Generation Bio Announces the Presentation of Preclinical Data on iqDNA and ctLNP Platforms at the ASGCT 27th Annual Meeting
09 5월 2024 - 7:59PM
Generation Bio Co. (Nasdaq:GBIO) a biotechnology company
innovating genetic medicines for people living with rare and
prevalent diseases, today presented five posters on its
immune-quiet DNA (iqDNA) and cell-targeted lipid nanoparticle
(ctLNP) platforms as well as its enzymatic synthesis manufacturing
at the American Society of Gene and Cell Therapy (ASGCT) 27th
Annual Meeting. A sixth presentation on the company’s ctLNP
platform will be discussed in an oral presentation on Saturday, May
11.
“The data we’re presenting at ASGCT showcase some important
mechanistic underpinnings of our novel platforms, which we believe
have the potential to overcome the core challenges facing the field
of genetic medicine,” said Matt Stanton, Ph.D., chief scientific
officer of Generation Bio. “We believe immune-quiet DNA cargo and
highly selective, targeted LNP delivery are the tools required to
unlock the full potential of non-viral genetic medicines, and we
are applying them to our current portfolio of programs in T cells,
hematopoietic stem cells, and hepatocytes.”
Data included in the posters describe iqDNA as a modified,
structured, partially single-stranded DNA that evades innate
immunity while remaining transcriptionally active in the cell. In
mouse studies, the immune profile of iqDNA is consistent with
avoidance of key innate immune pathways such as cGAS-STING, which
is known to have a significantly lower binding affinity for
single-stranded DNA than double-stranded DNA. Given the
non-immunogenic nature of iqDNA, Generation Bio believes it can now
create novel therapies with a wide therapeutic index, which has
been a major challenge to the development of in vivo DNA-based
genetic medicines.
The company invented iqDNA using its proprietary rapid enzymatic
synthesis (RES) for DNA production. RES is a cell-free process that
permits chemical and structural changes with a high degree of
control, facilitating heightened DNA functionality through
engineering molecular design and components. Because RES does not
rely on cellular or viral components, it consistently produces 99%
pure material in approximately one week, marking a significant
improvement over biologic methods, which are often lengthy and
complex. Further, due to the nature of enzymatic processes, iqDNA
can be manufactured at scale to support clinical and global
commercial demands without the challenges faced by cell-culture
based production.
Generation Bio continues to leverage RES to advance its iqDNA
platform by optimizing various structural and chemical elements. In
new data presented at ASGCT, a second generation of iqDNA achieved
higher luciferase expression than first-generation iqDNA. The
company is currently testing formulations of second-generation
iqDNA encoding Factor VIII.
The company’s ctLNP platform is designed to reach a broad set of
target cell types and tissues with exquisite selectivity. Data
presented at ASGCT describe the foundation of the platform,
Generation Bio’s “stealth” LNP, which was engineered in part by
making structural modifications to the parental ionizable lipid
composition. Cell-specific targeting is achieved by optimizing
ligand selection and linker chemistry. Additional data on T cell
delivery, including the in vivo delivery of therapeutic transgenes
via ctLNP, will be discussed in an oral presentation on Saturday,
May 11.
Further information on the poster and oral presentations can be
found here. A copy of the presentation materials will be added to
the “Our Scientific Presentations” section of the company’s website
here on the day of each presentation.
Forward-Looking Statements
Any statements in this press release about future expectations,
plans and prospects for the company, including statements about the
company’s strategic plans or objectives, technology platforms,
research and clinical development plans, and preclinical data 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; uncertainties
regarding our novel platforms and related technologies; whether
results from preclinical studies will be predictive of the results
of later preclinical studies and clinical trials; 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; as well as the other risks and uncertainties set forth
in the “Risk Factors” section of the company’s most recent annual
report on Form 10-K, which is 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.
About Generation Bio
Generation Bio is innovating non-viral genetic medicines to
provide durable and redosable treatments for hundreds of millions
of patients living with rare and prevalent diseases. The company is
developing two distinct and complementary platforms: a potent,
highly selective cell-targeted lipid nanoparticle (ctLNP) delivery
system and a novel immune-quiet DNA (iqDNA) cargo produced by a
scalable capsid-free manufacturing process that uses proprietary
cell-free rapid enzymatic synthesis (RES). With these platforms,
Generation Bio aims to develop the next wave of non-viral genetic
medicines to support its 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.
Investors and Media Contact Maren
Killackey Generation
Bio mkillackey@generationbio.com 857-371-4638
Generation Bio (NASDAQ:GBIO)
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Generation Bio (NASDAQ:GBIO)
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