Planning A Gene Therapy Trial? Ask An Expert — And Pay Attention

It’s been 46 years since researchers Theodore Friedmann and Richard Roblin published a landmark paper in the journal Science that posed a provocative question: “Gene therapy for human genetic disease?” And while clinical trials for gene therapy drugs have become much more common in the past 20 years — more than 2,400 had been conducted through 2017 — the science is still a long way from mainstream.

So if you’re initiating or planning a gene therapy trial, here are two pieces of advice:

1. Surround yourself with experts.
2. Listen to what they have to say.

Whether those experts come from a CRO or a regulatory consultancy, their help will be critical in helping develop the trial, meet with the Institutional Biosafety Committee, establish a timeline, and manage expectations. Here’s why.