In vivo gene editing in liver tissues may be able to rescue patients younger than 18 years who are not eligible for gene replacement therapies, with hemophilia as a prime example
Next-generation gene therapies incorporating chimeric DNA sequences in the transgene can increase clotting factor synthesis and secretion, and advance the efficacy, safety, and durability of gene replacement therapy for hemophilia A as well as other blood clotting disorders.
With the rapid growth of gene and cell therapies, biotechnology and pharmaceutical companies face a call to action: we must establish proper selection and monitoring protocols to provide patients with the safest and most effective therapeutic options for genetic diseases. This article presents two clinical programs for gene and allogeneic cell therapies and provides a primer for the relevance of precision medicine applications.
Cell therapies require precise screening and monitoring of patients to ensure that the transfer of either autologous or allogeneic cells to a patient results in a therapeutic effect to targeted organs or tissues.