re: Generation

October 14, 2021

Changing the future of manufacturing at Generation Bio

BY Leslie Wolfe, Ph.D., Senior Vice President, Head of Chemistry, Manufacturing & Controls

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.

Safe Harbor Statement: Any statements in this press release about future expectations, plans and prospects for the company, including statements about our strategic plans or objectives, our technology platforms, our research and clinical development plans, the expected timing of the submission of IND applications and preclinical data, our manufacturing plans, our expectations regarding our new facility and other statements containing the words “believes,” “anticipates,” “plans,” “expects,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities, the initiation and completion of preclinical studies and clinical trials and clinical development of the company’s product candidates; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; uncertainties regarding the timing and ability to complete the build-out of the Company’s manufacturing facility and regarding the new manufacturing process; expectations regarding the timing of submission of IND applications; expectations for regulatory approvals to conduct trials or to market products; challenges in the manufacture of genetic medicine products; whether the Company’s cash resources are sufficient to fund the company’s operating expenses and capital expenditure requirements for the period anticipated, including the funding of the new manufacturing facility; the impact of the COVID-19 pandemic on the company’s business and operations; as well as the other risks and uncertainties set forth in the “Risk Factors” section of our most recent annual report on Form 10-K and quarterly report on Form 10-Q, which are on file with the Securities and Exchange Commission, and in subsequent filings the company may make with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company’s views as of the date hereof. The company anticipates that subsequent events and developments will cause the company’s views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the company’s views as of any date subsequent to the date on which they were made.

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