Generation Bio Announces Breakthrough in its Non-Viral Genetic Medicine Platform with Novel “Immune-Quiet” DNA
18 Octobre 2023 - 12:59PM
Generation Bio Co. (Nasdaq:GBIO), a biotechnology company
developing genetic medicines for people living with rare and
prevalent diseases, today announced the development of a
proprietary, novel DNA called immune-quiet DNA (iqDNA). iqDNA is an
optimized variant of the company’s closed-ended DNA (ceDNA) that
upon systemic administration with Generation Bio’s lipid
nanoparticle (LNP) delivery has shown cytokine levels and
tolerability comparable to chemically modified messenger RNA (mRNA)
in mice and non-human primates (NHPs).
Generation Bio’s non-viral genetic medicine platform aims to
advance durable, redosable, and titratable therapies to reach
potentially hundreds of millions of patients worldwide. The company
has made deep investments in its three proprietary platform
technologies, comprising novel DNA cargos, rapid enzymatic
synthesis (RES) manufacturing, and cell-targeted LNP (ctLNP)
delivery.
“Developing solutions that allow genetic medicines to avoid
innate immune stimulation is a shared and fundamental requirement
for enabling non-viral therapeutics, as exemplified by the
development of modified mRNA to enable a global modality,” said
Matt Stanton, Ph.D., chief scientific officer of Generation Bio.
“Our development of iqDNA is a similar step toward realizing the
full potential of non-viral DNA therapeutics. I’m excited to
further advance our platform as we seek to create safe and
effective medicines that could marry the scalability and drug-like
properties of mRNA with the durability of DNA-based genetic
medicines.”
Systemic dosing in mouse and NHP studies demonstrates that iqDNA
delivered via LNP evades detection by the key innate immune
pathways that are reactive to DNA. Figure 1 below shows a
comparison of IL-6 levels, representative of all cytokines
measured, in a cohort of NHPs that received an infusion of ceDNA,
iqDNA, or mRNA, each delivered using the same LNP at 1 mg/kg and
containing luciferase as a reporter gene.
Figure 1: IV infusion 1 mg/kg
Importantly, companion mouse studies demonstrate that iqDNA
conserves the key features of ceDNA that afford therapeutic
benefit, including expression.
Due to the superior tolerability profile of iqDNA, Generation
Bio is discontinuing development of prior ceDNA constructs and is
advancing iqDNA as the cargo for its lead program in hemophilia A
as well as for all other programs. Scaling of iqDNA factor VIII for
NHP testing is underway, leveraging existing RES systems and
processes.
“The identification of iqDNA is the culmination of six years of
work at Generation Bio to address the central challenge of immune
activation for DNA-based genetic medicines,” said Geoff McDonough,
M.D., chief executive officer of Generation Bio. “We expect that
iqDNA will significantly enhance the tolerability profile of our
lead program in hemophilia A allowing us to leverage our broad
portfolio of LNPs to advance this and our other programs.”
Dr. McDonough continued: “In parallel with cargo optimization,
we have made substantial investments in the translation of our
ctLNP delivery system from mice to NHPs, initially motivated by
avoiding the innate immune stimulation caused by off-target
delivery of ceDNA to immune cells. This led to the development of
our stealth LNP plus targeting technology, which unlocks the
potential for reaching non-liver cell types and tissues. Our
collaboration with Moderna aims to create highly specific in vivo
delivery to T cells and other immune cell types with both iqDNA and
mRNA cargos. We are excited to share updates on our iqDNA and ctLNP
platforms as we make progress across our liver and non-liver
programs.”
Cash runway extended into 2026
Generation Bio believes it has sufficient cash to extend the
funding of its operations into 2026 based on income from the
Moderna collaboration, including the initial upfront payment, as
well as interest income and expense control. Concurrent with
extending its cash runway, the company is increasing investment in
its iqDNA and ctLNP platforms.
Upcoming events and presentations
- AIchE Conference, October 18: Dr. Stanton will
present the data on iqDNA at the American Institute of Chemical
Engineers' (AIchE) 6th International Conference on CRISPR
Technologies in Boston, Mass. A copy of this presentation is
available on Generation Bio’s website.
- ESGCT Annual Congress, October 26: On
Thursday, October 26 Dr. Stanton will give an oral presentation at
the European Society for Gene and Cell Therapy (ESGCT) 30th Annual
Congress in Brussels, Belgium. The presentation will cover the
development and progress of Generation Bio’s ctLNP platform,
including its stealth and targeting delivery system. A copy of the
slide presentation will be made available on Generation Bio’s
website following the event.
- Webcast R&D Event, November 1: On
Wednesday, November 1 the company will host a webcast-only R&D
deep dive and Q&A session to provide further detail on its
platform capabilities across iqDNA, RES manufacturing, and ctLNP.
Registration information for the webcast can be found on the Events
page of Generation Bio’s investor website.
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 genetic medicine
platform incorporates a novel immune-quiet DNA called iqDNA; a
unique cell-targeted lipid nanoparticle delivery system, or ctLNP;
and a highly scalable capsid-free manufacturing process that uses
proprietary cell-free rapid enzymatic synthesis, or RES, to produce
iqDNA. This approach is designed to enable multi-year durability
from a single dose, to deliver large genetic payloads, including
multiple genes, to specific tissues and cell types, 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 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.
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, cash resources, technology
platform, 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; whether
results from earlier preclinical studies will be predictive of the
results of later preclinical studies and clinical trials;
uncertainties regarding our novel technologies, including iqDNA;
uncertainties regarding the RES manufacturing process; 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; 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.
Investors and Media Contact Maren
Killackey Generation
Bio mkillackey@generationbio.com 857-371-4638
A photo accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/b9bf80f8-5900-471d-b097-b2167f082be9
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