Cell therapy weekly: US FDA places clinical hold on BMN307 gene therapy study in adults with phenylketonuria

This week: the US FDA has placed a clinical hold on an investigational gene therapy study evaluating BMN307 in adults with phenylketonuria, NeuExcell Therapeutics and Spark Therapeutics – part of the Roche Group – join forces to develop a safe and effective gene therapy for people with Huntington’s disease, and an autologous CAR-T cell immunotherapy (termed relmacabtagene autoleucel) received NMPA approval in China. The news highlights: US FDA places clinical hold on BMN307 gene therapy study in adults with phenylketonuria NeuExcell links up with Roche on gene therapy for Huntington’s disease Autologous CAR-T cell immunotherapy receives NMPA approval in China...