Generation Bio Announces Plan to Adapt Next Generation Rapid Enzyme Manufacturing Process Across Its Portfolio, Provides Pipeline Update

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Company to Migrate All Programs in Its Portfolio to Rapid Enzyme Synthesis (RES), Improving the Quality, Scale and Speed ​​of Closed DNA Manufacturing

Signing of a lease agreement for the construction of an in-house RES manufacturing plant offering cGMP capacity and internal control over initial clinical and commercial supply

IND submission for hemophilia A is now scheduled for 2023; Factor VIII expression data with RES material in non-human primates expected at the end of 2021

CAMBRIDGE, Mass., July 14, 2021 (GLOBE NEWSWIRE) – Generation Bio Co. (Nasdaq: GBIO), an innovative biotechnology company in the field of genetic drugs for people with rare and widespread diseases, today announced its intends to deploy next generation rapid enzyme synthesis (RES) for the fabrication of its closed-end DNA (cDNA) constructs in all programs of the portfolio.

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

  • Quality: Consistently delivers very pure cDNA and reduces the variability inherent in cell-based manufacturing, which can have important regulatory and clinical benefits for Generation Bio’s entire pipeline, including its core program on hemophilia A;

  • Climb: Potential to expand the scale of manufacture of cDNA to hundreds of millions of doses, supporting the company’s intention to develop programs for prevalent diseases;

  • Speed: Shortens the cDNA 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 to build an approximately 104,000 square foot state-of-the-art cGMP (Current Good Manufacturing Practices) facility in Waltham, Mass. The facility is expected to be operational in 2023, allowing for new investigational drug (IND) application and clinical development for the company’s core hemophilia A program. The company uses existing infrastructure for SER production of materials. of research.

“We are proud of our pioneering work with our cDNA construction and cell-targeted lipid nanoparticle delivery system, and we bring the same level of innovation to manufacturing with RES. RES represents a sea change in the quality, scale and speed of cDNA production, and with our new manufacturing facility, we maintain rigorous internal quality control while achieving the scale and scope to match the promise of our platform, ”said Geoff McDonough, MD, CEO of Generation Bio. “We believe the benefits of RES merit adjusting the IND submission schedule for our hemophilia A program, and we expect the move to RES will accelerate overall development timelines in the remainder of. our pipeline.

Generation Bio expects to incur net rental costs of $ 104 million over 12 years and invest up to $ 45 million in the new manufacturing facility over the next two years. The company plans to continue its partnerships with contract manufacturing organizations during and after construction to ensure redundancy and secure an additional supply of cDNA. Spending for the new facility is not expected to impact Generation Bio’s cash flow path, which is sufficient to fund key milestones through 2024.

As Generation Bio transitions to RES, it plans 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 Hemophilia A Core Program. The company plans to report Factor VIII expression data to the aid of a drug product produced by RES in non-human primates by the end of the year.

About Generation Bio

Generation Bio innovates in the field of genetic medicines to provide durable and reusable treatments to people living with rare and widespread diseases. The company’s non-viral platform incorporates a new DNA construct called closed DNA or cDNA; a unique cell-targeted lipid nanoparticle delivery system, or ctLNP; and a highly scalable capsidless manufacturing process that uses its rapid proprietary cell-free enzymatic synthesis, or RES, to produce cDNA. The platform is designed to allow multi-year durability from a single dose, to deliver large genetic loads, including multiple genes, to specific tissues, and to allow titration and re-dosing to adjust or expand expression levels in each patient. RES has the potential to expand Generation Bio’s manufacturing scale to hundreds of millions of doses to support its mission of expanding the reach of genetic medicine to more people, living with more diseases, around the world.

For more information, please visit www.generationbio.com.

Forward-looking statements

All statements in this press release regarding the company’s future expectations, plans and prospects, including statements regarding our strategic plans or objectives, our technology platforms, our research and clinical development plans, the expected timeline submission of IND applications and preclinical data, our manufacturing plans, our expectations of 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 shown in these forward-looking statements due to various important factors, including: uncertainties inherent in the identification and product candidate development, including conducting research activities, launching t and the completion of preclinical studies and clinical trials and clinical development of the Company’s product candidates; uncertainties regarding the availability and timing of results from preclinical studies and clinical trials; whether the results of preclinical studies will predict the results of preclinical studies and subsequent clinical trials; uncertainties regarding the timing and ability to complete construction of the Company’s manufacturing facility and regarding the new manufacturing process; expectations regarding when to submit IND applications; expectations for regulatory approvals to test or market products; challenges in manufacturing genetic medicine products; whether the company’s cash resources are sufficient to finance the company’s operating expenses and capital expenditure needs for the expected period, including the financing of the new manufacturing plant; the impact of the COVID-19 pandemic on the business and operations of the company; as well as the other risks and uncertainties set out in the “Risk Factors” section of our most recent annual report on Form 10-K and the Quarterly Report on Form 10-Q, which are filed with the Securities and Exchange Commission , and in subsequent filings the company may make with the Securities and Exchange Commission. Further, the forward-looking statements included in this press release represent the views of the Company as of the date hereof. The company expects subsequent events and developments to cause a change in the company’s perspective. However, although the company may choose to update these forward-looking statements at some time in the future, the company specifically disclaims any obligation to do so. These forward-looking statements should not be taken as representing the views of the company as of a date subsequent to the date on which they were made.

Contact:

Investors
Maren killackey
Generation Bio
[email protected]
541-646-2420

Media
Alicia webb
Generation Bio
[email protected]
847-254-4275

Stephanie Simon
Ten communication bridges
[email protected]
617-581-9333


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