GENE THERAPY AND ITS SUPPLY CHAINS ARE NOT WELL UNDERSTOOD

Why are they being rushed through?

WHAT ARE THE ISSUES?

On Aug. 30, 2017, the Food and Drug Administration (FDA) approved the first gene therapy product to launch in the U.S. Novartis’ drug—brand name Kymriah—is now out of the clinic and available to patients. Eye-watering price aside, it has been hailed as a landmark event in the history of the U.S. pharma industry.

The clinical trials leading up to approval had to surmount the massive supply chain challenges posed by gene therapy, and other Advanced Therapy Medicinal Products (ATMPs), which can be summarized as follows:

It starts with the fact that ATMPs are biologics, made from living things, using processes far more variable and complex than the traditional small molecule products produced by chemical synthesis.

As biologics, their manufacture is an order of magnitude trickier than making drugs based on synthesis alone. Even seemingly minor alterations in the process can change the product, with potentially devastating effect. This has led to the mantra in biologics th…