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

Company to transition all portfolio programs to rapid enzymatic synthesis (RES), enabling improved quality, scale and speed of closed-ended DNA manufacturing

Lease agreement signed to build an in-house RES manufacturing facility providing cGMP capacity for and internal control over clinical and initial commercial supply

IND submission for hemophilia A now planned for 2023; factor VIII expression data with RES material in non-human primates expected year-end 2021

CAMBRIDGE, Mass., July 14, 2021 (GLOBE NEWSWIRE) -- 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.”