The New Era of Life Sciences: The Future is Now

Newsweek

16-05-2025

When we published our 2024 report, children suffering from Leber Congenital Amaurosis 4 (LCA4)—a severe inherited retinal dystrophy—had only hope that one day they might see the world. Today, thanks to an investigational gene therapy, we know that the 11 children who participated in the trials can, for the first time in their lives, discern a toy, recognize a human face and even spot a grain of sand. The biotech behind this achievement is MeiraGTx, which has now filed its therapy for approval under exceptional circumstances in the U.K., which would expedite the process.
Stories like that of MeiraGTx illustrate the transformative power of gene therapies on patients. More broadly, they highlight the potential for rare diseases R&D to emerge as the principal driver of therapeutic innovation. "Rare diseases have become a critical area for exploring new treatment modalities because, in many cases, there is no existing therapy, or even a model. For patients who have no options, innovative approaches are important," shares Marc Dunoyer, the CEO of Alexion, the rare diseases subsidiary of AstraZeneca. Ironically, the very severity and uncommonness of these illnesses can also be a blessing, as it allows for bolder approaches by both researchers and regulators. Moreover, rare disease drugs have historically had higher approval success rates compared to other drugs. The Tufts Center for the Study of Drug Development found that orphan-designated drugs had a Phase-1-to-approval success rate of 17 percent, compared to 7.9 percent for non-orphan drugs. Benefiting from faster approval pathways and enhanced regulatory incentives, entrepreneurs in the field face better odds of innovating successfully.
And when they do, the implications of their therapies go far beyond their original target. "Many of the advances seen today, such as gene editing and RNA-based therapies, originated in rare disease research before moving into larger indications," says Dunoyer. Arcturus Therapeutics, for instance, is addressing cystic fibrosis via a new delivery mechanism. "Our cystic fibrosis program is a prime example, where the ability to inhale mRNA safely could have a huge impact on the pharmaceutical industry. The rare disease field offers a shorter regulatory pathway to approval and a higher likelihood of success. This makes it an attractive space for evaluating next-generation technologies like mRNA therapeutics," shares Arcturus' CEO, Joseph Payne, as the company is expecting results from their Phase 2 trials in 2025.
Matt Sause, CEO, Roche Diagnostics. Credit: Courtesy of Roche Diagnostics.
Matt Sause, CEO, Roche Diagnostics. Credit: Courtesy of Roche Diagnostics.
We can perform comprehensive genomic profiling to uncover the molecular basis of a tumor. This allows doctors to provide tailored treatments. Over time, this will enable us to move to a future where we understand the molecular drivers for cancer and can deliver truly personalized healthcare.
Jacob Thaysen, CEO, Illumina. Credit: Courtesy of Illumina.
Jacob Thaysen, CEO, Illumina. Credit: Courtesy of Illumina.
Whether it is providing clarity for families dealing with rare genetic disorders or enabling rapid and precise cancer diagnoses, our goal is to ensure patients receive the answers they need when they need them.
This report has been paid for by a third party. The views and opinions expressed are not those of Newsweek and are not an endorsement of the products, services or persons mentioned.
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