New York Times, November 27, 2017
Gina Kolata

Links

Read the Full Article

Eager to speed development of revolutionary treatments, the Food and Drug Administration recently announced that it would expedite approval of experimental gene therapies. But the regulatory process may not be the biggest obstacle here.

Biotech companies have exciting plans to introduce treatments that may be transformative, sometimes curing genetic diseases with a single treatment. And the firms are itching to test their products.

But they are struggling to obtain a critical component of the therapy: the disabled viruses used to slip good genes into cells that lack them.

This delivery system lies at the heart of many forms of gene therapy; without the disabled viruses, there is no treatment. But manufacturing them is costly and onerous.

The genes intended to fix a defect in the body are carried into each cell by a modified virus, usually a disabled version of an adenovirus or a lentivirus. These viruses must be custom-made in specialized facilities for each treatment.

Few gene-therapy companies have the factories or expertise to make the viruses for use in clinical trials, where standards are exacting and comprehensive. The firms that can do it are swamped with orders and requests.

The result is a logjam. Firms exploring new gene therapies may wait for years in line for bespoke viruses [...]

biotechnology genetics health care finance innovation regulation research