Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine

Abstract excerpt

The evolution of medicines from small molecules to proteins drove increased therapeutic benefits, and the next generation of cell and gene therapies holds tremendous promise for patients. The Food and Drug Administration approved the U.S.'s first gene therapy, Novartis' tisagenlecleucel, and technologies like CRISPR-Cas9 are poised to create a wave of new medicines. Unfortunately, the vast majority of patients...

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