The Landscape of Early Clinical Gene Therapies outside of Oncology

Mol Ther. 2019 Oct 2;27(10):1706-1717. doi: 10.1016/j.ymthe.2019.09.002. Epub 2019 Sep 6.


The field of cell and gene therapy (GT) is expanding rapidly and there is undoubtedly a wave of enthusiasm and anticipation for what these treatments could achieve next. Here we assessed the worldwide landscape of GT assets currently in early clinical development (clinical trial phase 1/2 or about to enter clinical trial). We included all gene therapies, i.e., strategies that modify an individual's protein make-up by introducing exogenous nucleic acid or nucleic acid modifiers, regardless of delivery. Unmodified cell therapies, oncology therapies (reviewed elsewhere), and vaccine programs (distinct therapeutic strategy) were not included. Using a December 31, 2018 cutoff date, we identified 336 gene therapies being developed for 138 different indications covering 165 genetic targets. In all, we found that the early clinical GT landscape comprises a very disparate group of drug candidates in terms of indications, organizations, and delivery methods. We also highlight interesting trends, revealing the evolution of the field toward in vivo therapies and adeno-associated virus vector-based delivery systems. It will be interesting to witness what proportion of this current list effectively translates into new medicines.

Publication types

  • Review

MeSH terms

  • Clinical Trials as Topic
  • Drug Delivery Systems / classification*
  • Genetic Therapy / methods*
  • Genetic Vectors / administration & dosage
  • Humans
  • Molecular Targeted Therapy