A New Era of Gene Therapy: FDA’s Approval of Itvisma and What It Means

By Minsung Kim ’26 • Dec 5, 2025

Every generation witnesses a medical breakthrough—a moment when science pushes past long-standing limits and reshapes what we believe is possible. These breakthroughs are not merely incremental improvements; they redefine our understanding of disease, treatment, and hope. We don’t usually notice these shifts until one stands out so dramatically that it forces us to rethink what modern medicine can do.

Last week, such a moment arrived.

The U.S. Food and Drug Administration approved Itvisma, a gene therapy for spinal muscular atrophy (SMA), for patients aged two and older, including adults. This therapy delivers a functional copy of the SMN1 gene, directly addressing the genetic root of a condition that was once among the leading causes of infant mortality in the U.S.

“Today’s approval shows the power of gene therapies,” said Dr. Vinay Prasad of the FDA. His statement reflects a broader reality: the boundaries of medicine are expanding, and with them, the possibilities for patients who previously had no options.

SMA is a devastating neurodegenerative disorder. It leads to progressive muscle weakness, loss of motor function, and—in its most severe forms—death. For decades, effective treatment seemed out of reach. But the approval of Itvisma demonstrates how far our scientific capabilities have come. Its design uses an AAV vector to deliver therapy directly into the cerebrospinal fluid, allowing motor neurons to access the gene they lack. The treatment requires only a single intrathecal injection, independent of patient weight.

To many, this may seem like just another news headline. But beneath the technical terms lies a much deeper lesson: progress is rarely linear. Medical innovation often involves years of trial, failure, and reconsideration. Yet each step builds toward something greater.

I’m reminded of how easily we assume that change will come through dramatic leaps. In reality, breakthroughs like Itvisma are the result of countless small advances—experiments, adjustments, and persistence. Even the FDA relied on previous data from Zolgensma, another SMA gene therapy, to validate the safety profile of Itvisma. Every study, every dataset, every failed attempt became part of the foundation for this achievement.

And like scientific progress, our own growth works in a similar way. We often measure success by the final outcome—whether a disease is cured, whether a goal is reached. But the real transformation happens in the incremental steps leading there.

Itvisma’s development also reminds us of the importance of staying present. Researchers spent years focusing on each stage of improvement rather than rushing toward the final approval. When I study or work toward my own goals, I often fall into the trap of worrying about the future—grades, results, expectations. But breakthroughs, both scientific and personal, come from attention to what’s right in front of us.

The FDA emphasized that despite its promise, Itvisma requires caution, especially for adult patients who may face increased risks like hepatotoxicity. Progress always carries uncertainty. Yet uncertainty doesn’t negate possibility—it simply means we push forward carefully, one informed step at a time.

Ultimately, the approval of Itvisma marks more than just a new therapy. It represents a shift in how we confront rare diseases, blending innovation with persistence. It’s a reminder that the future of medicine is being shaped today, through deliberate effort, curiosity, and a willingness to challenge what once seemed impossible.

And just like science, our lives unfold in the present moment. That’s where progress happens—one small step at a time.