Generation Bio Presents Preclinical Data Demonstrating Broad Potential of Gene Therapy Platform at ASGCT and Reports First Qu...
May 12 2021 - 4:20PM
Generation Bio Co. (Nasdaq: GBIO), an innovative genetic medicines
company creating a new class of non-viral gene therapy, today
reported data from multiple digital presentations during the
ongoing 24th American Society of Gene and Cell Therapy (ASGCT)
Annual Meeting as well as first quarter 2021 financial results.
“We are excited to highlight two significant new developments
for our novel, non-viral gene therapy platform that showcase its
potential to address a broad range of diseases at a manufacturing
scale unprecedented for gene therapy,” said Geoff McDonough, M.D.,
president and chief executive officer of Generation Bio. “We have
long thought that therapeutic antibodies could be produced by the
liver, potentially expanding the durability and reach for certain
classes of biologics. Our data demonstrate endogenous therapeutic
antibody production from the livers of mice for anti-SARS-CoV-2
spike antibodies. Combining these data with our proprietary rapid
enzymatic synthesis method for closed-ended DNA (ceDNA) could open
the door to address a number of highly prevalent infectious
diseases. 2021 is set to be milestone-rich for the company, and we
remain on track to execute our goals for the year.”
Highlights from Digital Presentations at
ASGCT
- Novel non-viral gene therapy unlocks significant
potential for genetic medicine: In a digital presentation
entitled “Non-Viral Gene Delivery of Human FVIII to Hemophilia A
Mice and Non-Human Primates,” the company presented insights into
the scientific breakthroughs underpinning its unique non-viral
approach, specifically the on-target biodistribution of its
cell-targeted lipid nanoparticle (ctLNP) and the access to the
nucleus afforded by ceDNA. To view the digital presentation, please
visit Generation Bio’s website.
- Durable expression of therapeutically relevant levels
of anti-SARS-CoV-2 spike monoclonal antibody (mAb) expressed from
the liver in mice: Building on its core technologies,
Generation Bio has designed a ceDNA-based approach to generate
persistent expression of therapeutic antibodies from the liver.
Endogenous therapeutic antibody production may provide an
alternative means of delivering specific mAbs, equipping patients
with the ability to produce their own biotherapeutics for an
extended period of time per dose. In a digital presentation
entitled, “Vector Optimization for Non-Viral Antibody Gene Therapy
and Expression of Human Monoclonal Antibodies in Mice,” the data
presented show that ceDNA delivered via LNP enabled mice to
generate persistent anti-spike protein human antibody
concentrations reaching peak expression of 8 µg/ml, which
corresponds to a level that may be therapeutically relevant in
humans. Importantly, endogenously produced antibodies in the serum
of ceDNA-treated mice retained binding and functional activity,
neutralizing SARS-CoV-2 ex vivo at the same level as recombinantly
produced monoclonal antibodies. Generation Bio developed these data
as part of its collaboration with Vir Biotechnology, Inc. To view
the digital presentation, please visit Generation Bio’s
website.
- Next-generation rapid enzymatic synthesis further
expands efficiency and scale of manufacturing platform: In
the digital presentation on optimization of non-viral endogenous
therapeutic antibody production, Generation Bio outlined a
proprietary, next-generation rapid enzymatic synthesis of ceDNA
directly from nucleic acid components, without relying on Sf9
cells. The structure and sequence of ceDNA from enzymatic synthesis
is comparable to Sf9-produced ceDNA but shortens the ceDNA
production cycle from 28 days to one day. This enzymatic process is
similar to the one used to manufacture messenger RNA (mRNA)
vaccines, with what the company believes will be a comparable
trajectory of cost and scale.
- Construct optimization improves potency of ctLNP-ceDNA
in mouse models of hemophilia A: Generation Bio’s ceDNA
constructs are not constrained by the 4.7 kb transgene size
limitation faced by adeno-associated virus (AAV) gene therapy. In a
poster entitled, “When Size Matters: FVIII Construct Optimization
Leveraging ceDNA, a Non-Viral Gene Therapy Platform,” the company
described its semi-combinatorial approach to optimizing factor VIII
expressing ceDNA, which resulted in 34-times higher expression than
an analogous wild-type factor VIII construct. This demonstrates one
aspect of the potential that derives from ceDNA’s greater cargo
capacity, which includes improved construct potency, larger or
multiple genes and the inclusion of regulatory elements that can
modulate transgene expression. To view the digital presentation,
please visit Generation Bio’s website.
First Quarter 2021 Financial Results
- Cash
Position: Cash, cash equivalents and marketable
securities were $451.1 million as of March 31, 2021,
compared with $262.3 million as of December 31, 2020.
- R&D
Expenses: Research and development (R&D) expenses
were $18.8 million for the quarter ended March 31,
2021, compared to $13.4 million for the quarter ended March 31,
2020.
- G&A
Expenses: General and administrative (G&A)
expenses were $6.9 million for the quarter ended
March 31, 2021, compared to $4.6 million for the quarter ended
March 31, 2020.
- Net Loss: Net
loss was $25.6 million, or $0.46 basic and diluted net
loss per share, for the quarter ended March 31, 2021, compared
to a net loss of $17.7 million, or $3.22 basic and diluted net loss
per share, for the quarter ended March 31, 2020.
About Generation Bio
Generation Bio is an innovative genetic medicines company
focused on creating a new class of non-viral gene therapy to
provide durable, redosable treatments for people living with rare
and prevalent diseases. The company’s non-viral platform
incorporates a proprietary, high-capacity DNA construct called
closed-ended DNA, or ceDNA; a cell-targeted lipid nanoparticle
delivery system, or ctLNP; and an established, scalable capsid-free
manufacturing process. The platform is designed to enable
multi-year durability from a single dose of ceDNA and to allow
titration and redosing if needed. The ctLNP is engineered to
deliver large genetic payloads, including multiple genes, to
specific tissues to address a wide range of indications. The
company’s efficient, scalable manufacturing process supports
Generation Bio’s mission to extend the reach of gene therapy to
more people, living with more diseases, in more places around the
world.
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, 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;
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; 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, 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.
Contacts:
InvestorsMaren KillackeyGeneration
Bio541-646-2420mkillackey@generationbio.com
MediaAlicia WebbGeneration
Bio847-254-4275awebb@generationbio.com
Stephanie SimonTen Bridge
Communications617-581-9333stephanie@tenbridgecommunications.com
GENERATION BIO
CO.CONSOLIDATED BALANCE SHEET DATA
(Unaudited)(In thousands)
Earnings Release
Balance Sheet |
March 31, 2021 |
|
December 31, 2020 |
Cash, cash equivalents and marketable securities |
$ |
451,083 |
|
$ |
262,327 |
Working capital |
|
444,991 |
|
|
256,515 |
Total assets |
|
514,081 |
|
|
294,155 |
Total stockholders’
equity |
|
458,736 |
|
|
268,013 |
GENERATION BIO
CO.CONSOLIDATED STATEMENTS OF OPERATIONS
(Unaudited)
(in thousands, except share and per share
data)
|
Three Months Ended March 31, |
|
2021 |
|
2020 |
Operating expenses: |
|
|
|
|
|
Research and development |
$ |
18,753 |
|
|
$ |
13,394 |
|
General and administrative |
|
6,902 |
|
|
|
4,642 |
|
Total operating expenses |
|
25,655 |
|
|
|
18,036 |
|
Loss from operations |
|
(25,655 |
) |
|
|
(18,036 |
) |
Other income: |
|
|
|
|
|
Interest income |
|
93 |
|
|
|
319 |
|
Net loss and net loss
attributable to common stockholders |
$ |
(25,562 |
) |
|
$ |
(17,717 |
) |
Net loss per share
attributable to common stockholders, basic and diluted |
$ |
(0.46 |
) |
|
$ |
(3.22 |
) |
Weighted average common shares
outstanding, basic and diluted |
|
55,366,238 |
|
|
|
5,495,013 |
|
|
|
|
|
|
|
Comprehensive loss: |
|
|
|
|
|
Net loss |
$ |
(25,562 |
) |
|
$ |
(17,717 |
) |
Other comprehensive
income: |
|
|
|
|
|
Unrealized gains on marketable securities |
|
1 |
|
|
|
— |
|
Comprehensive loss |
$ |
(25,561 |
) |
|
$ |
(17,717 |
) |
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