Over the past decades, biopharmaceutical innovation has been redefining possibilities in healthcare. Advances in our understanding of human biology have enabled the development of therapies that target the root causes of disease, rather than merely alleviating symptoms. Cell and gene therapies, RNA-based approaches, and precision medicines are transforming patient outcomes with serious, rare, and life-threatening conditions.
At Vertex, we believe that the true value of the biopharmaceutical industry lies in scientific innovation. This belief is at the core of our mission and shapes every aspect of our strategy. We focus on diseases that we commit to deeply understand at a biological level and for which we can develop and rigorously evaluate therapeutic approaches using human cell–based models. Our R&D strategy leverages advanced technologies ranging from small molecules and cell therapies to gene editing and messenger RNA, with the ultimate goal of delivering the best possible outcomes for patients.
This long-term commitment is reflected in our investment profile. Approximately 60% of our global workforce is dedicated to research and development, while more than 70% of our operating expenses are allocated to R&D. We believe that sustained investment in science is essential not only for long-term business success, but also for addressing critical unmet medical needs and improving patients’ lives.
Through this approach, Vertex has achieved sustained innovation, with cystic fibrosis (CF) representing a defining example. Thanks to our commitment to understanding the pathological mechanism underlying CF, we have developed therapies that act directly on the cause of the disease transforming life expectancy and quality of life for the majority of people living with CF.
We are applying the same principles across other serious diseases, including alpha-1 antitrypsin deficiency, sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, and type 1 diabetes. Today, our portfolio spans ten therapeutic areas in clinical development, with significant progress expected in five additional disease areas over the next years.
Our commitment to innovation, however, extends beyond scientific strategy alone. It also encompasses meaningful engagement with the patient community. We work closely with patients and their families to understand the everyday challenges they face, ensuring that the solutions we develop respond to real needs.
Yet, without robust investment in pharmaceutical innovation, the benefits of any scientific advances will remain out of reach. It is important to note that this is innovation that is not on paper, or in theory. This is innovation that offers real positive impact on people’s lives, patients and caregivers, society as a whole and our countries’ health systems.
In Greece, pharmaceutical companies are required through mandatory clawbacks to cover a larger share of pharmaceutical expenditure, which in 2025 has raised to up to 74% in the hospital channel and up to 67% in the EOPYY pharmacies channel. This has created difficult environment for Greek patients to enjoy the benefits of innovation. At Vertex, our goal is the value of innovation for rare diseases, for Gene & Cell therapies, for cancer to be appreciated and nurtured earlier, so that patients can have access to this innovation as early as possible and can make a difference to the lives of those affected. We believe it is critical toseriously consider ways to provide meaningful access to innovative products.
As an active member of the PhARMA Innovation Forum (PIF), Vertex supports dialogue and policy initiatives that recognize innovation not as a cost, but as an investment in public health and society. The “Voices of Innovation” initiative highlights this shared commitment: by investing in pharmaceutical innovation, we can transform the lives of patients, tackle some of the most serious challenges in medicine, and continue delivering meaningful progress for years to come.
Panagiotis Nikakis, Country Lead, Vertex Greece
