FDA official offers insights into whether gene therapy makers could use common manufacturing process

WASHINGTON — A Food and Drug Administration official provided some insights this week into the FDA’s thinking about a groundbreaking approach to making gene therapies that could help make the products more profitable and attractive to investors.

At issue is whether makers of gene therapies may one day use a common manufacturing platform.

Gene therapies fix genetic defects, and they work well for treating extremely rare conditions, according to Peter Marks, the head of FDA’s biologics center. Rare diseases collectively affect millions of Americans, and there are many gene therapies in development for rare conditions. Lately, however, investors are not nearly as willing to fund cell and gene research as they once were, in part due to manufacturing challenges.

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