Deeper dive: Gene therapy manufacturing
Hari Pujar, Ph.D., Chief Technology Officer, discusses the unique manufacturing and scale-up challenges involved with cell and gene therapy production.
We all know what to expect from medical innovation in the movies. The low hum of a dimly lit lab after midnight. White boards covered with equations. The triumphant Eureka moment. The discovery is made, the problem is solved. Roll the credits.
If only it were so easy.
In reality, research in the lab is only the beginning and true medical innovation from idea to patient impact is complex, time-consuming, meandering and incremental. At Spark Therapeutics, a fully integrated, commercial gene therapy company, we credit decades of research in the field of gene therapy as the foundation for our potentially life-changing, one-time gene therapies. However, we believe that the only way to ensure that breakthrough science benefits patients is to make sure that innovation starts, but does not end, in the lab.
Spark Therapeutics is united by a mission to discover, develop and deliver one-time gene therapies that challenge the inevitability of genetic diseases, including inherited forms of blindness, hemophilia, and other progressive and debilitating diseases. We have hired some of the most creative and innovative minds in healthcare, and created an inclusive workplace where all ideas are heard and valued. Together, we are taking advantage of one of modern science’s biggest opportunities to dramatically transform the healthcare industry: turning the bits and bytes of the human genome into medicine.
The science behind gene therapy is highly complex, and our success is based on many years of lessons learned by our scientific and manufacturing teams as well as many others in the field. But a scientific and technological breakthrough that never reaches patients is not a breakthrough; it’s just a successful science experiment. Every healthcare stakeholder (and that’s everyone) needs to challenge the status quo to ensure future patients will see the benefit of a new generation of therapies in development. Because while Spark is currently the one of the few companies dedicated solely to gene therapy with an FDA approved product, Commissioner Scott Gottlieb recently said that he expects FDA to approve 40 gene therapies by 2022. As an industry, we must create the path that makes one-time gene therapies an option for many genetic diseases and an organization-wide focus on innovation saw us deliver multiple firsts on this path in recent years.
We collaborated with regulators to design a first-of-its-kind clinical trial and novel measures to determine whether a clinical trial is successful. And last year, the U.S. Food and Drug Administration (FDA) approved the first gene therapy for a genetic disease – a major milestone for everyone involved, and an important watershed for patients and families living with genetic conditions.
We sought input from the very stakeholders we hoped to positively affect: patients and families. Patient communities provide a holistic picture of patient need – we create a path of possibility, but they light our way. We can bring valuable innovation to patients and families by listening and learning, and then working with them to develop solutions for unmet needs. For example, we learned that genetic testing was a huge barrier for these families. We worked collaboratively with advocacy groups to develop free and simple genetic testing.
We met with and listened to healthcare professionals, payers, and policy makers. Their input helped us introduce first-ever payment models that recognize the unique nature of one-time gene therapies, including outcomes-based rebates with longer-term efficacy measures and a proposal to the Centers for Medicare & Medicaid Services (CMS) to spread the cost of potentially life-changing interventions over several years. The reception to these efforts has been strong and patients have already started to be treated. Importantly, we have helped ignite further discussion and collaboration that could profoundly impact the way society supports true innovation far beyond Spark, or gene therapy.
It is encouraging that the concept of innovation has become so ingrained in our culture that we can easily conjure the Hollywood image of a Eureka moment. If Hollywood can help us romanticize years of organizational alignment, insurance reform and government contracting, we will really be onto something. And that may make the biggest dream of all – a world where no life is limited by a genetic disease – a modern day reality.
Jeff Marrazzo is CEO of Spark Therapeutics and a champion of the potential benefits of precision medicine and healthcare reform for patients. Jeff can be found on Twitter @JeffMarrazzo.