What we owe ALS patients — and why one company fell short

Peter Marks wants drug developers to ask more stupid questions.

It’s part of the top Food and Drug Administration official’s plan to reinvigorate gene therapy, a field that has struggled despite significant technological advances. Some companies are shelving programs or going out of business, even when they have promising data. 

advertisement

The problems are numerous: The diseases are often exceptionally rare, limiting the potential market. Manufacturing at commercial quality is complex and expensive. Proving a drug works can be difficult, because there may be too few patients to run a traditional randomized study. 

Get unlimited access to award-winning journalism and exclusive events.