Cell and gene therapies: Technology and opportunities

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INTRODUCTION: WHAT ARE CELL AND GENE THERAPIES?
Cell and Gene Therapies (C&GT) are a growing category of medicines that enable genetic defects to be directly corrected in patients, thus enabling remediation of disease symptoms that typically have no other therapeutic option (1). Some of the diseases treated by C&GT have existing small or large molecule therapeutic options (e.g., Spinraza (2) from Biogen/Ionis for Spinal Muscular Atrophy, SMA, which can also be treated by the C&GT Zolgensema (3) from Novartis), but for many of the target conditions C&GT often represents the only treatment option for patients.