Science is all about change – making new discoveries that propel us forward. In my career, this change has been reflected in how we’re able to manufacture and deliver the medicines that people need.
Sometimes change is incremental. Other times, it launches us into a new era. That latter possibility is why I’m so excited about our recently announced next-generation rapid enzymatic manufacturing (RES) process at Generation Bio.
This is a major shift that will allow us to create higher quality products, much more quickly and efficiently, with the potential to increase scale to hundreds of millions of doses.
So why does this matter? Because our mission at Generation Bio is to unlock the full promise of genetic medicine, not just for some diseases and some patients, but for all. The manufacturing processes used currently for complex genetic therapies, like AAV-based gene therapies, simply are not able to produce enough doses to treat highly prevalent diseases. This is one reason why most genetic medicines in development are for rare diseases. Solving these manufacturing challenges could make access to genetic medicines widespread and sustainable.
At Generation Bio, we are transitioning all of our portfolio programs to RES for manufacturing of our closed-ended DNA (ceDNA). RES has three major advantages over the cell-based manufacturing methods currently employed by the gene therapy and biologics industry.
First, because it’s an enzymatic production process that doesn’t rely on cells, it avoids the inherent variability associated with biological processes and consistently yields highly pure ceDNA. This has important quality and regulatory implications as we move toward the clinic.
Second, it’s highly scalable, with the potential to produce up to hundreds of millions of doses. That could enable us to treat rare diseases on a global basis, and potentially to treat highly prevalent ones.
Third, it’s extremely fast. RES produces ceDNA in a single day (down from 28 days under our prior system), accelerating our drug discovery efforts tremendously. We can test hypotheses and answer questions much more quickly.
It’s important to note that we got here through the insights, ingenuity and hard work of many. I truly believe that these types of advances could only happen at a special place like Generation Bio. And in the beginning, it was just a shell of an idea jotted down on a napkin. Then we pulled in people across every function – manufacturing, molecular biology, preclinical, quality, regulatory and legal. We put everyone’s heads together to figure out a better way to manufacture our products. It was a hugely rewarding scientific experience.
From a few scribbled notes to a company-wide manufacturing shift, we’ve certainly come a long way – and we’re not stopping here. We’re in the process of building out a roughly 100,000-square-foot state-of-the-art manufacturing facility in Waltham, Mass. to scale RES for clinical and initial commercial supply.
I am so proud of what our team has been able to accomplish, and for what this ultimately could mean for patients. The opportunities ahead have never been brighter, and I’m thrilled to see what new changes the future holds.
