Vertex’s Paul Negulescu, Ph.D., Wins 2025 Gairdner Award for Cystic Fibrosis Breakthroughs
In a landmark moment for both Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and the broader scientific community, Paul Negulescu, Ph.D., Senior Vice President at Vertex, has been named a recipient of the prestigious 2025 Canada Gairdner International Award. This recognition honors Dr. Negulescu’s pioneering contributions to the understanding and treatment of cystic fibrosis (CF), a genetic disease that historically had limited treatment options and grim prognoses.
Negulescu shares this distinction with Michael J. Welsh, M.D., of the University of Iowa, another luminary in the field whose foundational research has also played a critical role in illuminating the molecular and cellular underpinnings of cystic fibrosis.
The Gairdner Foundation cited the award recipients “for pioneering research into the cellular and molecular mechanisms underlying the genetic disease cystic fibrosis, leading to the development of transformative drug therapies based on these mechanisms, thereby improving and saving countless lives.”
This international accolade is not merely a personal milestone for Negulescu, but also a profound validation of the multi-decade scientific enterprise that he has helped lead at Vertex. His work, and that of his collaborators, has fundamentally altered the therapeutic landscape for CF, shifting it from one of symptomatic relief to disease-modifying treatments targeting the root genetic cause.
From Gene Discovery to Molecular Insight: The Journey Toward a Cure
Cystic fibrosis is a life-threatening genetic disorder that primarily affects the lungs and digestive system. It is caused by mutations in the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene, which encodes a protein responsible for regulating the flow of salt and fluids in and out of cells. When CFTR is defective or absent, thick, sticky mucus accumulates in various organs, especially the lungs, leading to severe respiratory and digestive problems.
The CFTR gene was first identified in 1989, a watershed moment that ushered in new hope for families affected by CF. However, despite this genetic breakthrough, progress in developing treatments that addressed the root cause of the disease was slow. For years, available therapies focused on symptom management—antibiotics, mucus thinners, pancreatic enzyme replacements—without correcting the fundamental cellular defect.
It wasn’t until the early 2000s that significant strides were made toward disease-modifying therapies. That turning point began with the visionary research leadership of Dr. Negulescu at Vertex.
A New Therapeutic Era: Correcting the Defect, Not Just the Symptoms
Under Negulescu’s guidance, Vertex embarked on a mission to develop small-molecule drugs that could restore function to defective CFTR proteins—an approach once thought to be impossible. This bold direction led to the creation of the first CFTR modulators, therapies designed to either “potentiate” the function of the protein or “correct” its folding and trafficking inside cells.
The result of these years of research and testing has been nothing short of transformative. To date, Vertex has brought to market five CFTR-targeting therapies, each building upon the scientific insights and clinical successes of its predecessor.
The most advanced among these is a triple combination therapy, commercially known as Trikafta® (elexacaftor/tezacaftor/ivacaftor). Approved by the U.S. Food and Drug Administration in 2019 and subsequently in numerous global markets, this once-daily oral treatment is designed for people with one or more F508del mutations, the most common CF-causing genetic variant. It has the potential to treat more than 90% of the global CF population, representing an unprecedented leap in disease coverage and quality-of-life improvement.
Behind the Breakthrough: A Legacy of Vision and Collaboration
“For more than 20 years, Paul and the team of dedicated Vertex researchers have focused on discovering and developing breakthrough therapies for people living with cystic fibrosis,” said David Altshuler, M.D., Ph.D., Executive Vice President, Global Research, and Chief Scientific Officer at Vertex. “Paul’s outstanding vision, leadership, determination, and collaborative nature has resulted in what was once thought impossible — the discovery of the first-ever protein folding corrector medicines that treat the underlying cause of CF and have forever transformed the course of this disease.”
These innovations have not only extended the lives of patients but also fundamentally reshaped the medical understanding of how to target misfolded proteins in genetic diseases. Vertex’s CF research has served as a blueprint for other drug discovery programs aiming to address diseases at a molecular level.
In response to receiving the Gairdner award, Dr. Negulescu offered humble but heartfelt gratitude: “I am honored and humbled by this award that recognizes not only me, but also the dedication, creativity, and scientific excellence of the thousands of people across Vertex, and in the CF community, who have been steadfast in their commitment to bringing these medicines to people with cystic fibrosis around the world.”
Innovation Beyond the Present: Addressing the Remaining 10%
While the impact of Vertex’s CF therapies is far-reaching, the company’s scientific mission is far from complete. An estimated 10% of people with CF possess mutations that do not respond to currently approved CFTR modulators. These include nonsense mutations that result in truncated, nonfunctional proteins, as well as rare mutations with unique cellular profiles.
To address this unmet need, Vertex is investing in next-generation research, including novel therapeutic platforms like mRNA-based therapies. These therapies aim to deliver functional CFTR transcripts directly to cells, offering a potential solution for those whose mutations do not produce viable protein targets for existing drugs.
Clinical trials are currently underway to evaluate the safety and efficacy of these mRNA therapies, and early data suggests a promising path forward. Vertex is also exploring gene editing and gene therapy approaches to provide durable, possibly curative, solutions for all individuals living with CF.
The Gairdner Legacy: Recognizing the World’s Most Impactful Science
The Canada Gairdner International Award is one of the most esteemed honors in the global scientific community. It is bestowed annually by the Gairdner Foundation, which has spent decades identifying and celebrating trailblazing biomedical researchers whose work has significantly advanced our understanding of human biology and disease.
Since its inception in 1959, over 400 scientists have received the Canada Gairdner Award, and more than 90 of them have subsequently gone on to win the Nobel Prize, underscoring the award’s role as a harbinger of global scientific acclaim.
Recipients of the Gairdner are selected through a rigorous peer-review process that evaluates both the novelty and impact of the scientists’ work. The selection of Drs. Negulescu and Welsh places them among the ranks of the most influential scientists of our time.
A Milestone in Medicine, A Vision for the Future
The recognition of Paul Negulescu with the 2025 Canada Gairdner International Award marks a defining moment in the field of genetic disease research and pharmaceutical innovation. His contributions, together with those of his colleagues at Vertex and collaborators around the world, have transformed cystic fibrosis from a once-debilitating childhood disease to a manageable chronic condition for the majority of patients.
Yet perhaps even more important than past successes is the vision for the future: a world where all people with CF, regardless of mutation type, can access therapies that restore their health and prolong their lives. Through continued innovation, collaboration, and relentless pursuit of scientific understanding, that future draws ever closer.
As Vertex continues to push the boundaries of molecular medicine, the legacy of Dr. Negulescu’s work will endure — not only in the accolades it garners but in the countless lives changed, extended, and empowered by the therapies it inspired.
