Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry

Gene Ther. 2017 Sep;24(9):590-592. doi: 10.1038/gt.2017.36. Epub 2017 May 25.


For the last 20 years, academic research has been the major, and often only, driving force behind the spectacular development of gene transfer technology for the therapy of rare genetic diseases. Investors and industry became eventually interested in gene and cell therapy, due to the success of a series of pioneering clinical trials that proved efficacy and safety of last-generation technology, and to favorable orphan drug legislation in both Europe and the United States. Developing this forms of therapy is however complex and requires skills and knowledge not necessary available to the industry, which is better placed to develop processes and products and put them on the market. Cooperation between academia and industry is an opportunity to de-risk innovative approaches and ensure a faster and more economical development of therapies for diseases with high unmet medical needs and low-profit expectations.

Publication types

  • Review

MeSH terms

  • Academic Medical Centers / organization & administration*
  • Biomedical Research / methods
  • Biomedical Research / organization & administration
  • Drug Industry / organization & administration*
  • Genetic Therapy / methods*
  • Humans
  • Intersectoral Collaboration*
  • Rare Diseases / therapy*