Medicine in an era of genetic control

The potential of CRISPR technology in drug development


At a fascinating and wide-ranging talk on the future of the pharmaceutical industry, Menelas Pangalos, Executive Vice President of AstraZeneca, discussed the exciting new technology, known as CRISPR (clustered regularly-interspaced short palindromic repeats). CRISPR is a ground-breaking technique that can edit a cell’s DNA, subtly, and with precision.

Genetic modification was once seen as the Holy Grail of medical science. The idea that inborn errors of metabolism could be corrected, and the person cured, was fantastic. However, early attempts at gene therapy using viral-vectors resulted in some horrific off-target effects. CRISPR, on the other hand, has the potential to be much more targeted.

In the more immediate future, CRISPR technology may be used to improve the efficiency and safety of drug development. Using CRISPR to generate cell lines expressing specific genetic defects, the merits of targeted pharmacological treatments can be tested in more pathologically relevant pre-clinical models, thereby reducing candidate drug attrition.

Gene therapy may become a viable medical tool in the future but, in the meantime, CRISPR opens wonderful new worlds of possibility for drug development.

Based on a talk entitled “The challenge and excitement of discovering new medicines” given by Menelas Pangalos, at Churchill College, Cambridge, for the Cambridge Society for the Application of Research.

Further reading:

Lander ES. The heroes of CRISPR. Cell 2016; 164 (1–2): 18–28.

Savić N, Schwank G. Advances in therapeutic CRISPR/Cas9 genome editing. Transl Res 2016; 168: 15–21.

Dominguez AA, Lim WA, Qi LS. Beyond editing: repurposing CRISPR-Cas9 for precision genome regulation and interrogation. Nat Rev Mol Cell Biol 2016; 17 (1): 5–15.

Christopher Edge

Christopher Edge  PhD

Scientific Consultant

Feb 2016