Summarizing GSK’s vision to investors last month, CEO Emma Walmsley reaffirmed that the British Big Pharma had decided to “end our investment in cell and gene therapy.” While this was hardly news to the audience—especially coming months after GSK axed cell therapy deals with Lycell and Immatics—it still served as a stark reminder that the company’s development portfolio is swimming against the industry tide.
But rather than racing to get an “off-the-shelf” CAR-T into the clinic or going all-in on a solid tumor strategy, GSK believes its genetic expertise will offer the smoothest path to market success.
“About 70% of the entire GSK portfolio now has strong human genetic underpinning and even higher than that in the research phase,” the company’s head of research, John Lepore, M.D., tells Fierce Biotech in an interview.
The theory is that genetically validated targets have at least a twofold higher chance of becoming medicines.
“It’s not a cure-all,” says Lepore. “But on a portfolio basis, if you can have a twofold inflection, that’s fundamental for our company and for the industry in general—and we’ve made significant progress on that.”
It sounds simple, but there’s a reason not every Big Pharma is betting the house on genetic targets. Lepore says that as novel potential targets are identified through genetics, half of them can’t be targeted with a small molecule or an antibody.
That’s where oligonucleotides come in. These short strands of synthetic DNA or RNA can reduce, restore, or modulate RNA through several different mechanisms. Biotechs like Alynlam and Ionis were among the early pioneers in the field, but “the science of the platform of oligonucleotides is growing beyond what those two companies originally started with,” says Lepore.