It was a good year for new treatments for genetic diseases! Of the 44 FDA approvals of new drugs, 8 were for 6 single-gene diseases: DMD, beta thalassemia, cystic fibrosis, a form of amyloidosis, and two each for sickle cell disease and porphyria.
The eight approvals showcase the diverse therapeutic strategies that are finally leaping from clinical trial to clinical reality. That's important. DNA, RNA, and protein-based treatments face an especially high bar because of the perceived permanence of a correction at these levels of genetic information.
Slowing of disease progression or improvement in one or two symptoms are signs of success, but it might take time for some molecular corrections to translate into fading symptoms. That's why the multi-pronged strategies are critical.
If a gene therapy isn't leading to rapid or obvious improvement in a child with a brain or muscle disease, then perhaps RNAi, antisense therapy, or enzyme replacement therapy will. Better yet, instead of testing the technologies in tandem, do it in parallel.
I hope that this past year's progress isn't lost in the hype over gene editing in general, and CRISPR in particular. The media sometimes paints an unrealistic portrait of looming miracle cures and breakthroughs, which I analyzed last week here. New medical treatments are based on science, and that takes time – typically, three decades.
Here's a look at a handful of new treatments for genetic diseases approved in 2019, and how they work.
To continue reading, go to my DNA Science blog at Public Library of Science, where this post first appeared.