Durable, therapeutically relevant levels of anti-SARS-CoV-2 spike antibodies produced from the liver in a mouse model
Next-generation rapid enzymatic synthesis of closed-ended DNA further increases the efficiency and scale of the manufacturing platform
Potent factor VIII construct optimized by taking advantage of the increased cargo capacity of closed-ended DNA
CAMBRIDGE, Mass., May 12, 2021 (GLOBE NEWSWIRE) -- 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
First Quarter 2021 Financial Results
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.
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.
Ten Bridge Communications
GENERATION BIO CO.
CONSOLIDATED BALANCE SHEET DATA
|Earnings Release Balance Sheet||March 31, 2021||December 31, 2020|
|Cash, cash equivalents and marketable securities||$||451,083||$||262,327|
|Total stockholders’ equity||458,736||268,013|
GENERATION BIO CO.
CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except share and per share data)
|Three Months Ended March 31,|
|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||)|
|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|
|Other comprehensive income:|
|Unrealized gains on marketable securities||1||—|