focus
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.
Subscribe Log In
Previous article：
FDA moves to stop vape shops from selling Elf Bar, Esco Bars
Next article： Duchenne breakthrough therapy leaves behind pioneering families
Next article： Duchenne breakthrough therapy leaves behind pioneering families
