Posted by Michael Wonder on 23 May 2023
Rocket Pharmaceuticals receives FDA regenerative medicine advanced therapy designation for RP-L301 gene therapy for pyruvate kinase deficiency
23 May 2023 - Rocket Pharmaceuticals today announced that the US FDA has granted regenerative medicine advanced therapy designation to RP-L301, the Company’s investigational lentiviral-based gene therapy for pyruvate kinase deficiency, a rare blood disorder characterised by severe anaemia and excessive red blood cell breakdown.
Regenerative medicine advanced therapy designation designation was granted based on robust safety and efficacy data from the ongoing Phase 1 RP-L301 clinical trial and its potential to cure a life-threatening disease for which no curative therapies currently exist.
