Repair the gene therapy pipeline

Shortages of materials and skills are holding up treatments for genetic disorders, says Ian Campbell

It’s probably a medical researcher’s worst nightmare. Just months before you’re due to start toxicity studies prior to phase one clinical trials of a treatment, you’re told that the biological materials you need have been delayed by over a year and are going to cost a lot more.

Sadly, this is a common experience for those working on gene therapies, including one UK team funded by LifeArc that is studying a rare genetic immune deficiency disorder. These researchers were told they were facing an 18-month delay in the production of materials needed to prepare a potential treatment for clinical trials.