Rocket Pharmaceuticals (NASDAQ:RCKT) announces that the first patient has received investigational therapy in the Phase 1 clinical trial of RP-L301, the lentiviral vector (LVV)-based gene therapy for the treatment of Pyruvate Kinase Deficiency (PKD), a rare monogenic red blood cell disorder.
The trial is expected to enroll six adult and pediatric transfusion-dependent PKD patients in U.S. and Europe and will be comprised of three cohorts to assess RP-L301 in young pediatric (age 8-11), older pediatric (age 12-17) and adult populations.
Preliminary Phase 1 data are anticipated in Q4.
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