Abstract
Cell and gene therapies (CGTs) are new frontiers in the fight against serious diseases, including rare genetic disorders and cancers. CAR-T cell therapies continue to dominate the pipeline of genetically modified cell therapies, representing around 49% of the technology. CAR T cell products are human gene therapy products in which the T cell specificity is genetically modified to enable recognition of the desired target antigen for therapeutic purposes. Cell- and gene-based therapies are also called advanced therapeutic medicinal products (ATMP). These products pose unique challenges for pharmaceutical companies and patients. These CGT trials are often preceded by nonclinical programs that provide robust efficacy and safety data for the specific CGT and specific clinical indication using in vitro and in vivo models that were reasonably justified for the appropriate regulatory agency.
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