Generation Bio (GBIO) Announces Plan to Scale Next-Generation Rapid Enzymatic Manufacturing Process Across Portfolio and Provides Pipeline Update

Generation Bio Co. (Nasdaq: GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, announced today that it plans to deploy next-generation rapid enzymatic synthesis (RES) for manufacturing of its closed-ended DNA (ceDNA) constructs across all portfolio programs.

RES is a cell-free process that has the following advantages over cell-based manufacturing:

• Quality: Consistently yields highly pure ceDNA and reduces the variability inherent in cell-based manufacturing, which may have important regulatory and clinical benefits for Generation Bio’s entire pipeline, including its lead hemophilia A program;
• Scale: Potential to expand ceDNA manufacturing scale to hundreds of millions of doses, supporting the company’s intention to develop programs for prevalent diseases;
• Speed: Shortens the ceDNA production cycle from 28 days to one day, which will accelerate preclinical research and development across the company’s portfolio.

The company has signed a lease agreement to build out a state-of-the-art cGMP (current good manufacturing practice) facility of approximately 104,000 square feet in Waltham, Mass. to scale RES for clinical and initial commercial supply. The facility is expected to be operational in 2023, enabling an Investigational New Drug (IND) application and clinical development for the company’s lead hemophilia A program. The company is using existing infrastructure for RES production of research material.

"We are proud of our pioneering work with our ceDNA construct and cell-targeted lipid nanoparticle delivery system, and we are bringing that same level of innovation to manufacturing with RES. RES represents a step change in the quality, scale and speed of ceDNA production, and with our new manufacturing facility we maintain strong internal quality control while achieving scale and breadth to match the promise of our platform,” said Geoff McDonough, M.D., chief executive officer at Generation Bio. “We believe the benefits of RES merit adjusting the timing of the IND submission for our hemophilia A program, and we expect the shift to RES to accelerate overall development timelines across the rest of our pipeline.”

Generation Bio anticipates incurring net lease costs of $104 million over a 12-year period and investing up to $45 million in the new manufacturing facility over the next two years. The company plans to continue partnering with contract manufacturing organizations during and after construction to ensure redundancy and secure additional ceDNA supply. Expenditures on the new facility are not expected to impact Generation Bio’s cash runway, which is sufficient to fund key milestones into 2024.

As Generation Bio transitions to RES, it expects to update its program development strategy and timelines across its pipeline. Generation Bio is currently advancing its liver-directed, cell-targeted lipid nanoparticle delivery system with RES for the lead hemophilia A program. The company expects to report factor VIII expression data using RES-produced drug product in non-human primates by year-end.

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