Generation Bio Reports Business Highlights and Third Quarter 2023 Financial Results
09 Novembro 2023 - 6:15PM
Generation Bio Co. (Nasdaq: GBIO), a biotechnology company
innovating genetic medicines for people living with rare and
prevalent diseases, reported business highlights and third quarter
2023 financial results.
“The recent advances in our immune-quiet DNA (iqDNA) and
cell-targeted lipid nanoparticle (ctLNP) platforms represent two
breakthroughs in the field of genetic medicine,” said Geoff
McDonough, chief executive officer of Generation Bio. “Our iqDNA
has been shown to evade host innate immune sensors in mice and
non-human primates (NHPs), addressing a central challenge for
non-viral DNA therapeutics. Our ctLNP delivery system overcomes an
equally central challenge for delivery, which is the highly
selective targeting of DNA and/or RNA cargo to extrahepatic tissues
and cell types. The foundational properties of iqDNA and our ctLNP
have translated across mice and NHPs, and we now look forward to
generating additional NHP data with therapeutic payloads for each
platform.”
Business Highlights
- Developed iqDNA to Evade Innate Immune
Sensors: Generation Bio announced results from preclinical
studies in NHPs showing that an intravenous infusion of iqDNA
delivered by an LNP triggered little to no activation of a panel of
cytokines associated with the innate immune response. Innate
immunity has been a key challenge for DNA-based non-viral genetic
medicines as it has typically limited their therapeutic index.Given
the improved tolerability profile of iqDNA versus prior
closed-ended DNA (ceDNA) constructs, the company is advancing iqDNA
as its lead cargo across all programs, including its lead program
in hemophilia A. The company believes that iqDNA’s ability to evade
detection by key innate immune sensors while driving protein
expression will allow it to take advantage of a broader array of
LNP delivery systems. Planned next steps for the platform include
scaling iqDNA encoding Factor VIII protein for testing in
NHPs.
- Demonstrated Highly Selective T Cell
Transduction: At the European Society for Gene and Cell
Therapy (ESGCT) 30th Annual Congress, Generation Bio announced data
showing that 70% of circulating and splenic T cells were positive
for mRNA expression with less than 8% delivery to off-target immune
cells following dosing of its ctLNP in a humanized mouse model.
Furthermore, the profile of the underlying stealth LNP translated
to NHPs, with less than 0.1% of the dose taken up by off-target
tissues including the liver and spleen. The studies were conducted
as part of the company’s collaboration with Moderna, Inc. and
provide initial proof of concept for in vivo delivery to T cells
and the potential ability to apply the ctLNP platform to a broader
range of extrahepatic tissues and cell types.
Third Quarter 2023 Financial Results
- Cash
Position: Cash, cash equivalents, and marketable
securities were $291.0 million as of September 30,
2023, compared to $279.1 million in cash, cash equivalents, and
marketable securities as of December 31, 2022. The company
continues to believe that its cash position will fund its operating
plan into 2026.
- R&D
Expenses: Research and development (R&D) expenses
were $21.9 million for the quarter ended September
30, 2023, compared to $21.2 million for the quarter ended September
30, 2022.
- G&A
Expenses: General and administrative (G&A)
expenses were $11.6 million for the quarter ended
September 30, 2023, compared to $11.5 million for the quarter ended
September 30, 2022.
- Net Loss: Net loss was $28.3
million, or $0.43 basic and diluted net loss per share, for
the quarter ended September 30, 2023, compared to a net loss of
$31.3 million, or $0.53 basic and diluted net loss per share, for
the quarter ended September 30, 2022.
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, or 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; 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; 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 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 ContactMaren
KillackeyGeneration Biomkillackey@generationbio.com857-371-4638
|
GENERATION BIO CO.CONSOLIDATED BALANCE
SHEET DATA (Unaudited)(In
thousands) |
|
Earnings Release
Balance Sheet |
|
September 30, 2023 |
|
December 31, 2022 |
Cash, cash equivalents and marketable securities |
|
$ |
291,025 |
|
$ |
279,091 |
Working capital |
|
|
264,990 |
|
|
267,866 |
Total assets |
|
|
381,492 |
|
|
376,264 |
Total stockholders’
equity |
|
|
231,914 |
|
|
282,493 |
|
GENERATION BIO CO.CONSOLIDATED STATEMENTS
OF OPERATIONS (Unaudited)(in
thousands, except share and per share data) |
|
|
|
Three Months Ended September 30, |
|
|
2023 |
|
|
2022 |
|
Revenues: |
|
|
|
|
|
|
Collaboration Revenue |
|
$ |
2,146 |
|
|
$ |
— |
|
|
|
|
|
|
|
|
Operating expenses: |
|
|
|
|
|
|
Research and development |
|
|
21,862 |
|
|
|
21,192 |
|
General and administrative |
|
|
11,641 |
|
|
|
11,477 |
|
Total operating expenses |
|
|
33,503 |
|
|
|
32,669 |
|
Loss from operations |
|
|
(31,357 |
) |
|
|
(32,669 |
) |
Other income: |
|
|
|
|
|
|
Other income and interest income, net |
|
|
3,091 |
|
|
|
1,363 |
|
Net loss and net loss
attributable to common stockholders |
|
$ |
(28,266 |
) |
|
$ |
(31,306 |
) |
Net loss per share
attributable to common stockholders, basic and diluted |
|
$ |
(0.43 |
) |
|
$ |
(0.53 |
) |
Weighted average common shares
outstanding, basic and diluted |
|
|
65,907,000 |
|
|
|
58,872,334 |
|
|
|
|
|
|
|
|
Comprehensive loss: |
|
|
|
|
|
|
Net loss |
|
$ |
(28,266 |
) |
|
$ |
(31,306 |
) |
Other comprehensive loss: |
|
|
|
|
|
|
Unrealized gains on marketable securities |
|
|
19 |
|
|
|
37 |
|
Comprehensive loss |
|
$ |
(28,247 |
) |
|
$ |
(31,269 |
) |
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