CAMBRIDGE, Mass., Feb. 24, 2022 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, outlined its strategic priorities for 2022 and reported fourth quarter and full year 2021 financial results.
“Our primary focus is on diseases of the liver, where we are working to complete optimization of our cell-targeted lipid nanoparticle, or ctLNP, for the liver to advance our lead hemophilia A program as well as subsequent liver programs,” said Geoff McDonough, M.D., president and chief executive officer of Generation Bio. “In parallel, we are advancing our retina ctLNP to support our Stargardt program, and our vaccine ctLNP to enable our work in vaccines. We have an exceptional team and a strong balance sheet to drive toward achieving our vision.”
2022 Strategic Priorities
Generation Bio has created a unique non-viral genetic medicine platform comprised of closed-ended DNA (ceDNA), rapid enzymatic synthesis (RES) manufacturing for ceDNA and a proprietary portfolio of ctLNPs. The company is applying its platform technologies in three areas: the liver, retina and vaccines.
Fourth Quarter and Full Year 2021 Financial Results
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 DNA construct called closed-ended DNA, or ceDNA; 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 ceDNA. The platform is designed to enable multi-year durability from a single dose, to deliver large genetic payloads, including multiple genes, to specific 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.
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 platform, our research and clinical development plans, applications 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 the timing and ability to complete the build-out of the company’s manufacturing facility and regarding the new manufacturing process; 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 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.
Ten Bridge Communications
GENERATION BIO CO.
CONSOLIDATED BALANCE SHEET DATA
|Cash, cash equivalents and marketable securities||$||375,145||$||262,327|
|Total stockholders’ equity||381,746||268,013|
GENERATION BIO CO.
CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except share and per share data)
|Three Months Ended December 31,||Year Ended December 31,|
|Research and development||$||21,847||$||16,374||$||85,247||$||58,532|
|General and administrative||9,099||7,971||33,854||22,582|
|Total operating expenses||30,946||24,345||119,101||81,114|
|Loss from operations||(30,946||)||(24,345||)||(119,101||)||(81,114||)|
|Other income (expense):|
|Other income (expense) and interest income, net||3||119||(50||)||591|
|Net loss and net loss attributable to common stockholders||$||(30,943||)||$||(24,226||)||$||(119,151||)||$||(80,523||)|
|Net loss per share attributable to common stockholders, basic and diluted||$||(0.54||)||$||(0.53||)||$||(2.12||)||$||(2.95||)|
|Weighted average common shares outstanding,
basic and diluted
|Other comprehensive income:|
|Unrealized (losses) gains on marketable securities||(1||)||(17||)||(9||)||9|