For fans of both science and Marvel, the latest Spider-Man film, Brand New Day, offers a curious glimpse of a real medical frontier. Peter Parker's DNA goes haywire, and he turns to RNA interference—a genuine gene-silencing technique—to tame his spider instincts. But the technology behind the superhero's fix is no fantasy; it is already changing lives across Europe.
Gene therapy, at its core, targets the genetic roots of disease. Instead of managing symptoms, it introduces nucleic acids—DNA or RNA—into a patient's cells to correct, add, or silence genes. This can be done directly in the body (in vivo) or by modifying cells outside the body and then returning them (ex vivo). Delivery relies on viral or non-viral vectors, which act as molecular delivery trucks.
Alberto Auricchio, director of the Telethon Institute of Genetics and Medicine in Naples and president of the European Society of Gene & Cell Therapy, explains: “Gene therapy basically targets the root cause of a genetic disease.” The approach has expanded from rare monogenic disorders to potential treatments for cancer and HIV.
From lab bench to bedside in Europe
Europe has moved beyond clinical trials. “We are beyond clinical trials now. There are approved [gene therapy] products that are available to patients,” Auricchio told Euronews Next. The first in vivo gene therapy approved in the EU was Glybera, authorised in 2012 for a rare form of pancreatitis. The first ex vivo therapy, Strimvelis, followed in 2016, developed at the Hospital San Raffaele in Milan to treat severe combined immunodeficiency.
These milestones mark a shift from theory to practice, but the field still faces formidable obstacles. The most significant is cost. Developing these therapies is expensive, and the final price tags reflect that. Strimvelis, for instance, costs €594,000 excluding VAT. Auricchio notes, “These are very expensive drugs to be developed, and those that get to the market have very high prices, some of them matching millions of euros.”
Because many gene therapies target rare diseases, the market is small, which discourages traditional pharmaceutical investment. “These drugs are developed for rare diseases in many cases, which are not a very attractive market for typical developers like pharmaceutical companies; the high development cost of this drug is a challenge,” Auricchio said.
Despite the price, the potential payoff is durability. Unlike chronic medications, gene therapy aims for a one-time fix. “There are approved gene therapy drugs, for which we have decades of follow-up in patients where we know that the therapeutic effect is stable over decades, and in theory for the lifetime of the individual,” Auricchio explained.
In the film, Peter Parker has the luxury of a brilliant scientist like Bruce Banner to craft his treatment. Real-world patients and researchers must navigate a more complex landscape—one where scientific breakthroughs are tempered by economic realities. As Europe's gene therapy pipeline grows, the challenge will be ensuring that these life-changing treatments are accessible to those who need them, not just those who can afford them.
For more on how Europe is navigating health innovation, see our coverage of birdwatching as a therapy for aphasia and the EU defence ministers' talks.
