Cell and gene therapies: Technology and opportunities

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.

C&GT work by providing a functioning / non-defective copy of a gene directly to the patient, which can enable the relevant underlying biology to function correctly, thus remedying the symptoms caused by the genetic defects (1).

As such, these therapies provide a cure, as opposed to an ongoing treatment. There is also a growing sub-category of C&GT products based on gene-editing approaches (4), whereby the C&GT can repair or edit the defective gene, as opposed to substituting it.