After years of setbacks, the field is starting to deliver on its promises.
But the afterglow of that early achievement proved short-lived, as a labyrinth of technical challenges emerged. A common gene-therapy approach involves delivering a healthy gene to cells that have only defective copies in their genetic libraries. Once inside, the therapeutic gene instructs the cells to manufacture functional proteins instead of faulty ones.
Over the past decade, efforts to vanquish the spectre of failures past have started to pay off, heralding an era of optimism, particularly in the treatment of genetic eye disorders. There are almost 300 clinical trials worldwide investigating viral-vector-based gene therapies. Roughly half of these use modified adeno-associated viruses — small, well-studied viruses that do not cause disease in humans.
The Luxturna milestone laid the groundwork for tackling more-common eye disorders underpinned by multiple genes, says Edwards. “Inherited retinal diseases have sort of been the starting point, because they lend themselves to a gene-therapy approach,” he says. “We’re now seeing the technology being applied to more common diseases, which are more genetically complicated.”
Treating muscular diseases with AAV-based gene therapy is a fine balance; massive doses are needed to make a therapeutic impact, which increases the chance of adverse reactions, says Crudele. “You truly have to hit every single cell, which means that you have to give these really huge doses.”
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