Generation Bio Announces Plan to Scale Next-Generation Rapid Enzymatic Manufacturing Process Across Portfolio and Provides Pi...
July 14 2021 - 7:30AM
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.
About Generation Bio
Generation Bio is innovating genetic medicines to provide
durable, redosable treatments for people living with rare and
prevalent diseases. The company’s non-viral platform incorporates a
novel DNA construct called closed-ended DNA, or ceDNA; a unique
cell-targeted lipid nanoparticle delivery system, or ctLNP; and a
highly scalable capsid-free manufacturing process that uses its
proprietary cell-free rapid enzymatic synthesis, or RES, to produce
ceDNA. The platform is designed to enable multi-year durability
from a single dose, to deliver large genetic payloads, including
multiple genes, to specific tissues, and to allow titration and
redosing to adjust or extend expression levels in each patient. RES
has the potential to expand Generation Bio’s manufacturing scale to
hundreds of millions of doses to support their mission to extend
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
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.
Contact:
Investors Maren KillackeyGeneration
Biomkillackey@generationbio.com541-646-2420
Media Alicia WebbGeneration
Bioawebb@generationbio.com847-254-4275
Stephanie Simon Ten Bridge
Communicationsstephanie@tenbridgecommunications.com617-581-9333
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