-expected gene expression levels at lower doses for RP-A501 to treat Danon disease give the company a clear path forward in the rare cardiovascular disease.

Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) said late Tuesday that preliminary Phase I data for RP-A501 showed two patients treated in the low-dose cohort had LAMP2 expression levels above 50% that correlated with reductions in cardiac biomarker BNP and increases in cardiac output. ...