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Over the last 30 years, drugs and devices stemming from important scientific research have revolutionized the treatment of serious diseases, so much so that we often take for granted the tremendous improvements they bring to patients. The medicines we use today to treat diseases like heart attack, stroke and HIV/AIDS have reduced the rate of deaths by 70-80% in a generation. But what’s more exciting is that we’re on the cusp of another scientific revolution.

Cell and genetic therapies represent two rapidly emerging therapeutic modalities with the potential to treat several diseases.

Two programs at Vertex Pharmaceuticals that are currently making progress, for example, include hemoglobinopathies, with a focus on sickle cell disease and beta thalassemia, and type 1 diabetes (T1D). Learn more in the animated videos below.


Sickle cell disease and beta thalassemia are both blood disorders that are caused by a mutation in the beta-globin (HBB) gene. The mutation leads to issues with the production of hemoglobin, the protein crucial for allowing red blood cells to carry oxygen throughout the body. Vertex’s approach, which uses CRISPR/Cas9 gene-editing technology, aims to turn on the body’s natural machinery to make a type of hemoglobin that can carry oxygen.

Type 1 Diabetes

T1D is a chronic, autoimmune disease where the cells in the pancreas that produce insulin are destroyed. Vertex aims to address the underlying cause by developing cell therapies to potentially replace insulin-producing cells. Building on work pioneered in the laboratory of Dr. Douglas Melton and at Semma Therapeutics (acquired by Vertex in 2019), the team is developing novel stem cell-derived islet cells to hopefully achieve this goal.

Looking ahead

Vertex research teams are focused on making a transformative impact for patients, not just an incremental benefit. They bring together some of the best technologies and enhanced manufacturing capabilities in the hopes of bringing potentially transformative therapies to patients as quickly as possible. They have assembled one of the broadest and most diverse sets of cell and genetic therapy capabilities and teams and are well poised to deliver on the promise of these technologies for patients.

By taking a disciplined, scientific approach to some of the most serious challenges in medicine, and by bringing rigor and creativity to how they are addressed, Vertex research teams aim to improve the lives of millions of people around the world who are living with devastating diseases.

To learn more about Vertex’s work in cell and genetic therapies, visit

About Vertex Pharmaceuticals Incorporated

At Vertex, our differentiated strategy is to invest in scientific innovation to create transformative medicines for people with serious diseases in specialty markets. Each piece of that strategy is critical to the success of our business model and our ability to deliver new medicines — creating a virtuous cycle of innovation that enables us to sustainably discover and develop new medicines. This is the reason we’ve been able to get approvals for four innovative cystic fibrosis medicines in less than eight years, and it’s what we hope will allow us to do it again in multiple other serious diseases that fit our strategy.

Vertex is rapidly expanding — including the development of our new state-of-the-art research site in Boston — to bring together our industry leading portfolio of cell and genetic therapy technologies and teams.