Our Vision for the Future of Cell and Genetic Therapies at Vertex

December 12, 2019 8:00 am

Our Vision for the Future of Cell and Genetic Therapies at Vertex

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 in outcomes. 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.

Our knowledge of human genetics and the underlying biology of disease has exploded over the last decade. Coupled with the discovery and rapid evolution of new therapeutic technologies, the industry is poised to accelerate advances in medicine in ways we can only begin to appreciate today. Over the next 30 years, treatments will move beyond addressing the symptoms of a disease to actually preventing and curing it altogether. We’ll develop personalized therapies tailored to an individual’s genome. And we’ll live in a world in which the burden of diseases like sickle cell disease and diabetes are memories of the past.

It’s an incredibly exciting time to be in science and medicine, and an even more exciting time to be at Vertex.  

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. We focus our efforts on scientific innovation, because we believe that’s the only way to create real value in this industry. We only work on medicines that we believe will have a transformative impact for patients (we don’t develop “me too” drugs or reformulations), because those medicines bring the most value to patients and society. And we focus on serious diseases in specialty markets, meaning we can bring a medicine to patients with a relatively small sales and marketing budget and reinvest the vast majority of our product revenues into research and development. This creates a virtuous cycle of innovation that enables us to sustainably discover and develop new medicines.

It’s the reason we’ve been successful in discovering four breakthrough cystic fibrosis medicines in less than eight years, and it’s the secret sauce that will allow us to do it again in multiple other serious diseases that fit our strategy.

Genetic and cell therapies represent two rapidly emerging therapeutic modalities with the potential to treat—and even cure—several of the diseases we’re focused on at Vertex. But to be successful, we’ll need the best technologies, manufacturing capabilities and scientific expertise out there. That’s why we’re assembling “dream teams” in cell and genetic therapies. We’re scouring every corner of the globe to find the tools and people we need to succeed.

A perfect example is the work of our research team in conjunction with our acquisition of Exonics and partnership with CRISPR Therapeutics to build a leading gene editing platform for Duchennes muscular dystrophy (DMD) and myotonic dystrophy (DM1)—two devastating neuromuscular diseases.  We’re incredibly excited about the science the team is pioneering in these two diseases and we believe it could lead to transformative gene editing therapies for people living with DMD and DM1.

In the area of cell therapies, we have focused our initial efforts on type 1 diabetes, a devastating disease affecting more than 1.25 million Americans. We recently acquired Semma Therapeutics, which we believe has the leading technology for treating people with this disease,  through stem cell-derived islet transplantation. We hope to bring this cutting-edge technology to patients in the near future.

In order to realize the full potential of our vision in cell and genetic therapies, we need to substantially grow our R&D teams in these two areas. We’re actively looking for the best and brightest talent to join our team. Our plan is to grow our Vertex Cell and Genetic Therapies group to several hundred people over the next few years, and we’re building a dedicated new research site for that team. This new building, located at Innovation Square in Boston’s Seaport District, will be home to research, vector development and clinical manufacturing for cell and genetic therapies.

I’ve never been more excited about the future of Vertex and the promise of cell and genetic therapies. If you share my excitement, please consider joining our team.

To learn more about our openings in cell and genetic therapies, search “genetic therapies” in our available positions.