Gene Therapy Is Giving Us Incredible New Tools to Fight Disease. How Far Can It Go?

After decades of struggle, we’re finally seeing real-world, life-changing applications right here in Philadelphia. Has medicine finally got gene therapy right?

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dna in syringe

Think back to last spring. (You probably don’t want to, but humor me.) Those early months of the coronavirus pandemic in America weren’t all fear and chaos — though there certainly was that. There was also optimism, which, when one looks back on it now, seems completely untethered from reality.

You almost certainly heard it: Flu is worse. We are very, very ready for this. We’re just shutting down for two weeks. Considering our national death toll of more than half a million and counting, the optimism was clearly misplaced. Even so, one rosy prediction came true in a big way.

In March 2020, we were told a vaccine to prevent COVID-19 was a year to 18 months off — even though normal vaccine development takes years, often more than a decade, with no guarantee of success. We still don’t have vaccines to prevent AIDS or childhood RSV infections.

But on November 9th, pharmaceutical companies Pfizer and BioNTech announced that their two-dose vaccine was more than 90 percent effective in preventing COVID-19. Another company, Moderna, soon released similar news.

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