Generation Bio Co. (Nasdaq:GBIO), a biotechnology company
innovating genetic medicines for people living with rare and
prevalent diseases, announced that it has reached two significant
achievements through in vivo studies, including results generated
as part of its collaboration with Moderna, Inc. First, Generation
Bio has shown highly selective T cell targeting in vivo with its
cell-targeted LNP (ctLNP) platform in a humanized mouse model.
Separately, the company has confirmed the stealth properties of its
ctLNP platform in non-human primates (NHPs). Generation Bio will
present these data at the European Society for Gene and Cell
Therapy (ESGCT) 30th Annual Congress in Brussels, Belgium at 4:40
p.m. CEST today. Slides from the presentation will be made
available on Generation Bio’s website following the presentation.
Generation Bio’s ctLNP platform enables delivery of RNA and DNA
cargos to difficult to reach tissues and cell types by avoiding
off-target clearance by the liver and spleen, and by using
conjugated targeting ligands to drive selective, receptor-mediated
uptake. The ctLNP platform is the foundation of the immune cell
targeting programs in Generation Bio’s collaboration with Moderna,
which aims to leverage ctLNPs for in vivo delivery of nucleic acid
therapies to select immune cell types, including T cells.
“The data we’ve generated for our ctLNP platform demonstrate a
unique ability to target extrahepatic tissues and cell types,
giving us confidence as we advance genetic medicines for the
treatment of diseases beyond the liver, which is an exciting new
frontier for the field and for patients,” said Matt Stanton, Ph.D.,
chief scientific officer of Generation Bio.
As part of the company’s collaboration with Moderna, Generation
Bio has demonstrated that its T cell ctLNPs successfully drive
highly selective receptor-mediated uptake and expression of mRNA in
humanized mice upon systemic administration, as shown in Figure 1.
Furthermore, uptake and expression in T cells was shown to be
dose-responsive, without increasing delivery to off-target cell
types, as shown in Figure 2.
Figure 1: mRNA expression with T cell ctLNP in
humanized mouse model
Figure 2: Dose response with T cell ctLNP in
humanized mouse model
In a separate NHP study, the company confirmed the properties of
its stealth LNP, including long circulating half-life and very low
off-target delivery to liver and spleen, as shown in Figures 3a and
3b.
Figure 3a: Half-life of stealth LNP in NHPs and
mice
Figure 3b: Biodistribution of stealth LNP in
NHPs
“The mRNA expression data indicate the power of ctLNPs to
de-target the liver and spleen and reach specific extrahepatic cell
types by using a targeting ligand,” said Phillip Samayoa, Ph.D.,
chief strategy officer of Generation Bio. “Furthermore, species
translation for the stealth LNP underlying our ctLNP platform is
highly encouraging as we continue to move forward with our
cell-targeting work. We have made significant advances in
bioconjugation and ligand optimization, which we are applying to
develop ctLNPs for new targets.”
In addition to today’s presentation at ESGCT, the company will
host a webcast-only R&D deep dive and Q&A session on
Wednesday, November 1. This presentation will provide further
detail on Generation Bio’s ctLNP and iqDNA platforms as well as on
its RES manufacturing. Registration information for the webcast
R&D deep dive 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 novel immune-quiet DNA construct 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, the terms of the
research collaboration between Moderna and Generation Bio to
develop novel nucleic acid therapeutics, including the potential to
target immune cells with diverse nucleic acid cargos and the liver
for gene replacement, the targets to be developed under the
collaboration, and the potential benefits and results that may be
achieved through other 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: that the anticipated benefits of the
collaboration with Moderna may not be achieved on the anticipated
timeline, or at all; that data may not support further development
of the therapies subject to the collaboration with Moderna due to
safety, efficacy, or other reasons; 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 technologies;
whether results from earlier preclinical studies will be predictive
of the results of later preclinical studies and clinical trials;
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
Photos accompanying this announcement are available
at:
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