Spark's CSO is wrapping up animal studies on a gene therapy for Pompe disease as new work in the field sees explosive growth

When Federico Mingozzi joined Spark Therapeutics $ONCE last fall as their new chief scientific officer, he brought along several years worth of preclinical work on a new AAV gene therapy for Pompe disease. Now, he’s finishing that lysosomal storage discovery-stage effort with a flourish, unveiling late preclinical results for their drug SPK-3006, which they’re now steering toward the clinic in hopes of proving that they can completely surpass the pricey enzyme replacement therapy — and would-be successors in the clinic — used to keep patients alive.