“I think the most important thing for the Vertex community is to think about the patient as a whole person, and from there to understand that it’s still possible to do so much good work in this space. And we’re really excited that Vertex is taking that on.” – Pranav Saha, living with transfusion-dependent beta thalassemia

Everything we do is with patients in mind. We understand how important it is to see a patient as a whole person, and that’s why our teams are committed to continuing research with the aim of transforming the lives of people living with beta thalassemia. Not only are we dedicated to our research, but we have learned so much from working with the beta thalassemia community and with people like Pranav. 

A winning idea

Our commitments in this space go back nearly a decade, when a team member’s personal tragedy ignited the spark for progress and innovation at Vertex. In 2015, Vertexian Setu Roday pitched the idea of pursuing research on the disease as part of Vertex’s inaugural innovation challenge. Thalassemia was ever-present for his family in India where he grew up. His younger brother passed away from beta thalassemia at age six, and everyone in his family is a carrier for thalassemia.  

The innovation challenge gave Setu a platform to collaborate, innovate and pitch the winning idea to research, “A novel approach to the treatment of beta thalassemia.” While the initial idea was focused on a small molecule approach, our research strategy is modality-agnostic — we try to tackle diseases from many angles — and we began working on multiple research approaches with the goal of hopefully addressing the underlying cause of beta thalassemia later that same year. It’s remarkable to see how this combination of personal loss and a commitment to innovation across all of Vertex came together and sowed the opportunity to pursue treatments for beta thalassemia.

Our global commitment to you — the beta thalassemia community

Our commitments in research kicked off the beginning of our work with the beta thalassemia community. Through the years, as our research progressed, we also dedicated time and resources to supporting the community in a variety of ways. In 2022 and 2023, this included more than $1.2 million in independent grants toward global advocacy groups, medical education and conferences for beta thalassemia.

Throughout 2023, we collaborated with people impacted by beta thalassemia from across the globe to bring their voices and images into disease awareness campaigns. In the UK, Italy, France and Germany, we continue to foster strong patient advocacy group relationships by bringing groups together to share best practices, holding Community Advisory Boards to help us better understand their lived experiences and ensuring we always hear from the patient communities. Our partnership with the Thalassemia International Federation (TIF) brought the global patient voice to the forefront, sponsoring the International Patient Conference in Kuala Lumpur, Malaysia, discovering new insights in understanding the patient journey. We also collaborated with the Global Action Network for Sickle Cell and Other Inherited Blood Disorders (GANSID) to sponsor their Round Table Partnership, exploring beta thalassemia in the developing world.

We have welcomed the insights and guidance of those impacted by beta thalassemia through patient panels to help us develop better education and materials. In the U.S., we work closely with the Cooley’s Anemia Foundation, participating and sharing information at their annual Care Walks and Patient-Family Conferences. In October 2023, we were recognized at the Cooley’s Gala with an award for “individuals who make outstanding contributions to thalassemia.” It was an honor to receive this award because it reinforces our uncompromising commitment to people living with beta thalassemia, and we look forward to continuing to build relationships with the team at Cooley’s.

Hope for the future

Over the years, through all the community gatherings, roundtables and discussions, we’ve been able to better understand and support the needs of people living with beta thalassemia. It’s been so exciting to witness change in the community, as we see groups like Cooley’s shift their annual roll call from memorializing patients lost throughout the year to celebrating their milestone birthdays instead. We’re so happy to be a small part of this transition as patients can shift focus to the potential of their futures. 

And as we look to our future at Vertex, we remain steadfast in our commitments to beta thalassemia and the community. We’re proud to support them as they work on broadening support and community empowerment for beta thalassemia. We hope to continue elevating, amplifying and shining a light on each patient group and their dedicated efforts to raise awareness and change the course of this disease.

To everyone living with beta thalassemia — thank you for working with us as we strive toward a brighter future.