Nature Medicine asks leading researchers to name their top clinical trial for 2025, from genetherapies for prion disease and sickle-cell disease to digital tools for cancer and mental health.
2024’s big winners were the pharmaceutical companies behind the blockbuster weight-loss drugs semaglutide (Novo Nordisk’s Wegovy) and tirzepatide (Eli Lilly’s Zepbound). With other biotechnology and pharmaceutical companies now rushing to enter the weight-loss market, obesity will surely be a priority for 2025. Against this fizz of excitement, it is difficult to predict what other discoveries will attract the biomedical industry’s attention. We asked 11 experts which trials in the coming year are likely to have an outsized impact on medicine (Table 1).
Gene therapy for prion disease
Sonia Vallabh: I retrained from being a lawyer to do biomedical research after I discovered. I was at risk for dying of genetic prion disease.
Prion disease is not ultra-rare, since it kills 1 in 6,000 people, but it is rare. I helped establish the preclinical proof of concept that has led to a phase1/2 trial to evaluate the safety, tolerability
and pharmacokinetics of intrathecally administered ION-717 in patients with prion disease.
ION-717 is an investigational antisense oligonucleotide developed by Ionis Pharmaceuticals and was designed to inhibit the production of prion protein. As of right now, there are 16 clinical trial sites for ION-717 around the world. This trial has enrolled really quickly because this is a very active community that will show up. I see my work as campaigning for earlier treatment across the spectrum of neurodegeneration, as the earlier you get there, the more you can do to preserve brain function. We may see the first data by late 2025.
Sonia Vallabh is a senior group leader at The Broad Institute of MIT and Harvard, Cambridge, MA, USA.
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