Posted by Michael Wonder on 28 Nov 2018
Rocket Pharmaceuticals receives FDA regenerative medicine advanced therapy and fast track designations for RP-L102 gene therapy for Fanconi anaemia
27 November 2018 - Global trial of RP-L102 utilising no conditioning and “Process B” on track for early 2019.
Rocket Pharmaceuticals today announces that the U.S. FDA has granted regenerative medicine advanced therapy (RMAT) and fast track designations to RP-L102, the Company’s lentiviral vector-based gene therapy for the treatment of Fanconi anaemia. RMAT designation was granted based on the positive efficacy and safety results from the ongoing Phase 1/2 clinical trial of RP-L102 being conducted in Europe.
Rocket plans to initiate a clinical trial of RP-L102 for FA in early 2019 utilising no conditioning and “Process B” which incorporates higher cell doses, transduction enhancers, and commercial-grade vector manufacturing and cell processing. The global trial is expected to enrol approximately 12 FA patients at the Center for Definitive and Curative Medicine at Stanford University School of Medicine, Hospital Niño Jesús/CIEMAT, and other leading centers in the U.S. and in the EU.
